Don’t miss the start to the day with the Plenary Session. Enjoy your morning coffee as you listen to dynamic presentations intended to inspire and empower. Provided by ISPOR
Head to the exhibit hall to refuel, recharge and connect with fellow attendees and exhibitors over a steaming cup of coffee. Provided by ISPOR
As you enjoy your lunch in the Poster and Exhibit Hall, seize the opportunity to engage in meaningful conversations with fellow attendees. Take this time to exchange ideas, forge new partnerships, or simply enjoy causal conversation. Provided by ISPOR
Real-world data (RWD) is increasingly used to complement clinical trial data to assess the effectiveness and safety of therapeutic products. However, the potential of confounding from selection- and time-related biases, as well as unobservable patient characteristics, can pose challenges. Casual inference and casual assumptions provide a framework for addressing this – leveraging machine learning applications for powerful tools to improve the rigor and robustness of estimates.
The panel will explore the foundations and advances in causal machine learning methods, focusing on deriving patient insights and improving treatment decision-making.
1. Dr. Patrice Verpillat will discuss the regulatory perspective on AI, including examples of experimentation at the regulatory level. He will focus on EMA’s AI workplan, and initiatives from regulators to provide guidance to industry, as well as to drive awareness and stakeholder engagement.
2. Dr Katja Hakkarainen will provide the epidemiological perspective on the causal inference methods, especially the adoption of the target trial emulation framework in observational research. She will discuss the principles of target trial emulation, motivations for using the framework, and examples of applications of the framework in observational research.
3. Dr Andy Wilson will provide an overview of the statistical science and implementation of casual inference and describe emerging machine learning-based methods, such as targeted maximum likelihood aimed at addressing confounding. He will also present a case study on estimating vaccine effectiveness from observational data to illustrate the applications of these methods. He’ll conclude with an outline of promising innovative dynamic methods (including the concept of the modifiable ‘treatment policy’).
4. Ipek Özer Stillman will present a manufacturer’s assessment, focusing on evidence-generation activities to shape early-phase decisions, for example, protocol optimization and patient selection. Ipek will explore the potential impact of machine learning in causal inference, and how methods could be applied.
Sponsor: Parexel
Sponsor
Parexel
Moderators
Jackie Vanderpuye-Orgle, PhD
Parexel International, Billerica, MA, USA
Dr. Jackie Vanderpuye-Orgle is a health economist with over 20 years of progressive experience in the application of econometric principles and statistical analysis to both clinical and real-world data. In her role at Parexel, Jackie is responsible for providing technical expertise and oversight on network meta-analysis, advanced parametric modelling, predictive analytics, AI, and general statistical analysis workstreams. She focuses on identifying fit-for-purpose analytic solutions to leverage both clinical trial and real-world data to facilitate market access and to bring the right treatment to the right patient at the right time.
Panelists
Katja M Hakkarainen, PhD
Parexel International, Gothenburg, Sweden
Katja is Vice President in Epidemiology at Parexel’s Real-World Research team. She has over 15 years of experience in RWE, epidemiology, and pharmacoepidemiology in a variety of therapeutic areas, such as neurology, oncology, and endocrinology. Katja has conducted and lead numerous RWE studies globally using a range of data sources, including national health and demographic registers, pregnancy and disease registers, claims, chart reviews, and prospectively collected data from sites. Numerous of Katja’s studies have had a regulatory demand, and have been submitted to regulators. Katja has MScPharm and PhD in public health and epidemiology, and is a registered pharmacist in Finland and Sweden. Katja is based in Sweden.
Ipek Ozer Stillman, MS, MBA
Takeda, Cambridge, MA, USA
Ipek is an accomplished global pharmaceutical leader with two decades of experience in the areas of Value, Evidence, and Market Access. Her career journey is fueled by her passion for driving a positive, sustainable impact in healthcare globally. She thrives on developing innovative strategies that include multidisciplinary approaches, leveraging her expertise at the intersection of business strategy, data analytics, digital transformation, and health policy.
Currently, she is the Head of Global Health Economics at Takeda. She has worked at Takeda for about nine years, with a prior role within the Shire R&D organization as part of the Health Economics, Outcomes Research, and Epidemiology group. Before Takeda, Ipek worked at a large global research consultancy collaborating with global biotechnology and pharmaceutical clients for over ten years.
Ipek holds a Master of Business Administration degree from MIT Sloan School of Management, and a Master of Science degree in Mechanical Engineering from Northeastern University.
Patrice Verpillat, MD, MPH, PhD
European Medicines Agency (EMA), Amsterdam, Netherlands
Dr. Patrice Verpillat (MD, MPH, PhD) is the Head of the Real World Evidence (RWE) Workstream at the European Medicines Agency (EMA). He is a medical doctor, specialist in epidemiology. Before joining the EMA, he has worked during 20 years in the pharmaceutical industry where he had positions in several international companies, always dealing with real world data (RWD) and non-interventional studies (NIS) in order to bring RWE into research, access and life-cycle product management.
Dr. Verpillat has published over 70 articles in Medline referenced journals. He has been involved in many organisations such as ENCePP, ICH M14 working group, European pharma association (efpia) and ISPE
Andy Wilson, PhD, MStat
Parexel International, Waltham, MA, USA
Andy is currently the head of innovative RWD analytics for Parexel. He's also an adjunct professor of statistics at the University of Utah.
Using HRQoL Data From Children and Youth to Strengthen HTA: What Are the Barriers? How Can We Improve Current Practice?
Digital Conference Pass
Notwithstanding the increased availability of instruments for measuring and valuing children and adolescents’ HRQoL, the use of PRO instruments in the economic evaluation of health care technologies in pediatrics continues to show substantial gaps and weaknesses. Recent reviews of pediatric technology assessments submitted to England’s NICE and Australia’s PBAC found limited use of child-specific measures to generate health state utilities, considerable variation in the methods used to generate utilities, and frequent use of adult instruments and health state utilities. Use of poor-quality evidence in decisions regarding children’s health care could potentially lead to non-optimal resource allocation and disadvantage children.
The aim of this symposium is to examine the barriers to using existing age-appropriate tools for HTA in children and adolescents, to identify methods gaps that need to be addressed, and to explore ways that pediatric evidence-based decision-making can be strengthened. The symposium will present a range of perspectives, with speakers from HTA, industry, and academia.
Prof. Devlin will introduce the speakers and provide background for their presentations by describing some of challenges and recent advances in the pediatric PRO and HTA field. Dr. Shah will then talk about the issues faced by decision-makers in organisations such as NICE who have to deal with the reality of basing decisions on sometimes less-than-optimal evidence, and ways of addressing those difficulties, while Dr. Knies will describe efforts made by the National Health Care Institute of the Netherlands to extend formal product appraisal to pediatric health care. Dr. Chandler will provide an industry perspective on the challenges involved in assembling robust evidence in support of pediatric health-care products, and Prof. Ungar will explore ways in which the field could advance from a methodological perspective. Prof. Devlin will lead a Q&A session with the panel of speakers; questions and comments from the audience are welcomed.
Sponsor
EuroQol Research Foundation
Moderators
Nancy Devlin, PhD
School of Population and Global Health, University of Melbourne, Melbourne, Australia
Nancy is Professor of Health Economics at the University of Melbourne, and holds positions as Editor in Chief at Value in Health and Chair of the Board of the EuroQol Research Foundation. She served as the elected President of ISPOR (2019-2020). Her previous positions include being Director of Research at the Office of Health Economics (2009-2019) and Professor of Economics (and Head of the Economics Department) at City University of London (2001-2009). Her principal areas of research expertise are the measurement and valuation of patient reported health outcomes; the cost effectiveness thresholds used in making judgments about value for money; and priority setting in health care. She is currently leading a large programme of research to improve measurement and valuation of quality of life in children (QUOKKA), funded by the Australian Government. Nancy has published >200 peer reviewed journal papers on a wide range of empirical and theoretical topics in health economics. In 2024, Nancy was ranked 3rd in the world’s ‘Highly Ranked Scholars’™ in Health Economics, based on productivity, impact and research quality.
Panelists
Fleur Chandler, MSc
Sanofi and Patient Advisory Board Lead, Duchenne UK, Middlesex, United Kingdom
Fleur is a health economist with a wealth of outcomes research and health technology assessment experience, with over 30 years leading both global and local teams in the pharmaceutical industry. Fleur is especially driven by rare disease, a parent of a teenager with Duchenne Muscular Dystrophy, she is on the advisory board of patient organisation Duchenne UK. Bringing her professional and personal experience together, she conceptualised and leads Project HERCULES, a global collaboration generating gold standard disease level evidence for HTA in Duchenne.
Saskia Knies, PhD
Zorginstituut Nederland, Diemen, Netherlands
Saskia Knies is a coordinating advisor at the National Health Care Institute (Zorginstituut Nederland, ZIN) in the Netherlands. She is responsible for the broadening and improving the HTA tasks at ZIN and leader of WP 2 on Implementation of SUSTAIN-HTA. In addition, she was involved in the recent update of the health economics guidance that was published in January 2024. Saskia began her career in health economics/HTA in 2007 during her PhD research at Maastricht University. Since 2012 she is working as an advisor at the National Health Care Institute.
Koonal Shah, PhD
National Institute for Health and Care Excellence (NICE), London, England, United Kingdom
Koonal is an associate director (science policy and research) at NICE. His is actively involved in NICE's methods work on severity weighting, health inequalities, environmental sustainability and quality of life measurement. Koonal is an honorary senior research fellow and regular guest lecturer at the University of Sheffield.
Wendy Ungar, MSc, PhD
The Hospital for Sick Children Research Institute, Toronto, ON, Canada
Wendy Ungar is a Senior Scientist, SickKids Research Institute, and Professor, University of Toronto. She holds the Canada Research Chair in Economic Evaluation and Technology Assessment in Child Health and directs Technology Assessment at Sick Kids (TASK) where she applies economic methods to child health. Her team maintains the PEDE database, a searchable online database of pediatric economic evaluations used by global HTA agencies Dr. Ungar chairs the Ontario Genetic Testing Advisory Committee which makes funding recommendations on emerging genetic testing technologies for Ontario, Canada. Her book, Economic Evaluation in Child Health was published by Oxford University Press in 2010.
Meditation for Mental Clarity
This workshop is open to both novice and advanced practitioners where we integrate meditation with scientific insights, tailored for attendees of our scientific conference. Participants will acquire practical, evidence-based strategies for stress management and the enhancement of mental clarity. This session will highlight the fascinating connection between the breath and emotions and how we can leverage the former for managing the latter. Attendees will also experience guided meditation, specifically designed to induce mental focus. The workshop will conclude with a Q&A session, encouraging an exchange of questions and personal reflections on the meditative practice.
Leaders: The session will be led by Dr Jag Chhatwal and Dr. Rachael Fleurence. Dr. Chhatwal is an associate professor at Harvard Medical School and director of the Institute for Technology Assessment at Massachusetts General Hospital. He has been practicing meditation for two decades and is a certified meditation instructor with the Art of Living with 1000+ hours of training. Dr Fleurence is a senior advisor at the National Institutes of Health. She previously served at the White House on the launch of a national elimination plan for Hepatitis C. She has been practicing meditation for over 15 years.
ACCESS Forum Session
Introduction to the ACCESS Forum: A New Professional Society Focused on Optimizing Market and Patient Access Globally
Level: Introductory
This session will introduce ISPOR attendees to the Access Forum, a new professional society that aspires to be the global voice and Center of Excellence for promoting market and patient access to innovative healthcare products, technologies, and services. The panel will explore the evolving intersection of health economics and outcomes research (HEOR) with market access (MA), and some initial directions for the emerging collaboration between the AF and ISPOR. Panelists will reflect on an emerging vision for market access that involves cross-functional collaboration across multiple teams and departments. Presenters are drawn from experienced leaders from the HTA, HEOR, market access and life sciences community.
Sean Tunis will open the session with an introduction to the Access Forum and the panelists, and a review of the panel objectives and agenda. Joe Boswell will discuss the reasons for establishing the Access Forum, progress to date, and the rationale for collaboration with ISPOR. Omar Dabbous will present the strategic framework that is guiding the activities of the Access Forum, and key elements of the Access Vision 2030 that is being developed by a group of experienced, recognized market access professionals. Ravinder Dhawan and Erin Mistry will offer their perspectives on the current and emerging challenges for the market access function as it evolves from its positioning as a group of ‘technical experts in a support function’ to ‘strategic partners for the business’. Carole Longson will serve as a reactor to the initial comments drawing on her long tenure at the National Institute of Health and Clinical Excellence (NICE) and more recent consulting experiences. Following brief initial comments from each panelist, the remainder of the session will be interactive Q&A with the panelists and the audience. The discussion will aim to generate ideas on how ISPOR and Access Forum could work together to advance shared goals in market access.
Moderators
Sean Tunis, MD, MSc
Rubix Health, Baltimore, MD, USA
Sean Tunis, MD, MSc. is a Senior Fellow in the Tufts Center for the Evaluation of Value and Risk in Health where works on project involving health technology issues at the interface of regulatory and reimbursement policy, market access, comparative effectiveness, outcomes measurement and health technology assessment. He is also a Principal with Rubix Health where he consults on similar issues with public and private sector organizations. Dr. Tunis also serves as a Mentor-in-Residence at Johns Hopkins Tech Ventures, helping early stage companies to develop their initial reimbursement strategy and to align their clinical development programs with payer and regulatory requirements.
In 2020 and 2021, Dr. Tunis served as a Senior Advisor in the Office of the Principal Deputy Commissioner at the Food and Drug Administration, where he worked on the use of real-world data to inform regulatory and reimbursement decisions for medical devices and diagnostics.
From 2006 to 2019, Dr. Tunis was the Founder and CEO of the Center for Medical Technology Policy in Baltimore, Maryland. CMTP is an independent, non-profit organization that provides a neutral platform for multi-stakeholder collaborations that are focused on improving the quality,
relevance, and efficiency of clinical research.
Through September of 2005, Dr. Tunis was the Director of the Office of Clinical Standards and Quality and Chief Medical Officer at the Centers for Medicare and Medicaid Services (CMS). In this role, he had lead responsibility for clinical policy and quality for the Medicare and Medicaid programs. Dr. Tunis supervised the development of national coverage policies, quality standards for Medicare and Medicaid providers; quality measurement and public reporting initiatives, and the Quality Improvement Organization program. As Chief Medical Officer, Dr. Tunis served as the senior advisor to the CMS Administrator on clinical and scientific policy. He also co-chaired the CMS Council on Technology and Innovation
Dr. Tunis joined CMS in 2000 as the Director of the Coverage and Analysis Group. Before joining CMS, Dr. Tunis was a senior research scientist with the Technology Assessment Group, where his focus was on the design and implementation of prospective comparative effectiveness trials and clinical registries. Dr. Tunis also served as the Director of the Health Program at the Congressional Office of Technology Assessment and as a health policy advisor to the U.S. Senate Committee on Labor and Human Resources, where he participated in policy development regarding pharmaceutical and device regulation.
He received a B.S. degree in Biology and History of Science from the Cornell University School of Agriculture, and a medical degree and master’s in health services research from the Stanford University School of Medicine. Dr. Tunis did his residency training at UCLA and the University
of Maryland in Emergency Medicine and Internal Medicine. He is board certified in Internal Medicine and holds adjunct faculty appointments at Johns Hopkins, Tufts and the University of California San Francisco Schools of Medicine
Panelists
Joseph Bosewell, BS
GenMav, New York, NY, USA
Omar Dabbous, MD, MPH
Curestack, Tampa, FL, USA
Ravinder Dhawan, PhD, MS
Merck, Cokesbury, NJ, USA
Carole Longson, MBE
Carole Longson Consultant, Manchester, United Kingdom
Carole is a respected leader in health technology assessment with considerable achievements in research, public and private sectors. She has unparalleled insight into the health technology innovation pipeline having pioneered the development of NICE’s health technology evaluation programmes. After a career in drug discovery at GSK, Carole was an Executive Director at NICE from 2000-2018 and Chief Scientific Officer at the Association of the British Pharmaceutical Industry from 2018-2020. She was formerly President of Health Technology Assessment International and now is an independent advisor including Life Science Advisor at NICE.
Carole is currently vice chair of the Medicines Discovery Catapult in the UK, was previously on the Scientific Advisory Committee for the EU Innovative Medicines Initiative (IMI) and holds non-Executive Director and advisory roles in the UK and abroad. Throughout her career, she has championed the public and private sector collaborating to ensure medical innovation benefits patients in a way that is sustainable for healthcare systems.
Erin Mistry, MS, BSc
Coremedix, Boston, MA, USA
Member Group Meetings
ISPOR Italy-Rome Chapter Meeting
This meeting is open to everyone interested in connecting with the ISPOR Italy-Rome chapter. It offers a valuable opportunity to learn more about the chapter’s initiatives and explore how to participate in the chapter.
ISPOR Forums
Access to Medicines in Low- and Middle-Income Countries: What Is Being Done? What More Can Be Done? What Can ISPOR Do?
Digital Conference Pass
There is substantial evidence of delays in access to innovative medicines in low- and middle- income countries (LMICs). Ongoing monitoring by leading professional organizations such as the European Federation of Pharmaceutical Manufacturers, the Latin American Federation of the Pharmaceutical Industry, and the Pharmaceutical Research and Manufacturers Association of America have documented that delays to access are typically two years or more. Clearly, both demand-side and supply-side factors play a role though their relative importance is unclear. ISPOR has recently launched a new Special Interest Group on Global Access to Medical Innovation including a Working Group on Global Differential Pricing. This workgroup has gathered the available data and literature on access delays and is reviewing the aims, structure, and key features of programs that health technology developers (HTDs) and payers have established to improve access in LMICs. In this workshop, Mikkel Oestergaard, working group co-chair, will present their findings on these delays and the programs that companies, country payers, and regions have developed to address them. Kart Veliste, Silvana Lay, and Marlene Gyldmark are contributors to this literature and are experienced in the issues that payers and companies face in their access negotiations. The The key discussion question will be: given what we know about current access delays and barriers, what steps could be taken toward economists' universally-agreed target of efficient (or "fair") global differential pricing to support the global dynamic efficiency, i.e., the optimal rate of innovation?
This session will stimulate, within the ISPOR community, a discussion of potential policy changes that HTDs and payers could make to better use global differential pricing to improve access to innovative medicines in LMICs.
Moderators
Mikkel Oestergaard, PhD
MSD Innovation & Development GmbH, Zurich, ZH, Switzerland
Mikkel Oestergaard is an Executive Director in HTA Statistics at MSD and is based in Switzerland. He has worked in global pharma companies since 2012, and previously worked at the World Health Organization on health statistics and research policy. Mikkel has a PhD from the University of Cambridge.
Speakers
Marlene Gyldmark, MPhil
Idorsia Pharmaceuticals, Allschwill, BL, Switzerland
Marlene is a leader with several years’ experience in health service research, academia, and life science industry from both developing as well as developed countries.
Work experience includes building state-of-the art HEOR teams at Idorsia AG, Switzerland, at Roche Diabetes Care, Switzerland, and Roche Pharma global headquarters in Switzerland, as well as holding various positions within the field of Market Access, Pricing and HEOR at Roche Pharma, Novo Nordisk, and Pfizer, Denmark. Prior to the experience in industry, Marlene worked for more than 7 years in health service research in Denmark, Nepal and Tanzania.
In addition, Marlene also has many years of experience as an external lecturer in health economics at Copenhagen University, as well as leading an executive course in “Market Access for Pharmaceuticals” under Copenhagen University life-long learning.
Marlene is a former member of the Board of Directors at ISPOR, and also sits on the board of the patient organization "Institute of Neurodiversity (ION)".
Marlene is educated as master in economics and political sciences from University of Copenhagen, Denmark, and Health Economist from University of York, UK.
Silvana Lay Ma, MBA
FIFARMA, Frisco, TX, USA
Silvana Lay is an engineer from Universidad Agraria La Molina and holds an MBA with a focus on International Business from Tulane University. With over eight years of experience in the pharmaceutical industry, she has spearheaded strategies for medicine access and public policy at major companies like MSD and Roche Farma. Currently, she serves as Director of Access and Public Affairs at FIFARMA, where she is dedicated to enhancing systems that ensure timely access to innovative medicines across Latin America. Before her career in the health sector, she managed critical areas of sustainability, corporate social responsibility, communications, and compliance in the oil and gas industry, distinguishing herself through her ability to integrate innovation with social impact.
Kärt Veliste, BSc; MSc
Estonian Health Insurance Fund, Tallinn, Estonia
Key opinion leader/public payer
Advancing Real-World Evidence and Health Technology Assessment for Medical Devices and Diagnostics
Digital Conference Pass
Health technology assessment (HTA) evaluates the benefits of medical technologies and helps determine the value of a diagnostic or medical intervention and whether it should be made available in a health system. Real World Evidence (RWE) provides insights on how technologies are used and perform in a real world setting and is becoming increasingly important for reimbursement and regulatory decision making.
HTA submissions typically use generic frameworks for assessing medical innovations, which are not specific for diagnostics or medical devices, and may hinder how their value is measured and evaluated. Additionally, these frameworks may not include RWE, which is important to enable a complete evaluation of evidence, particularly for medical devices.
This forum aims to highlight opportunities for improvements to HTA frameworks to more fully capture the value of diagnostics and medical devices. Case studies will be used to illustrate uses of RWE and the importance of including a tailored evidence evaluation framework for diagnostics and medical devices. The forum will also include a state of the art discussion on the methods to evaluate diagnostic tests (Dr. Lavinia Ferrante), a presentation on the current challenges experienced by HTAs for the assessment on these technologies (Mr. David Tamblyn), and a discussion with two medical device companies (representatives: Lindsay Bockstedt and Chiara Capobianco) presenting their approaches in using RWE, and sharing how evidence standards may evolve as RWE becomes more widely accepted. The panel will conclude with questions from the moderator and the audience.
Moderators
Arturo Cabra, MSc
GE Healthcare, Miami, FL, USA
Arturo Cabra, BE, MSc.
Global Leader. Health Economics & Outcomes Research.
GE HealthCare, PDx
Arturo Cabra is Global Leader of Health Economics and Outcomes Research (HE&OR) at GE HealthCare PDx (Pharmaceutical Diagnostics). In this role, Arturo is responsible for leading the development and execution of evidence generation strategies to address patient, payer and physician needs across the GEHC-PDx portfolio.
Arturo is currently the Member Engagement co-chair of the ISPOR Medical Device & Diagnostics Special Interest Group.
Arturo holds a master’s degree in Healthcare Economics from Universidad Anahuac and advance training in Epidemiology and EBM.
Speakers
Chiara Capobianco, MSc
Boston Scientific, Milan, MI, Italy
Chiara Capobianco, M.Sc., is the Real-World Evidence EMEA team Lead at Boston Scientific (BSC) and board member at the Boston Scientific Foundation Europe.
Chiara holds a MSc in Economics and Health Care Management from the Università Cattolica del Sacro Cuore, Rome, Italy. She has more than ten years of international work experience in the health economics, market access and outcome research field across different therapeutic areas in the medical device and medical equipment space. During her experience, Chiara held leadership positions to drive market access strategies to enhance patient access to care.
Chiara’s professional areas of interests are in market access, health technology assessment, data generation and transformation to support decision-making by multiple stakeholders in healthcare across different countries and regions.
Lavinia Ferrante di Ruffano, PhD
York Health Economics Consortium, York, United Kingdom
Colleen Longacre, MPH, PhD
Medtronic, Mounds View, MN, USA
Colleen Longacre is a Senior Manager of Health Economics and Outcomes Research at Medtronic. In this role, Colleen is responsible for the development and execution of real-world evidence studies to fulfil global regulatory and reimbursement/HTA requirements for medical device technologies within Medtronic’s Cardiac Rhythm Management Portfolio. Colleen holds a PhD in Health Services Research, Policy, and Administration from the University of Minnesota and an MPH in International Health from Boston University.
David Tamblyn, MPH
The University of Adelaide, Adelaide, SA, Australia
How Do We Facilitate the Implementation of Needed Novel HTA Methodologies in National and EU HTA Practice?
Digital Conference Pass
The EU has invested in developing new health technology assessment (HTA) methodologies through IMI and Horizon EU projects for over ten years. Additionally, HTA bodies have started to work differently, from systematic reviews and ample indications to submission-based work for small and targeted populations. Moreover, the EU HTA Regulation, which will require the first joint assessments to take place in 2025, will affect how HTA bodies work nationally. Finally, there is a growing feeling that data from clinical practice (often referred to as real-world data/evidence) may have a role in HTA next to randomized controlled trials (RCTs).
However, developments in HTA methodologies may usually not match the needs of HTA bodies. How do we better align HTA needs to academic HTA methods development? In the panel, we will introduce a new EU Horizon Europe-funded project, SUSTAIN-HTA, that aims to bring academic developments in HTA methodology closer to the HTA needs. Two representatives from NICE and Central and Eastern Europe (Ukraine) will indicate the need for such a project. Additionally, a researcher from a well established research institute from Hungary will indicate the importance of SUSTAIN-HTA of bridging academic HTA development to HTA methods implementation and in particular how further development of transferability may ensure adequate and tailored HTA methods for the different contexts in Europe.
Moderators
Wim Goettsch, PhD
National Health Care Institute (ZIN); Utrecht University, Division of Pharmacoepidemiology and Clinical Pharmacology, Diemen, Netherlands
Professor Wim Goettsch is a key contributor to European HTA. At Utrecht University he is a Professor of HTA of Pharmaceuticals. He serves as a special HTA advisor to Zorginstituut Nederland (ZIN) and was former director of EUnetHTA JA3 Directorate.
Speakers
Dalia Dawoud, PhD
National Institute for Health and Care Excellence, London, LON, United Kingdom
Dalia Dawoud, PhD, is Associate Director (Research) at the National Institute for Health and Care Excellence (NICE). She has practised as health economist and researcher for over 20 years. Her current work is focused on advancing HTA and clinical guideline development methods through research.
Dalia leads NICE newly established HTA Innovation Laboratory (HTA Lab) as well as NICE’s portfolio of European Commission funded projects, such as IMI EHDEN and HORIZON 2020 HTx projects, with cumulative funding of over 5 Million Euros. She is widely published in the area of health economics and outcomes research and serves as Associate Editor of ISPOR journal Value in Health and as Director on ISPOR Board of Directors (2023-2026).
Zoltan Kalo, PhD
a) Semmelweis University; b) Syreon Research Institute, Budapest, Hungary
Zoltán Kaló is a Professor of Health Economics at the Center for Health Technology Assessment of Semmelweis University and the lead partner of Syreon Research Institute in Budapest, Hungary.
He has 30 years of international experience in academia and industry, specializing in health systems design, HTA implementation, health economics and outcomes research, patient access, and pricing policies of healthcare technologies.
Dr. Kaló serves as a policy advisor to public decision-makers, global health care corporations and scientific societies. Between 2016-2021 he was a Scientific Committee member of the Innovative Medicines Initiative (IMI). He was a Director and Board Member of ISPOR between 2012-2014, the founding Chair of ISPOR CEE Network Executive Committee between 2013-2015 and received the Marilyn Dix Smith Leadership Award of ISPOR for his contribution to the society in 2021.
Oresta Piniazhko, PhD
State Expert Centre of the Ministry of Health of Ukraine, Kyiv, Ukraine
Oresta Piniazhko, PhD, Director of HTA Department at State Expert Centre of Ministry of Health, Ukraine.
Oresta is an experienced expert in HTA, pharmaceutical policy and implementation practitioner. She holds a PhD degree in Pharmacoeconomics and is currently holding a position of Director of HTA Department at the State Expert Center of the Ministry of Health of Ukraine, ensuring management and implementation of the best international practices of HTA into health care system of Ukraine since February 2019. Being a dynamic communicator she is also a President of Ukraine ISPOR Chapter since 2017 and before ISPOR Ukraine Students Network (2015-2017). Oresta is visiting lecturer at The Institute of Business Education of Vadym Hetman Kyiv National Economics University and senior lecturer at Danylo Halytskyi Lviv National Medical University.
12:15 - 12:45
Exhibit Hall Theater
How to Transform Expert Knowledge Into Evidence for Health Technology Assessment: Optimizing Expert Elicitation Techniques
Digital Conference Pass
Level: Introductory
As our industry aims to provide earlier access to novel therapies for patients, there are often instances where there are little to no data available to inform Health Technology Assessment (HTA) decision-making, or there is substantial uncertainty associated with data at the time of submissions. Expert elicitation is an evidence generation technique that is used to elicit evidence using experts’ judgement in the absence of existing data and/or characterise uncertainty to support the assessment of new therapies/technologies. When conducted and applied appropriately, expert elicitation can be leveraged to reduce the impact of uncertainty within HTA submissions, to provide stakeholders with reliable information to inform their decision-making in the absence of patient-level data. For expert elicitation to be leveraged to its full potential in HTA decision-making, it is critical that we ensure suitable efforts are made throughout the elicitation process to adhere to available guidance and increase the confidence of authorities regarding the reliability and credibility of the data produced. This theatre presentation will highlight key considerations and learnings on the conduct of expert elicitation activities, to help provide data at the time of submissions to inform HTA decision-making. We will present recommendations from guidance documents available to help inform appropriate method selection and study design. One such document is the Medical Research Council protocol that has been developed to provide clarity on methods for collecting and using experts’ judgements. This presentation will share our experience in implementing this guidance based on real-world instances and practical examples, ensuring key components such as methods selection, material development, selection of experts, and analyses are appropriate in evidence generation activities. We will illustrate an elicitation exercise through a live practical exercise. A review of criticism found in HTA assessments will be outlined followed by suggestions regarding how they can be overcome.
Sponsor: Adelphi Values
Sponsor
Adelphi Values
Speaker
Louise Heron, MSc
Adelphi Values PROVE™, Bollington, United Kingdom
Louise’s responsibility includes driving innovation and growth across our different practice areas of Value Insight and Communication, Health Economics and Data Analysis, and Payer, Pricing and Reimbursement. She has extensive experience in leading and conducting landscaping and literature research projects to gain insight into product value, reimbursement opportunities and recommendations for market access strategies and in developing impactful health economic models and payer funding tools to communicate product value to payers.
Danielle Riley, MSc
Adelphi Values PROVE™, Macclesfield, United Kingdom
Danielle is an Associate Director in the PROVE™ practice within Adelphi Values. In this role, Danielle leads our expert opinion and expert elicitation service offerings and provides methodological, research and analysis support and insight across all aspects of project delivery. She has extensive experience in leading a variety of primary research projects designed to elicit key stakeholders input on market access challenges and exploratory research questions, Prior to joining Adelphi Values, Danielle gained a first class BSc honours degree in Psychology from Bangor University. She also holds an MSc in Cognitive Neuroscience from Durham University.
12:45 - 13:15
The Art of the Possible for Pre-License Access and Funding
Digital Conference Pass
Find out more on how to maximise early access funding opportunities as part of your commercialisation strategy.
Sponsor: WEP Clinical
Sponsor
WEP Clinical
Speaker
Penny Fairmann, BSc (Hons)
WEP Clinical, London, United Kingdom
Penny has been a successful market access specialist in the Biopharmaceutical industry for 20 years having extensive experience in design and delivery of value materials to support multiple specialised medicine launches. Penny has held a number of infield and senior strategic market access management roles across small and medium biopharmaceutical companies in the UK including UCB and Jazz Pharmaceuticals.
Fast Facts
Go Bold, Go Gold: Amplify Your Impact With Open Access
Join us for an insightful session where the Editor-in-Chief and Deputy Editor of Value in Health Regional Issues for North America & Western Europe explore exciting new opportunities for prospective authors. This presentation promises an in-depth discussion about the editors' vision for the journal, its expanded editorial scope, and the range of author benefits arising from its transition to a gold open access journal including fee structures and waiver/discount programs.
Discussion Leader
Manuel Antonio Espinoza, MD, MSc, PhD
Pontificia Universidad Católica de Chile, Santiago, Chile
Manuel Espinoza is Associate Professor in the Department of Public Health and Head of the Health Technology Assessment Unit, both at Pontificia Universidad Católica de Chile. Manuel is also Editor-in-chief of Value in Health regional issues and Chair of the HTAi Latinamerica Policy Forum, and co-chair of the ISPOR SIG on Health Equity Research. He was president of the ISPOR Latinamerica Consortium, founder of the Chilean ISPOR Chapter, and member of the board of directors of ISPOR.
Manuel holds a medical doctor degree and Master in Epidemiology both from Pontificia Universidad Católica de Chile; a Master in Biostatistics from Universidad de Chile, and Master and PhD in Health Economics, both from University of York in the UK. Manuel´s work is focused on methods and processes for prioritization in health care as well as applied economic evaluation and impact policy analysis.
Rafael Pinedo-Villanueva, PhD
University of Oxford, Oxford, OXF, United Kingdom
Dr Rafael Pinedo-Villanueva is Associate Professor and Senior Health Economist at the Centre for Statistics in Medicine, University of Oxford, UK. He is the head of the Health Economics and Outcomes Research group where he leads a portfolio of publicly and industry-funded research projects focusing on the use of real-world evidence for economic evaluations and analyses of healthcare interventions in the UK, Europe, and beyond. He is Deputy Editor of the North America and Western Europe region of Value in Health Regional Issues and a collaborator of the Global Burden of Disease Study.
13:00 - 14:00
Member Group Meetings
ISPOR Spain Chapter Meeting
This meeting is open to all who are interested in learning more about and getting involved in the activities of ISPOR Spain Chapter.
13:15 - 14:15
ISPOR Medical Devices and Diagnostics Special Interest Group
Join us for the ISPOR Medical Device & Diagnostics Special Interest Group Open Meeting. This session is a unique opportunity for professionals to engage with the MD&D SIG leadership team and to co-create, lead, and participate in shaping the future of our initiatives.
We'll kick off with an introduction sharing insights into our ongoing activities. This will be followed by updates on key projects and member project proposals, highlighting the value of diagnostic information and capacity-enhancing innovations. Additionally, a micro-course on economic evaluation for diagnostics will be introduced. We will also provide a summary of our recent forum on advancing Real-World Evidence and Health Technology Assessment for medical devices and diagnostics.
The meeting will conclude with breakout group discussions, enabling participants to delve deeper into topics of interest and engage in collaborative dialogues. This meeting is ideal for those in academia, industry, healthcare, and policy-making, offering a vibrant platform to exchange ideas and forge meaningful collaborations. Don’t miss the chance to help shape impactful initiatives in medical devices and diagnostics.
13:30 - 14:30
ISPOR Student Network Session
Advancing Evidence Generation Through HTA for Enhanced Health Outcomes: A Global Perspective
The ISPOR Europe 2024 Student Network Event will discuss generating evidence toward enhancing health outcomes, focusing on the translation of health technology assessment (HTA) from a global perspective. A panel of industry, regulatory, and academic experts will lead the discussion, focusing on the generation and implementation of HTA and the challenges faced in these areas. The session will showcase innovative approaches and real-world case studies that highlight successful HTA applications, explore the interface with other decision making on the technology, and examine the impact of HTA on health policy and future research. The speakers will engage in a discussion both with each other and with the audience through a question-and-answer format. This event is designed to educate not only students and early-career professionals, but also all other ISPOR members attending the conference interested in the making of HTA and its impact.
Moderators
Kanya Shah, PhD, PharmD, MBA, MS
University of Illinois Chicago College of Pharmacy, Chicago, IL, USA
Kanya Shah is currently a Ph.D. student at the University of Illinois Chicago (UIC) in the Pharmacy Systems Outcomes and Policy department. She previously earned a Doctor of Pharmacy (PharmD), Master of Science (MS) in Health Outcomes, and Master of Business Administration (MBA) from the University of Rhode Island.
Speakers
Michael Berntgen, PharmD, PhD
European Medicines Agency (EMA), Amsterdam, Netherlands
Michael Berntgen is Head of the Scientific Evidence Generation Department at the European Medicines Agency (EMA), Amsterdam. This department supports the development of medicines to ensure generation of robust and relevant scientific evidence, also in collaboration with other stakeholders (e.g. patients, HTAs). Activities include the provision of scientific advice and methodology qualification, management of the PRIME scheme, support to medicines for the paediatric population and for orphan diseases, as well as provision of expertise and support in translational sciences. Furthermore, Michael coordinates across the Agency the collaboration with HTA bodies and payers, to foster timely access to medicines.
Sandipan Bhattacharjee, PhD, MS
Bayer US LLC, Whippany, NJ, USA
Gihan Hamdy Elsisi, PhD
HTA office, Cairo, Egypt
Fang-Ju (Irene) Lin, PhD
Department of Pharmacy Systems, Outcomes and Policy, University of Illinois Chicago College of Pharmacy, Taipei City, Taiwan
Dr. Fang-Ju Lin (Irene), MSCP, PhD, is an Associate Professor at the Graduate Institute of Clinical Pharmacy, National Taiwan University. She specializes in pharmacoepidemiology and pharmacoeconomics, focusing her research on the safety and effectiveness of medications in cardiovascular diseases, cardio-oncology, and chronic obstructive pulmonary disease. By utilizing a variety of healthcare databases, her work seeks to deepen insights into drug outcomes in complex clinical settings, supported by her extensive background in clinical pharmacy.
13:45 - 14:45
Spotlight Session
Fit-for-Purpose Real-World Data: Principles and Developments
Digital Conference Pass
RWE to support decision making in HEOR relies on fit-for-purpose data. Much is talked about the quality of research data derived from clinical practice (Real-world Data). To avoid confusion, it helps to keep the inferential use case in mind and work backwards: To support causal conclusions in etiologic studies on drug effects information bias needs to be minimized. What are the measurement error mechanisms that lead to information bias, what are the metrics to quantify them, how do we assess measurement characteristics in validation studies, and how do we assess the impact of imperfect measurement on an effect estimate? Once this is understood the many supporting activities and terms currently used can be better contextualized. These issues will be developed in a logical way to develop a coherent and results-oriented data quality map. Using precise language on data quality and information bias provides clarity in the current mambo-jumbo of buzzwords.
Assessing the measurement characteristics in a given database is time-consuming and expensive. We highlight approaches to expediting data validations through stratified batch validation, through LLM support in establishing electronic health record (EHR)-based reference standards, and through claims diary reviews. To what extent can coding algorithms be shared across data sources and how can their transportability be assessed? We connect our points with recent FDA/EMA guidance documents, discuss implications for federated data analyses, how to prepare decision-makers as reviewers of EHR/claims data analyses, and how to improve your marketing position as a producer of RWD.
Moderators
Sebastian Schneeweiss, MD, ScD
Chief, Brigham and Women's Hospital, Boston, MA, USA
Sebastian Schneeweiss, M.D., Sc.D., is a Professor of Medicine at Harvard Medical School and Chief of the Division of Pharmacoepidemiology, Department of Medicine, Brigham and Women’s Hospital in Boston. He holds a secondary appointment in the Department of Epidemiology of the Harvard T.H. Chan School of Public Health.
His research focuses on assessing the effectiveness and safety of biopharmaceuticals in clinical practice. He has developed and applied analytic methods to improve the accuracy of estimating causal treatment effects of newly-marketed drugs using complex digital healthcare databases. His work is published in >500 articles and is used for regulatory and coverage decision making around the globe. He is funded by NIH, PCORI, Burroughs Wellcome, and the FDA where he is also a voting consultant. He is Principal Investigator of the FDA Sentinel Innovation Center and co-leads the RCT-DUPLICATE initiative and ENCORE to understand when and how real-world evidence studies can reach robust conclusions to support regulatory and HTA decisions.
He is Past President of the International Society for Pharmacoepidemiology and is a Fellow of the American College of Epidemiology, the American College of Clinical Pharmacology, and the International Society for Pharmacoepidemiology. In 2024, he is Visiting Fellow of the Center for Advanced Studies, Ludwig-Maximilians-Universität, Munich.
Speakers
Daniel Prieto-Alhambra, MD, PhD
Oxford University, London, United Kingdom
Professor Dani Prieto-Alhambra (MD, MSc (Oxon), PhD) is a clinician scientist with a long track record in pharmaco- and device epidemiology and real world evidence research. He leads the Health Data Sciences team at the Botnar Research Centre (University of Oxford) and recently took the role of Professor of Real World Evidence and deputy director for the EMA-funded DARWIN EU® initiative from the Medical Informatics department, Erasmus MC, Rotterdam.
Jeremy Rassen, ScD
Aetion, Inc., New York, NY, USA
Jeremy A. Rassen, MS, ScD is a pharmacoepidemiologist with 25 years of academic and industry experience. He is co-Founder, president, and chief technology officer at Aetion, a healthcare technology company that delivers real-world evidence for life sciences companies, payers, and regulatory agencies. Prior to founding Aetion, Dr. Rassen was assistant professor of medicine at Harvard Medical School, where he focused on methods to improve the quality and validity of real-world data studies. He also worked in Silicon Valley in a variety of tech companies. Dr. Rassen received his bachelor’s degree in computer science from Harvard College and his master’s and doctorate degrees in Epidemiology from the Harvard T.H. Chan School of Public Health.
Breakouts: IP, WS, & OBS
Cure Models for Health Technology Assessment: Can They Be Trusted for Decision-Making?
Digital Conference Pass
Level: Experienced
ISSUE:
New treatments for cancer and other diseases increasingly appear to offer the potential for cure. But enforcing an assumption of cure when fitting models to data from clinical trials is associated with substantial uncertainty due to small sample sizes and limited follow-up periods. Therefore, health technology assessment (HTA) decision-makers may be reluctant to utilise cure models. This Issue Panel will debate whether we should be more open to using cure models to inform HTA decision-making, and whether new methodological developments mean we can place greater trust in these models.
OVERVIEW:
Parametric survival models are used to extrapolate beyond clinical trial follow-up periods to estimate the long-term survival benefits associated with new health technologies. ‘Standard’ parametric models are likely to extrapolate poorly if some patients are cured. For new treatments that are potentially curative, such as immunotherapies and chimeric antigen receptor (CAR) T-cell therapies, using cure models to extrapolate survival is logical. However, cure models may estimate cure fractions inaccurately, leading to biased estimates of treatment effects and cost-effectiveness, especially when fitted to data with limited follow-up and small sample sizes. Dr John will introduce the session (2 minutes). Professor Latimer will present flexible parametric non-mixture cure models, arguing that these offer advantages over more commonly used models (11 minutes). Dr Felizzi will focus on novel ‘informed’ mixture cure models (11 minutes). Dr Whittington will draw on published examples of cure models used to support HTA decision-making, discussing their practical and technical strengths and limitations (11 minutes). Dr John will summarise the panel presentations, offering her perspective as a NICE Appraisal Committee Chair, and a general practitioner (8 minutes), and will lead the debate and discussion with the audience (17 minutes). Stakeholders with an interest in the assessment of potentially curative treatments will find this session interesting and stimulating.
Moderators
Megan John, MBBS, BSc, MRCGP
National Health Service and Chair of NICE Appraisal Committee D, Windsor , United Kingdom
Megan John is Chair of NICE HTA Appraisal Committee, a General Practitioner, commissioning leader and Associate Chief Medical Officer for transformation in the NHS.
Megan completed undergraduate medical and surgical studies at Imperial College London, with an additional business management degree focussing on health care management. Her medical studies started in the same year as the inception of NICE. This motivated Megan to pursue evidence based medicine and analysis in the provision of NHS therapies, and addressing equity of access to health care, as her continued fields of special interest.
She holds the lead role for priorities and policy for medicines and non-medicines with Frimley Health and Care Integrated Care Board (ICB) and for the South East England NHS collaborative. She is also ICB clinical lead for individual funding requests and the Evidenced Based Intervention program.
Since completing her training Megan has worked as a general practitioner and practice partner, with special interests in health care service commissioning, health inequalities, cross sector leadership and collaboration, minor surgery, dementia and sexual health.
Motivated, and experienced, in development and application of ethical framework decision making tools for the Health care sector, Megan is an adviser at Oxford University ETHOX department and is an associate for health inequalities. She has also previously been a member of the NIHR late stage funding committee.
Megan started with NICE as a committee member in 2018 on technology appraisal before moving in 2021 to position of chair.
Panelists
Federico Felizzi, PhD
Menarini GmbH, Basel, BS, Switzerland
Federico Felizzi is Global HEOR Franchise Lead - Oncology at Menarini. Previously he was a Global HEOR Director at Novartis, and he has served as HTA Statistician, Health Economic Modeler and HTA Evidence Lead at Roche. He has contributed to the submission of new interventions to HTA assessors in key markets. Through thought partnerships he helped co-build evidence packages with a strong focus on outcomes certainty.
He holds a Degrees in Mathematics a PhD in Systems Biology from ETH Zurich and an MBA at IE Business School.
Nicholas Latimer, PhD, MSc
University of Sheffield & Delta Hat Limited, Sheffield, United Kingdom
Nick is a Professor of Health Economics at the University of Sheffield, a Yorkshire Cancer Research Senior Fellow, and an Analyst for Delta Hat Ltd. He was a member of NICE Technology Appraisal Committee B for 5 years and is a member of the NICE Decision Support Unit (DSU). Nick’s research focuses on survival analysis in economic evaluations. He has authored four DSU technical support documents related to survival analysis, treatment switching, and partitioned survival models. His current work involves investigating the use of cancer registry datasets to estimate the comparative effectiveness of cancer treatments used in clinical practice.
Melanie Whittington, PhD, MS
Tufts Medical Center (CEVR), Boston, MA, USA
Dr. Melanie Whittington is the Managing Director and Head of the Leerink Center for Pharmacoeconomics where she leads pharmacoeconomic evaluations of in-development and recently approved pharmaceuticals. She is also a Senior Fellow at the Center for the Evaluation of Value and Risk in Health (CEVR) at Tufts Medical Center where she works to develop novel methods for value assessment. She was previously the Director of Health Economics for the Institute for Clinical Economic Review (ICER) where she led the cost-effectiveness analyses and budget impact analyses within ICER reviews. She is a leader in the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) and was the recipient of the ISPOR Bernie J. O’Brien New Investigator Award. She has a PhD in Health Services Research with a concentration in Health Economics and Outcomes Research.
Should We Account for Variation in Patient Preferences in Health Technology Assessment?Individual Preferences with Respect to the Characteristics and Possible Outcomes of a Healthcare Intervention May Vary
Level: Intermediate
ISSUE: Individual preferences with respect to the characteristics and possible outcomes of a healthcare intervention may vary. Some patients may have a preference for maximising the probability of response to treatment, whereas others may have a preference for avoiding certain adverse events. Some patients may have a preference to avoid injections, whereas others may wish to avoid frequent oral medication.
However, the standard decision rules for cost-effectiveness analysis identify a single technology as maximising expected health benefit. Decisions made on this basis may fail to recognise variation in preferences and circumstance and be economically inefficient failing to maximise patient utilities, compromise treatment adherence, and worsen health inequalities.
In this issue panel we will explore how heterogeneity in individual preferences might be investigated using qualitative and quantitative methods, including elicitation of personal utility functions, and accounted for in decision-making in terms of deliberation and ‘decision-rules’.
OVERVIEW: Ben Van Hout will discuss evolving methods to solicit more accurate estimates of utility weights using personal utility functions and their potential use in decision-making.
Neil Hawkins will moderate and will provide context by exploring how the ‘rules’ of cost-effectiveness analysis might be adapted to account for preference heterogeneity including the role of cost-minimisation, net-benefit, and ‘individualised cost-effectiveness” approaches.
Shelagh Szabo will describe methodologies that have been used to explore heterogeneity in patient preferences. These include qualitative methods using grounded theory and quantitative methods such as discrete choice experiments with latent class analysis.
Amanda Adler, as a clinician and an ex-chair of a NICE technology appraisal committee, will moderate and share her experiences in considering heterogeneity in preferences in real-world decision making. We will use real-time polling to allow the audience to express their own heterogeneity in preferences with respect to this topic and to allow the audience to act as a reimbursement ‘decision-maker’.
Moderators
Amanda Adler, MD, PhD, FRCP
Churchill Hospital, Oxford, Oxford, United Kingdom
Amanda Adler has previously been a chair of a NICE Technology appraisal committee and has extensive interest in the application of health technology assessment in resource allocation
Panelists
Neil Hawkins, MSc, PhD, MBA, CStat
University of Glasgow, Glasgow, United Kingdom
Shelagh Szabo, MSc
Broadstreet Health Economics & Outcomes Research, Vancouver, BC, Canada
Shelagh is a Scientific Director and Founding Partner at Broadstreet HEOR. She is an epidemiologist with over 15 years of experience in health outcomes research and consulting. Prior to co-founding Broadstreet, she was a Principal at ICON plc where she headed up the Epidemiology team and later was Head of Evidence Generation at Redwood Outcomes.
She has conducted studies in the areas of oncology, infectious diseases, rare diseases, diabetes, pediatrics, and ophthalmology, among others and her work has been published in journals including the Journal of Pediatrics, BMJ Open, Annals of Hepatology, Arthritis & Rheumatism, British Journal of Cancer, Clinical Pediatrics, and Value in Health.
Ben A Van Hout, PhD
University of Sheffield, Sheffield, United Kingdom
Ben van Hout combines a position of professor of Health Economics with that of Chief Scientific Officer in OPEN Health Evidence & Access. In 2020, he received the Donabedian Lifetime Achievement award for his contributions to the field of economic evaluations in health care. Over the last couple of years, he has taken his research to the next level by first having a myocardial infarction and second having a large motorcycle accident. Since then, he has taken more than 16,000 tablets, still counting, for prevention, pain and itch.
His current position allows him to only be involved in projects he fancies, “personal utility functions” being on the top of the list.
Balancing Budgets and Breakthroughs: Does Budget Impact Analysis Systematically Disadvantage Gene Therapies?
Digital Conference Pass
Level: Introductory
ISSUE:
Gene therapies are a type of advanced therapeutical medicinal product (ATMP) with the potential to revolutionise our approach to treating disease. They offer the possibility of curing, halting and intercepting disease, rather than managing symptoms over a patient's lifetime. Many healthcare payers around the world rely on health technology assessment (HTA) and budget impact analysis (BIA) to assess the value and affordability of new technologies, including gene therapies. Whilst research has been conducted concerning how the full value of gene therapies can be realised in HTA, much less attention has been given to the challenges posed by gene therapies to the development, use and interpretation of BIA. Current approaches to BIA may judge gene therapies as unaffordable due to BIA’s short-term time horizons and narrow scope. Given the long-term benefits and wider societal impact of gene therapies, there is some concern that BIA as used currently may be systematically disadvantaging this type of therapy. Several questions must be explored: What is the appropriate use of BIA? Is it and should it be used in decisions of reimbursement? Should BIA methodologies be adapted to account for the disadvantages faced by gene therapies, or does it serve its purpose(s) as currently deployed? What would be the implications of any changes?
OVERVIEW:
Following a brief introduction to the issues by the moderator (Grace Hampson, Associate Director, Office of Health Economics, UK) (4 mins), the panel will debate a set of proposed policy recommendations regarding the future of BIA for gene therapies, with reference to the questions above. Panellists will each speak for 12 minutes, providing their perspectives on current challenges of using BIA for gene therapies and their suggestions on what should be done (or not) about the key challenges. 20 minutes will be reserved for audience discussion.
Moderators
Grace Hampson, MSc
Office of Health Economics, London, United Kingdom
Grace is an economist with expertise in the methods and processes of value assessment, reimbursement and healthcare decision making. Her current research interests include interactions between health technology appraisal and novel payment mechanisms, particularly in relation to innovative technologies such as cell and gene therapies and new drugs to tackle Antimicrobial Resistance. Grace leads OHE’s Policy Organisation and Incentives in Health Systems research team.
Panelists
Tom Denee, PharmD, MBA
Johnson & Johnson’ Innovative Medicine Europe, Zeist, Netherlands
Tom Denee has a pharmacy (PharmD) and business (MBA) background and has over 11 years of experience in health economics, market access and reimbursement as well as over 14 years of experience in the pharmaceutical industry in different therapeutic areas and departments at Johnson & Johnson. He has presented multiple posters and organized multiple discussion panels at ISPOR Europe over the last 9 years. He has also been speaking at multiple conferences.
Scott Ramsey, MD, PhD
Hutchinson Institute for Cancer Outcomes Research, Fred Hutchinson Cancer Center, Seattle, WA, USA
Dr. Scott Ramsey is a Professor at Fred Hutchinson Cancer Center and the University of Washington. He directs the Hutchinson Institute for Cancer Outcomes Research, a multidisciplinary team devoted to clinical and economic evaluations of cancer prevention, screening and treatment.
His research portfolio includes SEER-Medicare/Cancer Registry data linkages, patient outcomes, economic modeling of interventions, cost-effectiveness analysis, quality of life assessment, health care utilization, economic burden, pragmatic trial design, early technology assessment, and stakeholder engagement.
David Sánchez González, BA
Retinosis FARPE, Madrid, Madrid, Spain
David Sánchez González is a dedicated advocate for patient rights and a prominent figure in the field of retinal diseases. Since 2008, he has been actively involved in patient advocacy, working tirelessly to improve the lives of those affected by retinal conditions. David was appointed president of RETIMUR, the Retina Association of Murcia, in 2011, where he has been instrumental in driving the organization’s mission forward.
In addition to his role with RETIMUR, David has been an integral part of the Spanish Federation of Rare Diseases (Federación Española de Enfermedades Raras) since 2016, currently serving as the treasurer. His commitment to the global retinal disease community is further evidenced by his position on the board of directors of Retina International, a role he has held since 2020.
David was honored to be named president of both the Federation of Associations for Hereditary Retinal Dystrophies (FARPE) and the Foundation for the Fight Against Blindness (FUNDALUCE) in March 2023. However, he had already been serving on the boards of both organizations for several years prior to his appointment as president. His leadership extends to the European network ENR-EYE, a network dedicated to rare ocular diseases, where he became a member in February 2024.
Originally from Catalonia, David has made Murcia his home for many years. He is married and a proud father of two daughters. Academically, David holds a degree in History from the University of Murcia. Outside of his professional and advocacy work, he is an avid sports enthusiast, particularly passionate about running.
David, who is personally affected by Retinitis Pigmentosa, remains deeply committed to improving communication and membership structures within the retinal disease community, driven by his personal experience and dedication to the cause.
Do Treatment Sequencing / Pathways Models Have a Place in Health Technology Appraisal?
Digital Conference Pass
Level: Intermediate
ISSUE:
The panellists will debate the use of treatment sequence models (also called “pathways models”), using examples from oncology. They will discuss important challenges with developing these models, and whether the issues can be sufficiently addressed to support their implementation in HTA.
OVERVIEW:
Treatment sequence models are intended to evaluate the cost-effectiveness of a sequence of therapies that comprise a clinical pathway. This mitigates the issue of potentially inconsistent assumptions and recommendations that can arise from performing multiple single technology appraisals. However, there are numerous issues with their implementation both methodologically and logistically. Our issue panel will discuss these issues and how/whether they can be overcome, with perspectives from industry, HTA and academia:
- The moderator (Dr Hugo Pedder, University of Bristol) will introduce the concept of treatment sequence models with an interactive example (10 minutes).
- Dr Jeroen Jansen (University of California) will discuss methodological challenges and some of the lessons learned so far in his work on developing sequence models for the Innovation and Value Initiative (10 minutes).
- Professor Dawn Lee (Peninsula Technology Assessment Group (PenTAG)) will discuss logistical challenges when implementing sequence models, including use of Real World Evidence and model validation, with experiences from a live NICE TA in advanced Renal Cell Carcinoma (aRCC) (10 minutes).
- Mark Harries (Ipsen) will provide an industry perspective on the use of these models for HTA, with his experience representing the company in the NICE aRCC pilot pathway TA submission (10 minutes).
We will follow the discussion with a debate on whether such models can have a place in HTA, and if so, what form that should take (15-20 minutes). Stakeholders working with HTAs or clinical guidelines may benefit from attending, whether from industry, public sector, or academia. Whilst we touch on technical details of economic modelling, the discussion will be of general interest.
Moderators
Hugo Pedder, PhD, MSc, BSc
University of Bristol, Bristol, BST, United Kingdom
Hugo is a Research Fellow at the University of Bristol, primarily developing methods for evidence synthesis. He sits on a NICE HTA Committee and Co-Director of the NICE Technical Support Unit, providing technical expertise to support NICE Clinical Guideline development. Hugo is a Statistical Ambassador for the Royal Statistical Society and is passionate about communicating research and data literacy.
Panelists
Mark Harries, BPharm (Hons), MSc, MRPharmS
Ipsen Ltd, London, LON, United Kingdom
Mark Harries is Head of Market Access, UK and Ireland for Ipsen Ltd. He has worked in market access for over 20 years in consultancy prior to joining Ipsen. He has directly been involved and either led or played a significant role in over 40 UK and Ireland HTA submissions including the NICE pathway’s pilot appraisal of aRCC.
Jeroen Jansen, PhD
University of California – San Francisco, San Francisco, CA, USA
Jeroen is a methodologist working at the intersection of evidence synthesis, biostatistics, and health economics. He has worked on treatment sequence models as part of the Innovation and Value Initiative. He is an Associate Professor in the Department of Clinical Pharmacy in the School of Pharmacy at the University of California, San Francisco. He also acts as Chief Scientist - Health Economics & Outcomes Research (HEOR) at the Precision Medicine Group.
Dawn Lee, MMath, MSc
University of Exeter, Exeter, United Kingdom
Dawn Lee is an Associate Professor of Health Economics NICE Evidence Assessment Group PenTAG. Prior to her current role Dawn spent 12 years in consultancy working her way up from a junior health economist to Chief Scientific Officer at BresMed. As an EAG member Dawn led the production of the model and assessment report for NICE’s pathways pilot appraisal of advanced renal cell carcinoma (aRCC) and a recent NICE pilot for late stage assessment of medical technologies. Dawn's key interest include survival modelling, immune-oncology evaluation, structured expert elicitation, sequencing models, surrogate endpoints and cancer diagnostics.
Can Generative AI Aid Readiness for Joint Clinical Assessment (JCA)?
Digital Conference Pass
Level: Introductory
ISSUE:
The Joint Clinical Assessment (JCA) framework aims to streamline the approval of life science innovations in Europe by reducing redundant practices and harmonizing evidence requirements. This single, transparent process can spur pharmaceutical innovation and expedite patient access to approved therapies. However, the process’s complexity, including tight timelines and increased data demands, requires careful planning to avoid inefficiencies and delays. This panel will explore how generative AI can support the readiness for JCA, addressing timeline and scope challenges, and opportunities within this ambitious framework.
OVERVIEW:
Ramiro Gilardino will introduce the JCA framework, its objectives, and its potential impact on pharmaceutical innovation and patient access. He will also introduce the panelists and outline the session structure. Ipek Ozer-Stillman will discuss how the industry can prepare for the JCA, emphasizing the need for system efficiencies. She will summarize key challenges and necessary preparations for submitting the JCA dossier, highlighting opportunities for process optimization. Jag Chhatwal will share his perspective on the technology, highlighting how generative AI can be a game-changer to accelerate the JCA preparations. He will demonstrate the use of generative AI to conduct simulations of different PICO scenarios, highlighting specific examples and potential outcomes of implementing such AI-driven approaches. Diana Delnoij will share the Health Technology Assessment (HTA) perspective, discussing AI’s dual role in evaluating healthcare solutions and supporting HTA activities. She will emphasize the need for capacity building within HTA agencies to effectively harness AI technologies to meet state-of-the-art, scientifically valid, transparent methods required by HTA bodies. Finally, Ramiro will conclude the session by summarizing the key insights from the panel, outlining next steps for stakeholders, and moderating the Q&A session. In conclusion
, this panel will provide a comprehensive overview of how generative AI can enhance JCA readiness, offering actionable insights and strategies to navigate the complexities of this initiative.
Moderators
Ramiro Gilardino, MD, MHS, MSc
MSD, Dubendorf, ZH, Switzerland
Ramiro Gilardino is an impact-driven healthcare executive with over 15 years of leadership in Global Market Access, Health Policy, and HEOR. He has successfully led strategies that improved patient access, drove reimbursement success, and shaped health policy in pharmaceutical, biotech, and global organizations. An expert in advancing HTA frameworks and forging stakeholder partnerships, he is dedicated to promoting health equity and delivering value-based healthcare solutions globally.
Panelists
Jag Chhatwal, PhD
Harvard Medical School and Value Analytics Labs, Wilmington, MA, USA
Dr. Chhatwal is an associate professor at Harvard Medical School and Director of the Institute for Technology Assessment at Massachusetts General Hospital. Dr. Chhatwal has co-authored over 120 original research articles and editorials in peer-reviewed journals. His work has been cited in leading media outlets, including CNN, Forbes, National Public Radio, New York Times, and Wall Street Journal.
Since 2011, Dr. Chhatwal has taught several workshops and short courses on decision modeling and AI at the ISPOR. He is a member of ISPOR AI Working Group. He also serves as an associate editor of Value in Health and is serving as the guest editor for special issues on AI in Value in Health.
Diana Delnoij, PhD
Dutch National Health Care Institute, Diemen, Netherlands
Diana Delnoij is a political scientist who has worked as a health services researcher for more than 35 years. She has published on various research topics, such as payment and insurance systems, quality of care from the patient´s perspective, value based health care, and the role of health technology assessment (HTA) and HTA agencies in health systems. Apart from that, as an editor of the European Journal of Public Health she holds a special interest in public health. At present, she is the Chief Scientific Officer of the National Health Care Institute (Zorginstituut Nederland), the Dutch HTA agency, and she has an endowed chair as professor of Governance on Healthcare Quality and Efficiency at the Erasmus School of Health Policy & Management. In addition, she chairs the International Horizon Scanning Initiative (IHSI), in which 9 European member states co-develop a repository of upcoming pharmaceutical products.
Ipek Ozer Stillman, MS, MBA
Takeda Pharmaceuticals, Cambridge, MA, USA
Ipek is an accomplished global pharmaceutical leader with two decades of experience in the areas of Value, Evidence, and Market Access. Her career journey is fueled by her passion for driving a positive, sustainable impact in healthcare globally. She thrives on developing innovative strategies that include multidisciplinary approaches, leveraging her expertise at the intersection of business strategy, data analytics, digital transformation, and health policy.
Currently, she is the Head of Global Health Economics at Takeda. She has worked at Takeda for about nine years, with a prior role within the Shire R&D organization as part of the Health Economics, Outcomes Research, and Epidemiology group. Before Takeda, Ipek worked at a large global research consultancy collaborating with global biotechnology and pharmaceutical clients for over ten years.
Ipek holds a Master of Business Administration degree from MIT Sloan School of Management, and a Master of Science degree in Mechanical Engineering from Northeastern University.
Podium Sessions
Health Utility Measurement and Methods
Digital Conference Pass
Studies on utility measurement in special populations such as pediatrics and caregivers.
Moderator
Borislava Mihaylova, DPhil
University of Oxford, London, LON, United Kingdom
I am Professor of Health Economics and Lead of Health Economics and Policy Research Unit at Queen Mary University of London, London, UK and Associate Professor and Senior Health Economist at the Health Economics Research Centre, University of Oxford. I study the impact of diseases and treatments on morbidity, mortality, health-related quality of life and healthcare/other resources and costs; cost-effectiveness of prevention and treatment strategies; and development of evaluative frameworks and disease models to assess net effects of policies and treatments. Assessing heterogeneity of effects across population categories/ individuals is a research focus in my work.
P12: How Does Unpaid Caregiving Affect EQ-5D? Does the Average Treatment Effect Differ for 3L and 5L?
2:30PM - 2:45PM
Pennington B, Hernandez M
University of Sheffield, Sheffield, UK
OBJECTIVES: It is recognised that unpaid caregiving can affect health-related quality of life (HRQoL). It is also understood that using EQ-5D-5L instead of EQ-5D-3L to measure patient HRQoL shifts mean utility scores up the scale towards full health and compresses them into a smaller range.
We compared the average treatment effect of different caregiving intensities on EQ-5D-3L and EQ-5D-5L, to understand whether the effect of caregiving on HRQoL differs between the two measures.
METHODS: We used causal inference methods in our cross-sectional observational UK dataset of 3,536 carers and 39,901 non-carers. We conditioned on potential confounders: sex, age, family status, education, and diagnosis of a health condition. We used inverse probability weighting with a multinomial logit model to predict the probability of belonging to the weekly hours of caregiving intensity classes. We predicted EQ-5D-3L and -5L index scores using regression and the probability of reporting full health using a probit model. We checked for balance and overlap.
RESULTS: People providing upwards of 5 hours/week unpaid care had statistically significantly worse EQ-5D-3L and 5L index scores than non-carers. The average treatment effect was smaller for all intensities using 5L than 3L: a maximum of -0.157 for 50+ hours for 3L compared to -0.109 for 5L. Carers were less likely to report full health for 3L and 5L and the effect size was bigger for 3L: up to -0.165 compared a maximum of -0.133 for 5L.
CONCLUSIONS: Unpaid carers have worse HRQoL than non-carers and the utility decrement increases as intensity of care increases. Using EQ-5D-5L instead of EQ-5D-3L reduces the utility decrement associated with caregiving and reduces the decrease in the proportion of respondents reporting full health. Using EQ-5D-5L instead of EQ-5D-3L will reduce the cost-effectiveness of interventions that decrease the caregiving burden, all other things being equal.
P10: Responsiveness of Key Quality-of-Life Measures to Important Changes in the Lives of People With MS: The EQ-5D-3L and Multiple Sclerosis Impact Scale Eight Dimensions
2:00PM - 2:15PM
Goodwin E1, Michalowsky B2, Middleton R3, Hawton A4
1University of Exeter, Exeter, DEV, UK, 2German Center for Neurodegenerative Diseases (DZNE), Greifswald, Germany, 3Swansea University, Swansea, Wales, UK, 4University of Exeter, Exeter, Devon, UK
OBJECTIVES: For healthcare treatments to be approved for use in the UK NHS, robust evidence is required of their impact on health-related quality of life (HRQoL). The National Institute for Health and Care Excellence (NICE) recommends the use of the EQ-5D. Concerns have been raised about the responsiveness of the EQ-5D in the context of multiple sclerosis (MS), however empirical evidence is limited. This research aims to assess the responsiveness of the EQ-5D-3L compared to an MS-specific utility measure, the Multiple Sclerosis Impact Scale Eight Dimensions (MSIS-8D), in people with MS.
METHODS: Analysis was undertaken using data from the UK MS Register (UKMSR), a prospective, longitudinal, cohort study with over 10,000 participants. We investigated changes in EQ-5D-3L and MSIS-8D values before and after (i) commencement of disease-modifying treatment and (ii) onset of new symptoms; and (iii) between time-points with/without a contemporary relapse. Responsiveness was evaluated using standardised response means (SRM), standardised effect sizes (SES) and paired t-tests.
RESULTS: Neither measure appeared responsive to DMT initiation (ie p > 0.05). Both measures were responsive to experiencing a relapse, with the MSIS-8D (mean change -0.047, p = 0.0001, SRM -0.330, SES -0.228) showing greater responsiveness than the EQ-5D (mean change -0.047, p = 0.0054, SRM -0.226, SES -0.139). Neither measure was responsive to experiencing new symptoms (EQ-5D SRM -0.095, SES -0.070; MSIS-8D SRM -0.138, SES -0.118), although the reduction on both measures was statistically significant (p<0.05). In all analyses, 95% confidence intervals for the SRMs indicated that the MSIS-8D was significantly more responsive than the EQ-5D-3L.
CONCLUSIONS: Although the MSIS-8D appears to offer greater responsiveness compared to the EQ-5D-3L, the responsiveness of both measures in this analysis was limited. This adds weight to existing concerns about the ability of QoL measures used in healthcare decision-making to fully capture treatment effects in MS.
P11: EQ-5D-Y in Children With Asthma or Eczema: Does Perspective Matter in Proxy-Reported Outcomes Assessment of Pediatric Populations?
2:15PM - 2:30PM
Lee JJ1, Tan RLY1, Soh JY2, Tham EH2, Ho MSL3, Yew YW3, Tan SL3, Goh ENA4, Herdman M1, Luo N1
1National University of Singapore, Singapore, Singapore, 2National University of Singapore, Singapore, Singapore, Singapore, 3National Skin Centre, Singapore, Singapore, Singapore, 4KK Women’s and Children’s Hospital, Singapore, Singapore, Singapore
OBJECTIVES: We compared the measurement properties of two different proxy versions of EQ-5D-Y-3L (Y-3L) and EQ-5D-Y-5L (Y-5L) in caregivers of paediatric patients with asthma or eczema. Using the proxy-proxy version (P1), caregivers rate the child’s health from their (the proxy’s) point of view; using the proxy-patient version (P2), the caregivers are required to imagine how the child would rate their own health.
METHODS: We recruited paediatric asthma/eczema patients and their parents/legal guardians from the specialist outpatient clinics of two tertiary hospitals in Singapore. Patients completed Y-3L and Y-5L. Proxies completed either the P1 or P2 version of Y-3L and Y-5L. Asthma control was assessed using the Global Initiatve for Asthma (GINA) assessment while eczema severity was assessed by the clinicians using the validated Investigator Global Assessment for Atopic Dermatitis (vIGA-AD). A subset of patient-proxy dyads was reassessed after 101.3 ± 63.6 days. Known-groups validity, responsiveness to improvement, and patient-proxy agreement were assessed for both proxy versions of Y-3L and Y-5L using Y-3L index score (3L-Index), Y-5L level sum score (5L-LSS), and EQ VAS.
RESULTS: We included 328 child-proxy dyads (P1: 154 dyads; P2: 174 dyads). P2 demonstrated lower ceiling effects [P1: 53.90%-63.90%; P2: 47.13%-56.32%]. All P1-derived scores differed as expected between known groups defined by GINA or vIGA-AD but not all P2-derived scores differed as expected. P1/P2 standardized effect sizes for improvement were 0.48/0.21 for 3L-Index and -0.28/-0.42 for 5L-LSS. Patient-proxy agreement was low-to-moderate in both proxy versions (ICC range: 0.22-0.42). All proxy-reported scores indicated better health as compared to self-reported scores regardless of which proxy version was used.
CONCLUSIONS: In this population, neither proxy version was superior on the psychometric tests performed. Either version may be suitable for use depending on the purpose of the study they are to be used in. Further research is needed to confirm these results.
P9: Recommended Standards for Managing and Reporting Missing Utility Data for Health Technology Appraisal
1:45PM - 2:00PM
Hansell N, Moss J, Butler K
York Health Economics Consortium, York, North Yorkshire, UK
OBJECTIVES: Health technology assessment (HTA) in the UK often requires that health-related quality of life is considered in a cost-utility analysis (CUA). Most studies contain missing data at some level. Missing utility data can misrepresent the denominator of the ICER. Despite this, there are no definitive guidelines on how to manage missingness for UK HTA, and analysts rely on judgment to address missingness. We intend for this research to formalise our recommendations for dealing with missing utility data for UK HTA.
METHODS: A simulated individual patient dataset, similar to those used to estimate utility values for a CUA submission for HTA in the UK, was developed. With this dataset, we simulated missingness at various levels and by different mechanisms. We assessed the performance of: complete case analysis (CCA), mean square estimation (MSE), linear mixed modelling (LMM) and multiple imputation via chained equations (MICE), and used this to make recommendations for handling and reporting of missing data in IPD that will be submitted to decision-makers in the UK.
RESULTS: Regardless of the mechanism or the level of missingness, MICE and LMM always resulted in substantially less error when calculating health state utility. Where data were missing at 30%, CCA and MSE were often associated with a substantially higher mean difference than MICE and LMM. The standard deviation was always substantially depressed regardless of mechanism or level of missingness when LMM was used.
CONCLUSIONS: It is our recommendation that an assessment of the mechanism and magnitude of missingness be made and reported for all data sets that are used to inform economic models for HTA in the UK as a minimum standard. Further, we recommend that CCA is almost never appropriate and our research, echoed by others supports the use of MICE as standard.
Individualizing Patient Outcomes
Digital Conference Pass
This session explores approaches to individualizing patient outcomes. Presentations will cover the standardized implementation of Goal Attainment Scaling (GAS) using inventory-facilitated methods, the development of GAS for specific conditions such as pediatric autism, stakeholder perspectives on presenting patient-reported outcomes in clinical practice, and methods for defining clinically meaningful changes in patient-centered measures. Together, these talks highlight creating, implementing, and interpreting individualized outcome measures to enhance patient-centered care and clinical decision-making.
Moderator
Lotte Klim
LEO Pharma, Copenhagen, Denmark
P14: A Mixed-Methods Analysis to Define Minimum Clinically Importance Differences (MCIDs) in Range of Motion, Physical Function, and Worst Stiffness in Patients With Tenosynovial Giant Cell Tumor (TGCT)
2:00PM - 2:15PM
Gelhorn H1, Cutts K1, Fikre T1, Han Y1, Tait C2, Zeringo N2, Saunders A2, Harrow B3
1Evidera, Bethesda, MD, USA, 2Deciphera Pharmaceuticals, LLC, Waltham, MA, USA, 3Deciphera Pharmaceuticals, LLC, Burlington, MA, USA
OBJECTIVES: Tenosynovial giant cell tumor (TGCT) is a locally aggressive neoplasm that involves the synovium, bursae, or tendon sheath. Tumor location varies and is often associated with joint destruction, pain, stiffness, and limited range of motion (ROM). The objective of the current analysis was to define a meaningful change in clinical outcome assessments (COAs) measuring active range of motion, physical function and stiffness among patients with TGCT.
METHODS: Qualitative embedded exit interviews were conducted with patients participating in the double-blind MOTION Phase 3 trial (NCT05059262) of vimseltinib vs placebo for TGCT. Patients were asked about meaningful changes in Patient Global Impression of Change (PGIC) and Patient Global Impression of Severity (PGIS) anchors, active ROM, physical function (Patient-Reported Outcomes Measurement Information System (PROMIS) – Physical Function (PF)), and stiffness (Worst Stiffness Numeric Rating Scale (NRS)). Subsequent quantitative anchor (using PGIS and PGIC) and distribution-based analyses of the clinical trial data, informed by the results of the qualitative interviews, were used to triangulate and define meaningful within-patient changes. Analyses were conducted on blinded data prior to trial primary analyses.
RESULTS: A total of 123 patients were randomized in MOTION with 96 (78%) completing an exit interview. The PGIC anchor variables had moderate correlations with changes in the COA measures (range: |0.38 to 0.54|). The data for both the PGIC-PF and PGIC-ROM support that most patients (PGIC-PF: 67%; PGIC-ROM 73%) considered “minimally improved” responses on these questions to represent clinically meaningful changes. Anchor- and distribution-based estimates for PROMIS-PF ranged from 1.45 to 4.9, for active ROM from 6.0 to 16.4, and Worst Stiffness from -2.3 to -0.5.
CONCLUSIONS: Combined evidence from anchor and distribution-based methods supported by patient insights from qualitative interviews informed the final selection of MCIDs of +3-points for PROMIS-PF, +10% for active ROM, and -2-points for Worst Stiffness NRS.
P15: Exploring Stakeholder Perspectives on Presentation of Patient-Reported Outcomes for Individual-Level Decision Making in Clinical Practice: A Qualitative Study
2:15PM - 2:30PM
Kannan S1, Ezendam N2, van Til J3, van Maaren M1, Mikhal J1, Groothuis-Oudshoorn K3
1University of Twente, Enschede, Netherlands, 2Netherlands Comprehensive Cancer Organisation (IKNL), Utrecht, Utrecht, Netherlands, 3University of Twente, Enschede, Overijssel, Netherlands
OBJECTIVES: As healthcare increasingly prioritizes patient-centered care, the use of patient-reported outcome measures (PROMs) to evaluate clinical practice has grown. A new area of research investigates whether PROM outcomes can inform consultations between patients and providers. However, limited evidence exists on how PROM data is presented to clinicians and patients. This study aims to understand current practices in PROM presentation in patient dashboards and explore the main challenges involved in designing these dashboards.
METHODS: We conducted semi-structured interviews with stakeholders involved in PROM dashboard design. The focus was on dashboards that communicate patient outcomes and are intended for use by clinicians and/or patients to inform disease management decisions.
RESULTS: Six dashboards were reviewed, and eight stakeholders were interviewed. Design decisions were predominantly derived from either the stakeholders' assumptions or specific testing with the users.
Analysis of interview data revealed four main themes:
- Graphical data presentation depends on the available data type. Bar charts are preferred for single data points, while line graphs are favored for longitudinal data.
- Stakeholders believe dashboards should be simple to minimize patient burden. This requires careful consideration of whether and when statistical measures and reference scores are helpful for patients.
- Data presentation should support patient comprehension. Highlighting concerning scores, using color shades, visual cues, descriptive labeling, and textual explanations are used to help patients interpret the data.
- Stakeholders feel dashboards should allow personalization, enabling patients to adapt data presentation to their preferences.
CONCLUSIONS: The study identifies key considerations in designing PROM dashboards. However, many current design decisions are based on assumptions about patients’ and clinicians’ needs, wants, and preferences which may not always align with actual user experiences. More research is needed to improve our understanding of how to present PROM data to aid user comprehension and facilitate decision-making.
P16: Standardized Implementation of a Personalized Endpoint: Inventory-Facilitated Goal Attainment Scaling
2:30PM - 2:45PM
Sevinc G1, Nesto S1, Chapman C1, Rockwood K2, Howlett SE2
1Ardea Outcomes, Halifax, NS, Canada, 2Dalhousie University, Halifax, NS, Canada
OBJECTIVES: Goal attainment scaling (GAS) is a clinician-facilitated patient-centric clinical outcome assessment that quantifies the impact of an intervention on personalized goals. Although it has many strengths, such as being highly responsive, inherently clinically meaningful, and non-arbitrary, a lack of standardization in its implementation may challenge its reliability and validity. Here, we utilized a goal inventory to standardize GAS implementation and assessed the acceptability of this approach from the patient/caregiver’s perspective.
METHODS: Twenty-six patient/caregiver dyads (65+ years) participated in goal-setting interviews with four clinicians trained in GAS. Dyads were given a GAS orientation handout (with or without a goal inventory) and asked to set at least one goal. The goal inventory included 37 goals covering multiple domains. GAS acceptability was assessed using multiple-choice surveys (n=10, n=6) and open-ended questions (n=11, n=7) at baseline and follow-up. GAS acceptability was scored, with higher scores representing greater acceptability. Data were analyzed using Mann-Whitney tests, and open-ended responses were analyzed thematically.
RESULTS: Participants set a total of 71 goals [range=1-4]. There was no statistically significant difference between the number of goals set between participants who used an inventory (x̄=2.9) and those who did not (x̄=2.5; p=0.13) or between the acceptability of inventory-facilitated (1.15±0.25; 1.27±0.34) and standard GAS (0.96±0.24; 1.42±0.33) at baseline (p=0.12) or follow-up (p=0.35) respectively. Setting treatment goals resulted in a better-than-usual relationship with the clinician independent of goal inventory use. An enhanced understanding of the scope of treatment and increased perceptions of being cared for by the clinician were commonly noted, but only for inventory users.
CONCLUSIONS: Utilizing an inventory during goal setting to standardize the implementation of GAS is acceptable. However, future work is needed to assess its impact on the goal type, goal scale quality, and the time taken to develop GAS scales and facilitate its use across the phases of drug development.
P13: Development of a Goal Attainment Scale (GAS) and Global Impression (GI) Items for Use As Outcome Measures in Intervention Studies in Pediatric Autism
1:45PM - 2:00PM
Staunton H1, McIver T1, Tillmann J2, Clinch S1, Hanrahan V3, Ewens B3, Averius C2, Barsdorf AI4, Baranger A5, Berry-Kravis E6, Charman T7, Singer A8, Neerland H5, Ventola P9, Williams ZJ10, Barrett L3
1Roche Products Limited, Welwyn Garden City, UK, 2Hoffmann-La Roche Limited, Basel, Switzerland, 3Modus Outcomes, Cheltenham, UK, 4Clinical Outcomes Solutions, Chicago, IL, USA, 5Autism-Europe, Brussels, Belgium, 6Rush University, Chicago, IL, USA, 7King’s College London, London, UK, 8Autism Science Foundation, Scarsdale, NY, USA, 9Cogstate and Yale Child Study Center, New Haven, CT, USA, 10Vanderbilt University, Nashville, TN, USA
OBJECTIVES: Existing clinical outcome measures of the core features of autism, social communication and interaction, as well as restricted and repetitive behaviours have shortcomings in assessing treatment effects. Goal attainment scaling (GAS) offers the potential to measure individual experiences, while Global Impression (GI) items provide a holistic assessment of treatment benefit. The objective of this study was to develop and cognitively debrief a novel GAS and GI items in paediatric autism.
METHODS: A comprehensive literature review was first completed to understand potential treatment benefits and goals in autism. This was followed by a qualitative study with two waves of interviews. The first ‘Concept Elicitation’ interview round (N=40) included autistic adolescents aged 12-17 years, parents of autistic children aged 5-17 years and clinicians, the purpose was to generate personalised goals and GI items. The second ‘Cognitive Debriefing’ interview round (N=39) was to obtain feedback on a draft GAS manual and GI items. An external expert panel provided input into all aspects of the study.
RESULTS: Twenty-five personalised goals were generated for core characteristics (communication, socialisation, restrictive and repetitive behaviours) and associated characteristics of autism for children and adolescents. Feedback from the cognitive debriefing supported the use of personalised goals. Adolescents/parents found these helpful, and clinicians recommended using these goals in clinical research. Nine GI items were generated for assessing impact/difficulty and change in symptoms; these can be rated by adolescents, parents of children with autism and clinicians.
CONCLUSIONS: As autism is a heterogeneous condition, use of personalised goal measures and tailored GI items may overcome some of the challenges with existing outcome measures. The FDA’s Patient-Focussed Drug Development Guidance-4 highlights the patient focused nature of outcomes like GAS in clinical trials. These findings will inform future measurement strategies for clinical studies in paediatric autism.
Breakouts: IP, WS, & OBS
Co-Creating a Clinical Outcome Assessment (COA) Strategy With Patient Partners: Guidance, Good Practice Methods, and Case Examples
Digital Conference Pass
Level: Intermediate
PURPOSE:
- Review good methods practices in co-creation with patient partners, particularly when developing a clinical outcome assessment (COA)-strategy as part of patient-focussed medical product development (PFMD);
- Review barriers to meaningful and successful co-creation partnerships with patients and possible solutions to overcome those barriers;
- Provide an interactive experience through case-example discussion with online polling; and
- Influence and improve future good practices in COA-strategy development through patient engagement and co-creation partnerships.
DESCRIPTION:
Globally, stakeholders recognise the need for early integration of patient engagement (PE) and active co-creation to achieve the outcomes that matter most to patients to ensure PFMD. COAs, measures describing how a patient feels, functions or survives, are a key component of PFMD. For development to be patient centered, the COA-strategy (i.e., COA identification, implementation, interpretation and communication) should include patients as partners. This workshop will begin by introducing and reviewing disease-agnostic existing co-creation guidance, methods and good practices to produce a COA-strategy co-created with patients. Speakers will then present 2 case examples of patients as partners in COA-strategy development, from a patient expert and regulatory standpoint, respectively. A critical evaluation of each example will be discussed, including:
- Assessment of whether or not good PE and co-creation methods practices were used;
- Identifying barriers encountered to meaningful PE and solutions leveraged, and
- How patient co-creation influenced the strategy.
The audience will participate in the exercise through discussion and real-time online polling. Speakers are experts in PE and COAs from the patient, industry, and regulatory community.
Discussion Leaders
Eleanor Perfetto, PhD, RPh, MS
University of Maryland School of Pharmacy, Baltimore, MD, USA
Eleanor M. Perfetto has worked in government, industry, consulting, and academia. Most recently, Eleanor was with the leading, U.S. nonprofit organization focused on patient issues as Executive Vice President for Strategic Initiatives at the National Health Council (NHC). Currently, she holds a faculty appointment at the University of Maryland School of Pharmacy where she is Professor of Pharmaceutical Health Service Research. Her research and policy work primarily focus on patient and family engagement, including comparative effectiveness and patient centered-outcomes research; patient-focused drug and medical-product development; clinical outcome assessment, selection, and development; and patient-centered value assessment and healthcare quality measurement.
As caregiver for her late spouse who suffered from early dementia due to chronic traumatic encephalopathy, Eleanor is a recognized advocate for patients with head-trauma-related dementia and their families. In May 2007, she received the Alzheimer’s Association New York City Chapter Advocacy Leadership Award. In 2010, she received the Alzheimer’s Association National Capital Area Chapter Award for Advocacy & Awareness. In 2012, she received the Legacy Award from the Concussion Legacy Foundation, for which she is a past board president and a two-term inaugural board member. Today, she serves on the board for the Brain Injury Association of America, where she chairs the Governance Committee and serves as Board Secretary. She also is a current board member for the Dan Lewis Foundation, which is dedicated to brain regeneration research.
Prior to joining the NHC and University faculty, Dr. Perfetto was with Pfizer Inc for almost eight years, most recently as Senior Director, Federal Government Relations; past positions were in Evidence-Based Strategies and Payment Policy Analysis. Early in her career, she served in the U.S. Public Health Service serving for 6 years as a pharmacist in the Indian Health Service in Pine Ridge, SD and Talihina, OK, and as senior pharmacoepidemiologist at the Agency for Health Care Policy & Research (now Agency for Healthcare Research & Quality). Her highest rank was Commander (05).
Dr. Perfetto has a distinguished record of service to professional and other organizations. Currently, she is Chair of the Patient-Centered Special Interest Group at the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) and served as co-chair for the 2016 ISPOR Annual Meeting. In the past, she has served as a CMS MedCAC member; co-chair of the National Quality Forum’s Alzheimer’s Disease and Related Dementias Project and on the Patient-Reported Outcomes Expert Panel; a past Pharmacy Quality Alliance and Drug Information Association board chair; and past member of the Center for Medical Technology Policy Board and Health Industry Council Board of Advisors. She was an Assistant Editor for the Journal of Managed Care and Specialty Pharmacy and has been a peer reviewer for numerous journals, the Patient-Centered Outcomes Research Institute, government granting agencies, and pharmacy professional organizations. She has contributed book chapters and published in peer-reviewed journals. She is a frequent speaker at numerous national and international scientific conferences.
Dr. Perfetto holds BS and MS degrees in pharmacy from the University of Rhode Island and in 2009 received the Distinguished Alumni award from the College of Pharmacy. She also holds a PhD in Public Health, concentrating in health policy and epidemiology, from the University of North Carolina at Chapel Hill.
Discussants
Gunnar Esiason, MBA, MPH
Boomer Esiason Foundation, New Canaan, CT, USA
Gunnar Esiason is a cystic fibrosis and rare disease patient leader. He is the head of patient engagement at RA Capital's venture group, RA Ventures.
Gunnar has been the face fundraising efforts for the Boomer Esiason Foundation, which has yielded close to $200 million raised for the fight against cystic fibrosis since 1993. He was a founding board member of the non-profit No Patient Left Behind, and previously led patient operations at a clinical trial technology startup.
Gunnar has been a leading voice on rare disease market access issues with health policies opinions featured in The Hill, Wall Street Journal, Nature Reviews Drug Discovery, and STAT News.
Gunnar holds an MBA and MPH from Dartmouth College and a BA from Boston College.
Angela Rylands, PhD, CPsychol
Kyowa Kirin International plc, Marlow, BKM, United Kingdom
International Director of Outcomes Research at Kyowa Kirin, responsible for developing and implementing patient-focused outcome research methodologies across product portfolios. Over 15 years experience working with clinical outcome assessments to measure the patient perspective of new and existing medicines or interventions, within the industry and from academic and healthcare provider positions. Completed a post-doctorate in Neuroscience from the University of Oxford, a PhD in Psychology with Neuroscience from the University of Manchester, and a BSc in Psychology from University College London. A registered BPS Chartered Psychologist and a co-chair of the ISPOR task force on using patient-reported outcomes in prospective real world studies.
Ashley Slagle, MS, PhD
Aspen Consulting, LLC, Steamboat Springs, CO, USA
Synthetic Preference Data in Digital Twins for Personalized Healthcare Decisions
Digital Conference Pass
Level: Introductory
PURPOSE: To present the benefits and applications of integrating synthetic preference data into digital twins for enhancing personalized healthcare decision-making. Synthetic data may solve data scarcity, privacy concerns, and evidence transportability. Synthetic data generation methods promise to replicate real-world scenario complexity and representativeness. Researchers can simulate alternative treatment plans and explore resulting outcomes to optimize decision-making by enabling the creation of digital twins. We will explore the innovative use of synthetic data for real-time elicitation of patient preferences in a wide range of decision-making settings. The application of digital twins in the studies of patient behavior and statistical methods available to develop synthetic preference data will be presented.
DESCRIPTION: Workshop attendees will obtain a working knowledge of the fundamentals of digital twins and the application of synthetic preference data. The workshop will review a). the basics of synthetic data, b). methodological advancement needed to develop synthetic data for preference studies c). practical applications of synthetic preference data in the further development of personalized healthcare. Dr Mühlbacher will chair the session, introducing key definitions and contextualizing the applicability of synthetic data in the field of preference studies (10 min). Dr Manca will illustrate the causal inference underpinning the validity of the models that produce predictions to generate synthetic data and make generalizable statements (15 min). Dr Kolasa and Dr Zah will exemplify the benefits of incorporating synthetic patient preferences in personalized healthcare, enhancing medication adherence, and managing chronic diseases through personalized interventions (15 min). The highlight of the workshop is the audience participation, involving the identification of problems and solutions for a hypothetical case study in surgical planning and risk management (20 min). This interactive workshop will be valuable for researchers, clinicians, and industry analysts interested in integrating synthetic data with real-world data for a broader range of decision-making applications.
Discussion Leaders
Vlad Zah, PhD
ZRx Outcomes Research Inc., Mississauga, ON, Canada
Vladimir Zah, PhD, brings 25+ years of evidence generation through HEOR, RWE and decision modeling. Since 2000, in various roles as Health Economist, Project Manager and Chief Investigator, Vlad executed more than 250 evidence based projects of Rx, Dx, MolDx and digital pill, in the product phase 1, 2, 3 and 4 settings, across various disease areas for top 20 global pharmaceutical companies, as well smaller pharma, bio and medical device companies; work supported with over 70 publications.
Discussants
Katarzyna Kolasa, PhD
Kozminski University, Warsaw, Poland
Katarzyna Kolasa PhD
Professor of Health Economics at Kozminski University Warsaw Poland
Digital Health Start Me Up Course leader
ISPOR Digital Health SIG founder
“The Digital Transformation of the Healthcare System” Routledge; 1st edition (July 31, 2023)
Driven with passion to improve healthcare, Katarzyna has focused her academic and busi-ness career on the health economics.
She has been working with multiple pricing & reimbursement challenges worldwide for the last 25 years, while holding various Regional and Global leadership positions at Astra Zeneca, BMS, Biogen Idec, Lundbeck, GE Healthcare, Straub Medical, BD and the Swedish county council of Kalmar. Katarzyna is mentor and consultant to start ups involved in the development of innovative medical devices and digital health solutions from both Holland and Poland.
Since 2000 she has been academic teacher and supervisor for over 30 MBA and Phd stu-dents. In partnership with the Polish Medical Research Agency, Deloitte Digital and the Polish Central Hospital of Ministry of Interior Affairs she founded the first Digital Health six months educational program designed for digital transformation leaders working in the healthcare sector. Katarzyna developed innovative Master Program Health Economics & Big Data (HEBDA) which first edition was financed by EU Power Grant 2018 as well.
She is the founder of the Global Special Interest Group Digital Health and short courses “The Role of Digital Endpoints in the Value Generation for Health Technologies”, “Risk-Sharing/Performance-Based Arrangements in Developing Countries” at International Society of Pharmacoeconomics and Outcomes Research (ISPOR). She is the current member of Edu-cational and post member of Health Science Policy Councils at ISPOR as well.
Katarzyna has dedicated her academic research towards the methodological advancements into the value assessment of pharmaceuticals, medical devices, and digital health solutions. Passionate about Big Data, she led the first project of machine learning adaptation for the optimal utilization of CT scanners granted by the Polish Ministry of Health as well. Since January 2022, she is the leader of AI special interest group at the Polish Chamber of Physi-cians. With the patronage of the Polish Parliamentary Commission for Innovation & Digitali-zation, she organized the first dialog about the societal preferences towards the adoption of AI in the healthcare in Poland.
Being coauthor of more than 50 IF publications, she presented her research at more than 60 international scientific conferences. As of 2022, Google Scholar reports over 730 cita-tions to her work.
Lucy Mosquera, BSc, MSc
Aetion, Ottawa, ON, Canada
Lucy Mosquera was the first employee at the Canadian start up Replica Analytics where she led the development of synthetic data generation algorithms and privacy assessment tools for applications in the health data space. Replica Analytics was acquired by Aetion (an American Real World Data company) in early 2022. Now Lucy is a Senior Director at Aetion, focusing on Data Science and Operations for their synthetic data software group. Lucy is also a research fellow at the Electronic Health Information Laboratory at the University of Ottawa. Her work on synthetic data generation and the assessment of the utility and privacy of synthetic data spans a dozen publications, webinars and conference presentations as well as co-authoring the O'Reilly textbook "Practical Synthetic Data Generation".
Axel Mühlbacher, PhD, MBA
Hochschule Neubrandenburg, Neubrandenburg, Germany
Axel Mühlbacher, Dr. rer. oec., is professor of health economics and health care management at Hochschule Neubrandenburg and adjunct professor in the Duke Department of Population Health Sciences, Duke University (USA). He is a CHPIR Senior Research Fellow in the Center for Health Policy and Inequalities Research at the Duke Global Health Institute. His research focuses on methodological advances in decision analysis, operations research, econometrics and choice experiments and their application in the health domain. Recent work includes stated preferences research, empirical research to understand the behavioral aspects of health and problems of multi-criteria decision making to inform the optimal design of policies and regulatory decision making. Past positions include assistant professor at Technische Universität Berlin and research fellow, German Coordinating Agency of Public Health, Albert-Ludwig Universität Freiburg. Axel Mühlbacher holds a M.Sc. in business administration from Eberhard-Karls Universität (Tübingen) and a Ph.D in economics from the Technical University Berlin. From 2010-11 he was a Harkness Fellow in Health Care Policy and Practice. He served as a member of the scientific advisory board of the Institute for Quality Assurance and Transparency in Healthcare (IQTiG) and is chair of the board of the International Academy of Health Preference Research (IAHPR). He currently serves as co-editor of Value in Health, and on the editorial advisory board of PharmacoEconomics, and The Patient – Patient Centered Outcomes Research. Axel Mühlbacher is author of several books and more than 100 peer-reviewed articles. Global Health Institute, Duke University (USA). He serves as a member of the scientific advisory board of the Institute for Quality Assurance and Transparency in Healthcare (IQTiG) and a board member and chair of the International Academy of Health Preference Research (IAHPR). He currently serves as associate editor of Value in Health and on the editorial advisory board of the Journal of Choice Modelling (JOCM), PharmacoEconomics, Int. Journal of Integrated Care (IJIC) and The Patient – Patient Centered Outcomes Research. Axel Mühlbacher is author of several books and more than 100 peer-reviewed articles.
Enhancing Health Technology Assessment Methods: Navigating Surrogate Endpoints and Associated Statistical Standards
Digital Conference Pass
Level: Intermediate
PURPOSE:
The workshop will introduce the rationale, objectives and preliminary recommendations from the ISPOR Statistical Evaluation of Surrogate Endpoints for HTA Decision Making Good Practices Task Force. The preliminary recommendations will be linked to the recent joint recommendations issued from an international HTA collaboration. The audience will gain a deeper understanding of statistical methods for clinical and economic value demonstration when surrogate endpoints are employed for licensing purposes.
DESCRIPTION:
Those conducting HTA around the globe often have to make access and reimbursement decisions based on surrogate endpoints that have been accepted by regulators. Despite many HTA agencies publishing guidelines on surrogate endpoints, few HTA submissions explicitly present evidence supporting the translation of the treatment effect on early/surrogate endpoints to the treatment effect on late/target outcomes for clinical value and economic value demonstration. Dr. Ciani will introduce the session (6 min). Ms. Kusel will discuss how the taskforce recommendations link to the work published by a collaboration between UK’s NICE, US’s ICER, Canada’s CDA-AMC, the Netherland’s ZIN and Australia’s PBAC on science and methods development for surrogacy (10 minutes). Dr. Bujkiewicz will discuss statistical approaches available for surrogate endpoint evaluation, including considerations for predictions, associated uncertainty and emerging novel tools to tackle less optimal data scenarios to support decision making (12 minutes). Using two existing technical appraisals, Dr. Ouwens will discuss how surrogacy analyses can be used for health economic modeling of relative treatment effects on target outcomes or to support implicit and explicit assumptions for modelled relationships (12 minutes). Dr. Ciani (20 minutes) will open the interactive polling and Q&A session on the task force’s recommendations. This informative workshop is designed for researchers, payers, regulators, and technology assessors, as well as for those working in the pharmaceutical industry or those interested in HTA and market access of health technologies.
Discussion Leaders
Oriana Ciani, PhD
SDA Bocconi School of Management, Milan, MI, Italy
Oriana Ciani is Associate Professor of Practice at SDA Bocconi in Milan. She holds a MSc in Biomedical Engineering from Politecnico di Milano and postgraduate degree in Healthcare Management from Bocconi University. She received her PhD from the University of Exeter with a thesis focusing on the evaluation of surrogate end points. She has been 2020 Fulbright Research Scholar at Yale School of Medicine and Yale School of Public Health. Oriana's research interests are centred on the use of evidence synthesis techniques to inform policy decisions, health technology assessment (HTA) and healthcare policies evaluation.
Discussants
Sylwia Bujkiewicz, PhD, MSc
University of Leicester, Leicester, United Kingdom
Sylwia Bujkiewicz is Professor of Biostatistics in the Biostatistics Research Group at the University of Leicester. She leads a research team working in the area of methods development of Bayesian multi-parameter evidence synthesis for combining data from diverse sources of evidence, including multivariate meta-analysis (of multiple outcomes including surrogate endpoints), network meta-analysis and meta-analysis of data from mixture of study designs. Sylwia is a member of NICE Decision Support Unit and ISPOR Task Force on surrogate endpoints.
Bart Heeg, MSc, PhD
Cytel Inc., Rotterdam, ZH, Netherlands
Bart is Vice President HEOR at CYTEL. Bart has a PhD in health economics. He has over 20 years of experience in heath economic and outcomes consulting. He focusses on indirect treatment comparisons, surrogacy analyses and health economic modelling.
Jeanette Kusel, MSci, MSc
National Institute for Health and Care Excellence, London, United Kingdom
Jeanette has led the scientific advice, now NICE Advice, business unit at NICE since 2018. Jeanette has led many of the digital health initiatives at NICE and spend a period of time as acting Medtech Director. Prior to NICE, she spent 9 years working with consultancy providing HTA and Health Economics services. She has a Masters in Health Economics for Healthcare Professionals at the University of York.
14:45 - 15:15
Coffee and Connect
Head to the exhibit hall to refuel, recharge and connect with fellow attendees and exhibitors over a steaming cup of coffee. Provided by ISPOR
Get Involved—Make the Most of Your ISPOR Membership
Are you an ISPOR member or considering becoming an ISPOR member? Stop by our booth to discover how you can make the most of your member experience! Meet with ISPOR staff to explore exciting opportunities to volunteer, join special interest groups or chapters, and publish your work in ISPOR’s esteemed journals.
14:45 - 15:45
Member Group Meetings
ISPOR Korea Chapter Meeting
The ISPOR Korea Chapter warmly invites all those interested in connecting with us. Established in 2010, the chapter proudly boasts a strong membership base. Join us to explore the key healthcare challenges in Korea and discover potential solutions by benchmarking European cases. We’ll also delve into the latest developments in the HEOR field, offering valuable insights into how these trends are shaping healthcare in Korea.
ISPOR Brazil Chapter Meeting
Join the ISPOR Brazil Chapter as they present and discuss their upcoming projects and activities aimed at advancing HEOR in Brazil. Attendees will gain insight into future collaborations, events, and opportunities for involvement that will shape the direction of HEOR in Brazil. Whether you’re a member or new to the chapter, this session is a valuable chance to engage with leading experts and contribute to the chapter’s exciting projects.
15:00 - 16:00
ISPOR Strategic Plan Session
ISPOR’s New 2030 Strategy: Raising the Global Profile and Impact of HEOR
Level: Introductory
Join us for an exclusive Connection Zone session led by ISPOR executive leadership as we take a deep dive into ISPOR’s new Strategic Plan 2030. During this session, attendees will learn about ISPOR’s bold new vision, mission, and core values, as well as the overarching goals and objectives we will be pursuing over the next 5-6 years. Attendees will be encouraged to engage in an open dialogue and share their ideas about how to implement the plan.
Speaker
Rob Abbott
ISPOR, Lawrenceville, NJ, USA
Mr Abbott is a highly accomplished, visionary leader with more than 20 years’ experience providing executive-level strategic and operational leadership in businesses, nongovernmental organizations (NGOs), government environments, as well as professional societies and associations. Most recently, he served as executive director and CEO of Health Technology Assessment International, a global nonprofit society focused on health technology assessment (HTA). He is recognized for his knowledge in HTA, strategy, and stakeholder relations. He has successfully guided a number of organizations through their transformation and growth. He is also a pioneering social responsibility thinker and advisor who has authored 2 books and numerous articles on the nature of business and society. Mr Abbott holds an undergraduate honors degree from the University of Victoria and graduate degrees from the University of Toronto. Additionally, he holds professional designations as both a management consultant and an ICF-accredited coach.
Mitchell Higashi, PhD
ISPOR, Lawrenceville, NJ, USA
Mitch Higashi, PhD, is ISPOR's Associate Chief Science Officer. In this role, Dr. Higashi oversees the development and deployment of ISPOR’s science and content strategies. He also manages the operations of the Society’s member-driven Institutional Council, Special Interest Groups, and HTA Initiatives, to advance initiatives that lead to increased awareness, understanding, and application of HEOR around the world. Dr. Higashi has more than 20 years’ of industry experience leading HEOR and market access functions at the senior executive level. Dr. Higashi earned his PhD in Health Economics and Outcomes Research at the University of Washington. He has been an active member at ISPOR during his career, serving on the Society’s Health Science Policy Council from 2012 – 2017 and most recently as an ISPOR 2023 conference committee co-chair.
Eberechukwu Onukwugha, PhD
University of Maryland School of Pharmacy, Baltimore, MD, USA
Eberechukwu Onukwugha, PhD is a Professor in the Department of Practice, Sciences, and Health Outcomes Research and is the Executive Director of Pharmaceutical Research Computing at the University of Maryland School of Pharmacy. She received a Master of Science in agricultural and applied economics as well as a Doctor of Philosophy in economics (concentration: econometrics) from Virginia Tech. Dr. Onukwugha has approximately 20 years of experience conducting health economics and outcomes research. She examines the costs and health outcomes associated with health-related decisions as well as the institutional and environmental context framing these decisions.
Brian O'Rourke, PhD
RCMS, Ottawa, ON, Canada
Dr. Brian O’Rourke is an independent healthcare advisor. He served as the President and Chief Executive Officer of CADTH from 2009-2020, following a distinguished career as a pharmacist and healthcare executive with the Canadian military. He also served as the Board Chair for the International Network of Agencies for Health Technology Assessment (INAHTA) from 2014 to 2018. With over 40 years of experience in healthcare, Dr. O’Rourke is a leading expert in the science and practice of health technology assessment (HTA). He has a Bachelor of Science in Pharmacy from Dalhousie University and a Doctor of Pharmacy from the University of Toronto. Following his retirement from CADTH, Dr. O’Rourke continues to play an active role in the global healthcare and HTA communities. He is Chair of the HTA Steering Committee and a member of the Scientific Advisory Council at the Centre for Innovation in Regulatory Science (CIRS). He also served as Chair of the Health Technology Assessment Council at the Professional Society for Health Economics and Outcomes Research (ISPOR) from 2019-2022 and as the 2023-2024 President of ISPOR. In November 2020, Dr. O’Rourke was appointed Colonel Commandant (Honorary) of the Royal Canadian Medical Service, and in November 2022, he was appointed as an Emeritus member of the Canadian Health Leadership Network. Dr. O’Rourke was the 2023 recipient of the Dr. Jill M. Sanders award of excellence in health technology assessment.
Laura Pizzi, PharmD, MPH
ISPOR, Lawrence Township, NJ, USA
Dr. Laura Pizzi is Chief Science Officer for ISPOR and Research Professor at Rutgers University. For nearly 25 years, she has led interdisciplinary teams of methodologists, statisticians, and clinicians to design and conduct economic and outcomes analyses on healthcare interventions and is a frequent author, speaker, and mentor on the topic.
At ISPOR, she leads the organization’s scientific strategy and initiatives in collaboration with ISPOR's global members and stakeholders.
15:15 - 15:45
Exhibit Hall Theater
Enabling Innovative Research With Complete EHR Data, Including Notes, Images and Mother-Child Pairs
Digital Conference Pass
Truveta Data is comprised of complete EHR data – including notes and images – linked with SDOH, mortality, and claims data for more than 100 million patients. This level of clinical depth – made available to researchers in a clean, research-ready, and daily-updated format – is unlocking novel insights related to safety, HEOR and market access, clinical trials, care quality, and more. Dive into recent research showcasing the value of these data and their potential to accelerate therapy approval and adoption and improve patient care.
Sponsor: Truveta
Sponsor
Truveta
Speaker
Ryan Ahern, MD
Truveta, Bellevue, WA, USA
Dr. Ryan Ahern is Co-founder and Chief Medical Officer of Truveta and has more than a decade of experience in healthcare data and clinical research. At Truveta, Ryan leads the Life Science Partner team which focuses on developing new partnerships and ensuring those partners are successful in their research. He is currently a Clinical Assistant Professor of Medicine at the University of Washington School of Medicine and practices at Harborview Medical Center, Seattle’s large county hospital. Prior to this, he trained in internal medicine at the Massachusetts General Hospital and has served as a Clinical Assistant Professor of Medicine at Weill Cornell Medical Center and New York Presbyterian.
15:15 - 16:15
Educational Symposia
Ready for 2025? How Lessons Learned From National System Reforms Can Guide Our Response to the New EU HTA Regulations
Digital Conference Pass
Anticipating the imminent launch of the joint clinical assessment (JCA) in January 2025, the healthcare industry in Europe is bracing for a transformative shift in how innovations are evaluated and approved.
In light of the recent reforms in key European countries such as Germany, Spain, France and Poland, our panel of experts will delve into the evolving landscape.
Join us as we unravel the essential healthcare system requirements and policies crucial for accelerating patient access to groundbreaking medicines.
What attendees will come away with:
- Fresh insights into how Germany, Spain, France and Poland have reformed their HTA processes and the impact of these reforms on healthcare access, innovation, and pricing
- Strategies to align national systems with EU regulations to ensure smooth implementation
- Insights on how national reforms can act as a blueprint for adapting to these evolving challenges and leveraging opportunities for improving patient outcomes
Sponsor: Cencora
Sponsor
Cencora
Moderators
Herbert Altmann, PhD
Cencora, England, United Kingdom
Since Feb 2023 Herbert has taken the position as VP Market Access & Healthcare Consulting Europe for Pharmalex/Cencora. His objective is to successfully engage with Pharma and BioTech companies, bringing innovative medicines to patients with unmet medical need as fast as possible.
After leaving Munich University with a PhD in Chemistry (thesis: Gentechnology), Herbert filed a patent and co-founded a Biotech company. The following 10 years he worked for 3M Innovation and Amgen in different international/global leadership roles and the last 17 years at Novartis - last 10 years as member of the European Executive Committee in the role of Head of Value & Access Europe.
Panelists
Max Brosa, MBA
Self-Employed, Sant Vicenç de Montalt, B, Spain
Max Brosa holds a Master’s in Business Administration from EADA and is a recognized specialist in health economics and market access. Since 1989, he has conducted research and consulting across various public and private organizations within these fields. In 2005, he founded Oblikue Consulting, a market access consulting firm, which he led until its integration into CencoraPharmalex in 2021, subsequently managing the Spanish affiliate until July 2024. He has authored over 50 publications on the economic evaluation of health interventions and multiple facets of market access. Currently, he serves as an independent consultant specializing in market access for the Spanish healthcare sector.
Iga Lipska, MD, PhD, MPH
Health Policy Institute, Medical University of Gdańsk, Gdańsk, Poland
Iga Lipska is a Medical Doctor by education with Master of Science in public health. She graduated from the University of Utrecht in the Netherlands, where she defended her PhD thesis on health technology assessment of new medicines.
Iga has broad international and Polish experience in the healthcare sector, with a main focus on HTA at national and hospital level, drug pricing and reimbursement, health insurance, public health and evidence-based decision making process in health care.
She contributed her efforts significantly to the role of HTA in evidence based decision making processes on pharmaceutical reimbursement in Poland. She played a major role in HTA capacity building in Poland supporting the establishment of Polish national HTA Agency.
She also contributed her leadership and networking skills to the implementation of HTA in Central Eastern Europe and internationally advising in e.g. Croatia, Moldovia, Bulgaria, Turkey.
She served in variety of managerial roles in public and private sector supporting evidence based decision making in health care in Poland and abroad. She was the Director of Drug Policy and Pharmacy Department at Ministry of Health, the Director of HTA Department at Polish HTA Agency and the Director of Control Department at National Health Fund (public payer in Poland). She negotiated pharmaceutical reimbursement prices and Risk Sharing Schemes on behalf of Minister of Health while being the Economic Committee Member. She supported the establishment of global HTA benchmarking program at Center for Innovation in Regulatory Science in London, UK.
She has been co-chairing ISPOR HTA Roundtable Europe for 3 years’ term (from 2019 to the end of 2022).
Starting June 2022 she has been co-chairing HB-HTA Interest Group at Health Technology Assessment international for 3 years’ term.
Recently she has been affiliated to Medical University of Gdańsk which is also her Alma Mater.
She has just been nominated the Chairwoman of the Board at Health Policy Institute in Poland with her main role to contribute to evidence based policy in health care and build capacity in that area based on her international and national experience.
François Meyer, MD
FIPRA Public Affairs, Paris, France
François Meyer spent 10 years as a physician in the teaching hospitals of Montpellier, France, followed by 5 years in R&D for a Swiss pharmaceutical company. In 1997, he joined the French Medicines Agency as Deputy Director of the Drug Evaluation Department. In 2005, he was appointed Director of the HTA Department of the Haute Autorité de Santé (HAS) and, in 2011, Advisor to the President of the HAS for International Affairs. He was a member of the Board of Directors of HTAi and has played a leading role in European HTA cooperation since 2006. Since 2023, Dr Meyer has been an independent consultant and special advisor to the RWE4Decisions project coordinated by FIPRA, a Brussels-based public affairs agency.
Mihai Rotaru, MSc
European Federation of Pharmaceutical Industries and Associations, Brussels, Belgium
Mihai Rotaru works as a Director Market Access at EFPIA (European Federation of Pharmaceutical Industries and Associations) where he is currently responsible for overall industry engagement in the implementation of the HTA Regulation at EU level. In addition, Mihai is also responsible for oncology policy working with the EFPIA Oncology Platform to drive industry engagement at EU and national levels, biotherapeutics as well as various supply chain issues (Falsified Medicines Directive, shortages and anticounterfeiting efforts).
Oriol Solà-Morales, MD, PhD
Health Innovation Technology Transfer, Barcelona, B, Spain
Putting Breast Cancer Into a Societal Perspective: How Can We Account for Its Full Impact in HTA?
Digital Conference Pass
Health Technology Assessment (HTA) can adopt different ‘perspectives’ on the appropriate scope of a value appraisal, from a relatively narrow ‘payer’ perspective, considering direct impacts on patients and the healthcare system, to a much broader ‘societal’ perspective that can include direct and indirect impacts on patients, their carers and families, employers, government departments, and society as a whole. Different HTA bodies adopt different perspectives, with some bodies taking a much broader perspective – and accounting for more aspects of value – than others.
This session will demonstrate the importance of recognising value drivers beyond health-related quality-of-life and direct medical costs for diseases with substantial 'spillover effects', using breast cancer as a case study. The efficacy of current breast cancer treatments in terms of survival means that payers see relatively low unmet needs around the treatment of breast cancer. However, there are important value elements in breast cancer that receive limited recognition under a narrow 'payer perspective' on value. A broader perspective would better account for the full value of more efficacious and more tolerable treatments. These broader value elements can relate to spillovers around family effects, informal caregiving, productivity, and fiscal impacts on society. Recognition of this value would appropriately incentivise innovation that can produce benefits across society.
Sponsor: Office of Health Economics
Sponsor
Office of Health Economics
Moderators
Chris Skedgel, PhD
Office of Health Economics, London, United Kingdom
Chris Skedgel is a Director at the Office of Health Economics and has over 25 years of applied health economics experience in consulting and academia. He has made methodological and applied contributions to using public preferences to promote health equity and maximise the societal value of healthcare. He sits on the editorial board of Pharmacoeconomics and has been a Lead Economic Reviewer for the pan Canadian Oncology Drug Review and the Canadian Common Drug Review.
Panelists
Conchi Biurrun, MA
Federación Española de Cáncer de Mama, Santander, Spain
CONCHI BIURRUN
Cbiurrun57@gmail.com
Born in Pamplona, Spain in 1957
Master degree in Marketing and International Trade
Languages: Spanish, French, English and Italian
Working experience International trade specialist at the Navarra Chamber of Commerce and Industry from 1978 till 2014
Working status: retired
Breast cancer survivor since 2010.
Patient advocate since 2013
Volunteer and treasurer at Federación Española de Cáncer de Mama (Spanish Federation of Breast Cancer Association, FECMA).
Member of BIG PPI (Breast International Group, Patient Partnership Initiative)
Expert patient homologated by EMA
EORTC patient collaborator
Member of Patient Advisory Group of OPTIMA European Project
Member of Oncology Patient Advisory Group of H2O (Health Observatory Outcome)
Member of different Patient Advisory Board’s (Roche, Pfizer, Novartis, Pierre Fabré, Menarini Steamline, Astra Zeneca
Participation as speaker in a round table on Liquid biopsy from a patient’s perspective at ENA in September 2022 in Barcelona.
Attendance to different Congress and Conferences, UICC World Cancer Congress, ESMO, ESMO BREAST, EBCC, etc.
Meindert Boysen, PharmD, MSc
Boysen Consulting International, London, England, United Kingdom
Meindert was responsible at NICE for international partnership working, developing NICE’s role in international fora and representing NICE at key international meetings, further sustaining and developing NICE’s international reputation. Until recently, Meindert was the director at NICE responsible for delivery of the programmes that together form the centre for health technology evaluation. Including the technology appraisals, highly specialised technologies, medical technologies evaluation, diagnostic assessment and interventional procedures programmes. After qualifying as a pharmacist from Utrecht University, he worked in a hospital in the Netherlands, and held positions in health outcomes and sales in the pharmaceutical industry. Meindert completed the MSc in Health Policy Planning and Financing at the London School of Hygiene and Tropical Medicine and the London School of Economics & Political Sciences and briefly worked for the King’s Fund before starting at NICE in 2004. Meindert was a member of the Board of NICE and a director of the board of the International Society for Pharmacoeconomics and Outcomes Research (2017-2020).
Antonio Llombart Cussac, MD, PhD
Arnau de Vilanova Hospital, Valencia, Spain
Melanie Whittington, PhD, MS
Tufts Medical Center (CEVR), Boston, MA, USA
Dr. Melanie Whittington is the Managing Director and Head of the Leerink Center for Pharmacoeconomics where she leads pharmacoeconomic evaluations of in-development and recently approved pharmaceuticals. She is also a Senior Fellow at the Center for the Evaluation of Value and Risk in Health (CEVR) at Tufts Medical Center where she works to develop novel methods for value assessment. She was previously the Director of Health Economics for the Institute for Clinical Economic Review (ICER) where she led the cost-effectiveness analyses and budget impact analyses within ICER reviews. She is a leader in the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) and was the recipient of the ISPOR Bernie J. O’Brien New Investigator Award. She has a PhD in Health Services Research with a concentration in Health Economics and Outcomes Research.
ISPOR Forums
Landing HTA Regulation: Insights Into European Policies and Implementation in Southern Europe – Cases of Spain and Italy
Digital Conference Pass
The Health Technology Assessment (HTA) Regulation aims to enhance the availability of innovative health technologies, such as medicines and certain medical devices, for patients across the European Union (EU). Additionally, it seeks to ensure the efficient use of resources and strengthen the quality of HTA throughout the Union. HTA provides comprehensive information on the clinical, economic, social, and ethical aspects of health technology usage, aiding Member States in making evidence-based decisions. This ensures healthcare remains accessible, sustainable, and of high quality.
In this forum, we will review the current status of the regulation’s implementation and provide a detailed explanation of the European regulation. Join us to explore the progress and challenges faced by individual Member States and understand how these regulations are shaping healthcare delivery across the EU.
Moderators
Paloma González García, MSc
Edwards Lifesciences, Valencia, Valencia, Spain
Speakers
Juan Antonio Blasco Amaro, MD, MSc
AETSA (Andalucian Public Foundation Progress and Health- FPS, Sevilla, Spain
Juan Antonio Blasco MD, MPH, Master in Health Economics and in international HTA; is coordinator of an HTA Agency in Andalucia, AETSA, and Member of the Spanish Network of HTA Agencies, RedETS and the former European Network for HTA (EUnetHTA) and member of the JSC Subgroup in the frame of the HTAR.
Marco Marchetti, MD, PhD
National Agency for Regional Services (AGENAS), Roma, Italy
- Director of the HTA Unit, National Agency for Regional Services / Agenzia Nazionale per i Servizi Sanitari Regionali (AGENAS)
- Co-chair of the Member State Coordination Group on Health Technology Assessment (HTACG) for the implementation of the EU HTAR
- Former Director of the National Center for Health Technology Assessment, National Institute of Health / Istituto Superiore di Sanità (ISS)
- Former Director of the HTA Unit (UVT), IRCCS – Sacro Cuore University
Sonia Pulido Sanchez, PharmD, MBA
AEMPS, Madrid, M, Spain
Sonia Pulido Sanchez holds a Bachelor of Science Degree in Pharmacy from Complutense University of Madrid, a Masters degree in Business Administration in the Pharmaceutical and Biotechnological Industry from Talento-EPHOS Business School and a Diploma in Health Technology Assessment from UNED University.
She started her career in clinical operations, focused in observational studies in neurology, phase II and III clinical trials for oncology and ophthalmology, among others. She was also an early adopter of Risk Based Monitoring strategies.
She then moved into the Health Technology Assessment area at AEMPS, as a Project Manager for national HTA reports. She was actively involved in EUnetHTA 21 and later in the Joint Scientific Consultations Subgroup.
She is currently the Co-Chair of the Joint Scientific Consultations Subgroup under the HTA Regulation working towards the successful implementation of the EU HTAR.
Data Analysis and Real-World Interrogation Network – European Union (DARWIN EU®) – Where Are We Now and What Is in Store for the Future?
Digital Conference Pass
The European Medicines Agency (EMA) and the European Medicines Regulatory Network established a coordination center (DARWIN EU) to provide timely and reliable evidence on the use, safety, and effectiveness of of medicines from real world healthcare databases across the European Union (EU) to inform decisions across a medicine’s lifecycle. DARWIN EU has been working on pilot projects like European Health Data Space (EHDS) HealthData@EU Pilot which will run until September 2024 and is led by the French Health Data Hub (HDH). Pilot projects, use cases, and other developments within DARWIN EU have a significant impact on the future of medicines development, lifecycle management and access. Thus, it is critically important to understand the implications of DARWIN EU from different perspectives. This forum will provide an update on what has been done so far and what is in store for the future, discussing the implications of DARWIN EU for different stakeholders from a regulatory (Dr. Verpillat), industry (Dr. Sen), and maintenance perspective.
Moderators
Phani Veeranki, MD, DrPH
Optum LifeSciences, CYPRESS, TX, USA
Dr. Phani Veeranki is a physician-scientist with more than 10 years of experience in clinical epidemiology, policy analysis and economic valuations to demonstrate the value of health care interventions.
Dr. Veeranki has served as an advisor and investigator for numerous clinical trials, several cohort studies and cross-sectional surveys, along with hospital-based quality improvement projects.
He is a thought leader in conducting interventional and observational research using real-world data and offers innovative clinical solutions, research approaches and strategies to clients and internal partners. He has deep knowledge and expertise in methodological approaches to address client needs in multiple therapeutic areas including respiratory, oncology, cardiovascular, neurology, psychiatry and rare/genetic diseases.
Additionally, he is an expert in retrospective and prospective cohort studies, clinical trials, medical chart review studies, preference-elicitation methods, expert-elicitation methods and health care economic models.
He has authored or coauthored more than 80 peer-reviewed publications and over 100 conference abstracts or presentations. His work has been published in leading journals including Journal of the American Medical Association, CHEST, BMJ and American Journal of Public Health.
In his current role with Optum life sciences, Dr. Veeranki serves a thought leader and provides scientific consultation on several client-funded projects and internal projects.
Before joining Optum, he held research positions in both academia and the life sciences industry. He holds both a doctorate and a Doctor of Public Health in epidemiology, and completed a fellowship in health outcomes research from Vanderbilt University Medical Center.
Speakers
Daniel Alhambra, MD, PhD
Oxford University, London, OXF, United Kingdom
Professor Dani Prieto-Alhambra (MD, MSc (Oxon), PhD) is a clinician scientist with a long track record in pharmaco- and device epidemiology and real world evidence research.
He leads the Health Data Sciences team at the Botnar Research Centre (University of Oxford) and works also as Professor of Real World Evidence at the Department of Medical Informatics, Erasmus MC University.
He recently took the role of Deputy Director for the Data Analysis and Real World Interrogation Network (DARWIN EU®)
Shuvayu Sen, PhD
Merck & Co., Inc., Kenilworth, NJ, USA
Patrice Verpillat, MD, MPH, PhD
European Medicines Agency, Amsterdam, NH, Netherlands
Dr. Patrice Verpillat (MD, MPH, PhD) is the Head of the Real World Evidence (RWE) Workstream at the European Medicines Agency (EMA). He is a medical doctor, specialist in epidemiology. Before joining the EMA, he has worked during 20 years in the pharmaceutical industry where he had positions in several international companies, always dealing with real world data (RWD) and non-interventional studies (NIS) in order to bring RWE into research, access and life-cycle product management.
Dr. Verpillat has published over 70 articles in Medline referenced journals. He has been involved in many organisations such as ENCePP, ICH M14 working group, European pharma association (efpia) and ISPE
Current Regulations and Best Practices for Defining, Measuring, and Reporting Medication Adherence in Clinical Trials
Digital Conference Pass
Medication non-adherence poses a significant challenge in clinical trials, leading to biased results, compromised study outcomes, and increased costs. This forum, organized by the ISPOR Medication Adherence and Persistence Special Interest Group, will offer a comprehensive overview of current guidelines from the European Medicines Agency (EMA) regulatory authority and health technology assessment (HTA) bodies. It will also cover best practices for defining, measuring, and reporting medication adherence in clinical trials.
The forum will begin by highlighting the importance of medication adherence in clinical trials, discussing the critical role it plays and the consequences of neglecting this issue. It will then provide an overview of the current regulations from the EMA and leading HTA bodies in the EU regarding approaches to considering medication adherence in clinical research. To offer practical guidance, the forum will introduce the ABC Taxonomy for Medication Adherence, which includes recommendations on defining medication adherence. Additionally, the Timelines-Events-Objectives-Sources (TEOS) Framework will be presented to guide the operationalization of medication adherence concepts in clinical research. The discussion will then shift to promoting transparent reporting methods, based on the International Society for Medication Adherence (ESPACOMP) Medication Adherence Reporting Guideline (EMERGE), to ensure clarity and accuracy in adherence data reporting.
The forum will utilize case studies to highlight common problems associated with medication non-adherence in clinical trials and provide examples of successful strategies to address these issues. Real-time polling will be used throughout the forum to facilitate interactive audience discussions on the challenges of medication adherence in clinical research. Participants will be encouraged to share their experiences and perspectives. The concluding segment of the forum will allow time for wrap-up and provide opportunities for questions and answers. This will enable attendees to clarify any doubts and reinforce their understanding of the key points discussed.
Moderators
Mickaël Hiligsmann, PhD
Maastricht University, Maastricht, LI, Netherlands
Dr Mickaël Hiligsmann is Associate Professor in Health Economics and Health Technology Assessment (HTA) at the Department of Health Services Research, CAPHRI Care and Public Health Research Institute, Maastricht University. As a researcher, he has more than 15 years of experience in health economic evaluation including cost-effectiveness analyses, decision-analytic modelling, valuation of health care (e.g. discrete-choice experiments, best-worst scaling) and medication adherence.
Speakers
Tamas Agh, MD, PhD
Syreon Research Institute, Budapest, PE, Hungary
Tamás Ágh graduated from the Semmelweis Medical University (MD, 2006), the Corvinus University of Budapest (MSc Physician-Economist, 2010), the Semmelweis University School of PhD Studies (PhD in Pharmacoeconomics, 2013) and the University of Pecs Doctoral School of Health Sciences (DrHabil in Health Sciences, 2020). He specialized in family medicine in 2010. Dr. Ágh is a director of strategy at the Syreon Research Institute and has been practicing as a medical doctor since 2006. He is also a research associate professor and the head of the Medication Adherence Research Group at the Center for Health Technology Assessment and Pharmacoeconomic Research at the University of Pécs. With a professional background spanning over 15 years, his areas of specialization in health economics and outcome research including medication adherence, patient-reported outcomes, health technology assessment and evidence synthesis. He serves as the chair-elect of the ESPACOMP International Society of Medication Adherence and the past-chair of the ISPOR Medication Adherence and Persistence Special Interest Group.
Bijan Borah, PhD
Mayo Clinic College of Medicine and Science, Rochester, MN, USA
Dr. Bijan Borah is a Professor of Health Services Research at the Mayo Clinic College of Medicine and Science. He is also the Endowed Scientific Director of the Clinical and Economic Evaluation Program within the Kern Center for the Science of Health Care Delivery at Mayo Clinic. He is also a leadership team member in the Special Interest Group for Medication Adherence and Persistence within ISPOR.
Lina Eliasson, PhD, CPsychol
Sprout Health Solutions, Pinner, LON, United Kingdom
I specialise in understanding, measuring and improving treatment adherence and in identification and validation of COAs for clinical trials. Following the completion of my PhD from UCL School of Pharmacy on adherence to oral oncology drugs, I worked as a post-doc at Imperial College London. I then joined an agency that specialised in treatment adherence where I oversaw the development of patient support programs. Before founding Sprout in 2017, I led the European COA team for one of the world’s largest CROs. Sprout provides patient engagement services throughout the drug lifecycle.
15:45 - 16:15
Exhibit Hall Theater
Generative AI: The Next Frontier in Health Economic Model Conceptualization
Digital Conference Pass
Health economic models (HEMs) are indispensable tools for assessing the cost-effectiveness of healthcare interventions. These models provide critical insights into the relative costs and benefits of new health technologies compared to existing alternatives, thereby informing health policy decisions, reimbursement strategies, and market access for pharmaceuticals and medical devices. However, as healthcare pathways become increasingly complex, the traditional methods for developing HEMs are being challenged. In this context, there is a growing need for innovative approaches that can not only handle this complexity but also uncover unseen patterns and generate novel solutions that add significant value.
In recent years, advances in artificial intelligence (AI), particularly with large language models (LLMs) such as the Generative Pre-trained Transformer 4 (GPT-4), have shown potential to revolutionize various domains by automating complex cognitive and reasoning tasks. The aim of this session is to discuss how LLMs can be utilised in HEOR reasoning problems, specifically related to conceptualising the natural history of the disease and recommending potential model structures.
The session will cover the following topics:
- Overview of human way of model conceptualisation for de novo models
- AI integration frameworks and approaches (such as human in loop, AI in loop)
- Reasoning algorithms for LLMs to be utilized for reasoning problems such as model conceptualization (e.g., chain of thoughts, tree of thoughts, graph of thoughts)
- Overview of HEM-XTM – a proprietary tool of ConnectHEOR trained for model conceptualization (using a case study demonstration)
- Further developments and scope of LLMs for reasoning problems in HEOR
Sponsor: ConnectHEOR
Sponsor
ConnectHEOR
Moderators
Shilpi Swami, MSc
ConnectHEOR, London, United Kingdom
Shilpi Swami, a seasoned Health Economics and Outcomes Research (HEOR) expert, currently serves as Vice President, Consulting and HTA Strategy at ConnectHEOR. She is also a Member Engagement Co-Chair at ISPOR Oncology Special Interest Group.
With a comprehensive global track record in leading HTA submissions and market access strategies, Shilpi's diverse experience spans academia, consulting, and biopharma, allowing her to offer strategic insights from multiple perspectives.
Formerly a Research Fellow at the University of York, Shilpi has contributed significantly to public health projects as well.
She remains dedicated to improving healthcare through data-driven insights and evidence-based research.
Speakers
Vladimir Babiy, PhD
Novartis, London, United Kingdom
Vladimir Babiy brings over a decade of experience in HEOR and Market Access across various levels and geographies in industry and consultancy. A pharmacist by training, he currently works at Novartis. His primary interest lies in exploring the synergy of human and artificial intelligences to advance HEOR.
Tushar Srivastava, MSc
ConnectHEOR, London, United Kingdom
Tushar Srivastava is the Director at ConnectHEOR, a leading global consultancy in HEOR and RWE. In his role, Tushar leads a dynamic team committed to pioneering innovative approaches to revolutionize healthcare. His team's focus encompasses health economic modeling, indirect treatment comparisons, RWE, patient preference studies, evidence review, and strategic consulting, supporting decision-making for life science companies, policymakers, and healthcare providers. Prior to his tenure at ConnectHEOR, Tushar amassed a wealth of experience across the pharmaceutical industry, academia, and policy-making, enhancing his multifaceted perspective on complex healthcare challenges. He has extensive experience of working on global pipeline projects with HTA experience of more than 25 countries. Outside of work, Tushar is an avid badminton player, keen non-fiction reader, and dedicated meditation enthusiast.
16:00 - 16:45
Poster Tours
Pricing, Reimbursement, and Access Poster Tour
Posters Featured in this Tour:
PT19 Emerging Trends in the Adoption of Alternative Financing Models for Advanced Therapy Medicinal Products: A Scoping Review
PT20 Impact of Huge-Seller Repricing on Price Trend for Reimbursable Cancer Drugs in Japan
PT21 Promoting Access to Innovative Drugs: A Review of National Drug Price Negotiation in China
PT22 Evaluating Reimbursement Pathway of Digital Health Technologies (DHT) Across Five European Countries
PT23 The Evolving German DiGA Market: Insights From a Claims Data Analysis
PT24 From French Early Access to Reimbursement: A Comparison of Early Access Prices and List Prices
Oncology Poster Tour
Posters Featured in this Tour:
PT13 Cost-Effectiveness Analysis of Stool-Based Colorectal Cancer Screening Tests for Individuals Aged 45 Years
PT14 Economic Evaluation of an eHealth Application Fostering Palliative Care for Cancer Patients in Greece
PT15 Utilization Trends and Access Disparities in CAR T-Cell Therapy: Real-World Insights From an All-Payer US Healthcare Database (2017-2024)
PT16 Economic Influences on Medical Decision-Making in Cancer Medicine: An Analysis of Qualitative and Quantitative Data
PT17 Leveraging External Priors to Address Population Variability in a Network Meta-Analysis: A Case Study in Advanced Esophageal Squamous Cell Carcinoma
PT18 Preferences for and Importance of Early Oncology Trial Endpoints to Canadians With Early-Stage Cancer: An Interim Analysis of a Qualitative Patient Preference Study
Moderator
Min-Hua Jen, PhD
Eli Lilly and Company, Uxbridge, LON, United Kingdom
Dr Min-Hua Jen is currently Senior Director - Real-World & Access analytics at Eli Lilly, leading the International Business team on Market Access/HEOR/Medical affairs statistical support. She has extensive experience applying statistics to clinical research, epidemiology and health economics and outcomes research in academia and industry settings. She is an active member in the PSI/EFSPI HTA Special Interest Group (SIG) and the engagement chair of the ISPOR Oncology SIG. She was trained in Epidemiology and Statistics and obtained her PhD at University of Bristol. Her research interests including indirect treatment comparisons and network meta-analysis; particularly incorporate external data for time to event outcomes, surrogacy analyses, multilevel modelling and health economic modelling.
Special Onsite Join / Renewal Event for ISPOR Membership
Special Onsite Promo! Stop by the ISPOR Booth to join or renew your ISPOR membership! We might just have a surprise for you...
16:00 - 19:00
Poster Session 2
Live
16:15 - 17:15
Member Group Meetings
ISPOR Patient Engagement in HEOR
ISPOR recently held a Patient-Centered Research Summit to provide a global platform to share developments in patient-centered research, patient-engagement methods, and policies that support the incorporation of this evidence into regulatory and value and health technology assessment processes. This Open Meeting will explore how insights from the Summit can address European healthcare challenges and to continue advancing patient-centered research by fostering collaboration between researchers and diverse patient communities. Experts from academia, healthcare, and regulatory bodies will discuss differences in patient engagement between the U.S. and Europe, aiming to tailor strategies to regional needs. The session will identify synergies and enhance patient involvement in healthcare decision-making within HEOR.
ISPOR Nordic Chapter Meeting
Join us for the ISPOR Nordic Chapter meeting, an inclusive event for anyone interested in engaging with this recently established chapter. Representing Denmark, Finland, Greenland, Iceland, Norway, and Sweden, the chapter offers a platform to collaborate across the Nordic region. This is a unique opportunity to learn about the chapter’s initiatives, explore upcoming activities, and discover how you can contribute to its growth and success.
17:00 - 18:00
Breakouts: IP, WS, & OBS
Are the Current Approaches to Accelerated Approval for Oncology Drugs Still Appropriate?
Digital Conference Pass
Level: Intermediate
ISSUE: Global oncology spending is continuously rising. Simultaneously, drugs are increasingly approved based on less comprehensive evidence to ensure timely access, hampering assessments of their added benefit and often resulting in negative ratings. Concerns are growing regarding the financial strain on health systems and misalignment between incentives in the pharmaceutical market and patient interests, i.e. access to beneficial drugs for critical needs.
OVERVIEW: First, Francine Brinkhuis will present findings (~10 minutes) from a BMJ-published study*, co-authored by herself and Wim Goettsch, revealing that many EMA-approved oncology drugs (1995-2020) lack demonstrated added benefit, especially those approved via expedited pathways. These pathways aim to accelerate access to drugs for unmet medical needs but often lead to expensive drugs without substantial clinical benefits. Further, while revenues seemingly align with added benefit, most oncology drugs recover R&D costs within a few years. All this underscores the policy gap between drug approval/reimbursement and patient/societal benefit.
Next, three panelists share their insights (~10 minutes each), moderated by Wim Goettsch:
- Michael Berntgen discusses regulatory incentives for unmet medical needs. Are current incentives adequate to drive innovation for beneficial drugs? How can regulatory processes be adjusted to prioritize patient/societal value?
- Matteo Scarabelli provides an industry perspective. What are the challenges/opportunities faced by industry under the current regulatory/HTA environment? How can industry incentives be aligned with delivering high-value oncology drugs?
- Bettina Ryll focuses on a patient perspective on access to oncology drugs. What changes are needed to ensure that patient needs are better met?
The panel will then engage in a debate including audience participation, fostering a discussion on the challenges of incentivizing drug development for unmet medical needs and concerns about sustainable health systems. The audience will gain a deeper understanding of the challenges/opportunities within regulatory/HTA frameworks, providing key takeaways for more informed decision-making in oncology drug development/reimbursement. *
https://www.bmj.com/content/384/bmj-2023-077391 https://www.bmj.com/content/384/bmj.q511
Moderators
Wim Goettsch, PhD
Utrecht University, Utrecht, Utrecht, Netherlands
Professor Wim Goettsch is a key contributor to European HTA. At Utrecht University he is a Professor of HTA of Pharmaceuticals. He serves as a special HTA advisor to Zorginstituut Nederland (ZIN) and was former director of EUnetHTA JA3 Directorate.
Panelists
Michael Berntgen, PharmD, PhD
European Medicines Agency (EMA), Amsterdam, Netherlands
Michael Berntgen is Head of the Scientific Evidence Generation Department at the European Medicines Agency (EMA), Amsterdam. This department supports the development of medicines to ensure generation of robust and relevant scientific evidence, also in collaboration with other stakeholders (e.g. patients, HTAs). Activities include the provision of scientific advice and methodology qualification, management of the PRIME scheme, support to medicines for the paediatric population and for orphan diseases, as well as provision of expertise and support in translational sciences. Furthermore, Michael coordinates across the Agency the collaboration with HTA bodies and payers, to foster timely access to medicines.
Bettina Ryll, MD, PhD
Melanoma Patient Network Europe, and Past Chair of the Patient Advocates Working Group, European Society for Medical Oncology, Uppsala, Sweden
Dr. Ryll holds a medical degree from the Free University of Berlin, Germany, and a PhD in Biomedical Sciences from University College London, UK. After losing her husband to Melanoma, she founded the Melanoma Patient Network Europe and developed a special interest in patient-centric clinical research and innovation, innovative trial designs and novel drug development concepts. Between 2019 and 2021, she was member of the first EU Cancer Mission Board where her particular interest has been how to best leverage the potential of personalized medicine for patients and society through novel forms of collaboration and the support of health policy and governance. Currently, she works as strategist at SIR, Stockholm School of Economics' Research Institute for Mission Zero Cancer, an innovation ecosystem in health financed by Vinnova, Sweden's innovation agency.
Matteo Scarabelli, PhD
EFPIA - European Federation of Pharmaceutical Industries and Associations, Brussels, Belgium
Matteo Scarabelli is Market Access Associate Director for EFPIA - EU Federation of Pharma Indudries.
After a Phd in Philosophy at the Sorbonne University and a first experience in a EU-focused think tank, he has built his expertise in the pharmaceutical market at Eurordis, the EU organisation of rare disease patients, as responsible for the involvement of patients in Regulatory and HTA procedures in view of access decisions for orphan drugs.
He contributed designing the strategy of rare disease organisations across Europe for the new EU HTA framework, also participating in the genesis and in the inter-institutional negotiations of the HTA Regulation.
Since two years now, at EFPIA, he have been working at the heart of pharmaceutical industry’s efforts to prepare for the new European HTA setting and its implementation into national pricing and reimbursement systems.
Are Existing Measurement Tools Accurately Measuring Health-Related Quality of Life of Informal Caregivers?
Digital Conference Pass
Level: Intermediate
ISSUE: As demands upon healthcare resources increase, informal caregiving has gained greater consideration in determining the impact of health conditions. However, not all informal caregiving is the same. Variation exists within and across health conditions. Three key areas of debate will be discussed. 1) conceptualizing informal caregiver HRQoL for use within economic models. 2) measurement of informal caregiver HRQoL. 3) valuation and incorporation of informal caregiver HRQoL in economic evaluations. This session will reflect upon lived experiences of being an informal caregiver of a person with a rare, progressive life-limiting condition, explore the differences of informal caregiving across the different groups, and consider how to account for these differences when selecting measures and creating economic models to ensure the correct values are captured, and reflect upon whether values attributed to informal caregiving should be the same across health conditions.
OVERVIEW: Fleur Chandler will first offer an overview of the lived experience and variability of being a caregiver (15 minutes), reflecting upon key domains of HRQoL affected. Jill Carlton will discuss the suitability of existing instruments to measure informal caregiver HRQoL, focusing upon content validity and acceptability. She will reflect upon whether societal views of informal caregiver HRQoL impact should differ according to issues such as rare conditions, and/or life-limiting conditions. Alice Biggane will discuss the need for wider conversation and collaboration between sponsors and payers to ensure alignment on how to capture caregiver HRQoL and the impact of potential treatments in health technology assessments and cost-effectiveness analyses, without leaving value behind (10 minutes). Alice Biggane, Jill Carlton and Fleur Chandler will be giving their thoughts on these areas from the different perspectives; industry, academia and from lived experience during a panel discussion lead by Sally Vincent. Sally will also facilitate questions from the audience (20 minutes).
Moderators
Anna Richards, MA
Vitaccess Ltd, London, LON, United Kingdom
Panelists
Alice Biggane, MPH, PhD
Pfizer, Tadworth, SRY, United Kingdom
Alice is a Senior Manger in Outcomes Innovation at Pfizer UK. In this role Alice is focused on ensuring that patient and caregiver perspectives and experiences are identified and measured, for inclusion in UK access and reimbursement strategies across numerous disease areas and patients populations. Alice has presented at several national and international conferences, and published extensively in peer-reviewed journals across a range of issues relating to patient-centered outcomes research.
Jill Carlton, BMedSci, MMedSci, PhD
University of Sheffield, Sheffield, Yorkshire, United Kingdom
Jill is a Professor of Health Outcomes Research at the University of Sheffield, UK. Jill’s research interests lie in measuring and valuing health, primarily through the development of patient reported outcome and preference-based measures. She is a mixed methods researcher who has led projects across numerous health conditions. This includes children and carers, as well as in rare, progressive life-limiting conditions. She has presented at national and international conferences and has published widely in high quality clinical and methodological journals.
Fleur Chandler, MSc
Sanofi and Patient Advisory Board Lead, Duchenne UK, Middlesex, United Kingdom
Fleur is a health economist with a wealth of outcomes research and health technology assessment experience, with over 30 years leading both global and local teams in the pharmaceutical industry. Fleur is especially driven by rare disease, a parent of a teenager with Duchenne Muscular Dystrophy, she is on the advisory board of patient organisation Duchenne UK. Bringing her professional and personal experience together, she conceptualised and leads Project HERCULES, a global collaboration generating gold standard disease level evidence for HTA in Duchenne.
Will the Revision of the EU General Pharmaceutical Legislation Improve Access, Affordability, and Availability of New Medicines? An Interactive "Moot" Style Debate
Digital Conference Pass
Level: Intermediate
ISSUE: Stakeholders from across the healthcare and life science spectrum are watching as the European Parliament and the Member State representatives in the Council debate the legislative proposal of the Commission to substantially review the EU’s legislative framework for pharmaceuticals. The common goal is to ensure better and faster access, availability and affordability of new medicines in all EU Member States. But how well are the needs of patients and the member states’ healthcare systems addressed? Will the proposals and counter-proposals really meet their needs?
OVERVIEW:
- Voices from the key healthcare communities will offer their perspectives on the proposed legislative changes and discuss how they might impact patient access at a national level.
- Opening (12 minutes) : the moderator (Petra Wilson, FTI) will open the proceedings by outlining the case that has arisen and presenting the positions of the European Commission and European Parliament. Following these position statements, the moderator (moot judge) will call for expert evidence to help the audience to vote.
- Expert Evidence from Panelists (3 x 12 minutes):
- The needs of patients (Strammiello – EPF)
- The needs of the rare disease community (Bros-Facer – EURORDIS)
- The needs of the national buyers (Ponten – Sweden)
- The moderator will ask the Jury (audience) if they need additional information before voting - 10 minutes questions and interventions from the floor
- To make this session more interactive, the voting will take place on-line using the mobile phones on the ISPOR platform. On the screen in the room it will be possible to follow the voting, put questions and comments forward and use it as a platform to comment on the presentations and agree/disagree with the promotor and the opposer. This should take place “real-time” in a lively way to imitate the type of debates that would normally take place in a national parliament.
Moderators
Petra Wilson
Health Connect Partners, FTI Consulting, Brussels, Belgium
Petra Wilson is co-founder and managing director of Health Connect Partners, a boutique consultancy which focusses primarily on helping clients understand the European health policy environment for digital health. Petra also acts as a Senior Advisor to FTI Consulting in Brussels, working with the Health Care and Life Sciences team lead by Antoine Miahle. With FTI, Petra engages with a wide range of pharmaceutical and medical technology companies, supporting them in understanding the EU political environment for their products and services. Alongside these roles Petra serves on the WHO’s Digital Health Technical Advisory Group; is a Board Member of the European Association for the Study of Diabetes (EASD), and serves on the Ethics Boards of several EU funded projects.
Petra’s professional experience includes eight years in the European Commission, where she focussed particularly on the use of information society technologies in healthcare; seven years as Senior Director of Connected Health at Cisco, where Petra’s team supported clients in making best use of new communications technologies to drive safer and more efficient access to healthcare. Petra also has deep experience of the health services sector, having worked on both the patient and provider side as CEO of the International Diabetes Federation; as well as experience in academia having been member of the teaching staff of both the law school and medical school of Nottingham University.
Petra holds a Doctorate in Public Health Law from Oxford University, she has British and Belgian nationality, lived in Brussels for over 20 years, now lives in England and works in English, German and French.
Panelists
Maria Cavaller Bellaubi, PharmD
EURORDIS, Barcelona, B, Spain
Maria is part of the Patient Engagement team based in Barcelona and coordinates the Patient Engagement Managers across the organization. She also manages the process of patient engagement in medicines development procedures at the European Medicines Agency (EMA), mainly protocol assistance and the coordination of patient engagement for European initiatives namely IMI-PARADIGM, IMI-c4c, H2020-ERICA and MoCA. She is currently a member of the Therapies Scientific Committee of IRDiRC (International Rare Disease Research Consortium); Patient Advocacy Committe at the European Hematology Association (EHA) and part of the multidisciplinary and cross-functional European Expert Group on Orphan Drug Incentives. Maria is also member of the WHO Novel Medicines Platform WG3 on sustainability of healthcare systems.
Maria is responsible for following the development of orphan medicinal products as an expert on the Committee for Orphan Medicinal Products (COMP) at the EMA. She coordinates the group of high-level EURORDIS representatives/volunteers who sit on the various scientific committees/working parties at the EMA, known as the EURORDIS Therapeutic Action Group, and work on topics linked to therapeutic development, regulatory policy and access to medicines. Maria holds a degree in Pharmacy (PharmD) and a Sc. MBA in Health Economics and Global Market Access. Before joining EURORDIS, Maria has worked at the EMA, in pharmaceutical companies and in community pharmacy.
Johan Pontén, MA
Medicine Evaluation Committee (MEDEV), Stockholm, Sweden
Johan Pontén is Senior Manager of International Affairs at The Dental and Pharmaceutical Benefits Agency in Sweden since 2014. He is since 2021 co-chair of the Medical Evaluation Committee, MEDEV, a network of 22 national authorities from 18 Member States and Norway bringing together all the relevant institutions (national HTA agencies and social health insurers-payers) responsible for the assessment, pricing and reimbursement of medicines in Europe. He has also supported the FINOSE collaboration between Nordic HTA agencies from the start in 2017. He has had a career in the Swedish Public Service and holds a Master in Pedagogic Leadership.
Valentina Strammiello, MA, BA
European Patients’ Forum, Brussels, Belgium
Valentina Strammiello works as Head of Programmes and oversees the EPF project portfolio and Youth Strategy. She represents EPF in HTA-related activities and in the European Health Parliament initiative.
Before joining EPF, Valentina has had a long collaboration with the CIPI Network and has worked as freelance OSINT analyst on Security and Energy issues. Self-trained in website management and web 2.0 applications, she is among the founders of ecpa2.0 (European Center for Public Affairs 2.0), a forum of young professionals. She holds a BA in International Relations and a Master’s Degree in European Studies.
Valuing Our Value: Can We Demonstrate the Return on Investment of HEOR?
Level: Intermediate
ISSUE: After decades of investment in the HEOR function, the biopharma industry appears to have entered a stage in which it is actively questioning what it has gotten in return. To date, little has been done to demonstrate the return on investment (ROI) of spending on HEOR personnel and research activities. This contrasts sharply with other components of biopharma spending, in which measurement of financial impact and calculation of ROI are routine. Do we need to demonstrate the ROI of HEOR? Can we?
OVERVIEW: Since individual health economists first started getting hired by industry in the 1980s, the HEOR function has grown to become a sizable component of the typical biopharma company org structure and annual budget. Core elements of HEOR—economic modeling, patient-reported outcomes, real-world evidence, and the like—have become fundamental for demonstrating product value to health system stakeholders. The beneficial impact of HEOR has largely been taken as a given rather than open to question, amid the common notion that HEOR activities and data are table stakes to successful launch and market uptake of biopharmaceutical products. This issue panel will consider the importance of demonstrating the ROI of HEOR and the difficulties in framing the calculation. Laura Pizzi, the panel moderator will outline key questions for the discussion, including: What is currently known about the ROI of HEOR? How can we formalize ROI calculations for HEOR work? Sissi Pham will provide an overview of current thinking within the biopharma industry on the role and value of the HEOR function. David Thompson will outline alternative approaches to ROI calculation and corresponding estimation challenges. Jens Grueger will provide a management consulting perspective on how HEOR and related functions can be championed within biopharma organizations. The moderator will solicit audience interaction and feedback on the alternative viewpoints.
Moderators
Laura Pizzi, PharmD, MPH
ISPOR, Lawrence Township, NJ, USA
Dr. Laura Pizzi is Chief Science Officer for ISPOR and Research Professor at Rutgers University. For nearly 25 years, she has led interdisciplinary teams of methodologists, statisticians, and clinicians to design and conduct economic and outcomes analyses on healthcare interventions and is a frequent author, speaker, and mentor on the topic.
At ISPOR, she leads the organization’s scientific strategy and initiatives in collaboration with ISPOR's global members and stakeholders.
Panelists
Jens Grueger, PhD
Boston Consulting Group, Zürich, Zürich, Switzerland
Jens Grueger is an Affiliate Professor of Health Economics at the Comparative Health Outcomes, Economics and Policy (CHOICE) Institute at the University of Washington, and Director and Partner at Boston Consulting Group (BCG). As a Senior Expert for Pricing & Access in Healthcare, he works with stakeholders to find innovative ways of accelerating and expanding equitable access for patients to medical innovation in areas of unmet need. His 30 years of experience in the field, and significant experience engaging experts across all areas of expertise, enable him to bring together different stakeholder voices to discuss solutions to challenges.
Sissi V Pham, Pharm.D.
AESARA, Chapel Hill, NC, USA
Sissi Pham is a founder-CEO of AESARA. Prior to AESARA, Sissi provided Value & Access consulting solutions to pharmaceutical, biotechnology and device partners. Sissi began her career with a major pharmaceutical company in the UK and at the end of her tenure she managed the global and health outcomes and market access support function based in the US. She is experienced in designing and implementing frameworks for robust Value & Access strategies, timely and relevant evidence generation plan, and effective communication of Value & Access information.
Sissi has also served on the benefits working group of the Pharmaceutical Research and Manufacturers of America that built a general framework for translating medical benefits into new stakeholder-centric value propositions during drug development. In addition, she has served as the Institutional Council Chair for the International Society of Pharmaceutical Outcomes Research, where she initiated the development of a supplement reviewing trends in health technology assessments (HTA) around the world that was published in Value in Health. This publication has contributed to the comparative effectiveness dialogue in the US and the evolution of HTA.
Sissi has an appointment of Associate Clinical Professor at the College of Pharmacy, University of Arizona. Sissi’s Value & Access experiences have been acquired through her career experiences in pharmacoeconomics fellowship, pharmaceutical industry, and strategic consulting.
David Thompson, PhD
Rubidoux Research LLC, Manchester, MA, USA
Dave is a health economist by training, with 35+ years of experience conducting real-world research and HEOR consulting for clients in the biopharmaceutical sector. He was Editor-in-Chief of the ISPOR publication Value & Outcomes Spotlight from 2008 to 2020 and a member of the ISPOR Board of Directors from 2020 to 2023. In addition, he serves on the advisory boards and belongs to the working groups of various RWE policy organizations. He has presented scores of workshops, issue panels, and educational symposia at the ISPOR Annual, EU, Asia Pacific & Latin America conferences, and he has published more than 50 papers in the peer-reviewed medical literature.
Is the Current EU Joint Clinical Assessment Process Supporting Accelerated Patient Access in the EU or Is It a Further Hurdle?
Level: Intermediate
ISSUE: EU JCA aims at establishing a framework that promotes research, innovation, and more equitable access to products with large unmet medical needs. Process and timelines for submission and review have been recently finalized. While the 27 member states are still finalizing national processes and regulations, the fundamental question remains: is the designed process going to expedite patients’ access in EU?
New medicines generally launch first and faster in US, with a substantive delay between regulatory approval and access varying from 133 to 899 days in Europe. The objective of the panel is to compare the perspectives and approaches of both large and small health technology developers and oncology versus gene therapy (Gergana Zlateva and Francis Pang) in regard to EU JCA since heterogeneity may be expected. Fabrizio Gianfrate will provide the local payer perspective not just on local implementation, but also the trade-off between accelerating access and meeting the needs of all member states.
OVERVIEW: provided by Cristina Masseria. Processes and timelines were designed to guarantee high quality and a voice for all member states, providing an opportunity for raising the EU perspective, maximizing synergies, and eliminating inequity across the EU. However, the maximum number of Population, Intervention, Comparison and Outcomes (PICOs) has not been set and will vary by therapeutic areas. The potential requirements of Indirect Treatment Comparison to meet all the PICO requirements may be substantial in terms of additional RCT statistical analyses and data gathering. The opportunity for the pharmaceutical company to engage with the EU is limited, with a very tight submission timeline. Companies are incentivized to initiate local reimbursement at the same time in all member states following EU JCA complete assessment, removing the possibility for accelerated submission. Moreover, there is risk of losing innovation funding for certain therapeutic areas with implications on pipeline selection.
Moderators
Cristina Masseria, MSc, PhD
AESARA, New York, NY, USA
Panelists
Fabrizio Gianfrate, PhD, MSc
University of Ferrara, Ferrara, Italy
Professor Gianfrate is a Professor of Health Economics and Outcome Research with experience at both the national AIFA P&R committee and at regional HTA and formulary committees and as an ex-director of the Italian Ministry of Health. He has experience evaluating digital data collected for evaluation of pharmaceuticals for diabetes.
Francis Pang, MBA
Orchard Therapeutics Ltd, London, LON, United Kingdom
Francis Pang is Senior Vice President, Global Market Access and International Geographic Expansion at Orchard Therapeutics and has more than 20 years of experience in pricing and reimbursement, market access, health economics and corporate development, encompassing leadership roles at Shire Human Genetic Therapies, Biogen and Amicus Therapeutics. Francis served as a founding member of the NICE Highly Specialised Technologies (HST) Committee as the Healthcare Industry Representative and Economic Lead for 10 years. Francis currently chairs the EU Access and Value Recognition Committee for the Alliance for Regenerative Medicine (ARM). Prior to joining the biopharmaceutical industry, Francis was the inaugural Pharmacoeconomics Research Fellow at the Centre for Health Economics, University of York and Monbusho Scholar at Kyoto University. Francis’s advanced degree background allows him to operate at the intersection of science (University of Cambridge), business administration (INSEAD) and health economics (University of York).
Gergana Zlateva, BA, MPA, PhD
Pfizer Inc., New York, NY, USA
Gergana Zlateva is VP, Global Access Strategy & Pricing, Oncology at Pfizer. Gergana is proud to have helped increase patient access to medicines across the globe throughout her 20-year career with Pfizer and to have worked across multiple therapeutic areas. Gergana holds a PhD in Economics from Fordham University, NY and a BA and MPA from Southern Illinois University, IL. Gergana’s CV includes more than 60 peer-reviewed manuscripts.
Innovation or Stagnation? Unpacking Issues and Solutions in the Uptake of Advanced Methods for Real World Evidence
Digital Conference Pass
Level: Introductory
ISSUE: The implementation of advanced methods in real-world evidence (RWE) generation presents opportunities and challenges from the perspectives of HTA/regulatory submissions and industry stakeholders, with potentially substantial resource requirements, uncertainties, and risks.
From an HTA and regulatory standpoint, RWE faces hurdles stemming from data quality concerns, including bias, small sample sizes, and interpretability. HTA/regulatory bodies demand robust evidence to support decision-making, necessitating transparency, reproducibility, and alignment with established guidelines. To address these challenges, innovative methodologies such as quantitative bias analysis (QBA), target trial emulation, Bayesian borrowing, and transportability have all been proposed, even being cited in official guidelines. However, why has the implementation of these methods in-practice been limited? This issue panel sets the stage for a comprehensive discussion, where experts from HTA/regulatory bodies, industry, and academia will share insights, experiences, and best-practices in navigating the evolving landscape of RWE methodologies. Through these multiple perspectives and collaborative solutions, stakeholders can overcome barriers to the uptake of advanced methods in RWE generation, paving the way for more robust, transparent, and impactful evidence to inform healthcare decision-making.
OVERVIEW: Dr. Duffield begins by describing the current landscape of submissions regarding advanced methods, and the challenges NICE faces in their evaluations (15m). Dr. Dixon will prove an industry perspective on the obstacles related to resource constraints, and challenges with organizational inertia in embracing novel approaches. Prof. Dickerman will highlight the foundational elements needed and hurdles in executing advanced methods such as target trial emulation (20m). Ms. Hsu will discuss the time and resource implications of implementing advanced methods through the medium of past case studies (15m).
By delving into the critical challenges of uptake barriers and potential solutions in leveraging advanced methodologies for RWE through multiple perspectives, this issue panel aims to foster collaboration and innovation in evidence generation.
Moderators
Stephen Duffield, PhD, MD
National Institute for Health and Care Excellence, Liverpool, United Kingdom
Stephen is Associate Director of Real-world methods at NICE. His role involves the continuing development of NICE’s real-world evidence (RWE) framework, collaboration on RWE demonstration projects, and helping to transform NICE’s use of real-world data across guidance products. He is also involved with upskilling individuals within and externally to the organisation, contributing to training workshops and technical forums. Stephen has a degree in medicine and a PhD in public health. Previously, he worked as a clinical doctor and a guideline developer in NICE Centre for Guidelines.
Panelists
Barbra Dickerman, PhD
Harvard T.H. Chan School of Public Health, Cambridge, MA, USA
Barbra Dickerman’s research is focused on using causal inference methods and large health databases to improve health decision-making. She is Assistant Professor of Global Cancer Prevention in the Department of Epidemiology at the Harvard T.H. Chan School of Public Health (HSPH) and affiliated with the Zhu Family Center for Global Cancer Prevention and CAUSALab. She also serves as co-director of the VA-CAUSAL Methods Core, an initiative of the U.S. Veterans Health Administration to integrate high-quality data and explicitly causal methodologies in a nationwide learning health system. She teaches causal inference methodology at HSPH, where she also helps to train junior investigators.
Jonas Häggström, PhD
Cytel, Stockholm, Sweden
Grace Hsu, MSc
Cytel Inc., Waltham, MA, USA
Grace is Director of Real-World Evidence at Cytel providing statistical consulting and guiding project strategy for the application of advanced analytics to clinical and RWE generation. She also develops statistical communications and training for non-statisticians. Examples of her peer-reviewed publications include work on COVID-19, synthetic/external control arm comparative effectiveness analysis, quantitative bias analysis, Bayesian borrowing and other methods for comparative studies for both pharmaceutical research and HTA/regulatory submissions.
Grace holds a MSc in Statistics from SFU and BSc in Mathematics from UBC. Prior to joining Cytel, Grace was a senior statistician at MTEK Sciences (acquired by Cytel in 2019), a boutique analytics firm, where she worked with the Bill and Melinda Gates Foundation (BMGF), providing simulations and adaptive trial design for large-scale BMGF-funded clinical trials and analyzing national-level registries.
How Do We Unleash the Ambition of the EU HTA Regulation Through Practical Methodological Solutions?
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Level: Intermediate
ISSUE: Wide variations in clinical practice and the availability of treatments across the EU will likely result in many PICOs being identified when scoping Joint Clinical Assessments (JCAs). This has been confirmed in multiple PICO simulation exercises including the ones undertaken in the EFPIA and Evidera report. Whilst good-quality direct comparative evidence is optimal, the likely number of PICOs mean this will not fully address the scope of many JCAs underscoring the need to understand the experience and current use of state-of-the-art evidence synthesis methods, and their strengths and limitations to facilitate decision-making in the context of JCAs.
OVERVIEW: EFPIA and Evidera conducted a simulation to explore the impact of the proposed EUnetHTA21 methodologies on several widely reimbursed oncology treatments. The simulation of the scoping process resulted in many potential PICOs for each product, ranging from 16 up to 57 depending on the indication and line of treatment.
Context is key to the use of different evidence synthesis methodologies, the simulation demonstrated the central role indirect treatment comparisons, real-world evidence, single-arm studies, and oncology-relevant endpoints play in country appraisals to help address uncertainty. The simulation found that a wide range of evidence synthesis approaches will be essential to conduct comprehensive JCAs and underlined the importance of considering the totality of evidence or endpoints available at the time of submission. With the publication of methodological guidelines over the next few months, it will be important for assessors and technology developers to understand how best to use state-of-the-art methodologies in JCAs and apply learnings from these previous experiences.
The issue panel will start with a short presentation highlighting the issues identified followed by a structured panel discussion with recommendations and audience questions. This panel will be beneficial to stakeholders involved in evidence generation, HTA, and JCA processes.
Moderators
Martin Parkinson, MRPharmS
Evidera, part of PPD clinical research business, Thermo Fisher Scientific, London, London, United Kingdom
Panelists
Anna Chaimani, PhD
Inserm, Université Paris Cité, Paris, 75, France
Anna is a Senior Researcher at the Institute of Health and Medical Research (Inserm) in Paris and the Leader of the Evidence Synthesis Group of the Center of Research in Epidemiology and Statistics (CRESS). She also holds a position as Associate Professor at the Oslo Center for Biostatistics and Epidemiology (OCBE) of the University of Oslo. She has extensive experience in the field of evidence synthesis with several methodological contributions and has served for several years as a co-convenor of the Cochrane Statistical Methods group and of the Cochrane Comparing Multiple Interventions Methods group. Her latest research interests lie in the development of new methods for ranking interventions, synthesis of data on complex interventions, combination of randomized and non-randomized studies, and development of novel interactive knowledge translation tools.
Niklas Hedberg, MSc
Dental and Pharmaceuticals Benefits Agency (TLV), Stockholm, Sweden
Niklas Hedberg is the Chief Pharmacist
at the Swedish governmental authority,
the Dental and Pharmaceuticals Benefits
Agency (TLV).
Niklas is the co-chair for medicinal products in the Member States Coordinaton Group on HTA.
Niklas was the chair of the EUnetHTA JA3 and EUnetHTA21 Executive Board from 2018 to 2023.
Niklas has been involved in the national
pricing and reimbursement decision
making for pharmaceutical products
since 2001, and for medical devices
since 2009. He has a broad experience in
different aspects of value-based
evaluation.
James Ryan, MSc
AstraZeneca, Cambridge, CAM, United Kingdom
James represents AstraZeneca on regional and global HTA Policy around the world and is a co-lead on EFPIA’s HTA Working Group’s methodology workstream on the EU HTA Regulation. With over 25 years of experience in health economics and payer evidence generation, James has extensive knowledge and experience of making HTA submissions across Europe and beyond. In addition to his policy role, he also leads the oncology global HTA and Modelling Science team at AstraZeneca.
Podium Sessions
Preference Research in Chronic Diseases
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This session showcases preference studies in cancer, hypercholesterolemia, haematology, and HIV.
Moderator
Sophie Van Tomme, PhD
Sanofi, Amsterdam, NH, Netherlands
As Health Value Translation Lead in Sanofi's Patient-Informed Development team, I integrate the Patient Voice into our development programs. We create strategies to generate meaningful Patient Experience Data, such as patient-reported outcomes and preferences. With this approach we aim to support patient-friendly clinical trial design and develop differentiating products that meet patient demands and have higher access probability.
I hold a Pharmacy degree from the University of Ghent and a PhD from Utrecht University. My background includes roles in pharmaceutical product development and project management at various small and mid-size biopharma companies.
P19: Exploring Preferences and Barriers to Lung Cancer Screening Participation: A Mixed-Method Study
5:30PM - 5:45PM
Michiels L1, Duchateau J2, Vangilbergen A2, Dooms C3, Van Hal G4, Huys I5
1Department of Pharmaceutical and Pharmacological Sciences, KU Leuven, Leuven, VBR, Belgium, 2Department of Pharmaceutical and Pharmacological Sciences, KU Leuven, Leuven, Vlaams-Brabant, Belgium, 3Department of Respiratory Diseases and Chronic Diseases and Metabolism, Leuven, Vlaams-Brabant, Belgium, 4Department of Family Medicine and Population Health, University of Antwerp, Antwerp, Belgium, 5Department of Pharmaceutical and Pharmacological Sciences, KU Leuven, Leuven, Flemish-Brabant, Belgium
OBJECTIVES: In Flanders, population screening programs for breast, cervical, and colorectal cancer are well-established. Lung cancer screening has not yet been implemented, despite its potential to significantly reduce lung cancer mortality through low dose computed tomography (LDCT) screening. Policymakers are evaluating whether a comprehensive lung cancer screening program could be introduced soon. To ensure successful implementation, it is crucial to understand the preferences and concerns of eligible participants. This study will identify key factors influencing screening participants' engagement, enabling healthcare professionals (HCPs) to address critical issues and enhance the screening program's overall effectiveness.
METHODS: An umbrella review and semi-structured interviews with eligible lung cancer screening participants (n=15) and HCPs (n=15) in Flanders will be held. Interviews will be transcribed ad verbatim and analyzed using thematic framework analysis. The themes identified from both the umbrella review and interviews will be categorized according to the CFR 2.0 framework [1]. The results will be narratively synthesized.
RESULTS: Preliminary findings from the umbrella review highlight key barriers to lung cancer screening. These include widespread concerns such as fear of diagnosis, concerns about radiation exposure, the financial burden associated with screening procedures, and logistical challenges such as transportation issues. Further insights and detailed results from the ongoing literature and planned interview study will be presented during the ISPOR 2024 congress.
CONCLUSIONS: To establish an effective lung cancer screening program in Flanders, addressing barriers such as fear of diagnosis, radiation exposure concerns, financial limitations, and logistical challenges is crucial. Considering these preferences and concerns can enhance the development of a more effective screening program in Flanders. In a next step, this approach facilitates tailoring screening recommendations to each person's individual needs during shared decision-making counseling visits.
[1] Damschroder LJ, Reardon CM, Widerquist MAO, Lowery J. The updated Consolidated Framework for Implementation Research based on user feedback. Implementation Science 2022;17. https://doi.org/10.1186/s13012-022-01245-0.
P18: Understanding the Challenges Around Implementing PROMs for Patients Receiving Novel Immunotherapies in Hematology
5:15PM - 5:30PM
Duncan E1, Joyner K2, Broekhuizen C3, Kyriakou C4, Nier S5, Oehrlein E6, Oerlemans S3, Reese E6, Morgan K2
1Myeloma Patients Europe, Brussels, Belgium, 2Myeloma Patients Europe, Brussels, Brussels, Belgium, 3Netherlands Comprehensive Cancer Organisation (IKNL), Utrecht, Utrecht, Netherlands, 4University College London Hospitals NHS Trust, London, England, UK, 5Acute Leukemia Advocates Network, Bern, Bern, Switzerland, 6Applied Patient Experience, LLC, Washington, DC, USA
OBJECTIVES: Newer immunotherapies, such as chimeric antigen receptor T-cell therapy (CAR-T) and T-cell engaging bispecific antibodies (BsAbs), offer innovative ways to treat hematological cancers (i.e., myeloma, leukemia, and lymphoma). While these novel immunotherapies offer promising survival outcomes, they are not without potentially serious side effects and impacts on Quality of Life (QoL). There are currently no specific Patient Reported Outcome Measures (PROMs) for reflecting these treatments. This research aimed to understand the challenges around implementing PROMs while patients receive novel immunotherapies. We sought to identify ways to encourage alignment, consistency, and interpretability of data collected using PROMs for novel immunotherapies.
METHODS: In March 2024, we held two two-hour virtual workshops with participants representing the myeloma, leukemia, and lymphoma patient communities, regulators, payers/ HTA bodies, industry, academic researchers, clinicians, and trialists.
RESULTS: Twenty-nine representatives participated across the online workshops. Key takeaways include: 1) PROMs in use today do not capture all symptoms and side effects important to patients and other stakeholders; 2) current measures do not sufficiently capture patients’ short-and long-term novel immunotherapy treatment experiences; 3) while there is no consensus regarding timing and frequency of long-term follow-up, workshop participants thought a reasonable approach to measuring long-term effects could include administering PROMs every 6 or 12 months during long-term follow-up, as well as an annual assessment of role and cognitive function.
CONCLUSIONS: PROMs currently used in studies about novel immunotherapies do not address all patient-important outcomes or treatment-related side effects, which can severely impact patients’ QoL. Current QoL assessment approaches do not address the unique attributes of novel immunotherapies. Assessments should be tailored to understand treatment experiences and patient outcomes. Collaboration between patients, clinicians, researchers, and industry representatives is essential to develop and refine PRO tools and accurately reflect real-world experiences of patients undergoing treatment with novel immunotherapies.
P20: Lipid-Lowering Agent Preferences Among Patients With Hypercholesterolemia: A Focus Group Study
5:45PM - 6:00PM
Yiu HHE1, Deng K2, Fung LW3, Ye X3, Blais JE3, Wei Y4, Lee KJ5, Tse HF3, Wong MCS6, Yan BP6, Wong WCW3, Li X3, Wong CKH3, Wong CK3, Chan EW3
1The University of Hong Kong, Hong Kong, Hong Kong, 2Monash University, Parkville, VIC, Australia, 3The University of Hong Kong, Hong Kong SAR, Hong Kong, 4Laboratory of Data Discovery for Health (D24H), Hong Kong, Hong Kong, 5The University of Hong Kong, Pokfulam, Hong Kong, 6The Chinese University of Hong Kong, Hong Kong SAR, Hong Kong
OBJECTIVES: Non-adherence to lipid-lowering agents presents significant risks to patients and compromises the effectiveness of treatment. This study aims to qualitatively identify the barriers to lipid-lowering medication adherence, understand the considerations of patients with hypercholesterolemia when selecting these agents, and guide the design of a medication preference study.
METHODS: Focus group interviews were conducted with Cantonese-speaking patients diagnosed with hypercholesterolemia in Hong Kong. These participants were recruited via convenience sampling by cardiologists at a university-affiliated hospital. The interviews were structured into three parts: exploring patients' perceptions of their condition and treatment, identifying crucial factors that influence their selection of lipid-lowering agents, and engaging in medication preference tasks designed using the Discrete Choice Experiment (DCE) method. Thematic analysis was applied to organize the codes from the transcripts into broader themes.
RESULTS: Twenty patients took part in the focus group interviews conducted on the university campus between January and March 2023. Four primary themes emerged: issues in medication management, patients' medication preferences, and the structure and understanding of preference tasks. Barriers to adherence included insufficient knowledge, high pill burden, poor communication with healthcare providers, minimal involvement in treatment decisions, limited access to information about medications, side effects, and forgetfulness. Factors influencing medication choice included the treatment regimen, effectiveness, side effects, doctors' recommendations, drug interactions, and out-of-pocket costs. Despite suggestions for altering attributes and levels, the medication preference tasks effectively captured patients' trade-offs.
CONCLUSIONS: Understanding the barriers to medication adherence and the factors that influence medication choice underscores the importance of considering patients' viewpoints. These findings can guide decision-makers in choosing medications that align with patient preferences, ultimately fostering better adherence.
P17: Preferences of People Living With HIV-1 for HIV Treatment Regimen Attributes: A Qualitative Interview Study
5:00PM - 5:15PM
Donatti C1, Ravi M2, Correa SC3, Rodriguez-Leboeuf AM4
1ViiV Healthcare, TWICKENHAM, UK, 2IQVIA, Bangalore, India, 3IQVIA, Barcelona, Catalonia, Spain, 4IQVIA, Madrid, Madrid, Spain
OBJECTIVES: This qualitative interview study described overall preferences of people living with HIV-1 and healthcare providers (HCPs) regarding attributes associated with HIV-1 treatment, particularly long-acting injectable options, and factors influencing these preferences.
METHODS: Individual qualitative interviews with people living with HIV-1 and HCPs in the United States were conducted to assess overall preferences when considering a treatment switch for the following attributes related to HIV-1 treatment previously identified from a targeted literature review: mode of administration, treatment administration setting, treatment administration frequency, injection location on body, and side effects. Influencing factors and spontaneously mentioned attributes were also recorded.
RESULTS: Interviews were conducted with 50 people living with HIV-1 and 5 HCPs (physicians, n=3; nurse practitioner, n=1; physician assistant, n=1). The majority of people living with HIV-1 were male (60%), with no prior injectable experience (62%), and had a mean age of 48.6 (range: 23-70) years. Both groups ranked side effects as highest priority, followed by treatment administration frequency. Highest-ranked spontaneously mentioned attributes among people living with HIV-1 were cost and number of injections. Regarding side effects, people living with HIV-1 were most concerned about weight gain, diabetes, and adverse gastrointestinal effects. Most people living with HIV-1 preferred 4- and/or 6-month intervals for HCP-administered injections (96% and 95%, respectively), citing alignment with appointments and HCP relationships, and 3-month intervals for self-administered injections (94%), considering storage and disposal. Oral administration was preferred by 92% of people living with HIV-1.
CONCLUSIONS: People living with HIV-1 prioritize minimizing side effects and reducing injection frequency when making treatment decisions. A planned quantitative discrete choice experiment will assess generalizability of these findings in a larger sample with broader geographical reach and provide evidence of risk tolerance and perspectives on benefit regarding different HIV-1 treatment modalities for health technology assessment and regulatory bodies.
RWE Methods and Applications
Digital Conference Pass
The session presents current considerations of use of RWE decision making followed by applications and methods of use of RWE in evidence generation.
Moderator
Maciej Niewada, PhD
Medical University of Warsaw, Warszawa, Poland
P24: Innovations in Automated Survival Curve Selection and Reporting of Survival Analyses Through Generative AI
5:45PM - 6:00PM
Wu Y1, Klijn S2, Teitsson S3, Malcolm B4, Jones C5, Rawlinson W6
1Estima Scientific Ltd, South Ruislip, LON, UK, 2Bristol Myers Squibb, Utrecht, ZH, Netherlands, 3Bristol Myers Squibb, Uxbridge, LON, UK, 4Bristol Myers Squibb, Middlesex, LON, UK, 5Estima Scientific Ltd, London, LON, UK, 6Estima Scientific Ltd, London, UK
OBJECTIVES: Survival analyses are a core part of many HTA submissions where extrapolation of time-to-event clinical endpoints is required. The purpose of this research was to explore automation of survival analysis reporting using Generative Artificial Intelligence (GenAI). Following published best practices for curve selection, GenAI was leveraged to recommend an appropriate extrapolation curve and provide justifications.
METHODS: Data were taken from a previously accepted HTA survival analysis report (NICE TA817) for patients treated for resectable urothelial cancer (PD-L1 ≥1%), with a minimum follow-up of 11-months. GPT-4o was provided with survival analysis outputs, including statistical tests, survival probability estimates, and figures, to assess proportional hazards (PH) and goodness-of-fit. Prompted with relevant content, GPT-4o was asked to; 1) assess PH, 2) select suitable extrapolation models (dependent vs. independent), 3) consider external data, then 4) select an appropriate curve. To validate accuracy, GPT-4o’s results were compared with results in the original report, the report published by NICE, and assessed against the opinion of three expert health economists.
RESULTS: GPT-4o’s interpretation of log-cumulative hazard plots, Schoenfeld residual plots, and Grambsch-Therneau test results aligned with interpretations made by the three health economic experts, the human produced report, and the NICE committee. GPT-4o concluded that the PH assumption might be violated, therefore suggesting consideration of both dependent and independent parametric models. Based on a comprehensive analysis of goodness-of-fit, visual fit, and long-term external survival data, GPT-4o recommended the same survival curves as those selected in the original report and by the NICE Committee. Notably, 13/13 statements or decisions made by GPT-4o were consistent with the original report or expert opinion.
CONCLUSIONS: The results suggest automation of curve selection and reporting of survival analyses is possible. However, more research is required to determine generalizability with differing levels of data maturity and to test the performance of alternative GenAI models.
P21: A Novel Framework for Quantitative Bias Analysis in the Presence of Non-Proportional Hazards Using the Accelerated Failure Time Model
5:00PM - 5:15PM
Macdougall A1, Soutar S2, O'Reilly JE2, Wallis J2, Carpenter L2
1Arcturis Data Ltd, Kidlington, UK, 2Arcturis Data Ltd, Oxford, UK
OBJECTIVES: Unmeasured confounding presents a key challenge when performing treatment comparisons using real world data. Quantitative bias analysis (QBA) is an important tool for assessing the robustness of treatment effect estimates obtained from such studies, but no published method is applicable to time-to-event (TTE) outcomes when the proportional hazards (PH) assumption is violated – a common circumstance when therapies with different This study developed, and assessed (with a simulation study), a method for QBA which used the accelerated failure time (AFT) model in order to relax the PH assumption.
METHODS: A two-step method was developed in which, firstly, the expectation-maximisation (EM) algorithm was used to impute an unmeasured binary confounder, U, with a known association with treatment assignment and the outcome. An AFT model was used for the TTE outcome, and a probit model for U. In the second step, an AFT model was fitted to the imputed data and a treatment effect obtained which was consequently adjusted for U. The method was using simulated data from a Weibull distribution over two data generating mechanisms which violated the PH assumption: delayed and waning treatment effect.
RESULTS: When using this two-step approach bias was small, particularly for the delayed treatment effect. Relative bias was 0.01% for delayed treatment effect and -0.10% for waning. Coverage achieved the expected level of 95% for both scenarios.
CONCLUSIONS: This study demonstrates a valid QBA framework which can be applied to TTE outcomes, without assuming PH. The framework can be readily applied, given the widespread use of both AFT models and the EM algorithm in statistical software; as well as being straightforward to interpret. This method could be applied to, for example, external control arm studies to formally demonstrate the robustness of results to unmeasured confounding.
P22: Current and Future Use of RWE in HTA Decision-Making: Payers View Globally
5:15PM - 5:30PM
Heinz S1, Kumari C2, Ataide J2, Bourakkadi M2, Castellano G2
1Ipsos GmbH, London, LON, UK, 2Ipsos UK, London, London, UK
OBJECTIVES: Using real-world data and evidence (RWD/RWE) in healthcare decision-making is increasing. However, acceptance of RWE varies among HTA agencies. Some agencies are more willing to consider RWE for evaluating new technologies and making decisions about healthcare resource allocation efficiently. We aim to explore the payer perspective on the barriers and opportunities of incorporating RWE as evidence submission in reimbursement decision-making by HTA bodies.
METHODS: We fielded an online survey with payers from the Ipsos payer panel across multiple EU and non-EU member states. Questions concerning the importance, acceptance, and aspects of RWE studies that are of concern for payers in HTAs were asked in this survey.
RESULTS: Payers indicate that national guidelines guide RWE use in HTAs in country where available (Canada, UK), while international, mainly EMA, guidelines are followed in countries such as Spain and Denmark. Major barriers to RWE adoption include data quality, methodology, and expertise. Transparency and data sources are crucial for payers, with pragmatic trials and registries being the preferred source to support recommendation decisions. RWE is considered valuable for indirect comparisons and treatments needing long-term management, in rapidly evolving landscapes, and with curative benefits after prolonged treatment. Some payers may accept to make HTA recommendations based only on RWE in some situation, such as rare diseases and gene therapies, when conducted according to guidelines.
CONCLUSIONS: The integration of RWE into HTA and access programs is advancing through initiatives aimed at improving data quality, methodology and relevance. Despite these efforts, RWE remains primarily supplementary to RCTs, except in specific cases. The ongoing focus on enhancing RWE’s credibility and use suggests a growing, although cautious, acceptance. Continued multi-stakeholders collaboration and development of standard frameworks to use the appropriate types of RWE and data sources in HTAs is a crucial step for making informed decisions.
P23: Integrating Machine Learning and Propensity Score-Based Methods for Making Causal Inferences From Real-World Data
5:30PM - 5:45PM
Gallinaro J1, Shah V1, Lee H1, Kreif N2, Guerra I1
1IQVIA, London, LON, UK, 2University of Washington, Seattle, WA, USA
OBJECTIVES: Causal inference methods allow for comparative analyses using real world data (RWD) when trials are unfeasible. Propensity scores (PS) are routinely used in RWD applications for tasks such as mitigating confounding, evaluating treatment effectiveness, and constructing external comparator arms in the absence of a suitable control group. Machine learning (ML) has the potential to improve these analyses by handling complex relationships in large datasets. This review examined ML methods for PS estimation in causal inference analyses.
METHODS: We performed secondary research to identify (i) simulation papers comparing non-parametric ML with logistic regression for PS estimation, (ii) theoretical papers discussing the application of ML in doubly robust estimators for both PS and outcome model estimation, and (iii) applied papers that use ML for PS estimation in real-world comparative effectiveness research.
RESULTS: Out of 9 applied papers analyzed, 5 used the super learner, 2 used neural networks and 2 used random forests for PS estimation. However, 5 simulation studies showed mixed results when comparing logistic regression or nonparametric ML models for PS estimation in terms of estimated effect bias, covariate balance and PS overlap. Three theoretical papers discussed potential bias in treatment effect estimation from using non-parametric ML for PS estimation due to regularization bias and overfitting risk. These issues can be mitigated in doubly robust methods with cross-fitting, leveraging non-parametric ML for both PS and outcome models, providing estimators robust against model misspecification.
CONCLUSIONS: Under certain conditions, ML can improve the validity of PS in estimating treatment effects from RWD. However, the importance of the “human factor”, such as variable selection based on expert knowledge, is crucial. Doubly robust methods, particularly when used with cross-fitting, can produce less biased treatment effect estimates. Future research should focus on the less-explored topic of using ML for PS estimation in the context of external comparator arm studies.
Breakouts: IP, WS, & OBS
Beyond "Guesstimates" in Long-Term Survival Extrapolation: Introducing a Comprehensive Step-By-Step Elicitation Framework
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Level: Introductory
PURPOSE: This workshop aims to discuss best practices and challenges in expert elicitation for long-term survival estimates. We will present a comprehensive, step-by-step elicitation framework, providing attendees with essential practical knowledge of this method, its benefits, and implementation considerations. Perspectives from industry and health technology assessment (HTA) agencies will also be discussed.
DESCRIPTION:
Estimating long-term survival in the absence of data is a persistent challenge in economic modelling for HTA and a common source of uncertainty within decision making. Expert elicitation is recommended in the NICE Methods Guide to bridge this data gap, and obtaining clinical experts' judgments has become standard practice for assisting in survival extrapolation. The ISPOR Structured Expert Elicitation (SEE) Task Force is currently developing guidance on SEE for healthcare decision-making. However, existing elicitation methodologies often result in estimates perceived as mere "guesstimates," which undermines confidence in these expert-elicited values. To address this critical methodological gap in HTA, we will provide an interactive demonstration of a novel elicitation framework for long-term survival estimates. This framework enhances the credibility, accuracy, consistency, and transparency of expert judgments. Min-Hua Jen will provide an overview of current approaches for eliciting long-term survival, highlighting limitations and practical challenges (10 minutes). Jacoline Bouvy will introduce NICE's expectations for incorporating SEE in survival outcomes and its envisioned use in HTA submissions (10 minutes). Kate Ren and Jessica Forsyth will present a rigorous elicitation framework for long-term survival estimates. A live demonstration using a recent case study will be conducted, with polls used to gather input for the demonstration (30 minutes). Finally, the panel will discuss the application of the framework, followed by a Q&A session (10 minutes). This interactive workshop will be valuable for researchers and industry analysts interested in conducting formal expert elicitation for long-term survival estimates.
Discussion Leaders
Jessica Forsyth, PhD
University of Sheffield, Sheffield, United Kingdom
Jessica Forsyth has a background in medical physics, Bayesian statistics and embryology and currently is a Research Associate in Statistics within the Sheffield Centre for Health and Related Research (SCHARR) at the University of Sheffield. She works as a member of SCHARR-TAG (an External Assessment Group for NICE Technology Appraisals), critiquing statistical methods used in NICE single technology appraisal submissions. She has also been involved in the development and conduct of multiple expert elicitation workshops for long-term survival outcomes.
Discussants
Jacoline Bouvy, PhD
National Institute for Health and Care Excellence, London, LON, United Kingdom
Jacoline is the Programme Director Medicines Evaluation at NICE. She is responsible for the delivery of medicines guidance produced by NICE’s Technology Appraisals and Highly Specialised Technologies work programmes.
Min-Hua Jen, PhD
Eli Lilly and Company, Uxbridge, LON, United Kingdom
Dr Min-Hua Jen is currently Senior Director - Real-World & Access analytics at Eli Lilly, leading the International Business team on Market Access/HEOR/Medical affairs statistical support. She has extensive experience applying statistics to clinical research, epidemiology and health economics and outcomes research in academia and industry settings. She is an active member in the PSI/EFSPI HTA Special Interest Group (SIG) and the engagement chair of the ISPOR Oncology SIG. She was trained in Epidemiology and Statistics and obtained her PhD at University of Bristol. Her research interests including indirect treatment comparisons and network meta-analysis; particularly incorporate external data for time to event outcomes, surrogacy analyses, multilevel modelling and health economic modelling.
Kate Ren, PhD
University of Sheffield|ConnectHEOR, Sheffield|London, England, United Kingdom
Kate Ren is a Senior Research Fellow in Statistics at the University of Sheffield and also the Director of Statistics at ConnectHEOR. She is a member of SCHARR-TAG (an external assessment group for NICE technology appraisals), and has extensive experience in critiquing statistical methods used in NICE single technology appraisal submissions. She is also a member of NICE Appraisal Committee C. Her research interests include indirect treatment comparisons, survival extrapolation and structured expert elicitation.
Broader Value Elements: Methods to Quantify Each and Their Relevance for European Markets
Digital Conference Pass
Level: Intermediate
PURPOSE:
Despite the ISPOR Value Flower being a mainstay in the health economics and outcomes research community, broader value elements are still not regularly quantified by many health technology assessment (HTA) bodies. This workshop will provide attendees with recommended best practices to quantify each value element within the ISPOR Value Flower and will then discuss their likelihood of implementation and relevance for European HTA bodies.
DESCRIPTION:
Dr. Willke will introduce the workshop by briefly describing Value Flower history and the current state of play for the consideration of the broader value elements by HTA bodies in the UK, France, Germany, Spain, and the US. (9 minutes) Dr. Shafrin will then present the recommended method and associated best practices for quantifying the following ISPOR Value Flower elements: severity of disease, value of hope, value of knowing, insurance value, and fear of contagion. (12 minutes) Dr. Whittington will then present the recommended method and associated best practices for quantifying the remaining ISPOR Value Flower elements, including productivity, family spillovers, equity, real-option value, and scientific spillovers. (12 minutes) Dr. Steuten will conclude the workshop with her perspective on the likelihood that these broader value elements will be adopted by HTA bodies in the UK, France, Germany and Spain with a discussion on the applicability of the broader value elements to each HTA body’s decision making. (12 minutes). Audience Q&A (15 minutes). Polling questions will be used after the panelists’ presentations to (i) verify audience understanding of methodologies for estimating broader value elements, and (ii) assess audience opinions on the relevance of these broader value elements for European markets. Workshop attendees will receive QR code that links to an abbreviated user guide for estimating each broader value element. This workshop will be relevant for health economists, HTA bodies, manufacturers, payers, and patient advocacy organizations.
Discussion Leaders
Richard Willke, PhD
Scintegral Health Economics, Lawrenceville, NJ, USA
Dick is currently principal at Scintegral Health Economics, LLC; he became the first Chief Science Officer for ISPOR in 2016, served in that role until mid-2023 and then as CSO Emeritus until retiring in 2024. Dick’s responsibilities were to develop, lead, and support strategic initiatives related to research, scientific, and content priorities. Prior to joining ISPOR he was employed for 25 years in the pharmaceutical industry with Pfizer and its legacy companies, where he retired as a vice president in the HEOR group. He received a Ph.D. in economics from Johns Hopkins University, has been a member of the economics faculty at Ohio State University (OSU), and senior economist at the American Medical Association. He has served as a co-editor for Value in Health, on AHRQ, NIH, and PCORI project review study sections, is past chair of the OSU Economics Advisory Board, and has over 100 scholarly publications.
Discussants
Jason Shafrin, PhD
University of Southern California, Los Angeles, CA, USA
Jason Shafrin, Ph.D. is a Senior Managing Director at FTI Consulting's Center for Healthcare Economics and Policy. Dr. Shafrin has over 20 years of health economics research experience serving as trusted advisor and expert to a wide variety of healthcare and life sciences companies, governments and non-governmental organizations (NGOs). Dr. Shafrin is an Adjunct Professor at the University of Southern California, the former Director of Research at the Innovation and Value Initiative and the Founder and Editor of the Healthcare Economist blog.
Lotte Steuten, PhD
Office of Health Economics, London, LON, United Kingdom
Lotte Steuten, PhD, is Deputy Chief Executive of the not-for-profit Office of Health Economics (OHE) and the scientific and business lead for its international research-led work program—maintaining its reputation for objective, innovative, and high-quality research, and meeting its charitable goals. She is also an Honorary Visiting Professor at City, University of London (UK) contributing to its teaching programmes in health economics.
For more than 20 years, Lotte has worked in academic, research, and consulting HEOR roles with one focus — improving healthcare decision-making through high-quality research and analysis. To achieve that, she has collaborated with HTA authorities, pharmaceutical and medical device companies, patients, payers, policymakers, academic researchers, clinical specialists, and venture capitalists. She has gained broad HEOR expertise of various technologies: precision medicine, oncology, vaccines, antibiotics, diagnostics, digital health, and medical devices.
Lotte’s career has been based in the United States, the Netherlands, and the UK. Collaborations with HEOR experts in Southeast Asia, Africa, and Latin America have provided her with deep insights into the differences and commonalities between the role of HEOR in different healthcare systems, cultures, and societies. With this background, she has: led diverse international teams; developed strategies and delivered on program and organisational missions, values, and objectives; acquired research funding and managed budgets; and been responsible for executive decision-making as well as legal and fiduciary matters.
As an active ISPOR member since 2004, Lotte has served on various Taskforces and Committees, delivered Short Courses, was co-Chair of ISPOR EU 2021, and is currently a member of the Board of Directors, and an Associate Editor for Value in Health. She aims to be a role model for diversity and played an active role in the 'Women in HEOR' initiative.
Before joining OHE, Lotte worked in Seattle (US) as an Associate Professor at the University of Washington and at the Fred Hutch Cancer Research Center. She graduated cum laude with her PhD from Maastricht University (NL) and then worked at Brunel University (UK) and Twente University (NL).
Melanie Whittington, PhD, MS
Tufts Medical Center (CEVR), Boston, MA, USA
Dr. Melanie Whittington is the Managing Director and Head of the Leerink Center for Pharmacoeconomics where she leads pharmacoeconomic evaluations of in-development and recently approved pharmaceuticals. She is also a Senior Fellow at the Center for the Evaluation of Value and Risk in Health (CEVR) at Tufts Medical Center where she works to develop novel methods for value assessment. She was previously the Director of Health Economics for the Institute for Clinical Economic Review (ICER) where she led the cost-effectiveness analyses and budget impact analyses within ICER reviews. She is a leader in the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) and was the recipient of the ISPOR Bernie J. O’Brien New Investigator Award. She has a PhD in Health Services Research with a concentration in Health Economics and Outcomes Research.
18:00 - 19:00
Welcome Reception
Join us for a lively Welcome Reception, a perfect kick-off to connect, unwind, and gear up for what's ahead.
Tue 19 Nov
7:00 - 9:00
Coffee and Connect
Don’t miss the start to the day with the Plenary Session. Enjoy your morning coffee as you listen to dynamic presentations intended to inspire and empower. Provided by ISPOR
7:00 - 17:00
Registration Hours
8:30 - 9:45
Plenary Session
ISPOR President's Address and Avedis Donabedian Lifetime Achievement Award Presentation
Digital Conference Pass
Welcome & ISPOR President’s AddressEberechukwu Onukwugha
Professor
University of Maryland, Baltimore
Avedis Donabedian Lifetime Achievement Award Presentation
Awardee: C. Daniel Mullins, PhD
Speaker
Eberechukwu Onukwugha, PhD
University of Maryland School of Pharmacy, Baltimore, MD, USA
Eberechukwu Onukwugha, PhD is a Professor in the Department of Practice, Sciences, and Health Outcomes Research and is the Executive Director of Pharmaceutical Research Computing at the University of Maryland School of Pharmacy. She received a Master of Science in agricultural and applied economics as well as a Doctor of Philosophy in economics (concentration: econometrics) from Virginia Tech. Dr. Onukwugha has approximately 20 years of experience conducting health economics and outcomes research. She examines the costs and health outcomes associated with health-related decisions as well as the institutional and environmental context framing these decisions.
Ready, Set, Go: The Last Sprint for the EU HTAR
Digital Conference Pass
Welcome & ISPOR President’s AddressEberechukwu Onukwugha
Professor
University of Maryland, Baltimore
Avedis Donabedian Lifetime Achievement Award Presentation
Awardee: C. Daniel Mullins, PhD
Plenary Panel
With only a few more months before the start of the EU HTAR in January 2025, everyone is in full speed ahead towards successful implementation in all organizations. The HTA Coordination Group, it’s subgroups and the European Commission are working hard to finalize all documents, guidances and implementing acts required for a well-functioning system. The session will explore if we are advanced enough and if we are ready for the HTAR to be applied. The session will specifically address how the stakeholders are preparing for the HTAR in their organizations and how they are implementing changes, as well as what key challenges are still ahead. These challenges will differ between the stakeholders. How is industry preparing for the high number of PICO and related analysis they see coming and is AI part of this adaptation? How are HTA bodies getting ready, specifically in Eastern EU countries, the late-access countries, how do they anticipate the usability of the JCA? How are patients and patient organizations preparing and what factors impact a sustainable future for patient input under the HTAR? Lastly, the panel will explore how the HTAR links to already existing regional initiatives, e.g Beneluxa as well as the International Horizon Scanning Initiative (IHSI). Are they mutually beneficial to each other, and how can we leverage the benefits of these collaboratives under the HTAR?
Moderators
Anne Willemsen, MSc
Dutch National Healthcare Institute, Diemen, Netherlands
Anne Willemsen holds double Masters Degrees in Health Sciences, specialising in both Health Technology Assessment and Health Policy. This led her through research and project management roles within the Dutch National Healthcare Institute (Zorginstituut Nederland) over the past years. Between 2016 and 2023 she has been working for EUnetHTA and has been coordinating Joint Clinical Assessments (JCA). Since April 2023, she was elected as the co-chair of the HTA Coordination Group JCA subgroup under the HTA Regulation.
Speakers
Roisin Adams, MPharm, MSc., PhD
National Centre for Pharmacoeconomics, Dublin, Ireland & Discipline of Pharmacology and Therapeutics, Trinity College Dublin, Dublin, Ireland
Vanessa Schaub, PhD
F. Hoffmann-La Roche Ltd, Basel, Switzerland
Dr Vanessa Schaub, Head of Global/EU HTA Strategy, Roche | Dr Vanessa Schaub has been working at Roche for almost 20 years, starting at the German affiliate where she held diverse access and commercial positions. In her current role, she ensures fit for purpose Global HTA strategies to stay ahead of emerging health systems trends and patient needs globally. Since August 2021 she also leads a cross functional Access SQUAD for external and internal EU HTA readiness. Her passion is around working in partnership with internal and external partners to foster rapid, broad and sustainable patient access to Health care innovations. Her team is guiding portfolio-related HTA strategies on the local market level as well as overseeing the implementation of above country level HTA approaches with a current strong focus on EU HTA. A health economist by training, Vanessa has disease area experience in oncology, neurology and respiratory diseases.
Michal Stanak, Dr.phil, AKC
National Institute for Value and Technologies in Healthcare (NIH), Bratislava, Slovakia
Michal Stanak has been the director of the Slovak national HTA body, National Institute for Value and Technologies in Healthcare, since January 1st 2022. In the years leading up to it, he served as the director of the HTA department at the Slovak Ministry of Health. From 2015 to 2020, he worked as a researcher at the Austrian Institute for HTA, where he worked on a number of HTA assessments for Austria, Germany and EUnetHTA. Since 2019, he has been a lecturer at the Comenius University, Bratislava, Slovakia.
Michal Stanak studied in England and Austria. He received a bachelor's degree in Philosophy from King's College London, a master's degree in Politics, Philosophy and Health economics from University College London and a doctorate in applied ethics in the field of decision-making in healthcare from University of Vienna.
Valentina Strammiello, MA, BA
European Patients’ Forum, Brussels, Belgium
Valentina Strammiello works as Head of Programmes and oversees the EPF project portfolio and Youth Strategy. She represents EPF in HTA-related activities and in the European Health Parliament initiative.
Before joining EPF, Valentina has had a long collaboration with the CIPI Network and has worked as freelance OSINT analyst on Security and Energy issues. Self-trained in website management and web 2.0 applications, she is among the founders of ecpa2.0 (European Center for Public Affairs 2.0), a forum of young professionals. She holds a BA in International Relations and a Master’s Degree in European Studies.
9:30 - 19:00
Exhibit Hall Open
9:45 - 10:15
Coffee and Connect
Head to the exhibit hall to refuel, recharge and connect with fellow attendees and exhibitors over a steaming cup of coffee. Provided by ISPOR
10:15 - 11:15
ISPOR New Professional Session
Three Things You Need to Know About Networking, But Were Afraid to Ask
In this session we explore why growing your network is an even more important skill than you realize. Your network has value for you in many more ways that just as a vehicle for seeking out jobs or project opportunities. Good networkers think hard about their priorities, build some basic skills, and see past the barriers that hold many of us back. We’ll share some perspectives and ideas from people with expertise in the subject and discuss with current ISPOR members the ways that they are growing the value of their networks. This session is organized by the ISPOR New Professionals, and all interested in advancing their HEOR careers are encouraged to attend.
Moderators
Ian Bradley, BSc
P31 Academy for Consulting Performance Excellence, Peterborough, CAM, United Kingdom
Ian Bradley is a Partner of P31 Consulting, LLC. Ian has two decades of experience in leadership development, coaching and organisational change, especially in the pharmaceutical and medical device industries. He has worked with major pharma companies, CROs, life science & biotechnology companies and their advisors and partners, in Europe, the US and around the world.
Ian is in demand for his work on communication, persuasion and negotiation, and besides delivering training he is often asked to deliver coaching and leadership interventions for senior teams.
Ian was a Principal with IMS Consulting for three years, where he led and delivered projects in market access and evidence development for global pharma companies. Ian worked early in his career in the Technology Transfer office of a UK university. Following that, Ian’s consulting career included roles with PA Consulting Group (1997-2003, Principal Consultant) and Arthur D Little (2006-2007, Practice Head / UK Healthcare).
Ian has a BSc(Hons) from the University of Durham in the UK in Engineering and Management and is based near Cambridge in the UK.
Speakers
Alice Kim, MSc
Charles River Associates, London, LON, United Kingdom
Riku Ota, MPH
Novo Nordisk, Brøndby, Denmark
Riku Ota is a dynamic and dedicated public health professional, with a comprehensive career spanning the pharmaceutical sector and consultancy across the Asia Pacific and Europe. His journey began with a focus on the clinical development of cutting-edge treatments for inflammatory bowel diseases (IBD), laying a solid foundation for his expertise in health outcomes and evidence generation.
In his pivotal role at Novo Nordisk's Japanese affiliates, Riku served as a Health Economics and Outcomes Research (HEOR) manager, where he played a critical role in expanding patient access to healthcare by highlighting the clinical and economic benefits of novel health technologies. Progressing within Novo Nordisk A/S in Denmark, he now excels as the Global Payer Evidence Lead, overseeing the MASH/NASH therapeutic area. This position underlines his commitment to integrating clinical value with strategic market access initiatives on a global scale.
An engaged member of the Health Equity Special Interest Group (SIG) within ISPOR for the Asia-Pacific region, Riku contributes to advancing health equity discussions and initiatives. His dedication to the field is further evidenced by his selection as a member of the New Professional Steering Committee for ISPOR for the 2023-2024 term, where he plays a significant role in steering the organization's future direction and initiatives.
Riku has also led the Health Technology Assessment (HTA) Taskforce for the EFPIA Japan Access Committee, demonstrating his leadership in one of the pharmaceutical industry's most influential bodies. His academic foundation is robust, with a Bachelor of Science in Statistics from the University of Otago, New Zealand, and a Master of Public Health from King's College London, UK. These qualifications underscore his analytical prowess and understanding of global health landscapes.
Currently, Riku is enhancing his strategic and leadership skills through a hybrid part-time Master of Business Administration program at IE Business School, Spain. His primary interests lie in global public health and addressing health disparities, particularly those arising from socioeconomic factors. Riku Ota's multifaceted career and ongoing academic pursuits highlight his commitment to making a significant impact on public health and healthcare accessibility worldwide.
Natalia Scherff
Coach Scherff, Frankfurt, HE, Germany
Natalia is an independent ICF certified career and leadership coach, working predominantly in the Pharmaceutical Industry across the Globe.
She has 20+ years of experience in corporate talent management, top executive search consulting firms, Russell Reynolds Associates, leadership assessment, and career development coaching.
For the last 4 years, while working for Novartis, Natalia has held strong reputation of the SME in executive recruitment for Value & Access, focusing on Global & Countries Access & HEOR leadership roles.
Natalia has two Business Master‘s Degrees obtained in Moscow and Frankfurt, and she is fluent in English, Russian, and German.
Yan Zhi Tan, MSc, BSc (Pharm) (Hons)
Lumanity, Utrecht, Netherlands
Member Group Meetings
ISPOR Clinical Outcome Assessment Special Interest Group
The ISPOR Clinical Outcome Assessment Special Interest Group invites you to join their Open Meeting to connect with the new leadership team, explore exciting key project proposals from fellow members, and dive into discussions about future collaboration ideas for the group. This meeting will allow you to brainstorm, share ideas, and contribute to innovative projects that will push the field of clinical outcome assessment forward. This is a valuable opportunity for members to engage with the group's initiatives and help shape its future direction.
ISPOR Greece Chapter Meeting
Join us for the ISPOR Greece Chapter meeting, an event open to all those interested in advancing HEOR within Greece. Established to foster collaboration among healthcare professionals, the chapter focuses on addressing healthcare challenges unique to Greece. This meeting provides a valuable opportunity to learn about the chapter’s initiatives, share knowledge, and explore the latest developments in HEOR. Attendees can discover how they can actively participate in shaping the future of healthcare in Greece.
Breakouts: IP, WS, & OBS
Comparing and Contrasting RWE Guidance: What Researchers Need to Know Considering the Global Picture
Digital Conference Pass
Level: Intermediate
ISSUE:
In the last five years, there has been a proliferation of RWE guidance issued by regulators and HTA agencies covering use cases, and design, data, and submission considerations for decision-grade RWE. For researchers, it may be challenging to track and operationalize guidance, particularly when designing a comprehensive evidence strategy for multiple stakeholders. As examples, different terms are used for similar concepts (e.g., data quality), and the same concept is described with variable emphasis. Stakeholders have called for more harmonized and efficient provision of guidance. To address this need, this panel will compare and contrast available guidance and propose areas for harmonization. OVERVIEW:
The moderator (Ulka Campbell) will present a review of recent guidance on real-world data (RWD) quality from the FDA, EMA, NICE, HAS, and IQWiG. RWD quality will be used as an example to focus the conversation, but the panel will cover other topics covered in guidance. The panel will discuss the similarities and differences among the guidances. Solange Corriol Rohou will discuss how they interpret and operationalize these guidances for different decision-makers. Páll Jónsson and Patrice Verpillat will provide their perspectives on how researchers should consider similarities and differences in guidance when designing real-world studies. The moderator will then facilitate a debate on areas for harmonization and more operationalized recommendations.
Moderators
Ulka Campbell, PhD, MPH
Aetion, Inc, New York, NY, USA
Ulka Campbell is an epidemiologist and the Head of Scientific Strategy at Aetion (a healthcare technology and research services company) providing methods and regulatory support across therapeutic areas and leading research to inform regulatory RWE best practices. Previously, she was at Pfizer for 14 years leading regulatory studies and serving as the Head of Safety Surveillance Research, overseeing a team responsible for post-approval safety studies obligated to FDA and EMA. She has co-authored several publications and taught courses on pharmacoepidemiology, standards for decision-grade real world studies, causal inference, and epidemiologic methods, and is an Adjunct Assistant Professor of Epidemiology at Columbia University. She received an MPH from the University of Michigan and a PhD from Columbia University in epidemiology.
Panelists
Solange Corriol-Rohou, MD, PhD
Pharmaceutical Company: AstraZeneca, GMD, Paris, 75, France
Solange Corriol-Rohou, a pulmonologist/immuno-allergist by training, joined AstraZeneca R&D in 2004 and is currently Sr. Regulatory Affairs & Global Policy Director, with responsibilities in the Respiratory/Infection and Vaccine/Immune franchises. Over the past 20 years, moving from the French Medicines Agency/EMA and academia to the pharmaceutical industry, she has gained strategic experience in drug development. She is quite active within EFPIA, ICH and IMI/IHI, and passionate about paediatric drug development, rare/ultra-rare diseases and drug development optimisation.
Pall Jonsson, BSc, PhD
National Institute for Health and Care Excellence (NICE), Manchester, LAN, United Kingdom
Pall Jonsson is Programme Director for Data and Real World Evidence at the National Institute for Health and Care Excellence (NICE). He has a strategic role in ensuring NICE is at the forefront of harnessing new and emerging opportunities for using real world data to inform NICE’s guidance to the health and care sectors.
Pall has a PhD in bioinformatics from the University College London. Before taking up his current role, he was Associate Director for Science Policy and Research, responsible for NICE’s portfolio of international research projects in areas such as big data and real-world evidence. Earlier experience includes work in academia, biotech and the pharmaceutical industry.
Patrice Verpillat, MD, MPH, PhD
European Medicines Agency (EMA), Amsterdam, Netherlands
Dr. Patrice Verpillat (MD, MPH, PhD) is the Head of the Real World Evidence (RWE) Workstream at the European Medicines Agency (EMA). He is a medical doctor, specialist in epidemiology. Before joining the EMA, he has worked during 20 years in the pharmaceutical industry where he had positions in several international companies, always dealing with real world data (RWD) and non-interventional studies (NIS) in order to bring RWE into research, access and life-cycle product management.
Dr. Verpillat has published over 70 articles in Medline referenced journals. He has been involved in many organisations such as ENCePP, ICH M14 working group, European pharma association (efpia) and ISPE
EU Joint Clinical Assessment: Indirect Treatment Comparisons for Evaluating Relative Clinical Effectiveness Across Multiple PICO Questions
Digital Conference Pass
Level: Intermediate
ISSUE: The European Union (EU) Health Technology Assessment Regulation (HTAR) will be applied from January 2025. From that date, all advanced therapy medicinal products and medical devices will have their relative clinical efficacy evaluated through a new EU Joint Clinical Assessment (JCA) pathway. Indirect treatment comparisons (ITCs) are expected to be frequently required to address the numerous PICOs (Population, Intervention, Comparator, Outcomes) requested in the JCA scope.
OVERVIEW: Member states will request relevant policy-driven PICOs to be consolidated and shared with the Health Technology Developer. Ideally, PICOs would be assessed using randomized head-to-head evidence, which is often impossible, as standards of practice vary from country to country, making ITCs essential. However, contextual data limitations are likely to be present in most ITCs. Additionally, substantial resources are required from all stakeholders to conduct and assess the necessary ITCs, either due to the number of PICOs requested or the number of ITCs considered necessary to address each PICO. The panelists will provide an overview of available HTA ITC guidelines and previous experience with EUnetHTA relative effectiveness assessments. They will highlight the rationale behind the design of joint clinical assessments and discuss how member states will likely handle various ITC methods. The panel will summarize the expected opportunities and challenges, from their respective and diverse viewpoints, in using ITCs to assess the relative clinical effectiveness for different stakeholders. Sophie Van Beekhuizen will moderate the issue panel. Wim G. Goettsch will offer a broader HTA perspective, addressing EU and national issues on the JCA process and its implications. Mahmoud Hashim will present a Health Technology Developer's viewpoint on how to be well-prepared for multiple PICOs & ITCs within the EU JCA. Finally, from an industry perspective, Thomas Wilke will outline the long-term and short-term activities required to successfully deal with the future JCA challenges.
Moderators
Sophia van Beekhuizen, MSc, MPhil
Cytel, Rotterdam, Netherlands
Sophia is Director & Research Principal at Cytel. She has over a decade of experience in consulting with focus on ITC and HTA submissions across Europe
Link to her profile: https://www.linkedin.com/in/sophie-van-beekhuizen-97727854/
Panelists
Wim Goettsch, PhD
Utrecht University, Utrecht, Utrecht, Netherlands
Professor Wim Goettsch is a key contributor to European HTA. At Utrecht University he is a Professor of HTA of Pharmaceuticals. He serves as a special HTA advisor to Zorginstituut Nederland (ZIN) and was former director of EUnetHTA JA3 Directorate.
Mahmoud Hashim, PhD, MD
Johnson & Johnson, Rotterdam, Rotterdam, Netherlands
Dr. Mahmoud Hashim is a medical doctor with a PhD in Health Economics and Outcomes Research and two Master’s degrees. With 12+ years of experience, he specializes in Health Economics and Outcomes Research (HEOR) and leads global HEOR projects across various therapeutic areas. Currently the Director of Market Access Analytics and RWE at Johnson & Johnson.
Thomas Wilke, Prof. Dr.
GIPAM, Wismar, Germany
Are There More Optimal Approaches to Managing Pricing and Reimbursement of Multi-Indication Medicines to Better Facilitate Patient Access?
Digital Conference Pass
Level: Intermediate
ISSUE:
Medicines with multiple indications are increasingly common
and can bring value through added treatment options for patients
and potential efficiency gains due to a well understood mechanism of action and safety profile. At the same time, the value of each indication can vary, so a single price cannot capture the value of individual indications. European countries have different ways of managing multi-indication medicines, but in practice lengthy negotiations and price erosion are still common, which can delay or restrict patient access and launch sequencing. Indication-based pricing
applies distinct prices for different indications and
has been suggested as a solution, however, it is difficult to implement, and most countries adopted a blended approach. There is a need to explore solutions to manage the pricing and reimbursement (P&R) of multi-indication medicines in a more optimal way for all stakeholders. This session therefore brings together different stakeholder perspectives to discuss potential solutions for the management of multi-indication medicines.
OVERVIEW:
An overview of multi-indication medicines based on a scoping literature review will be presented, including key P&R challenges and proposed solutions (10 minutes, moderator). Challenges and solutions will first be discussed from a broad, international perspective, highlighting the views of different stakeholders (13 minutes, Amanda Cole). They will then be examined from a country-specific perspective (Italy) to explore what the solutions could solve and what is feasible in practice (13 minutes, Claudio Jommi). Finally, the challenges and solutions will be commented on from the industry perspective (13 minutes, Julien Patris). The audience will then be invited to comment and ask questions to the panellists. The intention of the discussion will be to explore what potential solutions for multi-indication medicines are effective and feasible, and can benefit all stakeholders (11 minutes). This panel session may benefit payers, policy makers, patient organisations and manufacturers.
Moderators
Jens Grueger, PhD
University of Washington, Seattle, WA, USA
Jens Grueger is an Affiliate Professor of Health Economics at the Comparative Health Outcomes, Economics and Policy (CHOICE) Institute at the University of Washington, and Director and Partner at Boston Consulting Group (BCG). As a Senior Expert for Pricing & Access in Healthcare, he works with stakeholders to find innovative ways of accelerating and expanding equitable access for patients to medical innovation in areas of unmet need. His 30 years of experience in the field, and significant experience engaging experts across all areas of expertise, enable him to bring together different stakeholder voices to discuss solutions to challenges.
Panelists
Amanda Cole, PhD
Office of Health Economics, London, LON, United Kingdom
Amanda Cole is Associate Director at the Office of Health Economics, and Honorary Professor of Practice at University College London. Amanda’s research interests include the economics of innovation, the use of real-world evidence to support HTA and product development; novel pricing and reimbursement mechanisms for pharmaceuticals, and the interaction between HTA policy and optimal R&D decisions by industry. Before joining OHE in 2014, Amanda was a research fellow at the University of Birmingham where she focused on the HTA of medical devices and embedding health economic evaluation early in product development. Amanda holds a PhD in health economics from the University of Birmingham.
In this panel, Amanda Cole will provide a perspective from her 10+ years of expertise in economic evaluation, health technology assessment, health state valuation, and analysis of critical health policy issues in general. Specifically, she will draw on her work on understanding different stakeholder perspectives and seeking expert consensus on the pricing and reimbursement challenges and solutions for multi-indication medicines.
Claudio Jommi, MSc
Università del Piemonte Orientale, Novara, Italy
Claudio Jommi is Professor of Management at the Department of Pharmaceutical Sciences, and Director of Master of Regulatory Sciences and Market Access for Medicines and Biotechnologies (DRMKA), Università del Piemonte Orientale, Novara, Italy. He is also member of the Patent and Spin Off Committee of the same University. He was Professor of Practice of Health Policy at SDA Bocconi School of Management, where he held the position of Director of the Master of International Health Care Management Economics and Policy (Mihmep), and Director of the Health Policy Area and Scientific Coordinator of the Pharmaceutical Observatory at Cergas (Centre for Research on Health and Social Care Management). He was the President of the Italian Health Economics Association. He is the Editor in Chief of GRHTA (Global and Regional Health Technology Assessment). His research activity is focused on Pharmaceutical Economics, Policy and Management, Health Technology Assessment, Innovation management. He has had his work published in many international and national journals, including Applied Health Economics and Policy, BMC Health Services Research, BMJ Open, Drug Design Development and Therapy, Frontiers in Pharmacology, European Journal of Health Economics, Health Policy, International Journal of Health Planning and Management, Journal of Medical Marketing, Pharmacoeconomics and Pharmacoeconomics Open, PLOS and PLOS Neglected Diseases, Public Money and Management, Social Science and Medicine, Value in Health.
Julien Patris, MA
argenx, Brussels, Belgium
Julien Patris is the Head of Patient Advocacy, Public Policy and Corporate Communication at argenx; where he also serves as Head of Digital & Patient Solutions. Julien is a Guest Lecturer at ESSEC Business School.
Julien has served in various biopharmaceutical companies over the past two decades at international, Europe and national level. Julien's experience spans across Corporate Affairs, Market Access, Commercial and Country Management.
Julien has hands on experience in pricing and reimbursement and negotiations across multiple products and countries in Europe. He has authored and contributed to several peer-reviewed publications in the field of access, access policy and HTA. An economist by background, Julien has graduate from the College of Europe-Bruges (Economics), Sciences Po Strasbourg (Business, Finance) and Claude Bernard University Lyon (Pharmacy, Market Access).
Exploring the Broader Impact of Health Technologies on Health Inequalities: Perspectives, Measures, Tradeoffs, Barriers, and Solutions
Digital Conference Pass
Level: Introductory
ISSUE:
Health inequities denote systematic disparities in health among diverse population groups, imposing substantial social and economic costs. Healthcare interventions can influence these inequities and may extend their impact beyond the health sector, potentially affecting education and the labor market, where decisions are made by different bodies with separate budgets.
OVERVIEW:
In this panel, we will analyze regional variations in health technology assessment, focusing on benefit-harm-cost-equity trade-offs. Using examples, we aim to discuss the necessity of broader perspectives recognizing additional values and inherent trade-offs in health interventions. We will also explore barriers posed by limited real-world evidence for comprehensive distributional cost-effectiveness analysis (DECA) and address questions on measuring disparities. We will set the scene with three (10-minute) impulse talks, followed by a moderated discussion with prepared questions and an audience Q&A session (30 minutes). The first speaker will present a systematic review and DECA of hearing healthcare in Chile, addressing key questions: What are the main determinants of hearing health in Chile? What barriers exist to equal access to advanced hearing loss treatment? How can health inequality, a major policy concern for low- and middle-income countries (LMICs), be reduced shifting between sectors. Our second speaker will discuss methods for evaluating the equity impact of vaccination programs in LMICs. Topics include incorporating health equity into economic evaluations of vaccines and engaging local stakeholders. A case study on the Brazilian National Immunization-Program will highlight challenges in developing equity-informed economic evaluations and informing policy decisions. Next, our third speaker will discuss the evaluation of infectious-disease measures in Europe, highlighting their varied impact on socioeconomic groups. Using recent insights from the German OptimAgent population model, this case example will demonstrate benefit-harm-cost-equity trade-offs and the use of aggregated and non-aggregated disparity measures. This issue panel aims to foster a diverse and multi-stakeholder dialogue across regions and disciplines.
Moderators
Uwe Siebert, MD, MPH, MSc, ScD
Center for Health Decision Science and Dept. of Epidemiology and Health Policy & Management, Harvard T.H. Chan School of Public Health, Institute for Technology Assessment and Department of Radiology; Massachusetts General Hospital; Harvard Medical School, Boston, USA
Uwe Siebert, MD, MPH, MSc, ScD, is a physician by training and Professor of Public Health, Medical Decision Making and Health Technology Assessment (HTA), Chair of the Department of Public Health, Health Services Research and HTA at UMIT TIROL - University for Health Sciences and Technology in Austria, and Director of the International Continuing Education Program in HTA and Decision Science (htads.org). He is also Adjunct Professor of Epidemiology and Health Policy & Management at the Harvard T.H. Chan School of Public Health.
Prof. Siebert is President-Elect of ISPOR – The Professional Society for Health Economics and Outcomes Research and former President of the Society for Medical Decision Making (SMDM).
His research interests include applying evidence-based quantitative and translational methods from epidemiology, causal inference, real-world-evidence, health decision science and modeling, benefit-harm assessment, health economics, health services & outcomes research, public health, and artificial intelligence in the framework of health care policy advice and HTA as well as in the clinical context of routine health care, public health policies and patient guidance. He teaches courses for these topics at several universities and for industry in Europe, USA, South America, Africa, and Asia. He has worked with several HTA Agencies in Europe, Brazil, US and Canada and he advises public and government agencies, academic institutions and industry regarding the conduction of HTAs and their impact on policy and reimbursement decisions.
He has authored more than 400 publications (> 30,000 citations, H index > 80) and is Editor of the European Journal of Epidemiology and editorial board member of several scientific journals.
Panelists
Beate Jahn, Assoc.Prof. Dipl.Math. oec. Dr.rer.soc.oec
Institute of Public Health, Medical Decision Making and Health Technology Assessment; Department of Public Health, Health Services Research and Health Technology Assessment, UMIT TIROL – University for Health Sciences and Technology, Hall in Tyrol, Tyrol, Austria
Beate Jahn, Dipl.-Math. oec., Dr.rer.soc.oec., is an Associate Professor of Public Health and Data & Decision Science, the Coordinator of the Program on Personalized and Precision Medicine and Director of the Practical Hands-on Workshop 'Modeling Approaches for HTA' at the Institute of Public Health, Medical Decision Making and Health Technology Assessment at UMIT TIROL, Austria. She is Past President of the Austrian Regional ISPOR Chapter, a member of the ISPOR-SMDM Modeling Good Research Practices Task Force, a member of the COVID-19 Future Operations Platform (FUOP) and serves as expert in the European Commission Initiative on Colorectal Cancer (ECICC).
Klaas Kiesewetter, MA
MED-EL Medical Electronics, Innsbruck, Austria
Natalia Kunst, PhD
University of York, York, Yorkshire, United Kingdom
Valentina Strammiello, MA, BA
European Patients’ Forum, Brussels, Belgium
Valentina Strammiello works as Head of Programmes and oversees the EPF project portfolio and Youth Strategy. She represents EPF in HTA-related activities and in the European Health Parliament initiative.
Before joining EPF, Valentina has had a long collaboration with the CIPI Network and has worked as freelance OSINT analyst on Security and Energy issues. Self-trained in website management and web 2.0 applications, she is among the founders of ecpa2.0 (European Center for Public Affairs 2.0), a forum of young professionals. She holds a BA in International Relations and a Master’s Degree in European Studies.
Podium Sessions
Advances in Health Economics Modeling
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This session covers methods for conceptualizing and building health economic models.
Moderator
Praveen Thokala, MASc, PhD
The University of Sheffield, Sheffield, United Kingdom
Praveen Thokala joined the School of Health and Related Research (ScHARR) at the University of Sheffield after completing an MASc from the University of Toronto and a PhD from the University of Southampton.
During this time, he has completed several health economics projects including single technology appraisals (STA), multiple technology appraisals (MTA), and diagnostic assessment reports (DARs) for NICE. He has also recently led a modeling project for Institute for Clinical and Economic Review (ICER) in the US. His research interests include health economic modeling, multi-criteria decision analysis (MCDA), discrete event simulation modeling, and optimization. He co-supervised six PhD students to completion and currently co-supervises four PhD students.
In terms of MCDA, he has worked with the National Institute of Health and Care Excellence decision support unit (NICE DSU) in the UK on exploring the applicability of MCDA in HTA. He co-chaired the ISPOR Task Force on the use of MCDA in healthcare decision making and has been involved in several MCDA studies including supporting priority setting and benefit-risk analysis. He also co-edited a book titled, Multi-Criteria Decision Analysis to Support Healthcare Decisions.
P26: Comparison of Cost-Effectiveness Model Outcomes Using Two Individual-Level Simulation Models in Obesity
10:30AM - 10:45AM
Ni L1, Harris M2, Cotterill G3, Hoog M4, Basarir H1, Deger K2, van Hest N3, Johansson E5
1RTI Health Solutions, Manchester, UK, 2Evidera, Bethesda, MD, USA, 3Costello Medical, London, UK, 4Eli Lilly and Company, Indianapolis, IN, USA, 5Eli Lilly and Company, Solna, AB, Sweden
OBJECTIVES: Obesity is a complex disease that can reduce patient quality-of-life and contribute to increased risk of complications such as type-2 diabetes (T2D), cardiovascular (CV) disease, and cancer, which are associated with economic and societal impact. To fully capture its many consequences, cost-effectiveness models for obesity mirror this complexity. The aim of this study is to compare the outcomes of two different modelling approaches for assessing the cost-effectiveness of tirzepatide in managing obesity.
METHODS: Two individual-level simulation models previously developed to assess cost-effectiveness of tirzepatide against diet-and-exercise (D&E) were compared using a United Kingdom population with obesity and overweight: a state-transition model and a discrete-event simulation model. Both models track individual metabolic factors such as body weight, high-density lipoprotein, and systolic blood pressure, to estimate how many patients in a simulated cohort would develop obesity-related complications over their lifetimes. Using the same risk equations and patient characteristics, models were restricted to consider common complications: T2DM onset, CV events, and onset of sleep apnoea. This allows evaluation of the impact of modelling approach on predicted outcomes.
RESULTS: Both models showed tirzepatide reduced the number of T2D and CV events such as stroke and myocardial infarction compared to D&E. However, the discrete-event simulation model estimated higher incidences of T2D and sleep apnoea across all treatment arms compared to the state-transition model. Time-to-event outcomes were estimated in the discrete-event simulation model only, demonstrating that tirzepatide is associated with delays in the onset of complications. While the state-transition model partitioned the CV events once, the discrete-event simulation model used a two-step partitioning approach. Similarly, the approaches in estimating non-CV deaths also differed between the two models.
CONCLUSIONS: Obesity is a complex disease associated with numerous complications. Despite the differences in approaches, both models provided directionally consistent results, validating their use in obesity cost-effectiveness studies.
P27: Considerations for Model Conceptualisation When Using Surrogate Outcomes to Inform HTA Decision-Making
10:45AM - 11:00AM
Salih F1, Flight L2, Garrett Z2, Collins S2, Guo Y1, Mccracken F2, Knies S3, Efe R3, Lee K4, Husein F4, Hogan ME4, Morenz E4, Kaunelis D5, Gates M5, Bryan M5, Ollendorf D6, Tunis S7, Richardson M6, Coory M8, Aung PT8, Robayo A9, Dawoud D1
1National Institute for Health and Care Excellence (NICE), London, UK, 2National Institute for Health and Care Excellence (NICE), Manchester, UK, 3Zorginstituut Nederland, Diemen, NH, Netherlands, 4Canada's Drug Agency, Toronto, ON, Canada, 5Canada's Drug Agency, Ottawa, ON, Canada, 6Institute for Clinical and Economic Review, Boston, MA, USA, 7Tufts Center for Evaluation of Value and Risk in Health, Boston, MA, USA, 8Australian Government Department of Health and Aged Care, Canberra, Australia, 9Institute of Technological Evaluation in Health- IETS, Bogota, Colombia
OBJECTIVES: Many health technology assessment (HTA) agencies provide guidance on evidence standards for the use of surrogate outcomes. However, the level of detail varies, and there is limited guidance on considerations for economic models. With the increasing use of novel surrogate outcomes in clinical trials and consequently in economic models for HTA submissions, there is a need for further clarity around what to consider when utilizing these outcomes in economic models to inform decision-making.
A working group of representatives from HTA agencies and associated organizations was convened to produce recommendations and considerations for model conceptualization to inform HTA decision-making. METHODS: We conducted three scoping reviews; a review of regulatory standards for the validation of surrogate outcomes, a review of guidance from HTA agencies for the use and validation of surrogate outcomes and a review of statistical methods that have been proposed to validate surrogate outcomes. These reviews were supplemented with a qualitative study exploring how surrogate outcomes are dealt with in HTA evaluations by different HTA agencies and any lessons learned. The findings of these four strands of work were evaluated by the working group to inform the recommendations.
RESULTS: We developed recommendations outlining what should be considered alongside existing economic modelling guidance in HTA, when surrogate outcomes are used. This included considerations around their definition, justification, adoption, statistical validation, incorporation, reporting and approaches to quantify and present the uncertainty surrounding estimates based on their use.
CONCLUSIONS: The use of surrogate outcomes can pose a challenge for HTA agencies, because of the additional uncertainty in the relationship with longer term outcomes. Our recommendations can support economic modelers and decision makers especially for interventions in conditions where there are no long-term data available and when novel surrogate outcomes need to be used.
P25: REEEVR: A Tool for Automatic Conversion of Models for Health Technology Assessment From Excel to R
10:15AM - 10:30AM
O'Donnell M1, Xin Q1, Rogers G2, Haji-Ali AL3, Thom H1
1University of Bristol, Bristol, UK, 2The University of Manchester, Manchester, Greater Manchester, UK, 3Heriot-Watt University, Edinburgh, UK
OBJECTIVES: Complex Excel-based cost-effectiveness models in health technology assessment (HTA) suffer from prohibitive computational run-time to perform probabilistic sensitivity analysis (PSA). Previous work aimed to shift Excel users to the R programming language through tutorials, example models, and R tools that automate PSA. Our objective is to circumvent this process with the REEEVR tool that can convert Excel models into R automatically.
METHODS: Using concepts from programming language interpreters we parse, tokenise, generate abstract syntax trees, and translate each cell of complex Excel-based models into R. This output is then ordered, streamlined, and combined with libraries. The process is wrapped into a graphical user interface.
We tested our tool on an Excel-based 4-state Markov model comparing four implants for hip replacement surgery, using 1000 PSA samples. This followed models from academia, industry, and the UK National Institute for Health and Care Excellence (NICE) Guidelines Development Group (GDG). We compared total costs, quality adjusted life years (QALYs), and net monetary benefits (NMB) at £20,000/QALY to verify the conversion. A hand-coded R model was also used for comparison.
RESULTS: The output of the converted R model exactly matched those of the Excel-based model for all implants; in both models, the NMB for the best implant was £209,790 (95% credible interval £201,143 – 205,310). The run-time was reduced from approximately 20.5 seconds in Excel to 0.26 seconds in converted R, an 80 times reduction. This was also substantially lower than approximate 1.37 second run-time of the hand-coded R model.
CONCLUSIONS: Our REEEVR tool can exactly convert a probabilistic Markov model from Excel into R with a greatly reduced run-time. The converted R model can also be more efficient than hand-coded R models. The example was informed by academia, industry and the NICE GDG, suggesting the REEEVR tool could have wide applicability in HTA.
P28: Development of De Novo Health Economic Models Using Generative AI
11:00AM - 11:15AM
Chhatwal J1, Yildirim IF2, Samur S3, Bayraktar E2, Ermis T2, Ayer T4
1Harvard Medical School and Value Analytics Labs, Wilmington, MA, USA, 2Value Analytics Labs, Boston, MA, USA, 3Value Analytics Labs, Chantilly, VA, USA, 4Value Analytics Labs and Georgia Tech, Atlanta, MA, USA
OBJECTIVES: Despite its potential, the use of Generative AI in health economic modeling is still emerging. Leveraging Generative AI to streamline health economic model development could significantly benefit stakeholders by reducing the time and expertise required. Our objective was to evaluate the feasibility and accuracy of using Generative AI to develop a de novo health economic model.
METHODS: We used hepatitis C treatment as a case study for developing a de novo health economic model. Python was employed to interact with GPT-4 to develop a model structure for the natural history of hepatitis C by searching for published health economic models on PubMed. For parameter estimation, we utilized a multi-agent pipeline and OpenAI libraries. These parameters were then transferred to the R programming language's Heemod package to construct and run the Markov model. All of these steps were packaged into a customized solution, ValueGen.AI. The platform generated health states, transition probabilities, state and treatment costs, discount rates, cycle lengths, and time horizons. We compared the Generative AI-based model's structure, input parameters, and generated outcomes against expert opinion and published studies.
RESULTS: The Generative AI platform constructed a Markov-based model structure for hepatitis C with 10-15 health states and over 22 transitions between states. The platform also provided selected published models used as references. Additionally, the platform estimated values for transition probabilities, costs, and utilities, referencing publications for selected parameters. We repeated the experiment five times and observed variability in the model structure, attributed to variability in the referenced published models. Overall, the generated models had face validity.
CONCLUSIONS: We demonstrate the feasibility of developing a de novo Markov model for a chronic disease using Generative AI. Future research should focus on reducing variability in the model development process and comparing AI-generated model outputs with published models.
Patient Voices in Research: Exploring Information Needs, Online Behaviors, and Unmet Health Needs
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Understanding how patients seek, process, and utilize health information is crucial for effective healthcare delivery and support. This podium session brings together four studies that illuminate the complex interplay between patient needs, information-seeking behaviors, and health outcomes across a spectrum of conditions. It aims to inform more effective health communication strategies, enhance patient support systems, and guide future research and healthcare interventions.
Moderator
Maria Dutarte, MS
EUPATI, Utrecht, Netherlands
Maria Dutarte is the Executive Director of the European Patients’ Academy (EUPATI) since 2020. Maria has previously worked for the European AIDS Treatment Group (EATG) coordinating patient involvement in a number of training initiatives and scientific projects. She has also worked for different scientific and international organisations, e.g. French National Research Center for Scientific Research (CNRS), International AIDS Society (IAS), The Global Fund to Fight AIDS, Tuberculosis and Malaria and The International Foundation for Science (IFS). Her educational background is in International Education and Communications.
P30: Remote Monitoring in Heart Failure: Assessing Willingness-to-Pay Among Dutch Patients
10:30AM - 10:45AM
Menkveld R1, Klinkert I2, van der Schans S3, Van der Pol S4, Boersma C5
1Open University, Department of Management Sciences, Haren, GR, Netherlands, 2Rijksuniversiteit Groningen, Faculty of Economics and Business, Groningen, GR, Netherlands, 3Health-Ecore, Groningen, GR, Netherlands, 4Health-Ecore Ltd, Zeist, UT, Netherlands; University of Groningen, Department of Health Sciences, UMCG, Groningen, GR, Netherlands, Groningen, GR, Netherlands, 5Open University, Department of Management Sciences, Heerlen, LI, Netherlands; Health-Ecore Ltd, Zeist, UT, Netherlands; University of Groningen, Department of Health Sciences, UMCG, Groningen, GR, Netherlands
OBJECTIVES: Heart failure (HF) significantly impacts global healthcare. Remote monitoring, which allows for monitoring of patients in the home setting, has shown potential in improving HF management and reducing costs. This study examines factors influencing the willingness-to-pay (WTP) for telemonitoring among HF patients in The Netherlands, with focus on perceived usefulness, ease of use, social influence, and trust as predictors of perceived value and WTP.
METHODS: A survey was conducted in a group of 70 HF patients being monitored from a peripheral hospital. The survey explored how perceived usefulness, ease of use, social influence, and trust affected perceived value and WTP, based on the Theory of Planned Behavior (TPB) and Unified Theory of Acceptance and Use of Technology (UTAUT).
RESULTS: Perceived usefulness, ease of use, social influence, and trust did not significantly predict WTP. However, the analysis revealed a very strong positive significant correlation (0.97) between perceived value and WTP. Despite high perceived value and satisfaction with remote monitoring, patients generally exhibited low WTP. This discrepancy suggests that the Dutch healthcare system focus on universal access and minimal financial burden influences patient expectations, resulting in low WTP for remote monitoring. A regression analysis showed that the regression of the mediator perceived value on the dependent variable WTP, while controlling for overall trust, was significant (p = <0.001).
CONCLUSIONS: These findings highlight the importance of considering perceived value and the healthcare system context when evaluating patient WTP for remote monitoring. Further research should focus on trust and ease of use related to telemonitoring and alternative patient outcomes based payment models This knowledge is crucial for promoting adoption and sustainable financing of telemonitoring services to meet patient needs and ensure system sustainability.
P29: Australian Vasculitis Patients Informational Needs Questionnaire (AVPINQ)
10:15AM - 10:30AM
Trippe K
Kathryn Trippe, Balmain, Australia
OBJECTIVES: The purpose of the study was to explore the informational needs of patients, carers, and partners of people living with vasculitis in Australia.
METHODS: The questionnaire was developed by adaptation of the Vasculitis Specific Informational Needs Questionnaire (VINQ) and the Vasculitis Patient-Powered Research Network (VPPRN). A link to the Australian Vasculitis Patients Informational Needs Questionnaire (AVPINQ) was posted on the websites of Australian and New Zealand Vasculitis Society (ANZVASC) and LinkedIn.com. Patients responded to questions about their disease history, experience of patient education and source, and identified whether they wanted more information in any of the following areas: disease information, investigative tests, treatment options, physical support, psychological support, social support, employment support and financial support.
RESULTS: Seventy-eight questionnaires were completed and available for analysis. More than 90% of respondents (n=72) had not heard of their type of vasculitis before they received their diagnosis. 51% of respondents received information about vasculitis at the time of diagnosis. All respondents went on to source their own information following diagnosis. Despite accessing this additional information, 74.4% of respondents wanted information related to psychological state, 69.2% of respondents wanted more disease information, and 61.5% wanted to know more about their physical health. The order of preference of source of information was reported as verbal information from a doctor, written material alone, written material and a verbal conversation together, internet search, video, an educational group, verbal conversion with a nurse or clinical educator, and a 1–2-day course.
CONCLUSIONS: This study shows that Australian patients, carers and partners need information about vasculitis and the implications of living with this rare chronic condition. Patient education is an important aspect of disease management and patient-centered care. Developing specific vasculitis patient educational resources like those associated with more common chronic illnesses would benefit this patient group.
P31: Assessing the Unmet-Health-Related Needs of Inheritable Arrhythmogenic Cardiomyopathy Carriers: The UR-HEART Study
10:45AM - 11:00AM
Locquet P1, Reckelbus M2, Borry P2, Claessens Z1, Doevendans P3, Robyns T4, van Tintelen P5, Te riele A3, Van Steijvoort E2, Huys I1
1Department of Clinical Pharmacology and Pharmacotherapy, KU Leuven, Leuven, Vlaams Brabant, Belgium, 2Department of Public Health and Primary Care, KU Leuven, Leuven, Belgium, 3University Medical Center Utrecht, Utrecht, Netherlands, 4University Hospitals Leuven, Leuven, Belgium, 5University Medical Centre Utrecht, Utrecht, Netherlands
OBJECTIVES: As initiatives progress in the development of gene therapy for inheritable arrhythmogenic cardiomyopathies, this study, part of the GEREMY (Gene Therapy for treatment of rare inherited Arrhythmogenic Cardiomyopathy) project, is designed to gather insights into the unmet health-related needs of carriers. Needs range from impact on physical health and quality of care to impact on work and beyond. Results aim to tailor (gene therapy) drug development, healthcare settings and treatment decision-making to the needs of these patients.
METHODS: A literature review with a systematic search is being conducted in Pubmed and Embase, using “Arrhythmogenic Cardiomyopathy” and its synonyms to identify existing needs of these patients. Subsequently, a generic survey developed within the NEED (Needs Examination, Evaluation, and Dissemination) assessment framework is being disseminated among individuals diagnosed with Arrhythmogenic Cardiomyopathy at UZ Leuven (Belgium) and UMC Utrecht (The Netherlands). The survey is available in Dutch, French and English and includes 71 questions focussing on health-, healthcare- and social needs of patients. Descriptive statistics will be used to describe characteristics of participants. Several statistical tests will be performed on a selection of variables in order to identify potential relationships between demographic characteristics, clinical factors and the EQ-5D-5L utility scores.
RESULTS: The study has been initiated and data collection is currently ongoing. Full results will be presented on the conference.
CONCLUSIONS: This research forms the first phase of the UR-HEART (Understanding the preferences and unmet health-related needs of patients with arrhythmogenic cardiomyopathy) study, aiming to identify disease- and treatment characteristics that determine the needs of carriers and their willingness to make trade-offs between potential side-effects and anticipated benefits of current management and a future gene therapy.
P32: A Phenomenological Study Utilizing the Patient Experience Mapping Toolbox: Treatment Trade-Off Perceptions in Chronic Disease Patients
11:00AM - 11:15AM
Escontrias O1, Oehrlein E2, Schoch S1
1National Health Council, Washington, DC, USA, 2Applied Patient Experience, LLC, Washington, DC, USA
OBJECTIVES: The National Health Council’s, Patient Experience Mapping Toolbox (PEMT) assists health economics and outcomes researchers (HEOR) in the acquisition of patient experience data (PED) to understand patients’ experiences in the health care ecosystem from before they receive a diagnosis to living with their diagnosis. Generating an informed decision and determining which treatment options are best for chronic disease patients is multifaceted. To better evaluate treatment options in the context of patients’ priorities, goals, and unique circumstances, we asked patients what factors are important when deciding among treatment options.
METHODS: From June 2022-August 2022, twenty-five key informant interviews were conducted with patients across various chronic disease areas (i.e., depression, atrial fibrillation, rheumatoid arthritis, asthma, etc.). Each interview was one hour and conducted via Zoom. This phenomenological study employed an inductive qualitative analysis to ascertain the perspectives of patients. Themes were derived on two topics: 1) treatment tradeoffs and 2) what questions about treatments and tradeoffs are most important to ask a health care provider.
RESULTS: For Treatments and Tradeoffs, three themes of importance were identified: 1) Insurance coverage and out-of-pocket costs, 2) Side effects and risks, and 3) Treatment efficacy and effectiveness. As for the type of questions that other patients should be asking health care providers, four themes arose: 1) Coverage and financial coordination of treatments, 2) Comparison and options of treatments, 3) Treatment/Knowledge about root causes of the disease, and 4) Alternative non-pharmacological treatments.
CONCLUSIONS: Cost, insurance coverage, and side effects are of high importance to patients when considering different treatment options. When patients were asked what they would tell other newly diagnosed patients to ask providers, these themes continued with additional perspectives on determining what factors caused the condition. PED derived through patient experience mapping is a way for HEOR to incorporate critical patient views and questions in models.
Breakouts: IP, WS, & OBS
Global Waves: The Ripple Effect of EU Joint Clinical Assessment on Healthcare Worldwide
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Level: Intermediate
PURPOSE:
This workshop aims to explore the ripple effect of the European Union’s Joint Clinical Assessment (JCA) on health technology assessment (HTA) in non-EU countries. It will include perspectives from the global market, established HTA markets, and regions where reimbursement decisions often lag behind the EU.
DESCRIPTION:
The EU JCA is at the forefront of discussions within the HTA and payer communities, aiming to enhance efficiency and synergize the evaluation of new treatments across EU countries. Despite uncertainties in its implementation, the JCA's influence is expected to extend globally, as HTA reports often serve as references for agencies and payers worldwide. This workshop aims to highlight the broader impacts of the JCA, such as harmonization of standards, HTA method development, horizon scanning, and collaboration both within and across geographic regions. The panel will explore the complexities, opportunities, and expected changes from an international viewpoint. Dr. Yeh (chair) will provide an overview of JCA, highlighting the challenges and opportunities of using HTA evidence across borders. Dr. Vidal will discuss how JCA impacts a pharmaceutical company's global support for its country affiliates. Additionally, she will present findings from a recent survey examining the anticipated impact of JCA on 14 non-EU countries. Dr. Shum will offer insights from the perspective of a mature HTA market, detailing how HTA reports from other regions are utilized in Canada and the anticipated effects of JCA. Dr. Lin will share the perspective of an HTA submission reviewer in Taiwan, where reimbursement decisions for new treatments are generally delayed relative to the EU. She will discuss the current utilization of HTA reports from other countries in Taiwan and the potential use of JCA reports. The workshop will employ live polling for audience input on JCA’s ripple effect, leading to an interactive discussion to conclude the session.
Discussion Leaders
Danny Yeh, PhD
AESARA, Burlingame, CA, USA
Danny Yeh is the Head of the Center of Excellence within AESARA's Value and Evidence Team. With over a decade of experience in the pharmaceutical industry, Danny brings a wealth of knowledge to his role. Most recently, he held the position of Healthcare System Solution Cluster Lead, effectively serving as the Head of Government Affairs at Roche Taiwan.
Prior to his tenure at Roche, Danny spearheaded the establishment and leadership of the Health Policy and Systems Research team and Technical Institute at Genentech, which served as a distinguished center of excellence for Real-World Data (RWD) analytics and economic modeling capabilities.
Danny's professional journey also includes significant contributions within the Health Economics and Outcomes Research (HEOR) function at Biogen and Allergan. Throughout his career, Danny has played pivotal roles in supporting assets at various stages, from early phases to launch, across diverse disease areas. His expertise spans regions including the US, Asia Pacific, and Taiwan, where he has cultivated firsthand collaborations with key stakeholders such as clinical experts, regulatory bodies, payers, academia, policymakers, hospitals, and patient advocacy groups.
Discussants
Fang-Ju (Irene) Lin, PhD
National Taiwan University, Taipei, Taiwan
Dr. Fang-Ju Lin (Irene), MSCP, PhD, is an Associate Professor at the Graduate Institute of Clinical Pharmacy, National Taiwan University. She specializes in pharmacoepidemiology and pharmacoeconomics, focusing her research on the safety and effectiveness of medications in cardiovascular diseases, cardio-oncology, and chronic obstructive pulmonary disease. By utilizing a variety of healthcare databases, her work seeks to deepen insights into drug outcomes in complex clinical settings, supported by her extensive background in clinical pharmacy.
David Shum, PharmD, MBA, MSc
Hoffmann-La Roche Canada, Mississauga, ON, Canada
David Shum is the Director of Strategic Access & Pricing at Hoffmann-La Roche. In this role, he is responsible for delivering on outcomes related to HTA, pricing, and negotiations. Prior to this, David worked in healthcare at the Ontario Ministry of Health, Telehealth Ontario, University Health Network, and the Mayo Clinic. David holds a BSc in Pharmacy and MBA from UofT, PharmD from Wayne State University, and an MSc in Health Economics from the University of York, UK.
Anouchka Vidal, PharmD, MBA
F. Hoffmann-La Roche LTD, Basel, BS, Switzerland
Anouchka Vidal - Global/EU HTA Pipeline Lead, Roche | Anouchka Vidal is an experienced Pharm D having worked 10 years at Sanofi and 10 years at Roche, starting at the French affiliate where she held diverse access positions. In her current role, she ensures fit-for-purpose Global HTA strategies focused on the evidence needed to be generated to stay ahead of emerging health systems requirements trends and patient needs globally. Since August 2021 she has also contributed to a cross-functional Access SQUAD for external and internal EU HTA readiness. Her passion is working at the early stage of product development with internal and external partners to foster rapid, broad, and sustainable patient access to Health care innovations. Anouchka is guiding cardiovascular and metabolic portfolio-related HTA evidence strategies overseeing the above-country level with a specific focus on EU HTA. She has diverse disease area experience in oncology, diabetes, cardiovascular and metabolic diseases.
Lessons From Climate Change Models: What Can Health Economists Learn From Environmental Economists’ Modeling Methodologies?
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Level: Intermediate
PURPOSE:
Recent decades have seen accelerating global efforts to develop innovative technologies to address climate change and unmet health needs. Economic modeling (e.g., cost-effectiveness analysis, benefit cost analysis) is commonly used in both environmental and health economics to quantify the societal costs and benefits of innovative technologies; both fields share conceptual and technical challenges including dealing with uncertainty in the evidence base in modeling long-term impacts and the extent to which broader societal impacts (e.g., productivity) should be explicitly considered. The purpose of this workshop is to bring together environmental and health economists to share promising methodological approaches used to address the common set of challenges in long-term economic modeling, and identify areas of methodological collaboration to better quantify the societal costs and benefits of climate change-delaying interventions as well as novel health technologies.
DESCRIPTION:
Lotte Steuten will first summarize the significant societal burden from climate change and unmet health needs and outline a common set of challenges in economic modeling (10 minutes). Elizabeth Kopits will discuss how environmental economists address the key challenges in (e.g., discounting, treatment of uncertainty), highlighting promising approaches and areas for methods and data research (10 minutes). Jason Shafrin will draw upon recent advances in health economic modeling (e.g., generalized cost-effectiveness analysis framework) to describe how these methodological advances have allowed health economists to better quantify the impact of outcome uncertainty and long-run dynamics (10 minutes). Meindert Boysen will reflect the similarities and differences in both fields in addressing the common modeling challenges, and propose areas for future collaboration between the two fields (10 minutes). The panel will end with moderated audience Q&A (20 minutes). Polling questions will be used throughout to examine views on the extent to which methodologies widely used in environmental economics would be applicable in the health economics.
Discussion Leaders
Lotte Steuten, PhD
Office of Health Economics, London, LON, United Kingdom
Lotte Steuten, PhD, is Deputy Chief Executive of the not-for-profit Office of Health Economics (OHE) and the scientific and business lead for its international research-led work program—maintaining its reputation for objective, innovative, and high-quality research, and meeting its charitable goals. She is also an Honorary Visiting Professor at City, University of London (UK) contributing to its teaching programmes in health economics.
For more than 20 years, Lotte has worked in academic, research, and consulting HEOR roles with one focus — improving healthcare decision-making through high-quality research and analysis. To achieve that, she has collaborated with HTA authorities, pharmaceutical and medical device companies, patients, payers, policymakers, academic researchers, clinical specialists, and venture capitalists. She has gained broad HEOR expertise of various technologies: precision medicine, oncology, vaccines, antibiotics, diagnostics, digital health, and medical devices.
Lotte’s career has been based in the United States, the Netherlands, and the UK. Collaborations with HEOR experts in Southeast Asia, Africa, and Latin America have provided her with deep insights into the differences and commonalities between the role of HEOR in different healthcare systems, cultures, and societies. With this background, she has: led diverse international teams; developed strategies and delivered on program and organisational missions, values, and objectives; acquired research funding and managed budgets; and been responsible for executive decision-making as well as legal and fiduciary matters.
As an active ISPOR member since 2004, Lotte has served on various Taskforces and Committees, delivered Short Courses, was co-Chair of ISPOR EU 2021, and is currently a member of the Board of Directors, and an Associate Editor for Value in Health. She aims to be a role model for diversity and played an active role in the 'Women in HEOR' initiative.
Before joining OHE, Lotte worked in Seattle (US) as an Associate Professor at the University of Washington and at the Fred Hutch Cancer Research Center. She graduated cum laude with her PhD from Maastricht University (NL) and then worked at Brunel University (UK) and Twente University (NL).
Discussants
Meindert Boysen, PharmD, MSc
Boysen Consulting International, London, England, United Kingdom
Meindert was responsible at NICE for international partnership working, developing NICE’s role in international fora and representing NICE at key international meetings, further sustaining and developing NICE’s international reputation. Until recently, Meindert was the director at NICE responsible for delivery of the programmes that together form the centre for health technology evaluation. Including the technology appraisals, highly specialised technologies, medical technologies evaluation, diagnostic assessment and interventional procedures programmes. After qualifying as a pharmacist from Utrecht University, he worked in a hospital in the Netherlands, and held positions in health outcomes and sales in the pharmaceutical industry. Meindert completed the MSc in Health Policy Planning and Financing at the London School of Hygiene and Tropical Medicine and the London School of Economics & Political Sciences and briefly worked for the King’s Fund before starting at NICE in 2004. Meindert was a member of the Board of NICE and a director of the board of the International Society for Pharmacoeconomics and Outcomes Research (2017-2020).
Ben Groom, PhD
University of Exeter, London, LON, United Kingdom
Jason Shafrin, PhD
University of Southern California, Los Angeles, CA, USA
Jason Shafrin, Ph.D. is a Senior Managing Director at FTI Consulting's Center for Healthcare Economics and Policy. Dr. Shafrin has over 20 years of health economics research experience serving as trusted advisor and expert to a wide variety of healthcare and life sciences companies, governments and non-governmental organizations (NGOs). Dr. Shafrin is an Adjunct Professor at the University of Southern California, the former Director of Research at the Innovation and Value Initiative and the Founder and Editor of the Healthcare Economist blog.
From Methods to Implementation: Real-World and Regulatory Applications of Patient Experience Data Through Patient Experience Mapping & Patient Involvement
Digital Conference Pass
Level: Intermediate
PURPOSE: To educate health economics and outcomes research (HEOR) professionals on the Patient Experience Mapping Toolbox (PEMT), a set of publicly available resources to help researchers engage and document patients’ experiences, its real-world applications, and how it complements other patient initiatives.
DESCRIPTION: Patient experience data (PED) and patient engagement are critical to patient-centered outcomes in healthcare delivery research, drug development, and regulation. Meaningful patient engagement is bi-directional, and patients must be included as equal partners from the inception. Developed by patients, the PEMT can be used to collect disease-specific PED to inform a range of uses, including drug development and joint clinical assessments. To date, the PEMT has been applied to studies that ascertain patients’ treatment tradeoffs, as well as the experiences of diverse patients with food allergies and chronic kidney disease in the U.S. The use of this tool could be beneficial to broader global audiences. Through this workshop, HEOR-EU professionals will hear from patient engagement experts demonstrating applications of the PEMT and identify opportunities to enhance their patient-centered research methods.
Ms. Schoch will provide an overview of the PEMT and its methodology, including reviewing case examples. Dr. Oehrlein will demonstrate how the PEMT has been applied in real-world settings to build evidence for drug development. Dr. Mitchell will discuss the relevance of the PEMT to European settings, its potential applications, and how it complements European patient involvement initiatives. Dr. Gilardino will explain how the PEMT complements other patient and public involvement activities, including pilot projects with NICE and PBAC led by HTAi’s Patient and Citizen Involvement Groups, to implement an international template supporting patient submissions in HTA. This workshop will feature real-time polling via Slido, allowing participants to engage on using PEMT in global real-world studies and HEOR. There will be a 15-minute interactive panel discussion responding to audience Q/A.
Discussion Leaders
Silke Schoch, MA
National Health Council, Washington, DC, USA
Silke Schoch is the Director of Research & Programs at the National Health Council. Ms. Schoch joined the National Health Council in 2017 and has managed many of the NHC’s patient engagement focused projects including the Patient Experience Mapping Toolbox, Patient-Centered Core Impact Sets Blueprint, and Patient-Centered Value Classroom. Her interests include patient engagement, qualitative research, and gender equity-centered research. She has published in The Patient and Value in Health. Ms. Schoch earned a Bachelor of Arts degree in International Studies from American University in 2017 and a Master of Arts degree in Public Management from Johns Hopkins University in 2023.
Discussants
Ramiro Gilardino, MD, MHS, MSc
MSD, Dubendorf, ZH, Switzerland
Ramiro Gilardino is an impact-driven healthcare executive with over 15 years of leadership in Global Market Access, Health Policy, and HEOR. He has successfully led strategies that improved patient access, drove reimbursement success, and shaped health policy in pharmaceutical, biotech, and global organizations. An expert in advancing HTA frameworks and forging stakeholder partnerships, he is dedicated to promoting health equity and delivering value-based healthcare solutions globally.
Derick Mitchell, PhD
IPPOSI, Dublin, Ireland
Derick Mitchell, PhD. is the Chief Executive of the Irish Platform for Patient Organisations, Science and Industry (IPPOSI - www.ipposi.ie). IPPOSI is a unique, patient-led partnership
organisation that works with patients, government, industry, science and academia to put patients at the heart of health policy and innovation.
Derick has a background in research, strategy and advocacy from over 12 years’ experience spanning a number of leadership roles in research and multi-stakeholder engagement in Ireland and at the EU-level. Derick believes in the power of a united voice when it comes to patient advocacy and the central role of patients and their representative organisations in healthcare and research.
Derick is a board member of PFMD - a global collaborative and non-competitive coalition to improve global health by co-designing the future of healthcare for patients WITH patients. Derick is a former board member of the European Patients Academy (EUPATI) Foundation, a global initiative which is training patients to become involved in the medicines R&D process, and has spread the IPPOSI public-private partnership model to over 20 countries. Derick is also currently a Patient Advisor to the OECD Patient-Reported Indicator Survey (PaRiS) initiative as well as a founding member of the European Patients’ Forum-led DataSavesLives.eu initiative.
Derick holds a Bachelor of Science degree in Biotechnology from NUI Galway and a PhD in Molecular Medicine from University College Dublin. Outside of IPPOSI, Derick spends his free time attempting to influence his two young daughters.
Elisabeth Oehrlein, PhD, MS
Applied Patient Experience, LLC, Washington, DC, USA
Advanced Methods for Comparing Treatment Sequences or Pathways Using Real-World Data
Digital Conference Pass
Level: Experienced
PURPOSE:
With an ever-increasing number of treatment options, assessing the impact of changing treatment sequences/pathways has become crucial in health technology assessment (HTA). A major challenge is the scarcity of clinical effectiveness evidence, as trials typically do not compare treatment sequences. Real-world data (RWD) offers an alternative for capturing evidence on treatment sequences but analyses require appropriate statistical methods/assumptions to address time-varying confounding and immortal time bias. Descriptive treatment pattern analyses (e.g. Sankey diagrams) are useful but do not answer the counterfactual (i.e., what-if) questions required for HTA, particularly what would have happened had patients received alternative treatment sequences and the attributable effect of different lines of treatment (LOTs). This workshop provides an in-depth discussion on recent advances in treatment sequence evaluation in HTA, with tutorials on advanced methods for comparing treatment sequences using RWD.
DESCRIPTION:
Workshop attendees will review the latest developments in treatment sequence evaluation and be introduced to newly proposed methods using RWD. The audience will participate in real-time polling to consider the broader use of these methods in HTA. Prof. Latimer will chair the session, providing an overview (9 mins). Mr. Brooke will summarize NICE’s Pathways Project, which aimed to develop multi-use economic models that included treatment sequencing to appraise multiple LOTs within a single disease area and discuss requirements of RWD for sequencing (12 mins). Ms. Chang will provide a tutorial on using Target Trial Emulation and causal inference methods to adjust for time-varying confounding, to derive the comparative effectiveness of treatment sequences using RWD (12 mins). Dr. Adamson will address the challenges of long-term survival estimation in transportability studies due to treatment sequences and the utility of the Flatiron database for treatment sequence evaluation (12 mins). The workshop will conclude with an audience discussion on the priorities for treatment sequence research using RWD (15 mins).
Discussion Leaders
Nicholas Latimer, PhD, MSc
Delta Hat Ltd, Nottingham, UK; University of Sheffield, Sheffield, Great Britain
Nick is a Professor of Health Economics at the University of Sheffield, a Yorkshire Cancer Research Senior Fellow, and an Analyst for Delta Hat Ltd. He was a member of NICE Technology Appraisal Committee B for 5 years and is a member of the NICE Decision Support Unit (DSU). Nick’s research focuses on survival analysis in economic evaluations. He has authored four DSU technical support documents related to survival analysis, treatment switching, and partitioned survival models. His current work involves investigating the use of cancer registry datasets to estimate the comparative effectiveness of cancer treatments used in clinical practice.
Discussants
Blythe Adamson, PhD, MPH
Flatiron Health, New York, NY, USA
Blythe Adamson is an epidemiologist and economist accelerating the development of valuable medicines and health policy. She serves as Head of Outcomes Research and Evidence Generation at Flatiron Health in New York. Dr. Adamson advances artificial intelligence applications of natural language processing and machine learning in the curation of electronic health records for real world evidence with a team of engineers, scientists, and clinicians. She designs treatment comparative-effectiveness studies, codes mathematical models of disease, and delivers research for global health technology assessment. Dr. Adamson served as a former member of the White House COVID Task Force on Healthcare Resilience while lead pandemic data scientist in the West Wing. She is an Affiliate Professor at the University of Washington in the Comparative Health Outcomes, Policy, and Economics (CHOICE) Institute. Dr. Adamson has held roles at the Bill and Melinda Gates Foundation Institute for Disease Modeling, Infectious Economics, the NIAID HIV Vaccine Trials Network, and Fred Hutch Cancer Center. She continues to serve as a leader in the International Society for Health Economics and Outcomes Research (ISPOR). Dr. Adamson holds degrees in pharmaceutical economics, public health epidemiology, and microbiology. She enjoys writing about the economics of infectious diseases at www.blytheadamson.com.
Adam Brooke, MSc
The National Institute for Health and Care Excellence (NICE), Manchester, United Kingdom
Adam Brooke is the senior technical advisor for methods for NICE's medicines evaluation team. His main interests include creating and maintaining disease-specific reference models, incorporation of routinely collected real world evidence in appraisals and sequencing of treatments.
Jen-Yu Amy Chang, PhD, MSc
University of Sheffield, Sheffield, YOR, Great Britain
Amy is a Research Associate at the University of Sheffield and a Health Economist at Lumanity. She holds a PhD in Health Economics and an MSc in Clinical Pharmacy. Amy has experience in health economics and pharmacoepidemiology research, as well as actively supporting local HTA submissions through health economic modeling and evidence generation. Her extended expertise includes Target Trial Emulation/Real-world study design and execution, fit-for-purpose real-world data scoping, and advanced survival analysis, particularly using advanced causal inference statistical methods for comparing treatment sequences/treatment-switching.
10:30 - 13:30
Poster Session 3
Live
11:15 - 13:15
Lunch
As you enjoy your lunch in the Poster and Exhibit Hall, seize the opportunity to engage in meaningful conversations with fellow attendees. Take this time to exchange ideas, forge new partnerships, or simply enjoy causal conversation. Provided by ISPOR
11:30 - 12:00
Fast Facts
Professional Development in the Times of HEOR Evolution
Join us in an inspiring conversation about your professional development in these unprecedented but promising times.
As both RWE and HEOR functions are dramatically evolving, we host a unique session that will allow you to develop concrete ideas about your own professional career. Leverage our experience and participate in the conversation, whether you are an expert or a leader, just starting your career or planning a next reasonable move. In this session we will review practical strategies which successful leaders and well-known experts use to grow and shape their profiles; we will share key success factors for career-development and address your questions and comments.
Discussion Leader
Natalia Scherff
Coach Scherff, Frankfurt, HE, Germany
Natalia is an independent ICF certified career and leadership coach, working predominantly in the Pharmaceutical Industry across the Globe.
She has 20+ years of experience in corporate talent management, top executive search consulting firms, Russell Reynolds Associates, leadership assessment, and career development coaching.
For the last 4 years, while working for Novartis, Natalia has held strong reputation of the SME in executive recruitment for Value & Access, focusing on Global & Countries Access & HEOR leadership roles.
Natalia has two Business Master‘s Degrees obtained in Moscow and Frankfurt, and she is fluent in English, Russian, and German.
11:30 - 12:15
Poster Tours
Student Research Poster Tour
Posters Featured in this Tour:
PT31 Reducing Health Inequality of Introducing SGLT2i Therapy in a Real-World Type 2 Diabetes Population With Diverse Patient Characteristics: A Distributional Cost-Effectiveness Analysis
PT32 Economic Evaluation of Abdominal Aortic Aneurysm Screening in the Czech Republic
PT33 Cost-Effectiveness of Adjuvant Radiation Therapy Versus Tamoxifen Alone in Older Adult Women With Early-Stage Breast Cancer
PT34 Evaluating the Healthcare Burden and Clinical Impact of Polypharmacy Among the Elderly in South Korea
PT35 Are Social Impact Bonds the Answer to Limited Healthcare Resources for Improving Health Promotion and Disease Prevention?
PT36 Assessing the Impact of Vision Impairment on Health-Related Quality of Life Among Community-Dwelling Older Population in Hong Kong: Preliminary Results
Moderator
Zeba Khan, RPh, MS, PhD
Center for Health Outcomes, Policy & Economics, Ernest Mario School of Pharmacy, School of Public Health, Rutgers University, Piscataway, NJ, USA
Dr. Zeba Khan is currently the Founder and CEO of Zebgene LLC, and the Interim Program Director for Rutgers University Center for Health Outcomes, Policy, and Economics (HOPE) at the Ernest Mario School of Pharmacy. She has more than 25 years of experience establishing and leading multi-disciplinary corporate functions for biopharmaceutical companies including health economics and outcomes research, market access, pricing and reimbursement, public policy, corporate responsibility, and global health. Zeba holds a PhD and MS in Health Economics and Health Outcomes from The University of Texas at Austin. Zeba graduated with a Pharmacy degree from the University of Utah.
Kanya Shah, PhD, PharmD, MBA, MS
University of Illinois Chicago College of Pharmacy, Chicago, IL, USA
Kanya Shah is currently a Ph.D. student at the University of Illinois Chicago (UIC) in the Pharmacy Systems Outcomes and Policy department. She previously earned a Doctor of Pharmacy (PharmD), Master of Science (MS) in Health Outcomes, and Master of Business Administration (MBA) from the University of Rhode Island.
Joint Clinical Assessment Poster Tour
Posters Featured in this Tour:
PT25 Potential for Standardization in Economic Assessment Across HTA Bodies: A Case Study in First-Line Treatments of Non-Small Cell Lung Cancer
PT26 From Clinical Assessment to Drug Reimbursement in Spain: Current Timelines and Potential Impact From the Joint Clinical Assessment
PT27 Time to Reimbursement in Ireland: Utilizing Regression Analysis to Assess Factors Contributing to Overall Timelines
PT28 Do We Say “Game Over” When OS Data Is Not Mature at Launch? A Review of Value Drivers for Oncology Products in the Absence of Significant OS Gains
PT29 From European Union Joint Clinical Assessments to Local Health Technology Assessments: An Environmental Scan of Methodological Guidance Across Key European Markets
PT30 Navigating the New European Member State Coordination Group on Health Technology Assessment Quantitative Evidence Synthesis Guidelines
Moderator
Kejsi Begaj, PharmD
Rutgers University, New Brunswick, NJ, USA
Alin Kalayjian, PharmD, MBA
GSK, Somerset, NJ, USA
11:45 - 12:15
Exhibit Hall Theater
Planning With Payer Insights: Even the Best Economic Models Need Validation
Digital Conference Pass
In today's healthcare payer landscape, characterized by evolving pricing thresholds and Health Technology Assessment (HTA) requirements in markets where economic models are a critical element of a submission, a well thought model concept with appropriate input parameters for model implementation are critical to increase the likelihood of success and obtain a favorable reimbursement decision.
Economic models play a crucial role in demonstrating the value of a new product at launch in HTA markets. To accurately illustrate incremental value of a new product relative to the standard of care (i.e., comparator), and evaluate its cost effectiveness in a specific population, health economists must navigate through many input parameters to implement in the economic model, as well as conceptualize the model framework, optimal for the product at hand.
This HEOR Theater presentation will explore the benefits of an integrated, cross-functional approach that incorporates payer insights upfront to inform and validate economic model framework and inputs from various sources which include clinical trials and real-world evidence. The session will demonstrate how this approach of planning for payer insights at various stages of economic model development enhances the likelihood of success and drives impact in reimbursement decisions. Speakers from the economic modeling consultancy and life sciences industries will share examples illustrating this concept.
By uniting economic modeling methodology and practical payer perspectives, the presenters aim to provide a comprehensive framework for developing robust economic models that stand up to scrutiny in the access and reimbursement environment. This approach will assist life sciences companies to avert the scenarios of heavy criticism and illustrate how insight-based economic model development may have helped.
Sponsor: Genesis Research Group
Sponsor
Genesis Research Group
Moderators
Priti Jhingran, PhD
Genesis Research Group, Hoboken, NJ, USA
With over 3 decades of experience working in the pharmaceutical industry, including consulting, Dr. Jhingran’s passion, energy, forward thinking approach, and willingness to learn new things make her a great fit to be the strategic leader for this engagement. Dr. Jhingran has focused her efforts to understand evidence needs and deliver tools/solutions, such as economic models, objection handlers, for access decision makers/HTAs in US and exUS markets. She has facilitated and led multiple enterprise level initiatives focused on value, evidence, and outcomes. She brings extensive launch support experience with 15+ pharmaceutical product launches in key markets. Dr. Jhingran has led, managed, and developed diverse teams of Health Outcomes scientists dedicated to generation, dissemination, and communication of evidence supporting optimal reimbursement/access decisions for pharmaceutical products. She is lead/participated in multiple external workshops and presentations, which include the ISPOR Short Course on US Payers as well as the HEOR Theatre Presentation at 2022 EU ISPOR. She has published in the areas of health economic evaluation, health policy,and health services research with over 75 peer-reviewed populations.
Speakers
Dominic Jones-Phillips, PhD
Genesis Research Group, West Orange, NJ, USA
Smita Kothari, PhD, MBA
Merck & Co. Inc., Rahway, NJ, USA
Carole Longson, MBE
Carole Longson Consultant, Manchester, United Kingdom
Carole is a respected leader in health technology assessment with considerable achievements in research, public and private sectors. She has unparalleled insight into the health technology innovation pipeline having pioneered the development of NICE’s health technology evaluation programmes. After a career in drug discovery at GSK, Carole was an Executive Director at NICE from 2000-2018 and Chief Scientific Officer at the Association of the British Pharmaceutical Industry from 2018-2020. She was formerly President of Health Technology Assessment International and now is an independent advisor including Life Science Advisor at NICE.
Carole is currently vice chair of the Medicines Discovery Catapult in the UK, was previously on the Scientific Advisory Committee for the EU Innovative Medicines Initiative (IMI) and holds non-Executive Director and advisory roles in the UK and abroad. Throughout her career, she has championed the public and private sector collaborating to ensure medical innovation benefits patients in a way that is sustainable for healthcare systems.
11:45 - 12:45
Member Group Meetings
ISPOR Real-World Evidence (RWE) Special Interest Group
The Real World Evidence Special Interest Group invites you to join an Open Meeting to connect with the group’s leadership team and representatives from the European Medicines Agency. Engage in dynamic breakout groups with your peers to explore collaboration opportunities, brainstorm future member engagement activities, and develop ideas for conference presentations.
ISPOR Turkey Chapter Meeting
ISPOR Forums
Current Landscape and Future Directions of Structured Expert Elicitation in Healthcare Decision Making
Digital Conference Pass
Structured expert elicitation (SEE) is increasingly recognized as a critical tool for informing healthcare decisions, especially in scenarios where empirical data is scarce or unavailable, which is often the case within oncology. However, the challenge lies in conducting SEE exercises that ensure credibility, accuracy, consistency, and transparency of expert judgments. This forum, jointly hosted by the ISPOR Oncology Special Interest Group and the SEE for Healthcare Decision Making Task Force, invites methodology experts, industry professionals, and Health Technology Assessment (HTA) bodies to a comprehensive discussion on the current landscape and future directions of SEE in HTA.
This interactive forum will provide attendees with invaluable insights into best practices and emerging developments in SEE, specifically tailored for HTA submissions. Min-Hua Jen will open the session by providing a concise overview of SEE and highlighting the necessity of high-quality SEE for HTA submissions from an industry perspective. Salah Ghabri will present an update on the ISPOR Good Practice Report, outlining key recommendations for the use of different SEE protocols in healthcare decision making. Kate Ren will introduce new developments in SEE specifically designed for long-term survival outcomes, focusing on innovative methodologies that ensure coherent estimates of long-term survival with meaningful expert rationale, thereby addressing a critical component of economic modelling in HTA. Dawn Lee will share her practical experiences of conducting SEE, providing real-world insights and lessons learned.
The session will conclude with an interactive Q&A segment, allowing attendees to engage directly with the experts, and discuss specific challenges and considerations in the application of SEE, such as estimating conditional outcomes, defining target populations for estimation, handling the impact of subsequent treatments on overall survival, and integration into subsequent modeling efforts.
Moderators
Min-Hua Jen, PhD
Eli Lilly and Company, Uxbridge, LON, United Kingdom
Dr Min-Hua Jen is currently Senior Director - Real-World & Access analytics at Eli Lilly, leading the International Business team on Market Access/HEOR/Medical affairs statistical support. She has extensive experience applying statistics to clinical research, epidemiology and health economics and outcomes research in academia and industry settings. She is an active member in the PSI/EFSPI HTA Special Interest Group (SIG) and the engagement chair of the ISPOR Oncology SIG. She was trained in Epidemiology and Statistics and obtained her PhD at University of Bristol. Her research interests including indirect treatment comparisons and network meta-analysis; particularly incorporate external data for time to event outcomes, surrogacy analyses, multilevel modelling and health economic modelling.
Speakers
Salah Ghabri, PhD, HDR
Haute Autorité de Santé (HAS), Paris, 75, France
Senior Health economist Researcher and scientific referent in economic evaluation - Department of Medical Evaluation - French National Authority for Health (HAS) since 2013. My research interests revolve around economic evaluation of health technologies and health programs, analysis of uncertainty and health economic modelling and budget impact analysis.
Dawn Lee, MMath, MSc
PenTAG, University of Exeter, Exeter, DEV, United Kingdom
Dawn Lee is an Associate Professor of Health Economics NICE Evidence Assessment Group PenTAG. Prior to her current role Dawn spent 12 years in consultancy working her way up from a junior health economist to Chief Scientific Officer at BresMed. As an EAG member Dawn led the production of the model and assessment report for NICE’s pathways pilot appraisal of advanced renal cell carcinoma (aRCC) and a recent NICE pilot for late stage assessment of medical technologies. Dawn's key interest include survival modelling, immune-oncology evaluation, structured expert elicitation, sequencing models, surrogate endpoints and cancer diagnostics.
Kate Ren, PhD
School of Health and Related Research, University of Sheffield; ConnectHEOR, Sheffield, NYK, United Kingdom
Kate Ren is a Senior Research Fellow in Statistics at the University of Sheffield and also the Director of Statistics at ConnectHEOR. She is a member of SCHARR-TAG (an external assessment group for NICE technology appraisals), and has extensive experience in critiquing statistical methods used in NICE single technology appraisal submissions. She is also a member of NICE Appraisal Committee C. Her research interests include indirect treatment comparisons, survival extrapolation and structured expert elicitation.
Go Broad: The Application of Indirect Burden of Disease Evidence in US Value Assessment and Ex-US HTA Submissions
Digital Conference Pass
Rare diseases often require a significant amount of medical care which leads to a higher burden of direct medical costs. Additionally, indirect costs such as productivity loss, absenteeism, lifestyle disruptions, and family and societal impacts, contribute to the overall burden of patients with rare disease and their caregivers. However, these broad indirect impacts are less understood and not often measured and thus burden of disease estimates utilized in evidence for value assessment and ex-US HTA submissions are thought to be underestimating the total burden of disease.
Laura Housman will moderate the panel and provide a 5 outline of the issue including the difference between direct medical costs, non-medical costs, and indirect costs. Taylor Schwartz will provide a 10 minute overview of recently completed studies that utilized novel methodologies to measure non-medical costs and the broad indirect burden of disease as components of the overall burden of disease. He will also provide a comparative perspective as to how this information can be utilized by US value assessment and payers. Jan McKendrick will spend 10 minutes discussing providing a comparative perspective as to how measuring the indirect burden of disease can be useful as part of the evidence submissions in ex-US HTA submissions and how these evidence requirements may vary from country to country. Karl-Johan Myren will spend a 10 minutes sharing the perspective of the manufacturer on the pragmatic application of utilizing data on the indirect burden of diseases in EU HTA submissions. and the importance of indirect disease burden measurement for patient-centered drug development.
Moderators
Laura Housman, MPH, MBA, DrPH(c)
Avalere, Washington, DC, USA
Laura T. Housman, DrPHc (ABD), MBA, MPH is Senior Vice President and Practice Director of Evidence and Strategy within Avalere Health, bringing broad and extensive expertise in strategy, evidence generation, reimbursement, patient engagement, population health and personalized medicine to drive access to innovation across healthcare. Laura received her Bachelor of Arts degree in Economics from the College of Arts and Sciences and a Minor in Business Administration from the School of Management from Boston University; her Master of Public Health from the Boston University School of Public Health; and her Master of Business Administration with honors from Boston University’s Questrom Graduate School of Management. Laura is currently ABD as a Candidate in the Doctor of Public Health (DrPH) program at Boston University School of Public Health.
Speakers
Jan McKendrick, BSc MSc PGDip
Avalere Health, London, LON, United Kingdom
Jan has more than 30 years’ experience in the pharmaceutical industry starting as a clinical trial statistician then moving to market access / HEOR. Jan has extensive experience in developing HTA submissions and in providing strategic input into projects, particularly around the effective generation and use of evidence to support the value of products. In her current role, she leads the Global HEOR practice – working with the team to design and deliver a range of projects including high quality economic modeling and both strategic and tactical RWE projects.
Karl-Johan Myren, MSc
Alexion AstraZeneca Rare Disease, Stockholm, Sweden
Karl-Johan (Kalle) Myrén heads up the International EUCAN HEOR team at Alexion AstraZeneca Rare Diseases. Kalle has been in his role for 6 years and leads a team of experienced health economists and outcomes researchers across the region. Prior to joining Alexion, Kalle has had leading HEOR and market access roles in industry and consulting spanning over 25 years.
Taylor Schwartz, MPH
Avalere, Washington, DC, USA
Taylor is a Managing Director at Avalere where he leads US HEOR/RWE engagements. As an epidemiologist by training, Taylor has spent his career in HEOR collaboratively working with clients to conceptualize, design, and execute epidemiological and health economics and outcomes research across a variety of therapeutic areas. Taylor also conducts strategic evidence generation planning and co-leads Avalere's US Value Assessment subject matter expertise team.
Using Health Preference Methods for Value Clarification in Patient Decision Support: Current Use and Future Developments
Digital Conference Pass
The International Patient Decision Aid Standards recommends that patient decision aids (PtDAs) include a value clarification method (VCM). The audience will learn about the current use of MCDA and other preference elicitation methods as VCMs, their feasibility and effectiveness in practice, and about novel applications that may overcome some of the limitations of frequently used health preference methods, such as discrete choice experiments (DCEs).
Dr. van Til will provide examples of frequently used methods identified in the SIG's systematic literature review and discuss how attribute importance and preferences for options are quantified. Interactive polling will be used to elicit attendees’ attitudes about using preference elicitation methods as VCM, then compare attendee attitudes with the evidence in literature.
Dr. Poulos will discuss development of a VCM using object-case best-worst scaling to support patient decision making on treatment of primary immunodeficiency diseases. She will describe how the formative research process influenced the design of the PtDA.
Ms. Thomas will outline how multi-dimensional thresholding can be used as a VCM with patient-specific outputs provided in a PtDA. She will walk through an example of the process for a patient and the types of outputs that could be provided.
Speakers will address the potential of preference elicitation methods as a VCM, areas requiring additional research (eg, recommending a treatment option based on preferences), and challenges (eg, in generating individual preference estimates with some methods). Audience discussion will follow the case study examples. Presented by the ISPOR Health Preference Research Special Interest Group
Moderators
Janine van Til, PhD
University of Twente, Enschede, Netherlands
Janine is a health scientist by training. She is currently an associate professor at the Technical Medical Center at the University of Twente. She has more than 15 years of experience in the design, conduct and analysis of health preferences studies and multi-criteria decision analyses. Her research interest include the use of health preference methods to support shared decision making, development of individual patient value clarification tools, the optimal design of patient decision aids for patients with low health, numerical and verbal literacy and the study of role preferences of different stakeholders in clinical and societal decision making.
Speakers
Christine Poulos, PhD
RTI Health Solutions, Research Triangle Park, NC, USA
Christine Poulos, PhD is Senior Economist and Vice President of Health Preference Assessment at RTI Health Solutions, Research Triangle Park, NC, USA. She has more than 20 years of academic, research, and government experience in health and environmental economics. She is an expert in the design of stated preference surveys and she has extensive experience in applying stated preference methods to measuring the benefit-risk preferences of patients and other health care decision makers. Her most recent work has applied these studies to support decision making throughout the clinical product development cycle for treatments for conditions including severe emphysema, Alzheimer’s’ disease, multiple sclerosis, endometriosis, type 2 diabetes, and infectious diseases. She is currently Chair for the Health Preference Research Special Interest Group of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR). Her research has been published in numerous health outcomes and medical journals.
Caitlin Thomas, MSc
Evidera, London, LON, United Kingdom
Caitlin is a Research Scientist within the Patient Centered Research team at Evidera. She leads preference studies globally across a range of disease areas and with multiple purposes, such as supporting regulatory and reimbursement decision-making. She is part of the ISPOR HP SIG, working on a project that aims to synthesize insights into how health preference methods are being used to support individual value clarification in shared decision-making. She collaborated on the development of a tool, StudyGage, that uses preference research to understand the impact that a study design has on willingness to participate in clinical trials. Caitlin has an MSc in health economics and decision science from the University College London and a BSc in economics from the University of Bath. Her work has been published in international peer-reviewed journals.
Educational Symposia
Missed Signals: The Economic Implications of Current Health Technology Assessment Protocols for Diagnostics
Digital Conference Pass
Health Technology Assessment (HTA) plays a crucial role in evaluating the benefits of medical technologies and determining their value in the healthcare ecosystem. From diagnostics to therapeutics, HTA systematically assesses value attributes and supporting evidence. However, assessing diagnostic technologies presents unique challenges due to their limited direct impact on health outcomes.
This educational symposium aims to delve into the methodologies, processes, and results related to HTA assessment specifically for diagnostic imaging technologies. We will review the results from research and explore whether existing methodologies adequately capture the full value of these diagnostic technologies.
After the presentation, a panel with stakeholders representing different perspectives will engage in a debate to discuss the benefits and drawbacks of different HTA approaches for diagnostics to identify the suitability of current frameworks and areas for improvement.
Join us as we explore the intersection of HTA, evidence, and healthcare outcomes in the context of diagnostic imaging.
Sponsor: GE HealthCare
Sponsor
GE Healthcare
Moderators
Ricardo De La Fuente, PharmD, PhD, MBA
GE Healthcare, Hampton, NH, USA
Global market access professional with extensive global experience leading drug and diagnostic technologies from development to commercialization, committed to ensuring patient access to new and innovative technologies.
International experience in obtaining reimbursement focused on value demonstration at unprecedented price levels. Proven track record bringing new technologies from clinical development to commercial.
Speakers
Montserrat Chivite, MS
GE HealthCare, Madrid, Spain
Montserrat Chivite is the International Market Access Lead at GE Healthcare’s Pharmaceutical Diagnostics division. With 20 years in the pharmaceutical industry, she has a solid track record in driving strategic initiatives and global market expansion. Montserrat has held roles in consultancies and pharma companies, developing effective access strategies in pricing and reimbursement worldwide. She holds a degree in Communication/Economics and a Master’s in International Relations. Her expertise spans oncology, neurology, and cardiovascular areas, focusing on both prevalent and rare diseases.
Alistair McGuire, BA, MLitt, PhD
London School of Economics, London, United Kingdom
Professor Alistair McGuire is Head of Department and Chair of Health Economics at the Department of Health Policy. Prior to this he was Professor of Economics at City University, London after being a tutor in Economics at the University of Oxford. Professor McGuire has also been a Visiting Professor at Harvard University, the University of Sydney, the University of York, and the Universitat Pompeu Fabra, Barcelona.
He has acted as an advisor to a number of governments and governmental bodies, including the United Kingdom government, the UK Competition Commission, the UK Medical Research Council (MRC), the German Institut fur Qualität Wirtschaftlichkeit im Gesundheitswesen (IQWiG), as well as for a number of international bodies (including the World Bank, the WHO, and the IMF) and pharmaceutical and health care insurance companies.
Professor McGuire has pursued an interest in the economics of health care for over thirty years. His research cover all topics in health economics, including international comparisons, economics of the hospital, public/private sector interface, cost-effectiveness analysis of health technologies, and health economics alongside clinical trials. He also has extensive teaching experience and has written numerous books, articles and reports on this. He has been involved in a number of major clinical trials as the lead health economist.
Alexander Natz, LLM
European Confederation of Pharmaceutical Entrepreneurs (EUCOPE), Brussels, Belgium
Alexander Natz is the Secretary General of the European Confederation of Pharmaceutical Entrepreneurs (EUCOPE) in Brussels and advises innovative pharmaceutical and biotech companies, including start-ups, in regulatory and pricing & reimbursement matters from the EU law and German law perspective.
From 2008 to 2013, he was Head of the Brussels Office of Bundesverband der Pharmazeutischen Industrie e.V. (BPI). Before, he worked as a lawyer at Sträter Law Firm in Germany with a special focus on managed entry agreements and licensing of pharmaceuticals. Dr. Natz has also worked in the field of competition law with the European Commission and in the pharmaceutical industry. As a research assistant at Duke University (USA) he has dealt with international pharmaceutical law. His doctorate was supervised by the former judge at the European Court of Justice, Prof. Dr. Ulrich Everling.
Laura Sampietro-Colom, MD, PhD
Hospital Clínic Barcelona, Barcelona, Spain
Dr Laura Sampietro-Colom is the Deputy Director of Innovation and Head of the Unit for the Assessment of Innovations and New Technologies at the Hospital Clinic of Barcelona (Spain). In her everyday work she is supporting clinicians to prove the value for their clinical practice of both health care technologies they are developing and for those new technologies that want to be introduced in the Hospital. She is also involved in Innovative Public Procurement and Value Based Purchasing with doctors and procurers at hospital. She is currently the President of AQuAS, the Catalan Agency for HTA and Quality
Advancing Evidence-Based Medicine With Generative AI (GenAI)
GenAI is revolutionizing HEOR and RWE by offering innovative methods to process and analyze vast datasets, model complex health economics phenomena, and enhance decision-making processes. GenAI tools powered by large language models (LLMs) facilitate the development of a deeper and more comprehensive understanding of diseases and treatment outcomes, and significantly improve the extraction of insights from diverse data sources such as electronic health records, claims data, and scientific literature. By harnessing the power of GenAI, researchers and practitioners in the field are unlocking new possibilities for advancing health care innovation and improving patient care. Participants in this symposium will explore the frontiers in GenAI, discuss key developments and challenges, and present examples of GenAI’s applications in HEOR and RWE research. Among these examples is Analysis Group’s own proprietary GenAI platform, which excels in text classification, research summarization, and rapid data analysis. GenAI can also streamline the automated screening of research published in various languages by providing accurate translations and summarizations, thereby informing decision making with comprehensive insights across diverse sources. These GenAI-powered capabilities have greatly improved the efficiency of HEOR and RWE research and offered powerful and creative insights into health care data and literature. The introduction of this and similar GenAI platforms is set to further empower the health care sector, offering more effective and efficient tools to researchers for streamlining complex analyses, enhancing research accuracy, facilitating evidence-based decision making, and deepening overall understanding of complex diseases and treatments, ultimately leading to more effective health care solutions and policymaking.
Sponsor: Analysis Group
Sponsor
Analysis Group
Moderators
Eric Wu, PhD
Analysis Group, Inc., Boston, MA, USA
Dr. Wu is a health economist with expertise in health economics and outcomes research (HEOR), market access,
real-world evidence (RWE) generation, AI applications, and scientific evidence strategy. He has conducted
research in more than 30 countries and contributed to over 300 publications. Dr. Wu focuses a significant portion
of his practice on developing new scientific methods to address challenges in medical big data, RWE, AI, and
innovative comparative-effectiveness research methodologies.
Speakers
Rajeev Ayyagari, PhD
Analysis Group, Inc., Boston, MA, USA
Dr. Ayyagari has experience applying statistical techniques and AI to the study of clinical and economic outcomes in a broad range of disease areas. His recent work includes using AI-based approaches in literature reviews and leading the development of software tools to communicate the economic value of therapies.
Jimmy Royer, PhD
Analysis Group, Inc., Montreal, QC, Canada
Jimmy Royer, PhD
Dr. Royer applies a broad range of AI and GenAI tools to address client needs in data science and HEOR. His recent work includes using AI models to predict treatment resistance and the potential future onset of rare or undiagnosed conditions. In addition, Dr. Royer has conducted extensive academic research and coauthored books and papers on using neural networks to predict treatment resistance in tuberculosis, using new AI advances in HEOR, using machine learning algorithms in propensity score models, and determining the impact of hypertension therapies on mortality, among other topics.
Song Wang, PhD
Takeda, Singapore, Singapore
Song represents Takeda in regional and global evidence generation. With over 15 years of expertise in health economics, real-world evidence (RWE), and new product planning (NPP), Song has extensive knowledge and experience in building and developing strategic approaches, functional structures, local evidence generation capacity and capability, and collaborative networks. These efforts advance priorities at the global, regional (APAC, LATAM, EMEA), and local (USA, China) levels.
Eric Wu, PhD
Analysis Group, Inc., Boston, MA, USA
Dr. Wu is a health economist with expertise in health economics and outcomes research (HEOR), market access,
real-world evidence (RWE) generation, AI applications, and scientific evidence strategy. He has conducted
research in more than 30 countries and contributed to over 300 publications. Dr. Wu focuses a significant portion
of his practice on developing new scientific methods to address challenges in medical big data, RWE, AI, and
innovative comparative-effectiveness research methodologies.
12:15 - 12:45
Exhibit Hall Theater
Time to Rethink Your Global Launch: Evidence, Access, Digital, Indication and Market Sequencing
Digital Conference Pass
Global launches require sophisticated plans which aim to prioritize indications and order of entry for countries. A clear concise integrated evidence plan allows for more informed decision making leading up to launch. Launch strategies can be further streamlined and enhanced using digital solutions inclusive of AI. Guidehouse provides a solution to bring together evidence and access to deliver insight on where and how to launch.
Sponsor: Guidehouse
Sponsor
Guidehouse
Speaker
Karla Anderson, BS, RDN
Guidehouse, London, United Kingdom
Andy Longosz, MPH, MBA
Guidehouse, Washington, DC, USA
Andy Longosz is a Director and Global Pricing and Access Lead in the Life Sciences practice at Guidehouse. His experience includes a variety of cross-functional projects spanning global geographies and multiple therapeutic areas. Prior to joining Guidehouse, Andy served as an Associate Principal at IQVIA within the Value & Access Practice. He also worked at the World Bank, National Institutes of Health, Johns Hopkins Hospital and Wiley Publishing. Andy received his B.S. in Kinesiology, Health Sciences, Chemistry and Pre-Med from the College of William and Mary. He also received an MPH from the Johns Hopkins Bloomberg School of Public Health and an MBA from the Johns Hopkins Carey Business School.
12:30 - 13:15
Special Onsite Join / Renewal Event for ISPOR Membership
Special Onsite Promo! Stop by the ISPOR Booth to join or renew your ISPOR membership! We might just have a surprise for you...
12:45 - 13:15
Exhibit Hall Theater
Using Generative AI Methods for Evidence Generation and Health Technology Assessment: Perspectives From NICE and Industry
Digital Conference Pass
Level: Intermediate
The capabilities of generative AI, particularly Large Language Models such as ChatGPT are evolving rapidly, and present new opportunities and challenges for evidence generation and healthcare decision making. This session will present an overview of the state of the art in using generative AI for real world evidence generation, and discuss the implications of using AI enabled evidence in health technology assessment, drawing on perspectives from NICE and industry.
Sponsor: LCP Health Analytics
Sponsor
LCP Health Analytics
Speaker
Benjamin Bray, MD, MSc, FFCI
LCP Health Analytics, London, United Kingdom
Emma Clifton-Brown, BSc, MSc
Pfizer UK, Tadworth, Surrey, United Kingdom
Emma Clifton-Brown is Head of Access & Value at Pfizer UK, where she leads a team committed to demonstrating the full value of our medicines and vaccines to accelerate and optimise access to Pfizer’s breakthroughs and improve patient outcomes in the UK. Emma has worked in market access and health economics for over 15 years, across UK and global roles and numerous therapy areas. She is passionate about the role that medicines and vaccines can play in creating a healthier and more productive UK population and a healthcare ecosystem that is sustainable, improves outcomes that matter to patients and incentivises innovation. Outside of work, Emma loves to ski, cycle and explore the wild with her husband and 2 children.
Stephen Duffield, PhD, MD
National Institute for Health and Care Excellence, Liverpool, United Kingdom
Stephen is Associate Director of Real-world methods at NICE. His role involves the continuing development of NICE’s real-world evidence (RWE) framework, collaboration on RWE demonstration projects, and helping to transform NICE’s use of real-world data across guidance products. He is also involved with upskilling individuals within and externally to the organisation, contributing to training workshops and technical forums. Stephen has a degree in medicine and a PhD in public health. Previously, he worked as a clinical doctor and a guideline developer in NICE Centre for Guidelines.
Fast Facts
Best Practices in Women-Led Healthcare Companies Focused on Enhancing Women’s Health
The BioRegion of Catalonia includes more than 1,300 companies in the life sciences and healthcare sector. Since 2010, on average, a new company has been created every week, and one in four of these are spinoffs from universities and research centers. Of the more than 1,300 companies in the BioRegion, the largest group is represented by 335 biotechnology companies, 76 of which are focused on the production of new therapies and diagnostic tool. Biocat, the organization that coordinates and promotes the life sciences and health sector in Catalonia, elaborated in 2022 the report:
The gender gap in the life sciences and healthcare sector: situation, challenges and recommendations. The main objectives of the report were to describe the situation of women in the sector, identifying good practices and elements of value and implementing concrete measures. The report from Biocat, identified several good practices in the healthcare sector coming from the public and private sector.
The session will be dedicated to showcase good practices for minimizing gender biases in companies within the healthcare sector through examples of startups led by women. One example will be an innovative startup whose mission is to eradicate infertility, a globally prominent condition that affects approximately 200 million people and causes significant financial, psychological, and physical strain on patients. Another example will come from the experience of a startup advancing in the validation and market launch of a diagnostic test for the improvement of endometrial cancer diagnosis, representing an innovative solution in women's health.
The Healthcare Businesswomen’s Association (HBA) is the global leader in gender equity, advancing women into positions of influence and decision-making to improve health equity and outcomes.
The organization is comprised of individuals and organizations in healthcare committed to:
- Achieving gender parity in leadership positions
- Providing equitable practices that enable organizations to realize the full potential of women.
- Facilitating career and business connections to accelerate advancement
This session will include a panel of Marina Rigau, CEO, Mimark; Robert Fabregat, CEO of Biocat; and Laura Requejo, HBA Chapter Leader.
Discussion Leader
Robert Fabregat, PhD
BIOCAT, Barcelona, B, Spain
Robert Fabregat (Barcelona, 1980) has a degree in Chemistry and a PhD in Pharmacy from the University of Barcelona (UB), plus a master’s in Project Management from BES-La Salle.
He was director general for Research and Innovation in Health for the Catalan Ministry of Health from 2019 to 2021, and head of cabinet of the Minister from 2018 to 2019. In October 2021, Robert Fabregat joined Biocat as chief executive officer.
He has extensive experience in innovation strategy and managing national and international R+I projects in the fields of healthcare and biomedicine, collaborating with various research centers, public bodies and private companies. He has also been a professor at the University of Barcelona (UB).
13:15 - 14:15
Member Group Meetings
ISPOR Digital Health Special Interest Group
The Digital Health Special Interest Group invites you to join their Open Meeting to connect with the leadership team, share learnings from the PICOTS-ComTeC Framework, and learn about the group’s next key project. Attendees will also participate in small group discussions to brainstorm and collaborate on exciting new ideas to boost future member engagement.
ISPOR New Professionals Open Meeting
Are you new to the HEOR profession? Come join us to learn more about ISPOR New Professional initiatives and how to get involved. Open to all interested in learning more about ISPOR New Professional initiatives.
13:45 - 14:45
Podium Sessions
International Reference Pricing and Biosimilars
Digital Conference Pass
Beyond HTA and negotiations there are other measures to influence pricing of pharmaceuticals: International reference pricing and the topic of biosimilars.
Moderator
Steffen Wahler, MD; MA
St. Bernward GmbH, Hamburg, Germany
Steffen is Managing Owner of Hamburg-based St. Bernward Consulting. He has held leading positions in German health insurance, medical device and pharmaceutical industry. He served as director in the German pharma trade association (vfa) and was in the curator-board of IQWiG and the DIMDI-HTA division. Steffen obtained his doctorate in medicine at Rostock University and specialized as diabetologist. He earned degrees in economics at Universities of California and York/England as well as at INSEAD, France. He is regularly publishing in his and adjacent fields, like epidemiology and care organization research and co-authored the German Health Economics guidelines, the Hanover Consensus.
P40: The Effect of Biosimilar Entry on Biologic Drug Prices in Medicare Part B
2:30PM - 2:45PM
Winn A1, Abdelaziz A1, Dusetzina SB2, Mitchell AP3
1University of Illinois Chicago, Chicago, IL, USA, 2Vanderbilt University School of Medicine, Nashville, TN, USA, 3Memorial Sloan Kettering Cancer Center, New York, NY, USA
OBJECTIVES: After introducing biosimilars, expectations were high for dramatic cost reductions for biologics. However, savings have been modest in the US. This may be due to the US reimbursement model. For physician-administered drugs, which includes many biologics, providers receive payment based on the percentage of the cost billed, incentivizing the highest-cost product. Currently, US policymakers are debating changing this policy. However, to understand changing the system, causal estimates of the savings seen in the current system are needed, which this study aims to provide.
METHODS: To model cost-savings associated with biosimilar introduction, we used Bayesian structural time series models (BSTS), which utilize information from control time series data that closely resembles biologics’ ASP trends before biosimilars’ market entry. We identified prices from Medicare Part B Drug Average Sales Price (ASP) Pricing datasets (2005 Q1 - 2024 Q2). We included the following biologics: bevacizumab, epoetin(ESRD and non-ESRD), filgrastim, infliximab, peg-filgrastim, rituximab, and trastuzumab. We identified a set of control medications for each biologic such that the controls’ ASP trends closely resemble those of their respective biologics and are unlikely to be influenced by biosimilars’ market entry. We generated the predicted counterfactual ASPs, accompanied by 95% prediction intervals. Since the included biologics had different post-biosimilar entry times, we reported the cumulative price difference between biosimilars and the corresponding parent biologic over 3-year, and all post-biosimilar times.
RESULTS: Except for filgrastim, all biologics had a significant decrease in their ASP per dose values after the entry of biosimilars. Three years after biosimilar entry, most biologics ASP per dose decreased by 10 to 19%, with epoetin (ESRD) and epoetin (non-ESRD) having the greatest reduction in ASP per dose: -19.3% [-25.1%, -6.3%] and -19.6% [-25.5%, -6.7%], respectively.
CONCLUSIONS: Savings from biosimilar introduction are modest. Policymakers should consider alternative reimbursement approaches that align provider and payer incentives.
P38: Assessing the Impact of Access Policies on Biosimilar Pricing: A Comparative Study of Five Major European Markets
2:00PM - 2:15PM
Lee MK, Della Loggia L, Yadav V, Straccia V
Inbeeo, London, UK
OBJECTIVES: Biosimilars hold much promise for healthcare systems by improving patient access to efficacious and established treatments whilst mitigating costs. Despite biosimilar policies implemented across the EU, the effects of these measures remains unclear due to complex interplays between prescribing requirements, switching guidelines and tendering. This study aims to assess the impact of biosimilar policies on pricing dynamics in France, Germany, Italy, Spain and the United Kingdom.
METHODS: A targeted literature review was conducted to explore policies, prescribing guidelines and reimbursement requirements for biosimilars across Europe. Following this, launch dates and list prices of biosimilars approved by EU in last 5 years were extracted from NAVLIN database. Analysis was conducted to assess the correlation between biosimilar policies and prices.
RESULTS: Across majority of scope countries, HTAs are not required for biosimilars, and switching is at physicians' discretion. However, in Italy, HTAs are mandatory, and prescribers must adhere to guidelines. In-patient setting tendering is applied in all counties but only France, Italy and Spain have regulated pricing policies. Financial models including benefit sharing models and prescription quotas were mostly implemented in Germany, France and the UK. From Jan 2019 till May 2024, a total of 17 biosimilars are available in the EU. The average per pack launch price ranged between €581 to €1,128. Biosimilars in UK experienced the highest price erosion (~60% discount from launch price), followed by France and Germany with 20% and 10% discount, respectively.
CONCLUSIONS: Despite relaxed price-setting in the UK, biosimilars there experienced the most significant price erosion compared to other European markets, likely due to centralized negotiations and switching polices mandated by NHS directives. To fully understand the underlying impact of these policies, further research is needed to investigate policy implementation at the sub-national level, particularly interactions between institutions, physicians, and pharmacists.
P39: Variation in the Formula Used to Calculate Prices Under External Reference Pricing (ERP): A Global Overview
2:15PM - 2:30PM
Izmirlieva M
GlobalData, London, London, UK
OBJECTIVES: Many countries use External Reference Pricing (ERP) – also known as International Reference Pricing (IRP) – to set the price of medicines. The formula used to set the price in the referrer country in relation to price of the same medicine in a basket of reference countries is one of the most important elements of the ERP system. This study aims to assess the variation in formula use globally.
METHODS: The IRP systems of 70 countries were examined using the GlobalData IRP 360 country profiles and IRP Matrix. Countries were split into groups based on their ERP formula archetype.
RESULTS: Referencing the lowest price among the basket of reference countries is still a popular formula – currently used by 22.9% of countries. An additional 12.8% of countries reference the Nth lowest or the average of N lowest prices. Some 31.4% reference the average, 1.4% – the median, and 2.9% – the highest. An astounding 12.8% of countries have dual formulas, meaning that a different formula would apply to different drug types or in case the price is calculated from neighbouring vs developed countries in the reference basket. Another 7.2% use formulas including “or”: for example, the average/median from the basket of reference countries or the price in a specific country, whichever of the two is the lower/average. For two other countries the formula is unspecified. Just one country sets its reference price under ERP at a percentile of the available prices. Notably, one country applies a different, more punitive, formula for drugs with a waiver from pharmaco-economic evaluation.
CONCLUSIONS: Formulas used the calculate the price under ERP are increasingly complex, with a significant proportion of countries applying a different formula for different therapeutic areas or for domestically produced originator medicines. Departure from referencing the lowest price improves alignment with ERP best practice guidelines.
P37: Could International Reference Pricing Influence Equitable Pricing of Innovation in Europe? An Income-Based Comparative Analysis
1:45PM - 2:00PM
Madkour M
University of Strathclyde, Glasgow, UK
OBJECTIVES: Equitable pricing aims to maximise access to innovation by pricing it according to the country’s willingness to pay (WTP). The objective is to investigate whether the current International Reference Pricing (IRP) practices promise fair pricing for countries in Europe or not.
METHODS: Recent IRP rules for 29 European countries were extracted from the literature. The ex-factory launch prices of the top 10 cancer drugs globally by revenue were extracted from countries' national price formularies or from pricing intelligence platforms. The gross-national income per capita (GNI/capita) was extracted from the World Bank and used as a proxy for WTP of countries. The comparative analysis included graphing the basket GNI/capita of each country against its own GNI/capita to observe if countries are pricing their drugs by referencing comparable countries or not. In a combined graph, the daily defined dose (DDD) ex-factory launch price of the 10 drugs was standardised to their mean for each country (scatter plot) and graphed against the GNI/capita of each country (bar chart) to compare the relative launch price ratio between 29 countries.
RESULTS: The basket GNI/capita of each country against its own GNI/capita showed that some wealthy countries attempted to “free-ride” by referencing mostly from countries of lower income. Most IRP formulas used the mean, which enabled some countries to price innovation below their actual WTP. The coefficient of variation of the relative launch price of DDD of 10 drugs across 29 countries was calculated at 2.22%. This low variation persists despite significant differences in GNI/capita among these countries (example: GNI/capita of Austria: $70,9k, while Bulgaria: $34,3k).
CONCLUSIONS: Though IRP is widely used in Europe as a cost-saving tool, it is vital to address the potential inequalities that arise from applying IRP. Redesigning IRP or navigating other policies that capture a country's WTP and value of innovation should be explored.
Improving Transportability of Evidence in HTA: Methodological Investigations
Digital Conference Pass
The session summarizes issues and methods used to assess and improve transportability of evidence in HTA across patient populations and jurisdictions, including evidence of treatment efficacy, and quality of life.
Moderator
Sofie Berghuis, PhD
GSK, Amersfoort, Netherlands
P34: Transportability Analyses in Action: A Landscape Assessment of Application Studies Using Real Data
2:00PM - 2:15PM
Vuong Q1, Metcalfe R2, Yan R1, Park J1
1Core Clinical Sciences, Vancouver, BC, Canada, 2Core Clinical Sciences, Calgary, AB, Canada
OBJECTIVES: Transportability analysis can facilitate health technology assessment (HTA) decision making by transporting the findings from randomized clinical trials (RCTs) or observational studies to a distinct target population. Recently there have been rapid developments in transportability analysis methods, but adoption of these methods is still in the early stages. We conducted a landscape review of applications of transportability analysis to gain an overview of how transportability analysis is being used in practice.
METHODS: We systematically searched PubMed for articles about transportability analysis. We then screened the abstracts and full texts of the returned articles to only include those that applied at least one transportability analysis method on real datasets collected from human populations. We extracted data from the screened articles on the clinical context, the characteristics of the source and target datasets, and the characteristics of the statistical analysis.
RESULTS: The PubMed search returned 1439 abstracts, from which we retained 90 for full text screening. We ultimately included 52 articles, three of which were identified through hand-searches. Among these articles, 81 separate transportability analyses were performed on 62 source/target dataset pairs. Among source datasets, 42 (67.7%) were from US populations, and 51 (82.3%) were from RCTs, with 8 (12.9%) from observational studies. Among target datasets, 40 (64.5%) were from US populations, and 41 (66.1%) were from observational studies, with 18 (29.0%) from RCTs. Most analyses were conducted using individual patient data (IPD) in both the study and target datasets. Weighting (43 analyses, 53.1%), g-computation (13 analyses, 16.0%), and augmented approaches (15 analyses, 18.5%) were most frequently used to conduct transportability analysis.
CONCLUSIONS: Applications of transportability analysis have not substantially expanded beyond US populations. More analyses which transport findings to non-US populations will be helpful in establishing the role of transportability analysis in global health.
P33: Transportability Analysis Methods for External Validity Assessment of Randomized Clinical Trial Evidence for Health Technology Assessment Decision-Making: A Simulation Study
1:45PM - 2:00PM
Vuong Q1, Metcalfe R2, Yan R1, Park J1
1Core Clinical Sciences, Vancouver, BC, Canada, 2Core Clinical Sciences, Calgary, AB, Canada
OBJECTIVES: Randomized clinical trials (RCTs) are an important source of evidence for health technology assessment (HTA), but their external validity is often uncertain. Assessment of RCT external validity can facilitate decision making by improving the relevance of RCT evidence to local contexts. Transportability analyses are causal inference methods used to assess the validity of RCTs in external settings. We conducted a simulation study to assess the performance of two novel methods, inverse odds of participation weighting (IOPW) and g-computation, for transporting original RCT findings to real-world data (RWD).
METHODS: Datasets from RCT (study) and RWD (target) populations were generated with different distributions of covariates and effect modifiers and different true average treatment effects (ATEs) for a continuous outcome. We assumed that the treatment and response variables were only observed in the study dataset. IOPW and g-computation methods were each used to estimate the ATE in the target population using its covariate data. Bias, coverage of 95% confidence interval (CI), and relative error of standard error (SE) were used to compare the performance of IOPW and g-computation versus naïve linear regression.
RESULTS: Compared to the naïve estimator, the IOPW and g-computation estimators performed considerably better on all metrics. There was considerably less bias, and the 95% CIs of the target ATE calculated using IOPW and g-computation empirically met the nominal level. The relative errors of SE were small in all methods. The performance of the transportability analysis methods is contingent on specifying the models correctly when using IOPW and g-computation.
CONCLUSIONS: Transportability analysis is an important tool that can facilitate HTA decision making. As seen in our simulation study, transporting results from RCT to real-world settings using IOPW and g-computation methods show great promise. Challenges in the analysis include correctly specifying factors that affect treatment assignment in the RCT and modify the treatment effect.
P35: Transporting EQ-5D Aggregated Utilities Across Different Value Sets: A Simulation Modelling Approach
2:15PM - 2:30PM
Estévez-Carrillo A1, Rand K2
1Maths in Health B.V., Klimmen, LI, Netherlands, 2Maths in Health B.V., Klimmen, Limburg, Netherlands
OBJECTIVES: Cost-effectiveness models typically use reported aggregate utility values from scientific literature. Ideally, these models should incorporate country-specific utility values. We introduce a novel copula-based simulation method to estimate mean EQ-5D utility values for any value set using reported mean values and the original value set. We demonstrate the usefulness of this approach by comparing estimated and observed mean values for arthritis and diabetes patient groups in the Multi Instrument Comparison study, using UK crosswalk values as input to estimate German and Spanish mean utility values.
METHODS: We generated EQ-5D data using a copula approach to reflect a natural distribution of responses modeled on self-reported health states in a mixed patient population. The modeled data included EQ-5D-5L questionnaire and EQ-VAS responses, maintaining correlations between dimensions and cumulative densities. The resulting simulated dataset was then re-weighted to produce mean values matching the target value in the target value set. Finally, mean values for alternative value sets were estimated for using the simulated data and fitted weights.
RESULTS: In our MIC data analysis, the observed mean utility values for arthritis patients were 0.626 (UK crosswalk), 0.733 (German 5L), and 0.697 (Spanish 5L). Our simulation estimated values of 0.742 (German) and 0.693 (Spanish). For diabetes patients, observed values were 0.695 (UK crosswalk), 0.793 (German 5L), and 0.754 (Spanish 5L), with estimated values of 0.799 (German 5L) and 0.754 (Spanish 5L). Across all disease groups, the mean absolute error of estimation was approximately 0.005.
CONCLUSIONS: Our copula-based simulation method provides reliable and precise estimates of EQ-5D utility values for different country-specific value sets using reported aggregate means from one value set. This approach can be further refined using empirical EQ-5D data from similar patient populations.
P36: Assessing Methods for Transporting Trial Treatment Effects to Target Real-World Populations Without Individual-Level Data
2:30PM - 2:45PM
Tikhonovsky N1, Ma F2, Zhang X2, Ramagopalan S2
1Lane Clark & Peacock, London, LON, UK, 2Lane Clark & Peacock, London, UK
OBJECTIVES: Transportability methods provide a tool to extend results from randomized controlled trials or observational studies to real-world populations, typically relying on individual-level data. This study sought to validate and compare existing transportability methods that use aggregate data from target populations in the context of a different therapy area.
METHODS: Using methods previously tested for a cardiovascular prevention trial, we extended a treatment effect from the NIDA-CTN-0044 trial to a target population of U.S. substance abuse treatment seekers, represented by the 2021 Treatment Episode Data Set (TEDS): Admissions dataset. Three methods using aggregate data from the target population (weighting using simulated individual data, weighting using method of moments, and post-stratification) were compared against the gold-standard method: weighting using individual-level data. Transported odds ratios were reported for each method alongside 95% confidence intervals using robust standard errors (except for post-stratification). Seven common covariates were included as categorical or binary variables.
RESULTS: The information reported here results from secondary analyses of data from clinical trials conducted by the National Institute on Drug Abuse (NIDA). Specifically, data from NIDA-CTN-0044 Web-delivery of Evidence-Based, Psychosocial Treatment for Substance Use Disorders were included. A total of 406 participants in the NIDA-CTN-0044 study and a target population of 350,536 in the TEDS were identified. Weighting using method of moments yielded estimates closest to the gold-standard estimate transported the trial. For the NIDA-CTN-0044 trial, the expected treatment effect (odds ratio (OR)=1.32, 95% CI 0.76-2.28) obtained for the target population using the gold-standard approach was mostly closely approximated by the method of moments (OR=1.31, 95% CI 0.70-2.43), followed by the simulated data approach (OR=1.28, 95% CI 0.72-2.29) and post-stratification (OR=1.46).
CONCLUSIONS: Weighting using method of moments provided transported estimates most comparable to the gold-standard method. Future research should further validate aggregate-based transportability methods across different therapy areas and settings.
Breakouts: IP, WS, & OBS
Role of Real-World Evidence and Artificial Intelligence in Accelerated Approvals in Oncology: Current Status and Next Steps
Level: Intermediate
PURPOSE:
- To explore current evidence of RWE-based accelerated assessment (AA) and associated regulatory and policy challenges for oncology products in Europe.
- To understand the scope of AI in AA.
- To propose recommendations for the implementation of AI and RWE in oncology AAs in Europe.
DESCRIPTION:
Accelerated assessment (AA) and priority medicines (PRIME) schemes in Europe expedite the regulatory approval of innovative oncology treatments. However, they often rely on limited clinical trial data, challenging the evaluation of long-term safety and efficacy. Strategic use of real-world evidence (RWE) in AA may overcome data challenges but may pose withdrawal risk due to a lack of clinical benefit. To meet stringent regulatory timelines and ensure early market access, health technology developers may deploy AI for AAs, especially within the PRIME scheme. However, the lack of clear guidance from health policy and regulatory viewpoints precludes RWE’s optimal use in oncology AAs. In the beginning,
Ramiro will provide a brief overview of the AA and PRIME scheme by the European Medicines Agency (EMA) (5 minutes).
Kapil will discuss findings from the current evidence on the use of RWE, clinical trial data, or hybrid data in AA, analyzing oncology AAs from EMA’s website (15 minutes). Jessica will discuss regulatory nuances related to AA, considering evidentiary requirements and exploring opportunities to use RWE with or without AI in future AAs for oncology products (15 minutes). Finally,
Shahid will deliberate on specific policy issues influencing the use of RWE and AI in oncology AAs from both regulatory and health technology assessment (HTA) perspectives (10 minutes). The session will conclude with consensus development on a policy roadmap for integrating RWE and AI in oncology AAs (15 minutes), followed by a 5-minute wrap-up by
Ramiro. This session will be useful for RWE experts, AI/ML researchers, and regulatory and HTA agencies.
Discussion Leaders
Ramiro Gilardino, MD, MHS, MSc
MSD, Dubendorf, ZH, Switzerland
Ramiro Gilardino is an impact-driven healthcare executive with over 15 years of leadership in Global Market Access, Health Policy, and HEOR. He has successfully led strategies that improved patient access, drove reimbursement success, and shaped health policy in pharmaceutical, biotech, and global organizations. An expert in advancing HTA frameworks and forging stakeholder partnerships, he is dedicated to promoting health equity and delivering value-based healthcare solutions globally.
Discussants
Kapil Khambholja, PhD
Catalyst Clinical Research, Vadodara, India
Kapil Khambholja PhD with about 20 years of professional tenure and currently serving as Executive Director & Head of Medical Writing, RWE & HEOR at Catalyst Clinical Research. Having experience covering varied aspects of preclinical discovery, clinical development, and post-launch activities, Kapil has worked in diverse Scientific and Medical Writing domains including regulatory and drug safety and different therapeutic areas (immune-oncology, cardiovascular, infectious, transplant etc). Having worked with both global pharma company and CRO, he has successfully contributed to building and expanding partnerships for technical and process excellence. In his previous leadership role, he was responsible for safety reports for the entire Immunology portfolio for a top 5 global pharmaceutical giant besides operational and leadership responsibilities. He contributed closely and oversaw critical deliverables and FDA/EMA/PMDA submissions for multiple blockbusters and first-in-class drugs along with other assets under development at various stages for Novartis, J&J, and others. Kapil is also Head of Product strategy working on automation initiatives in the domain.
Kapil has led several thought leadership and innovation initiatives including intersection of RWE, RCT, technology and diversity. Published more that 40 papers, book chapters and speaker at various professional conferences; aim to change status quo in pharma and healthcare industry to push for faster clinical development and improved access.
Carlos Martin-Saborido, PhD, MSc
Spanish Ministry of Health, Madrid, Spain
Jessica Santos, PhD, Ma, Bsc, CIPP
Oracle Life Sciences, Cambridge, Cambridgeshire, United Kingdom
Dr Jessica Santos is the Global Compliance and Quality Director and Data Protection Officer in Oracle Life Sciences, provides consulting and research to communicate the value and potential for products and services that fall under healthcare regulation and legislation. Jessica is providing oversight and support across global offices in regulation, ethics and compliance, and responsible for all activities regarding compliance, privacy, ethics and quality framework on clinical, HEOR, RWR and commercial Research.
Jessica is also an experienced statistician, analyst and methodologist. She gained her reputation through her publications and professional committee work in the industry. Jessica is a frequent speaker and contributor in major conferences. She has a PhD in Social Science and MRS fellowship.
Jessica is also the chair of ISPOR Code of Conduct Committee.
How Can We Move From Generating Robust Patient Preference Information to Producing Decision-Ready Outputs?
Digital Conference Pass
Level: Experienced
PURPOSE:
This workshop will discuss challenges and solutions to transforming patient preference information into decision-ready outputs.
DESCRIPTION:
Background: Two decades of advancements in stated preference methods have improved the reliability of patient preference information. To ensure these improvements are accessible to practitioners, they have been reflected in various ISPOR task forces, workshops, and issue panels. While the scientific community has since moved on from methodological discussions to reflecting on the use of preference information in decision making, the development of tools and approaches to generate decision-ready outputs has just started to receive attention. As a field, we therefore understand how to generate reliable data and how that data could be used, but still lack methods for generating fit-for-purpose outputs. This workshop will discuss and demonstrate various methods that can transform patient preference information into outputs that align with the needs for making a wide range of health care decisions.
Leader: Sebastian Heidenreich will build on recommendations from a recent ISPOR task force to discuss preference-based trial simulations to overcome current limitations in benefit-risk assessments. The discussion will specifically outline opportunities that result from the inclusion of preference elicitation methods in pivotal trials, to help facilitate the interpretation of clinical data. Paul Schneider will demonstrate the suitability of novel preference elicitation instruments to develop individual specific decision tools that can be used in advisory board settings and shared decision-making contexts. For this purpose, a live preference elicitation will be conducted with individual specific treatment recommendations based on unique preferences. Divya Mohan will draw on tools from economic evaluations by demonstrating how an individual level Markov Model can be used to quantify the costs and benefits of preference-based decision making to the health care system. The results will be contrasted with QALY-based models to interpret findings within an established decision-framework.
Discussion Leaders
Sebastian Heidenreich, PhD
Evidera, London, LON, United Kingdom
Sebastian is Senior Director and Global Head of Evidera’s Patient Preference Team. He is a leading expert in the use of patient preference information to support medicinal products across the product lifecycle. He has taught preference elicitation to a wide audience, including government, academia, and industry. His research has appeared in a wide range of international journals and informed decisions of regulators, HTA bodies, and prescribers. Alongside leading Evidera’s patient preference team, he holds an adjunct faculty position at the University of Calgary and leads various international initiatives (e.g., at ISPOR).
Discussants
Divya Mohan, PhD
OPEN Health, London, London City, United Kingdom
Paul Schneider, MD, PhD, Msc
Valorem Health, Bochum, NRW, Germany
Paul Schneider is a Postdoctoral Researcher at the University of Sheffield and Co-Founder of Valorem Health.
From Data to Decisions: The Potential of RWE Transportability in Healthcare Decision Making
Digital Conference Pass
Level: Introductory
PURPOSE: Health Technology Assessment (HTA) bodies increasingly consider supplementary evidence from real-world data (RWD) alongside traditional data sources like RCTs. However there remain concerns about bias and the applicability of RWD from other countries to the local settings. Further complicated by the uneven availability of high-quality RWD across different geographical regions. This workshop will discuss how transportability analysis, like methods used to adjust for confounding factors, can help bridge this gap. Recently NICE updated their real-world evidence framework to include transportability methods as a potential approach to adjust for external validity concerns.
DESCRIPTION: Workshop attendees will hear from different stakeholders across the industry including decision makers on how transportability methods can address the lack of quality local data within an HTA submission. The workshop will a) present a transportability framework and methods, and current stakeholder guidance and b) address how transportability methods can be applied in practice. Philani Mpofu will open the workshop with a brief introduction on the concepts (5 min). Alexandra Starry will draw learnings from previous NICE and G-BA appraisals and introduce a transportability analysis framework (15 min). Marian Eberl will discuss challenges from the industry perspective to address requirements for local data and present a pilot transportability analysis in lung cancer using US and German data as an example explaining how the study was adapted to the previously outlined framework (15 min). Stephen J. Duffield will discuss key considerations for the acceptability of international data and transportability analysis approaches from a decision maker perspective (10 min). Then Philani Mpofu will lead the audience participation, by walking them through a hypothetical case study, asking them to identify differences that should be considered across a multi-country transportability project (15 min). This interactive and informative workshop will inform researchers and industry analysts on how to design their own transportability study.
Discussion Leaders
Blythe Adamson, PhD, MPH
Flatiron Health, New York, NY, USA
Blythe Adamson is an epidemiologist and economist accelerating the development of valuable medicines and health policy. She serves as Head of Outcomes Research and Evidence Generation at Flatiron Health in New York. Dr. Adamson advances artificial intelligence applications of natural language processing and machine learning in the curation of electronic health records for real world evidence with a team of engineers, scientists, and clinicians. She designs treatment comparative-effectiveness studies, codes mathematical models of disease, and delivers research for global health technology assessment. Dr. Adamson served as a former member of the White House COVID Task Force on Healthcare Resilience while lead pandemic data scientist in the West Wing. She is an Affiliate Professor at the University of Washington in the Comparative Health Outcomes, Policy, and Economics (CHOICE) Institute. Dr. Adamson has held roles at the Bill and Melinda Gates Foundation Institute for Disease Modeling, Infectious Economics, the NIAID HIV Vaccine Trials Network, and Fred Hutch Cancer Center. She continues to serve as a leader in the International Society for Health Economics and Outcomes Research (ISPOR). Dr. Adamson holds degrees in pharmaceutical economics, public health epidemiology, and microbiology. She enjoys writing about the economics of infectious diseases at www.blytheadamson.com.
Discussants
Stephen Duffield, PhD, MD
National Institute for Health and Care Excellence, Liverpool, United Kingdom
Stephen is Associate Director of Real-world methods at NICE. His role involves the continuing development of NICE’s real-world evidence (RWE) framework, collaboration on RWE demonstration projects, and helping to transform NICE’s use of real-world data across guidance products. He is also involved with upskilling individuals within and externally to the organisation, contributing to training workshops and technical forums. Stephen has a degree in medicine and a PhD in public health. Previously, he worked as a clinical doctor and a guideline developer in NICE Centre for Guidelines.
Marian Eberl, MSc
Daiichi Sankyo Europe, München, Germany
Marian Eberl is the RWE Generation Lead for multiple lung cancer assets within the Global Oncology Value, Access & Pricing department at Daiichi Sankyo. In this role, he is responsible for strategic planning and project implementation using RWD and RWE analytics, focusing on secondary data studies. Additionally, he contributes to the development of analytic capabilities, toolsets, and processes related to RWE generation at Daiichi Sankyo.
Marian is trained as a cancer epidemiologist with a strong emphasis on study design methodology. He holds an M.Sc. in Epidemiology from Ludwig-Maximilians-Universität München and an M.Sc. in Health Systems and Public Policy from the University of Edinburgh.
Alexandra Starry, MPP
Cytel, Berlin, Germany
Alex, a Senior Research Consultant, is a highly skilled and experienced data analyst with a BA in Public Financial Management from Indiana University and a Master of Public Policy from the Hertie School in Berlin. Over the past four years at Cytel, she has led multiple medical chart reviews and claims data projects within various indications, focusing on treatment patterns, epidemiology, disease burden, and healthcare utilization and costs.
Spotlight Session
HEOR in the Era of Generative AI: Navigating the New Frontiers
Digital Conference Pass
Level: Introductory
ISSUE: The emergence of generative AI heralds a transformative era for HEOR, offering many opportunities to enhance methodologies across the discipline. This technology promises to revolutionize aspects such as evidence synthesis, real-world data analysis, health economic modeling, and health technology assessment (HTA). Generative AI can serve as a valuable auxiliary tool, facilitating economic evaluations and potentially streamlining HTA submissions. However, these advancements come with inherent challenges and limitations that need careful consideration. As generative AI technologies advance, it is crucial for the HEOR community to continuously evaluate and adapt to these changes, ensuring that their integration into our workflows is both effective and ethically sound. This panel, convened by the ISPOR AI Working Group leaders, aims to explore current and promising approaches to integrating generative AI within HEOR.
OVERVIEW: The session, moderated by Jag Chhatwal, will begin with an exploration of the current generative AI landscape and its intersections with HEOR. Rachael Fleurence will illustrate practical applications, as well as limitations, of generative AI in systematic literature reviews, evidence synthesis and real-world evidence. Xiaoyan Wang will discuss the potential of generative AI in developing synthetic or external control arms, which could significantly expedite the evidence generation process for clinical trials. Jag Chhatwal will present the current uses of generative AI in health economic modeling, highlighting both the current capabilities and challenges. Finally, Dalia Dawoud will discuss how HTA may adapt evaluation methods and processes in response to submissions that employ generative AI.
The session will feature interactive polling to engage attendees actively and gather their insights. By bringing together diverse expert views, this session aims to foster a comprehensive understanding of the multifaceted roles, ongoing challenges, and future prospects of generative AI in HEOR. It will also address the existing uncertainties and skepticism surrounding the adoption of generative AI in HEOR.
Moderators
Jag Chhatwal, PhD
Harvard Medical School and Value Analytics Labs, Wilmington, MA, USA
Dr. Chhatwal is an associate professor at Harvard Medical School and Director of the Institute for Technology Assessment at Massachusetts General Hospital. Dr. Chhatwal has co-authored over 120 original research articles and editorials in peer-reviewed journals. His work has been cited in leading media outlets, including CNN, Forbes, National Public Radio, New York Times, and Wall Street Journal.
Since 2011, Dr. Chhatwal has taught several workshops and short courses on decision modeling and AI at the ISPOR. He is a member of ISPOR AI Working Group. He also serves as an associate editor of Value in Health and is serving as the guest editor for special issues on AI in Value in Health.
Panelists
Dalia Dawoud, PhD
National Institute for Health and Care Excellence, London, United Kingdom
Dalia Dawoud, PhD, is Associate Director (Research) at the National Institute for Health and Care Excellence (NICE). She has practised as health economist and researcher for over 20 years. Her current work is focused on advancing HTA and clinical guideline development methods through research.
Dalia leads NICE newly established HTA Innovation Laboratory (HTA Lab) as well as NICE’s portfolio of European Commission funded projects, such as IMI EHDEN and HORIZON 2020 HTx projects, with cumulative funding of over 5 Million Euros. She is widely published in the area of health economics and outcomes research and serves as Associate Editor of ISPOR journal Value in Health and as Director on ISPOR Board of Directors (2023-2026).
Rachael Fleurence, PhD
National Institutes of Health, Bethesda, MD, USA
Dr Fleurence is a Senior Advisor at the National Institutes of Health where she is working on launching a national initiative to eliminate Hepatitis C in the United-States. Dr Fleurence is also affiliated with the National Institute of Biomedical Imaging and Bioengineering where she works on advances in Artificial Intelligence and Machine Learning. Dr Fleurence currently co-leads the ISPOR Task Force on the suitability of EHR data for Health Technology Assessments. Previously, Dr Fleurence served as a senior health advisor in the Biden-Harris White House, Dr. Fleurence received a BA from Cambridge University (United Kingdom), an MA in business management from ESSEC-Paris (France), and an MSc and PhD in health economics from the University of York (UK).
Xiaoyan Wang, PhD
IMO health, Rosemont, IL, USA
Breakouts: IP, WS, & OBS
Role of Global Indirect Treatment Comparison (ITC) Programs in Supporting Europe’s Joint Clinical Assessments (JCA): Insights From Global and EU Experts
Digital Conference Pass
Level: Intermediate
ISSUE:
The pharmaceutical and medical device industry strategically develop global indirect treatment comparison (ITC) programs to support product launches by providing nuanced understanding of a new intervention’s efficacy and safety in relation to existing treatments. Global ITC programs have evolved over time in terms of complexity and now include systematic reviews, ITC feasibility assessments, and various ITC analyses including network meta-analyses, matching adjusted indirect treatment comparisons, and real-world evidence (RWE)-based external control arm studies. Given the complex nature of global ITC programs and intricacies of global market access, global ITC programs have become instrumental in providing comparative and causally valid evidence for new products to payers and health technology assessment (HTA) bodies. Notably, insights gleaned from global ITC programs extend to EU’s joint clinical assessments (JCAs) where European Parliament aimed to substitute the parallel evaluations of clinical data conducted by multiple country-specific HTA bodies with a single harmonized relative effectiveness assessment. Well-designed global ITC programs can ensure that EU HTAs have high-quality comparative evidence for JCAs where head-to head trial data are unavailable.
OVERVIEW:
This panel will discuss the role of global ITC programs in supporting European JCAs. Dr. Chris Cameron will moderate and provide an overview of global ITC programs and HTA requirements for EU’s JCAs, and pose key questions for the panelists to debate, including:
- What are the opportunities with leveraging global ITCs for EU JCAs?
- What are the challenges with leveraging global ITCs for EU JCAs?
- How do multiple PICOS across EU increase complexities?
- Why might well-designed global ITC programs be more acceptable for EU JCAs?
- What are the challenges with harmonizing ITCs to support EU JCAs?
International experts will provide their unique perspective from ITC producer, industry and HTA perspectives. Target stakeholders include HTA agencies, regulators, academics, clinical guideline developers, patient representatives, and pharma/biotech manufacturers.
Moderators
Chris Cameron, MSc, PhD
EVERSANA, Sydney, NS, Canada
Dr. Chris Cameron is Chief Scientific Officer at EVERSANA. He is a global thought leader in health economics and outcomes research with over 15 years of experience. Prior to joining EVERSANA, Chris was a partner at Cornerstone Research Group Inc., and lead of Health Economics at CADTH. He has also been a consultant decision scientist for ICER, a member of the Economic Guidance Panel at pCODR, and served on PMPRB Technical Panel on drug pricing guidelines in Canada. Chris is an adjunct professor at Dalhousie University, and has authored over 180 peer-reviewed publications in many of the world’s top journals, including JAMA, Lancet, and BMJ. His research has been featured in the Globe and Mail and New York Times. He was recently named one of the most prolific authors in the world on indirect treatment comparisons and was a co-author on the PRISMA NMA reporting guidelines. Chris holds a bachelor’s degree in mathematics and an MSc and PhD in Epidemiology where he was a Vanier Canada Graduate Scholar. He also completed a fellowship on real-world data analytics at Harvard University and FDA’s Sentinel Initiative.
Panelists
Shannon Cope, MSc
Precision AQ, VANCOUVER, Canada
Shannon Cope, MSc, is a Senior Vice President with Precision AQ. Shannon has over ten years of experience consulting for pharmaceutical industry regarding evidence synthesis and economic evaluations to assess the value of new interventions.
Justin Doan, MSc, MPH, DrPH
Pfizer, Inc., New York, NY, USA
Justin Doan is a Team Lead at Pfizer managing a team of HEOR researchers focused on supporting Pfizer’s breast cancer portfolio. Dr. Doan has spent more than 20 years working across a number of biotechnology and pharmaceutical firms in both US and global roles with a focus on oncology research. His research interests include advanced survival analytics, value characterization, and pulling through the patient voice into trials and real-world research from concept development through to publication. He holds a DrPh and MPH from the University of South Florida and an MSc from the University of Florida.
Uwe Siebert, MD, MPH, MSc, ScD
Center for Health Decision Science and Dept. of Epidemiology and Health Policy & Management, Harvard T.H. Chan School of Public Health, Institute for Technology Assessment and Department of Radiology; Massachusetts General Hospital; Harvard Medical School, Boston, USA
Uwe Siebert, MD, MPH, MSc, ScD, is a physician by training and Professor of Public Health, Medical Decision Making and Health Technology Assessment (HTA), Chair of the Department of Public Health, Health Services Research and HTA at UMIT TIROL - University for Health Sciences and Technology in Austria, and Director of the International Continuing Education Program in HTA and Decision Science (htads.org). He is also Adjunct Professor of Epidemiology and Health Policy & Management at the Harvard T.H. Chan School of Public Health.
Prof. Siebert is President-Elect of ISPOR – The Professional Society for Health Economics and Outcomes Research and former President of the Society for Medical Decision Making (SMDM).
His research interests include applying evidence-based quantitative and translational methods from epidemiology, causal inference, real-world-evidence, health decision science and modeling, benefit-harm assessment, health economics, health services & outcomes research, public health, and artificial intelligence in the framework of health care policy advice and HTA as well as in the clinical context of routine health care, public health policies and patient guidance. He teaches courses for these topics at several universities and for industry in Europe, USA, South America, Africa, and Asia. He has worked with several HTA Agencies in Europe, Brazil, US and Canada and he advises public and government agencies, academic institutions and industry regarding the conduction of HTAs and their impact on policy and reimbursement decisions.
He has authored more than 400 publications (> 30,000 citations, H index > 80) and is Editor of the European Journal of Epidemiology and editorial board member of several scientific journals.
How Does Health Economics & Outcomes Research (HEOR) and Market Access Need to Evolve to Address New Challenges and Opportunities With New Health Technology Assessment (HTA) Processes Globally?
Level: Introductory
ISSUE: The role of HEOR and Market access is growing in importance. We are witnessing many reorganizations across pharma companies with the aim of better integrating access into the overall product strategy. Tatia Woodward and Indranil Bagchi, representing respectively global HEOR and Market Access, will discuss how these functions need to evolve and collaborate to create a holistic strategy that demonstrate the value of innovative products from pipeline until patent expiration. They will share their direct experience in terms of the impact and evolution of their roles, as well as perspectives on what more is needed to elevate the access function within the company. Brian O’Rourke will provide the HTA perspective in terms of evidence required for value demonstration and highlight how institutions like ISPOR can support this evolution.
OVERVIEW: We are witnessing a fundamental transformation of the global HTA landscape. Center for Medicare and Medicaid Services (CMS) is empowered to negotiate drug prices, the EU is creating a singular, comprehensive HTA process, Canada and the UK are updating their frameworks focusing on sustainability and innovation, and collaborations such as the Access Consortium are emerging in various markets. The need to demonstrate the value of new medicines, vaccines, and devices across the entire product lifecycle is becoming more urgent than ever. Real World Evidence (RWE) has emerged as a pillar to demonstrate effectiveness, long term safety, durability, and risk/benefit profile in populations not included in clinical trials, though acceptance of RWE across stakeholder groups remain variable. Value-based pricing is becoming a standard access strategy at launch, for expensive medications that obtain accelerated regulatory approval. The need to reconsider clinical trials program design, often designed just for fast regulatory approval, is forefront to allow collection of data also necessary to inform optimized access and pricing.
Moderators
Cristina Masseria, MSc, PhD
AESARA Inc., Madrid, M, Spain
Panelists
Indranil Bagchi, PhD
GSK, Philadelphia, PA, USA
Dr. Indranil Bagchi is the Global Head of Pricing & Market Access at GSK. In this role, Indranil drives the overall strategy on value demonstration and market access across all product areas in the GSK portfolio, to secure access to medicines for our patients and to maximize the value of our portfolio.
Indranil has more than two decades of experience in market access across several major companies in the pharmaceutical industry. In 2014, Indranil received the ‘Outstanding 50 Asian Americans in Business’ award and in 2010, Indranil was recognized in Pharmaceutical Executive magazine’s annual roster of Emerging Leaders, “The New Breed of Leadership.” Indranil is a frequent speaker and contributor to forums, articles and conferences addressing issues related to access to medicines.
Prior to GSK, Indranil was Senior Vice President and Worldwide Head of Value and Access at Novartis Oncology. Previously at Pfizer, he was Vice President and Global Head of Payer Insights and Access and prior to that, he was with GlaxoSmithKline in Health Economics and Outcomes Research. Dr. Bagchi has an undergraduate degree in Pharmacy, a master’s degree in Pharmacy and Healthcare Administration and a doctoral degree in Pharmaceutical Socioeconomics.
Patrick Hopkinson, BPharm, MBA, MSc
PHTA Consulting, WOK, United Kingdom
Patrick Hopkinson leads PHTA Consulting as an independent consultant. A senior leader in life sciences, he has extensive expertise from executive roles in global pharmaceutical organizations. As former VP of Global HEOR at Bristol Myers Squibb, he specializes in Market Access, HEOR, HTA Policy, and RWE, with broad commercial experience. An HTA expert, Patrick has successfully launched products globally, overcoming access challenges. He's contributed to industry policy groups and served on NICE's Appeal Panel. With experience at GSK and in the UK NHS, Patrick aims to transform healthcare stakeholder relationships, focusing on value and sustainability
Brian O'Rourke, PhD
ISPOR, Lawrenceville, GA, USA
Dr. Brian O’Rourke is an independent healthcare advisor. He served as the President and Chief Executive Officer of CADTH from 2009-2020, following a distinguished career as a pharmacist and healthcare executive with the Canadian military. He also served as the Board Chair for the International Network of Agencies for Health Technology Assessment (INAHTA) from 2014 to 2018. With over 40 years of experience in healthcare, Dr. O’Rourke is a leading expert in the science and practice of health technology assessment (HTA). He has a Bachelor of Science in Pharmacy from Dalhousie University and a Doctor of Pharmacy from the University of Toronto. Following his retirement from CADTH, Dr. O’Rourke continues to play an active role in the global healthcare and HTA communities. He is Chair of the HTA Steering Committee and a member of the Scientific Advisory Council at the Centre for Innovation in Regulatory Science (CIRS). He also served as Chair of the Health Technology Assessment Council at the Professional Society for Health Economics and Outcomes Research (ISPOR) from 2019-2022 and as the 2023-2024 President of ISPOR. In November 2020, Dr. O’Rourke was appointed Colonel Commandant (Honorary) of the Royal Canadian Medical Service, and in November 2022, he was appointed as an Emeritus member of the Canadian Health Leadership Network. Dr. O’Rourke was the 2023 recipient of the Dr. Jill M. Sanders award of excellence in health technology assessment.
Are Digital Endpoints Fit for Health Technology Assessment?
Digital Conference Pass
Level: Introductory
ISSUE: More clinical trials and real-world evidence studies are using digital endpoints, which are outcomes collected using digital tools such as wearables. These endpoints can provide useful information on how patients are functioning between clinic visits and do not rely on patient recall or opinion. There is little experience with them, however, so lack of validation and uncertainty over patient compliance mean they are not commonly considered by HTA bodies. This panel will discuss whether there are any scenarios where digital endpoints are ready to be considered in HTA, what the pros and cons are of using digital endpoints, and what researchers and HTA bodies need to do to allow digital endpoints to be used for decision making in the future.
OVERVIEW: Each panellist will have 10 minutes to outline their case. This will be followed by a debate amongst panellists, moderated by Jeanette Kusel. The audience will have the opportunity to pose questions to the panellists and polls will be used to elicit audience opinion. Dr Jacoline Bouvy will explain why HTA bodies are excited about the prospect of digital endpoint, but currently cautious about their use to inform decisions. She will discuss what is needed in terms of validation and links to health-related quality of life for HTA bodies to be more reassured of their use. Prof Lynn Rochester will highlight recent efforts that support cases where digital endpoints have been developed, validated and can be used in decision-making, highlighting the case study of the Mobilise-D initiative. Dr Kai Langel will highlight the work that is ongoing through the DEEP initiative to make digital endpoints fit for purpose for decision making. This panel will be of interest to all those involved in technology development and evaluation.
Moderators
Jeanette Kusel, MSci, MSc
National Institute for Health and Care Excellence, London, United Kingdom
Jeanette has led the scientific advice, now NICE Advice, business unit at NICE since 2018. Jeanette has led many of the digital health initiatives at NICE and spend a period of time as acting Medtech Director. Prior to NICE, she spent 9 years working with consultancy providing HTA and Health Economics services. She has a Masters in Health Economics for Healthcare Professionals at the University of York.
Panelists
Jacoline Bouvy, PhD
National Institute for Health and Care Excellence, London, LON, United Kingdom
Jacoline is the Programme Director Medicines Evaluation at NICE. She is responsible for the delivery of medicines guidance produced by NICE’s Technology Appraisals and Highly Specialised Technologies work programmes.
Kai Langel, BSc
Deep Measures Oy, Helsinki, Helsinki, Finland
Since 2000, Kai has been a pioneer in patient-facing solutions for clinical trials working with young innovative technology companies. In 2012, Kai co-founded eClinicalHealth, the developers of the Clinpal remote clinical trial platform. Through his involvement in technical, operational and regulatory roles, he has gained deep understanding of broad aspects of the drug development process and the ecosystem behind it.
Kai combines his innovation mindset and nimble execution with several years of experience working at Johnson & Johnson Innovative Medicine R&D in various roles. Today, Kai leads DEEP Measures Oy as a Founder and CEO, having started this initiative within J&J with DEEP becoming an independent company in 2022. DEEP’s core mission is to accelerate the adoption of harmonized digital measures of health to be transform the way human health is managed.
Lynn Rochester, PhD
Newcastle University, Newcastle upon Tyne, NT, United Kingdom
Professor Lynn Rochester is Professor of Human Movement Science at Newcastle University, as well as leading the Brain and Movement Research group (BAM) and acting as an Honorary Consultant Physiotherapist with the Newcastle Hospitals NHS Foundation Trust.
Her main research interests are in neurodegenerative disease focused on the motor and non-motor mechanisms of gait and falls, surrogate markers of disease and disease progression (in particular cognitive decline and falls risk), technological applications in healthcare using wearable sensors (digital biomarkers), and development and evaluation of novel interventions.
Lynn led the work to qualify Mobilise-D (digital endpoint) with the EMA.
Is It Time for More Non-profits to Lead Reimbursement and Commercialization of Gene Therapies to Promote Affordable and Sustainable Access?
Digital Conference Pass
Level: Introductory
ISSUE:
Licensed gene therapies have been withdrawn from the European market by for-profit (pharmaceutical or biotechnology) manufacturers for ‘commercial reasons’, including challenging reimbursement pricing, expensive post-launch data requirements, high manufacturing costs and small patient populations. Conversely, the non-profit, Telethon Foundation, recently took over the commercialisation of Strimvelis in Europe after it was discontinued by its for-profit manufacturer. Furthermore, the Access to Gene Therapies for Rare Diseases (AGORA) initiative was recently established to support non-profits with sustainable access to gene therapies, demonstrating the growing appetite for non-profit commercialisation. It will be important to understand whether non-profits have the resources and solutions to overcome the challenges of gene therapy commercialisation. Could the anticipated challenges of the upcoming Joint Clinical Assessment (JCA), including differing requirements for assessing orphan treatments across member states, be tougher for non-profit manufacturers than for-profit manufacturers? With patient communities most affected by the availability of gene therapies, what is their take on the issue?
OVERVIEW:
Kate Hanman will summarise the commercial challenges leading to gene therapy withdrawals (10 minutes). During the ensuing discussion (35 minutes), Annabel Griffiths will share a patient community’s experience of a withdrawn gene therapy and discuss the risks and benefits from the patient perspective around for- and non-profit-led commercialisation. Mohit Jain will discuss how for-profit manufacturers have the resources needed for sustainable commercialisation of gene therapy, including post-launch data requirements. Arjan Lankester will discuss how non-profits can support licensing, delivery and affordable access to treatments which are otherwise commercially unsustainable. Finally, Kate will ask the panellists how non-profit and for-profit manufacturers will navigate the anticipated challenges of JCA. The audience will vote on whether more non-profits should commercialise gene therapies at the start and end of the session, to see if the debate changes their opinion. Time for the audience’s questions will be allocated (15 minutes).
Moderators
Kate Hanman, BSc, MSc
Costello Medical, Cambrdige, CAM, United Kingdom
Kate is US Head of Rare Diseases at Costello Medical, a global healthcare consultancy providing support to pharmaceutical, public sector & not-for-profit organisations in the areas of market access, medical communications & evidence development. She leads the US team which leverages their specialist expertise to overcome the unique challenges facing the rare disease community, including the additional novel challenges encountered by stakeholders in the gene therapy space.
Panelists
Claire Booth, PhD, MSc, MBBS
UCL Great Ormond Street Institute of Child Health, London, United Kingdom
Mohit Jain, PhD, MBA
BioMarin Pharmaceutical, London, LON, United Kingdom
Mohit is responsible for Global Value, Access & Strategic Pricing in BioMarin. BioMarin is a company focused on developing life transforming therapies for patients suffering genetic diseases with a number of gene therapies in development. Mohit has been with BioMarin for over twelve years and prior to this worked in other companies including large pharma and consulting. By background a scientist and then worked in corporate finance before finding a calling in Market Access. Mohit is married with two lovely girls aged 14 and 16 years, occasionally trying to fit in a run.
Bob Stevens
The Society for Mucopolysaccharide Diseases (MPS Society), Amersham, United Kingdom
Indirect Effects in Health Economic Models: How Do We Include Them and What Are the Implications?
Digital Conference Pass
Level: Intermediate
ISSUE: The introduction of novel vaccines for infectious diseases leads to decreases in disease among vaccinated individuals due to direct effects. Changes in disease may also occur in unvaccinated individuals due to indirect effects, such as herd immunity and serotype replacement. On one hand, indirect effects are a key feature of infectious disease biology and health economics, extending the value of a vaccine or treatment beyond the individuals receiving it. On the other hand, indirect effects are difficult to quantify and incorporate into health economic models. This panel will examine the public health effects due to indirect effects and discuss the implications of their inclusion or exclusion from health economic models.
OVERVIEW: Historically, health economic models have primarily quantified direct effects, but there is increasing evidence of herd immunity and serotype replacement following vaccine introductions for different pathogens. The hurdles lie in the uncertainty of these effects and the intricate population and disease dynamics driving them, complicating the validation and assessment of modeling outcomes. From the public health, modeling, and health technology assessment perspectives, this panel will debate the approaches to including indirect effects in health economic models of infectious disease and the implications in medical decision-making. Dr. Huang will moderate the session and provide an overview of recent research and challenges and approaches in modeling indirect effects. Dr. Orenstein will share observations of indirect effects and other phenomena associated with vaccines from the public health perspective. Dr. Oidtman will discuss effects like herd immunity and serotype replacement in the context of health economic modeling and model validation approaches. Dr. Drummond will discuss how uncertainties associated with indirect effects manifest when demonstrating value to decision makers while emphasis on future research and validation efforts. The attendees will be invited to share their views and pose questions to the panelists.
Moderators
Min Huang, PhD
Merck & Co., Inc., Rahway, NJ, USA
Panelists
Michael Drummond, PhD
University of York, Lichfield, STS, United Kingdom
Michael Drummond is Professor Emeritus of Health Economics and former Director of the Centre for Health Economics at the University of York. His particular field of interest is in the economic evaluation of health care treatments and programmes. He has undertaken evaluations in a wide range of medical fields including care of the elderly, neonatal intensive care, immunization programmes, services for people with AIDS, eye health care and pharmaceuticals. He is the author of two major textbooks and more than 750 scientific papers, and has acted as a consultant to the World Health Organization and the European Union. He has been President of the International Society of Technology Assessment in Health Care, and the International Society for Pharmacoeconomics and Outcomes Research. In October 2010 he was made a member of the National Academy of Medicine in the USA. He has advised several governments on the assessment of health technologies and chaired one of the Guideline Review Panels for the National Institute for Health and Care Excellence (NICE) in the UK. Until recently he was Co-Editor-in-Chief of Value in Health and has been awarded 3 honorary doctorates, from City University (London), Erasmus University (Rotterdam) and the University of Lisbon.
Rachel Oidtman, PhD
Merck & Co., Inc., Rahway, NJ, USA, Rahway, NJ, USA
Walter Orenstein, MD, DSc (Hon)
Emory University, Atlanta, GA, USA
Walter A. Orenstein, MD, DSc (Hon)
Walter A. Orenstein, MD, is currently a Professor Emeritus of Medicine, Epidemiology, Global Health, and Pediatrics at Emory University. From 2008 through 2011, Dr. Orenstein was Deputy Director for Immunization Programs at the Bill & Melinda Gates Foundation. His primary focus at the foundation had been on polio eradication, measles control, and improving routine immunization programs. Prior to 2004, Dr. Orenstein worked for 26 years in the Immunization Program at the Centers for Disease Control and Prevention. From 1988-2004, he was the Director of the United States Immunization Program. He is a former Assistant Surgeon General of the USPHS. Dr. Orenstein successfully developed, promoted, facilitated and expanded new vaccination strategies to enhance disease prevention.
Dr. Orenstein has authored and co-authored numerous books, journals and reviews. Dr. Orenstein co-edited Plotkin’s Vaccines, 8th edition in 2024 – the leading textbook in the field. He is a past Chair of the WHO’s Poliomyelitis Technical Consultative Group. He served as the Chair of the National Vaccine Advisory Committee (NVAC) from 2012 to 2016. He is also currently a member of the WHO’s Strategic Advisory Group of Experts (SAGE) on Ebola Working Group. He is the former Chair of WHO’s Immunization and Vaccines Related Implementation Research Advisory Committee (IVIR-AC). Between July 1, 2016 and June 30, 2018, Dr. Orenstein was the President of the National Foundation for Infectious Diseases (NFID). He is a Member of the National Academy of Medicine (2006-IOM); a Fellow of the American Association for the Advancement of Science (2018); a Fellow of the American Academy of Pediatrics (1980); as well as numerous other prestigious organizations.
Dr. Orenstein served as Chairman of the Technical Consultative Group on the Global Eradication of Poliomyelitis of the World Health Organization (WHO) from 1996 – 2004 and a member of the WHO SAGE (Strategic Advisory Group of Experts) Working Group on Polio Vaccine from September 2008 – 2020. He was Chairman of the WHO Immunization and Vaccines-related Implementation Research (IVIR-AC) from 2019 to 2024.
Dr. Orenstein’s research focus has been on assessment of vaccine effectiveness in observational studies, methods to overcome vaccine hesitancy, ways to enhance uptake of recommended vaccines, and ways to facilitate polio eradication and sustain that eradication. In addition, Dr. Orenstein was a Principal Investigator for an NIH funded Center of Excellence for Influenza Research and Response (Emory CEIRR), with a focus on better understanding influenza pathogenesis and host response.
14:45 - 15:15
Coffee and Connect
Head to the exhibit hall to refuel, recharge and connect with fellow attendees and exhibitors over a steaming cup of coffee. Provided by ISPOR
14:45 - 15:45
Member Group Meetings
Arabic Network Open Meeting
The meeting will discuss about how to harness real-world data (RWD) for effective management of non-communicable diseases (NCDs) in the Arab world. Dr. Suliman Alghnam will introduce the topic and present the speakers. Dr. Sara Al Dallal will provide an overview of the current state of use of Real-World Evidence (RWE) in the UAE and the potential future outlook towards using data generation in NCD management. She will discuss the challenges facing on data collection, integration, and standardization, emphasize the importance of multistakeholder engagement, and provide insights into successful strategies for overcoming challenges and building robust RWD infrastructures at a country level. Dr. Anas Hamad will discuss how RWD can be leveraged to identify trends, improve patient outcomes, and inform policy decisions across the Arab region. Drawing on examples from his work in Qatar, he will demonstrate how integrating RWD into healthcare systems can lead to more personalized and effective NCD management. Dr. Hamad will provide a comprehensive overview of best practices and successful case studies, highlighting the potential for RWD to transform NCD management in the Arab world. Dr. Amal Yassine will present the specific barriers to the use of RWE and how RWE can be effectively integrated into the healthcare decision-making process in Morocco. There will also be a focus on the key levers and appropriate strategies to achieve this objective, in particular to improve the management of non-communicable diseases. Finally, Dr. Yassine will highlight the prospect of collaboration between countries in the MENA region and internationally, in order to adopt best practice and benefit from external expertise. The meeting will include an opportunity for Q&A for audience at the end. All interested in learning about this dynamic and rapidly evolving region are welcome to attend.
Moderators
Suliman Alghnam, PhD
Public Health Authority, Riyadh, Saudi Arabia
Speakers
Sara Al dallal, MBBS, MSc
Emirates Medical Association, dubai, United Arab Emirates
Anas Hamad, PhD, MSc, DMS, BSc, RPh
Hamad Medical Corporation, Doha, DA, Qatar
Dr. Anas is currently the Director of Pharmacy Department at Qatar's National Center for Cancer Care & Research and Head of Medication Safety & Quality Center at Hamad Medical Corporation's Pharmacy Executive Office. He holds an adjunct position at Qatar University as an Assistant Professor of Clinical Pharmacy & Practice. In addition, he is chairing the Standing Committee on Medication Services at Qatar Red Crescent Society. He is also the Vice President & Director of ISPOR Qatar Chapter.
Dr. Anas finished his BSc in Pharmacy at the University of Jordan. He obtained MSc in Clinical Pharmacy, International Practice & Policy from the University of London and PhD in Clinical Practice & Medication Use from King's College London. He acquired an Executive Diploma in Health Services Management from GSM London. He completed the Executive Leaders Program with Qatar Leadership Centre.
Dr. Anas is an active researcher with several publications and conference proceedings. He is an Editorial Board Member at International Journal of Clinical Pharmacy and Review Editor at Frontiers in Oncology. He regularly speaks at local and international conferences. He is a member of both HMC’s and QU’s Institutional Review Boards in addition to the MOPH National Clinical Research Review Committee. Moreover, he is a member of various committees/boards focusing on pharmacy practice, medication use, cancer care, research, safety & quality, medical affairs, health economics, and governmental tenders.
Amal Yassine, MD, PhD
National Health Insurance Agency, Mohammedia, MOH, Morocco
Amal Yassine holds an MD, Executive MBA in health governance and management, and PhD in Life and Health Sciences. She is Secretary General of Moroccan Society for Health Products Economics, and Vice-President for Africa of the Latin Association for the Analysis of Health Systems (ALASS)-Spain. Dr. Yassine has 15 years’ experience in health financing as a manager in the regulatory body of the health insurance system in Morocco, and she is a Member of ISPOR Maghreb Chapter.
ISPOR Student and Faculty Open Meeting
Come and meet the ISPOR Students and Faculty that are paving the way for the future of HEOR! This meeting will provide an overview of the ISPOR Student and Faculty activities and share information on how to get involved. Open to all interested in learning about ISPOR Student and Faculty initiatives.
15:15 - 15:45
Fast Facts
How to Get Published and Influence People!
In this session the Editors-in-Chief of Value in Health will talk about writing for impact, offering tips on what to do and not to do when preparing and submitting your papers. This includes how to write the “highlight” points, which we now require and publish. The session will also address how Value in Health and authors can work together to maximize the impact of your work. This session will be of interest to any author considering submitting papers to Value in Health. The session will include a Q&A session, providing authors with an opportunity to ask the Editors-in-Chief for advice about publishing in Value in Health.
Discussion Leader
Nancy Devlin, PhD
School of Population and Global Health, University of Melbourne, Melbourne, Australia
Nancy is Professor of Health Economics at the University of Melbourne, and holds positions as Editor in Chief at Value in Health and Chair of the Board of the EuroQol Research Foundation. She served as the elected President of ISPOR (2019-2020). Her previous positions include being Director of Research at the Office of Health Economics (2009-2019) and Professor of Economics (and Head of the Economics Department) at City University of London (2001-2009). Her principal areas of research expertise are the measurement and valuation of patient reported health outcomes; the cost effectiveness thresholds used in making judgments about value for money; and priority setting in health care. She is currently leading a large programme of research to improve measurement and valuation of quality of life in children (QUOKKA), funded by the Australian Government. Nancy has published >200 peer reviewed journal papers on a wide range of empirical and theoretical topics in health economics. In 2024, Nancy was ranked 3rd in the world’s ‘Highly Ranked Scholars’™ in Health Economics, based on productivity, impact and research quality.
C. Daniel Mullins, PhD
University of Maryland School of Pharmacy, Baltimore, MD, USA
C. Daniel Mullins, PhD is a professor at the University of Maryland School of Pharmacy. He is founder and executive director of the University of Maryland PATient-centered Involvement in Evaluating effectiveNess of TreatmentS (PATIENTS) Program. He received his BS in Economics from M.I.T. and his PhD in Economics from Duke University. His research and teaching focus on comparative effectiveness research (CER) and patient-centered outcomes research (PCOR). Dr. Mullins has received funding as a Principal Investigator from AHRQ, FDA, NHLBI, NIA, NIMHD, Patient-Centered Outcomes Research Institute (PCORI) and various patient advocacy organizations and pharmaceutical companies. He is the lead for the Community & Collaboration (C&C) Core of the University of Maryland Institute for Clinical and Translational Research (ICTR) and co-lead of the C&C Core for Johns Hopkins’ CTSA.
Professor Mullins is 1 of 2 editors-in-chief for Value in Health and is author of over 325 peer-reviewed articles and book chapters. At the University of Maryland Baltimore (UMB), he received the Dr. Patricia Sokolove Outstanding Mentor Award and the Dr. Martin Luther King Jr. Faculty Diversity Award. He was named Researcher of the Year at UMB and was awarded a University System of Maryland Wilson H. Elkins Professorship. He is a past recipient of the Dr. Daniel D. Savage Memorial Science Award, the Association of Black Cardiologists’ most prestigious annual award, and the ISPOR Marilyn Dix Smith Leadership Award.
Exhibit Hall Theater
Cell & Gene Therapies: Five Years On – Real-World Data, Outcomes, and Evolution of Payers' Expectations
Digital Conference Pass
Level: Introductory
This session will explore the 5-year long-term outcomes of CAR-T therapies and other cell therapies to discuss:
- The role of outcomes-based agreements (OBAs) in mitigating financial risk for payers and facilitating patient access to these therapies
- Real-world evidence on the durability of responses and long-term safety profiles of CAR-T therapies
- The implications of these outcomes for future access, pricing, and reimbursement decisions
This session will analyze whether these long-term outcomes justify continued access to CAR-T and cell therapies, considering both clinical results and cost-effectiveness data, and how OBAs may evolve to support sustainable access moving forward.
Sponsor: Lifescience Dynamics
Sponsor
Lifescience Dynamics
Speaker
Alberto Briones, BSc, PhD
Lifescience Dynamics, London, United Kingdom
15:15 - 16:15
Educational Symposia
Indirect Treatment Comparison Methodology Matters: Unlocking the Essentials for Robust Analysis
Digital Conference Pass
Indirect treatment comparisons (ITCs) are frequently required in health technology assessments due to the lack of head-to-head trials for interventions of interest. Methodological guidance for ITCs, such as the European Union Member State Coordination Group's guidelines in supporting Joint Clinical Assessment on quantitative evidence synthesis and NICE's Technical Support Documents on evidence synthesis and population-adjusted indirect comparisons, provide detailed advice on applying various ITC approaches in practice.
Network meta-analysis is the most widely used ITC approach, under the assumption that any variables that modify treatment effects are balanced across the included study populations. Recent population-adjusted ITC methods aim to relax this assumption, adjusting for differences between populations using individual participant data from one or more studies. Despite its limitations – such as requiring and being sensitive to population overlap, being restricted to estimates in aggregate study populations, and being limited to two-study indirect comparisons – matching-adjusted indirect comparison (MAIC) remains the most commonly used population-adjusted ITC approach. However, poorly conducted ITCs can have significant consequences, potentially leading to a potential cost-effective intervention not being reimbursed.
This session will explore innovations in ITC methodologies and introduce alternative approaches to MAIC, including simulated treatment comparison (STC) and multilevel network meta-regression (ML-NMR), with a special focus on applications to time-to-event outcomes. Case studies will be presented to highlight the limitations and pitfalls of MAIC, while demonstrating how alternative methods can address these limitations.
Attendees will gain a comprehensive understanding of the diverse range of ITC approaches and acquire practical knowledge of the key principles needed for performing robust ITC analyses.
Sponsor: BeiGene
Sponsor
BeiGene
Moderators
Shilpi Swami, MSc
ConnectHEOR, London, United Kingdom
Shilpi Swami, a seasoned Health Economics and Outcomes Research (HEOR) expert, currently serves as Vice President, Consulting and HTA Strategy at ConnectHEOR. She is also a Member Engagement Co-Chair at ISPOR Oncology Special Interest Group.
With a comprehensive global track record in leading HTA submissions and market access strategies, Shilpi's diverse experience spans academia, consulting, and biopharma, allowing her to offer strategic insights from multiple perspectives.
Formerly a Research Fellow at the University of York, Shilpi has contributed significantly to public health projects as well.
She remains dedicated to improving healthcare through data-driven insights and evidence-based research.
Speakers
David Phillippo, PhD, MSc
University of Bristol, Bristol, United Kingdom
David's research focuses on methods for evidence synthesis, Bayesian network meta-analysis (NMA), population adjustment methods for indirect comparisons, and assessing the impact of bias in clinical guidelines and decision making. He is the lead author of Technical Support Document 18 published by the NICE Decision Support Unit on population-adjusted indirect comparisons, providing guidance on the use of this new class of methods in NICE Technology Appraisals. He is a member of the NIHR Bristol Technology Assessment Group, a network member of the NICE Decision Support Unit, and he supports the development of NICE Clinical Guidelines through his involvement with the NICE Technical Support Unit based in Bristol. He is the author and maintainer of several freely-available R packages, including nmathresh for assessing sensitivity to biased evidence in NMA using threshold analysis, and multinma for performing NMA and multilevel network meta-regression with individual and aggregate data.
Kate Ren, PhD
ConnectHEOR, Sheffield, United Kingdom
Kate Ren is a Senior Research Fellow in Statistics at the University of Sheffield and also the Director of Statistics at ConnectHEOR. She is a member of SCHARR-TAG (an external assessment group for NICE technology appraisals), and has extensive experience in critiquing statistical methods used in NICE single technology appraisal submissions. She is also a member of NICE Appraisal Committee C. Her research interests include indirect treatment comparisons, survival extrapolation and structured expert elicitation.
Rhys Williams, PhD
BeiGene, San Mateo, CA, USA
Dr. Rhys Williams joined BeiGene as the Vice President, Integrative Evidence Generation and Health Economics, Global Medical Affairs in February 2023. Previously, he was the Vice President, Health Economics and Outcomes Research at Sunovion Pharmaceuticals. Prior to that, Rhys was an Executive Director in Global Health Economics at Amgen, where he worked for thirteen years. He’s also worked at Aetna health plans, Pfizer, BMS, Janssen and Knoll.
Rhys is a native of the UK and received his BS (honors) degree in Statistics from the Aberystwyth University in Wales. He has two Masters Degrees in Mathematics and Applied Statistics from Tulane University and a Doctoral degree in Epidemiology from Boston University. Rhys has authored over 70 peer-reviewed publications which have appeared in New England Journal of Medicine, Journal of Clinical Oncology and Archives of Internal Medicine, among others. He also serves as an ad hoc reviewer for the Journal of the American Medical Association, Stroke, Value in Health and Obesity Research.
Exploring Unmet Needs in Cancer Screening and the Value of Multi-Cancer Early-Detection Testing
Digital Conference Pass
Cancer remains the second-leading cause of death in the United States, striking about 1 in 3 women and 1 in 2 men in their lifetimes. Today, screening tests in the US are recommended for 5 cancers, but approximately 80% of cancer deaths come from cancers that have no recommended screening guidelines.
Recommended single cancer screening tests save lives, but this technology still only exists for a select number of cancer types. Instead of looking for cancer one-at-a-time, organ-by-organ, multi-cancer early detection technology has the ability to screen individuals for multiple cancers in a cost-effective manner. Adding these new technologies to single-cancer screening has the potential to change the cancer landscape and improve patient outcomes by helping to identify cancers at earlier, more treatable stages on a population scale.
Sponsor: GRAIL, Inc
Sponsor
Grail, Inc
Moderators
Anuraag Kansal, PhD
GRAIL, Inc, Piedmont, CA, USA
Speakers
J. Jaime Caro, MDCM, FRCPC, FACP
Evidera, Lincoln, MA, USA
J. Caro develops and applies novel techniques in modeling, health economics, comparative effectiveness, epidemiology, and outcomes research. To provide a better alternative to the well-known QALY, he is working on the BADI, a broader approach to valuing health benefits. He continues to develop DICE, the unified approach to health economic modeling that he created. Working with health technology assessment agencies and academic groups, he is formalizing this innovation to enable rapid, standardized and less error-prone development of decision-analytic models. Dr. Caro has adapted an engineering technique – discrete event simulation – to model diseases and their treatment and extended it to simulate the design of clinical trials and observational studies. He applied this technique to provide comparative effectiveness information in the absence of head-to-head trials in a new method called Simulated Treatment Comparisons and is applying simulation to make registries more feasible and efficient – an approach called SAVES. He proposed an innovative approach to the assessment of health technologies involving the efficiency frontier and is helping address the growing problem of judicialization of health care system decisions.
Laura Housman, MPH, MBA
Avalere, Washington, DC, USA
Laura T. Housman, DrPHc (ABD), MBA, MPH is Senior Vice President and Practice Director of Evidence and Strategy within Avalere Health, bringing broad and extensive expertise in strategy, evidence generation, reimbursement, patient engagement, population health and personalized medicine to drive access to innovation across healthcare. Laura received her Bachelor of Arts degree in Economics from the College of Arts and Sciences and a Minor in Business Administration from the School of Management from Boston University; her Master of Public Health from the Boston University School of Public Health; and her Master of Business Administration with honors from Boston University’s Questrom Graduate School of Management. Laura is currently ABD as a Candidate in the Doctor of Public Health (DrPH) program at Boston University School of Public Health.
Maarten IJzerman, PhD
Erasmus University, Rotterdam, Netherlands
Maarten J. IJzerman is Professor of Cancer Health Services Research and Dean of Erasmus School of Health Policy and Management in Rotterdam, the Netherlands. He is a part-time Professor of Cancer Health Services Research in the Faculty of Medicine, Dentristry and Health Sciences of the University of Melbourne in Australia.
He leads a data-driven research program focussing on the implementation of genomics enabled precision oncology.
ISPOR Women in HEOR Session
Gender Equity Along the Health Technology Development Pipeline: A Women in HEOR Forum
Digital Conference Pass
PURPOSE: The purpose of the ISPOR Europe 2024 Women in HEOR Forum is to facilitate a discussion among panelists about gender equity in
the health technology development pipeline, highlighting contributions by subject matter experts and ISPOR members.
DESCRIPTION: The vision of the Women in HEOR initiative is to support the contribution of women in our field by serving as a catalyst for leadership and a platform for mentorship, collaboration, and networking. This Women in HEOR Forum will bring into focus gender equity as it relates to the health technology development pipeline. Panelists will address several topics in early phases of development such as the role of funding support of women’s health research, pharmaceutical manufacturer investments in women’s health products/treatments, regulation policy to ensure gender-appropriate data collection and research to identify unmet medical needs among women. Panelists will also consider later phase topics such as research that examines women’s access to treatments, reporting guidelines to consider women in health research, and representation in clinical trials.
The agenda for the session will include: 1) an introduction to the Women in HEOR initiative and its objectives and activities 2) brief remarks by each panelist, and 3) a moderated discussion of the above topics throughout the healthcare technology development pipeline. The panelists will engage with the audience through live polling and open Q&A. Attendees will gain valuable perspectives on gender equity perceived challenges, potential solutions, and current initiatives to address the issue. This session is open to all conference attendees.
Moderators
Olivia Wu, PhD
University of Glasgow, Glasgow, United Kingdom
Olivia holds the William R Lindsay Chair of Health Economics and is co-Director of Health Economics and Health Technology Assessment (HEHTA) at the University of Glasgow. Her expertise is in the adaptation and application of HTA methodologies in context. Her research is multidisciplinary in nature, and spans across a wide range of clinical areas and different types of health interventions (e.g. pharmacological treatments, medical devices, diagnostic tests, and digital health interventions). She has led numerous health economic evaluations alongside clinical evaluations and trials, which work has informed clinical guidelines and health policy decisions, both at national and international levels. Olivia also has a body of work on novel and effective use of evidence synthesis approaches to maximise the value of existing data for informing clinical and economic evaluations. Currently, Olivia leads the Complex Reviews Synthesis Unit (CRSU) which hosts one of the nine Evidence Synthesis Groups funded by the National Institute for Health and Care Research (NIHR). Olivia is a long-standing advisor to HTA agencies within and out with the UK. She has previously served as member of the NICE Technology Appraisal Committee for 10 years.
Olivia has a strong commitment to developing an inclusive environment for the next generation of researchers within and out with the UK. She co-leads the ISPOR Women in Health Economics and Outcomes Research Initiative and is a steering member of the NIHR Race Equity and Diversity in Careers Incubator. Olivia is a strong advocate for mentoring and provides mentorship to academic and government agency researchers both at national and international levels.
Speakers
Ipek Ozer Stillman, MS, MBA
Takeda, Cambridge, MA, USA
Ipek is an accomplished global pharmaceutical leader with two decades of experience in the areas of Value, Evidence, and Market Access. Her career journey is fueled by her passion for driving a positive, sustainable impact in healthcare globally. She thrives on developing innovative strategies that include multidisciplinary approaches, leveraging her expertise at the intersection of business strategy, data analytics, digital transformation, and health policy.
Currently, she is the Head of Global Health Economics at Takeda. She has worked at Takeda for about nine years, with a prior role within the Shire R&D organization as part of the Health Economics, Outcomes Research, and Epidemiology group. Before Takeda, Ipek worked at a large global research consultancy collaborating with global biotechnology and pharmaceutical clients for over ten years.
Ipek holds a Master of Business Administration degree from MIT Sloan School of Management, and a Master of Science degree in Mechanical Engineering from Northeastern University.
Grammati Sarri, PhD, MSc, DiDS
Cytel, London, LON, United Kingdom
Grammati has 15+ years’ experience in epidemiology studies, evidence synthesis and decision making from her prior roles in academia, health technology assessment (NICE, UK), WHO and private health consultancies. Grammati has strong methodological experience in generating impactful evidence from different sources (clinical trials, real-world evidence, economic studies), clearly communicating findings in an impactful way and using complex analytical methods for health care-decision making. Grammati currently holds several positions in international organizations (ISPE), invited speaker to PhD training programmes in pharmacoepidemiology and current Advisory Board member to a EU funded trial on AI in breast cancer surgery (CINDARELLA). Grammati is working on EU JCA developments (produced blogs, delivered webinars, methods trainings) and topics around uncertainty, bias in both applications of non-randomized evidence and evidence synthesis. Publications and presentations related to RWE innovation: driving outputs on building knowledge around RWE methods guides, reliable RWE methods (framework for combining RWE with RCTs for decision-making [ISPE-endorsed], RWE quality assessment, statistical methods, data transferability, external controls for single-arm trials), and driving innovation in the use of RWE around topics such as patient experience, the lifecycle or living decision-making, health inequalities, data transferability across countries, specific challenges for RWE use in single-arm trials for rare and very rare diseases.
Julia F. Slejko, PhD
University of Maryland Baltimore, Baltimore, MD, USA
Julia F. Slejko, PhD is an Associate Professor of Practice, Sciences, and Health Outcomes Research at the University of Maryland School of Pharmacy and is Co-Director of the Patient-Driven Values in Healthcare Evaluation (PAVE) Center. Dr Slejko’s research is focused on innovative approaches for decision-analytic modeling for economic and health outcomes assessments. She holds a BA in Molecular, Cellular, and Developmental Biology from the University of Colorado Boulder. During her PhD training, she focused on pharmacoeconomics at the University of Colorado School of Pharmacy Center for Pharmaceutical Outcomes Research (CePOR). Her postdoctoral training was completed at the Pharmaceutical Outcomes Research and Policy Program in the University of Washington School of Pharmacy. Prior to her PhD training, she had a 7-year career in drug discovery at Array BioPharma. Dr Slejko serves as a Value in Health Associate Editor, Co-Chair of the ISPOR Faculty Advisor Council, co-lead of ISPOR’s Women in HEOR initiative, and is a member of ISPOR’s Health Science Policy Council.
ISPOR Forums
From Research to Policy to Patients: Measuring the Impact of Health Economics and Outcomes Research (HEOR)
ISSUE: Organisations working in the field of HEOR, such as ISPOR and its members, make tremendous efforts to achieve a positive impact on health policy and decision-making. But they are grappling with the challenge of answering a critically important question: what is the real-world impact of HEOR? And how can we measure this contemporaneously? As the field evolves and decisions are made about whether and how much to fund HEOR efforts, measuring and tracking our impact is essential. In this panel, we will give case examples of measuring HEOR impact, examples of some cutting-edge approaches such as AI-driven insights, and how these data can help inform approaches for HEOR to lead change in health policy worldwide.
OVERVIEW: Jan Hansen (Genentech) will moderate this session and set the context by explaining global shifts in HEOR organization and investment. Rob Abbott (ISPOR) will present the importance of measuring HEOR’s impact in the context of ISPOR’s Strategic Plan 2030.Laura Pizzi (ISPOR) will discuss the impact measures that ISPOR currently uses, and opportunities to improve them. Lotte Steuten (OHE) will discuss ways to measure the impact of HEOR on methods guidelines, healthcare policies and patients, using the example of an analysis with BMJ Impact Analytics that tracked the trajectory and use of HEOR research in real-time. James Chambers (Tufts University) will discuss the impact of HEOR on coverage decisions using data from the Tufts Medical Center Specialty Drug Evidence and Coverage (SPEC) Database. Drawing on these examples, panellists will debate the approaches to HEOR impact measurement, including the role of data-driven methods and AI-tools, and establish a call to action for the use of these measures as well as development of new measures.
Moderators
Jan E. Hansen, PhD
Genentech, South San Francisco, CA, USA
Jan Hansen, PhD, is Vice President of the Evidence for Access Unit within Genentech’s US Medical Affairs. Her unit is staffed with researchers who are uniquely positioned and skilled to address both the value and quality drivers of access in the US. These researchers are tasked to consider both value and quality evidence when they develop access strategies and tactics. Jan is a seasoned executive with more than twenty-five years of experience in the health care and pharmaceutical industries. Jan’s pharmacoeconomics and health outcomes expertise were acquired throughout her vast career experiences in patient-based research, health care consulting, sales strategy and execution, and in both global and US focused outcomes research. She has held positions with increasing leadership responsibility at several well-regarded companies, including PCS-Pharmaceutical Data Services (now CVS-Caremark), NDC Health, Wolters Kluwer Health, Glaxo Wellcome (now GlaxoSmithkline), and Allergan. Her professional accomplishments have been recognized by several professional organizations and resulted in numerous appointments, including: the University of Iowa College of Pharmacy Advisory Council, past Chair and current member of the University of Washington College of Pharmacy’s Corporate Advisory Council, Board member (representing Genentech) of the National Pharmaceutical Council (NPC), member of the Drug Information Association (DIA) Advisory Council of North America, past NPC Corresponding Officer and Research Committee member (representing Allergan), current member of the Academy Health Institution Council, and Foundation of Managed Care Pharmacy (FMCP) Board of Trustee. Jan is a licensed pharmacist and holds a Ph.D. in Pharmacy Administration from the University of South Carolina at Columbia. She is also a speaker and guest lecturer on topics related to pharmacoeconomics and outcomes research. Jan’s ultimate passion is “building”; she takes great pride in building organizations and fostering young, innovative-thinking, yet applied researchers. For Jan, there is no greater joy than watching these researchers flourish and making a difference to patients’ lives.
Speakers
Rob Abbott
ISPOR, Lawrenceville, NJ, USA
Mr Abbott is a highly accomplished, visionary leader with more than 20 years’ experience providing executive-level strategic and operational leadership in businesses, nongovernmental organizations (NGOs), government environments, as well as professional societies and associations. Most recently, he served as executive director and CEO of Health Technology Assessment International, a global nonprofit society focused on health technology assessment (HTA). He is recognized for his knowledge in HTA, strategy, and stakeholder relations. He has successfully guided a number of organizations through their transformation and growth. He is also a pioneering social responsibility thinker and advisor who has authored 2 books and numerous articles on the nature of business and society. Mr Abbott holds an undergraduate honors degree from the University of Victoria and graduate degrees from the University of Toronto. Additionally, he holds professional designations as both a management consultant and an ICF-accredited coach.
James Chambers, PhD, MPharm
Center for the Evaluation of Value and Risk in Health, Institute for Clinical Research and Health Policy Studies, Tufts Medical Center, Boston, MA, USA
James Chambers is an investigator at the Center for the Evaluation of Value and Risk in Health at Tufts Medical Center, and a Professor of Medicine at Tufts University School of Medicine. His research has highlighted variation in patients’ access to specialty and orphan medications and in the evidence base that health plans cite in their policies. He graduated from Queens University in Belfast with an MPharm degree and previously worked as a pharmacist in the UK and Ireland. James also obtained an MSc from the University of York and PhD from Brunel University, both in Health Economics.
Laura Pizzi, PharmD, MPH
ISPOR, Lawrence Township, NJ, USA
Dr. Laura Pizzi is Chief Science Officer for ISPOR and Research Professor at Rutgers University. For nearly 25 years, she has led interdisciplinary teams of methodologists, statisticians, and clinicians to design and conduct economic and outcomes analyses on healthcare interventions and is a frequent author, speaker, and mentor on the topic.
At ISPOR, she leads the organization’s scientific strategy and initiatives in collaboration with ISPOR's global members and stakeholders.
Lotte Steuten, PhD
Office of Health Economics, London, LON, United Kingdom
Lotte Steuten, PhD, is Deputy Chief Executive of the not-for-profit Office of Health Economics (OHE) and the scientific and business lead for its international research-led work program—maintaining its reputation for objective, innovative, and high-quality research, and meeting its charitable goals. She is also an Honorary Visiting Professor at City, University of London (UK) contributing to its teaching programmes in health economics.
For more than 20 years, Lotte has worked in academic, research, and consulting HEOR roles with one focus — improving healthcare decision-making through high-quality research and analysis. To achieve that, she has collaborated with HTA authorities, pharmaceutical and medical device companies, patients, payers, policymakers, academic researchers, clinical specialists, and venture capitalists. She has gained broad HEOR expertise of various technologies: precision medicine, oncology, vaccines, antibiotics, diagnostics, digital health, and medical devices.
Lotte’s career has been based in the United States, the Netherlands, and the UK. Collaborations with HEOR experts in Southeast Asia, Africa, and Latin America have provided her with deep insights into the differences and commonalities between the role of HEOR in different healthcare systems, cultures, and societies. With this background, she has: led diverse international teams; developed strategies and delivered on program and organisational missions, values, and objectives; acquired research funding and managed budgets; and been responsible for executive decision-making as well as legal and fiduciary matters.
As an active ISPOR member since 2004, Lotte has served on various Taskforces and Committees, delivered Short Courses, was co-Chair of ISPOR EU 2021, and is currently a member of the Board of Directors, and an Associate Editor for Value in Health. She aims to be a role model for diversity and played an active role in the 'Women in HEOR' initiative.
Before joining OHE, Lotte worked in Seattle (US) as an Associate Professor at the University of Washington and at the Fred Hutch Cancer Research Center. She graduated cum laude with her PhD from Maastricht University (NL) and then worked at Brunel University (UK) and Twente University (NL).
The Future of Single-Arm Trials and External Controls in Health Technology Assessment
Digital Conference Pass
Recent years have witnessed the release of several publications on the acceptance of single-arm trials (SATs), which has come about following the increasing use of SATs for marketing authorisation. Likewise, the inclusion of SATs in HTA submissions has increased dramatically over the last decade.
In March 2024, the European Commission ratified methodological guidelines specifying the statistical analyses for the new Joint Clinical Assessment (JCA). The guidelines address the inclusion of SATs and external controls through discussion on methods for analysing non-randomised data and disconnected networks.
However, the pre-conditions for the acceptance of non-randomised comparisons, which include pre-specification of the analysis methods, are not without their challenges, especially given the time available for the JCA. Moreover, such pre-conditions are likely to differ between EU member states.
With the new regulation taking effect in January 2025, there is an imperative to clarify the conditions under which the use of non-randomised evidence from SATs in benefit dossiers will be accepted. This session will serve as a pivotal platform for fostering dialogue between HTA bodies and the industry regarding expectations and achievable outcomes.
Mr. Scott will outline the current guidelines regarding the use of SATs and external controls, as well as offering insights into the ongoing discourse surrounding their acceptance. Dr. Jen, Dr. McConnell, and Dr. Ren will put forward their position regarding the conditions under which treatment comparisons from SATs using external controls can be relied upon for the purpose of providing evidence of additional benefit. The remainder of the session will be left for debate with the audience on the expectations and efforts required for HTA submissions, considering the challenges of clinical trial design, development costs, ethical considerations, and the imperative to expedite patient access to effective medications.
Moderators
Martin Scott, MSc
Numerus, Reutlingen, Germany
Martin Scott is the scientific director at Numerus in Germany. His role focuses on advising Clinical Development and Market Access Teams with regard the evidence requirements of HTA in Germany as well as for the future European Joint Clinical Assessment. Having worked as a statistician in the UK, Germany, Switzerland and Italy at pharmaceutical manufacturers and CROs, Martin has been responsible for the statistical analysis and design of clinical trials across all phases of development. Professional interests include the use of observational/pragmatic trials for regulatory submissions and the statistical methods employed therein.
Speakers
Min-Hua Jen, PhD
Eli Lilly and Company, Uxbridge, LON, United Kingdom
Dr Min-Hua Jen is currently Senior Director - Real-World & Access analytics at Eli Lilly, leading the International Business team on Market Access/HEOR/Medical affairs statistical support. She has extensive experience applying statistics to clinical research, epidemiology and health economics and outcomes research in academia and industry settings. She is an active member in the PSI/EFSPI HTA Special Interest Group (SIG) and the engagement chair of the ISPOR Oncology SIG. She was trained in Epidemiology and Statistics and obtained her PhD at University of Bristol. Her research interests including indirect treatment comparisons and network meta-analysis; particularly incorporate external data for time to event outcomes, surrogacy analyses, multilevel modelling and health economic modelling.
David McConnell, PhD
National Centre for Pharmacoeconomics, Dublin 8, D, Ireland
Dr. David McConnell is a Senior Statistician at the National Centre for Pharmacoeconomics (NCPE). David holds a PhD in mathematics from Trinity College Dublin and previously held lectureships at the University of Glasgow and Cardiff University. He joined the NCPE in 2019. His role at the NCPE involves evaluating statistical aspects of clinical trials, evidence synthesis and economic modelling in health technology assessments, as well as research and guideline development in these areas. David’s research interests include indirect treatment comparisons, survival extrapolation, and communicating uncertainty to decision-makers. He is a member of the Methodology Subgroup of the EU HTA Coordination Group, and previously contributed to methodological guideline development and scientific advice procedures as part of EUnetHTA21, including co-authoring the guidelines on Direct and Indirect Comparisons.
Sarah Ren, PhD
University of Sheffield, Sheffield, United Kingdom
15:45 - 16:15
Exhibit Hall Theater
Better and Faster: Automating Model Building and Statistical Analyses
Digital Conference Pass
Health economic models play a vital role in product development, providing valuable information and insights from early planning and portfolio selection to pricing analysis. Building models is complex, requiring integration of evidence from multiple sources, including results from de novo statistical analyses, into a computational framework that allows users to modify inputs and review results. The resulting modeling framework must have face validity, avoid programming errors and produce valid forecasts. Standardizing and automating model building and related statistical analyses facilitates faster model development, enhances quality and reliability, and expands accessibility to the development process.
In this presentation, we will explore approaches to automating model-building and required statistical analyses and introduce an innovative modeling platform. This platform automates model generation for a broad range of therapeutic areas and model types using a wizard for capturing model specifications and creating customized models while minimizing errors. Once the generated model is populated with inputs, the platform can run the model on a PC or a server-based environment to optimize run-times. Automation of the statistical analyses that inform model inputs will also be described and illustrated with an application for parametric fitting of time to event equations. Finally, these automation tools will be demonstrated to the audience by using the platform.
During the Q&A period, the advantages and challenges of automated model building and contrasts to AI-based model generation will be discussed.
Discover how an automated model-building tool can significantly expedite the creation of early health economic models that adhere to good practice guidelines and minimize errors. Furthermore, learn how automation tools leverage the computational power of server-based installations while preserving the Excel-based nature of models preferred by stakeholders.
Sponsor: Evidera
Sponsor
Evidera
Moderators
Ágnes Benedict, MSc, MA
Evidera, a part of Thermo Fisher Scientific, Vienna, Austria
Agnes Benedict, MSc, MA, is an Executive Director of Scientific Affairs with the Evidence, Modeling and Communication group at Evidera, a PPD business unit, and the Executive Director of the Centre of Excellence in Health Economics and is based out of Vienna, Austria. She directs strategic multi-workstream HEOR projects to support market access. She specializes in portfolio models and advanced modelling studies as well as HTA submission support, in any therapeutic area.
Ms. Benedict is responsible for internal knowledge sharing and cross-pillar collaboration within Evidera.
She is an Editorial Advisory Board member for Value and Outcomes Spotlight Magazine of ISPOR. Ms. Benedict holds a BS in economics; a Master's degree in public economics from the Budapest University of Economic Sciences, Hungary; and a Master’s in health economics from the University of York, UK, as well as a postgraduate certificate of statistics from the University of Sheffield, UK. She has presented studies at international conferences and is the co-author of abstracts and manuscripts that have appeared in the British Journal of Cancer, BJU International, Expert Opinion in Pharmacotherapy, International Journal of Clinical Practice, Journal of Affective Disorder, JNCCN and Pharmacoeconomics.
Speakers
Apoorva Ambavane, MSc, MPH
Evidera, Inc, London, United Kingdom
Apoorva Ambavane, MPH, is a Senior Research Scientist and Senior Director with Evidera in the Evidence Synthesis, Modeling, & Communications team. In her role at Evidera, Ms. Ambavane’s main area of interest is pharmacoeconomics and product value development. Her work focuses on healthcare costs and outcomes modeling, including cost-effectiveness analysis, budget impact analysis and costs comparison, and developing value proposition for drugs. Ms. Ambavane has extensive experience in designing and implementing economic models, preparing and supporting reimbursement submissions to HTA bodies, and dissemination of model results in the form of technical reports, and manuscripts. Ms. Ambavane has led and participated in the designing and implementation of health economic models in Microsoft Excel (Markov, microsimulation, discretely integrated condition event [DICE]). She specializes in oncology; however, has experience in auto-immune disorders, neurology, and nutrition products. Before joining Evidera, she was an intern with the global health economics and outcomes research team at Bayer Healthcare Pharmaceuticals in New Jersey. At Bayer, she conducted a retrospective claims database analysis and systematic literature reviews to support reimbursement claims in the U.S., the UK, France, Germany, Italy, and Sweden. Ms. Ambavane received her MPH from Columbia University. Her concentration was healthcare policy and management. Prior to studying at Columbia, she graduated with a BS from Texas A&M University in genetics and biochemistry.
K. Jack Ishak, PhD
Evidera, St-Laurent, QC, Canada
K. Jack Ishak, PhD, is Vice President, Statistical Methodology and Strategy at Evidera, a PPD business. Dr. Ishak works to help Evidera remain a scientific leader in the development and application of statistical methods for health technology assessment, real-world studies, and patient-reported outcomes by fostering flexible and integrated solutions to research projects and promoting best-in-class capabilities and training. He has been involved in methodological developments in indirect treatment comparison (simulated treatment comparisons), predictive analyses to model disease progression, projection of incomplete survival curves, sequential modeling of treatment lines, crossover adjustment methods in oncology, and the use of simulation for disease modelling and for testing novel study designs (e.g., Bayesian adaptive designs in the context of comparative effectiveness research). Dr. Ishak was involved in developing a forecasting tool combining growth and decay and dynamic disease transmission modelling to predict COVID case counts across a wide range of locations. His current work focuses on automation in statistical analyses for comprehensive, standardized, and reliable reporting of results, sample size planning for studies leveraging external comparator arms, and applications of the win ratio to optimize statistical power in rare disease trials. Dr. Ishak’s work has been published extensively in peer reviewed journals.
Dr. Ishak is based in Montreal, Canada; he received his undergraduate degree in statistics from Concordia University and obtained his master’s and doctoral degrees in biostatistics and epidemiology from McGill University.
16:00 - 16:30
Get Involved—Make the Most of Your ISPOR Membership
Are you an ISPOR member or considering becoming an ISPOR member? Stop by our booth to discover how you can make the most of your member experience! Meet with ISPOR staff to explore exciting opportunities to volunteer, join special interest groups or chapters, and publish your work in ISPOR’s esteemed journals.
16:00 - 16:45
Poster Tours
Case Studies Poster Tour: HEOR in Action
Posters Featured in this Tour:
PT43 Implementation of a Health Technology Assessment Unit: Experience in the Largest Private Healthcare Provider in Oncology in Brazil
PT44 Bridging HTA Assessment and Policy Appraisal and Decision
PT45 Don't Sleep on HTA: From HTA to Clinical Guidelines Within Treatment of Obstructive Sleep Apnea
PT46 Development of Economic Evaluation Studies (EEs) in Saudi Arabia
PT48 Use of an Individual-Based Approach in Oncology: A Case Study for the Treatment of Pediatric Patients With Gliomas
Moderator
Katja Rudell, PhD, MSc
Parexel International, LONDON, LON, United Kingdom
Katja Rudell cofounded the ISPOR Special Working Group in COA at ISPOR in 2019 and is a Senior Director of COA Science at Parexel. She holds a PhD from University of London, and has been working in Clinical Outcomes Research for 20 + years.
Real-World Evidence Poster Tour
Posters Featured in this Tour:
PT37 Comparison of ICD-10 Diagnoses and Longitudinal Estimated Glomerular Filtration Rate (eGFR) in the Detection of Incident Stage 3 Chronic Kidney Disease (CKD): A Retrospective Comparison Study Using UK Secondary Care Data
PT38 Is There an Increased Risk of Developing Hypertension After a COVID-19 Infection?
PT39 A Mixed-Methods Blueprint for Obesity Healthcare Policy: From Research to Action
PT40 Assessing the Value of Real-World Evidence for HTA-Decision Making
PT41 Benefits of Inhaled Corticosteroids (ICS) in COPD Maintenance Combinations: Real-World Evidence Using Longitudinal Targeted Maximum Likelihood Estimation
PT42 Disease Burden and Unmet Medical Need in Patients With Ulcerative Colitis in Greece: A Cross-Sectional Patient Survey
Moderator
David Thomson, BSc(Hons), MSc
National Institute for Health and Care Excellence, Manchester, United Kingdom
16:00 - 19:00
Poster Session 4
Live
16:15 - 17:15
Member Group Meetings
Latin America Consortium Industry Committee
The Role of Industry in Generating and Utilizing RWE for Decision-Making
During this meeting, we will explore the important role that industry plays in generating and utilizing Real-World Evidence (RWE) to inform decision-making processes. As healthcare systems increasingly rely on data-driven insights, the importance of RWE in shaping policy, clinical guidelines, and regulatory approvals has grown exponentially. Industry stakeholders are at the forefront of this movement, leveraging RWE to demonstrate the value and effectiveness of their products in real-world settings.
Various aspects of RWE, including data collection methods, analytical techniques, and the integration of RWE into regulatory and reimbursement frameworks in Latin America. Participants will gain insights into best practices for collaborating with healthcare providers, payers, and policymakers to ensure that RWE is utilized effectively and ethically. By sharing case studies and discussing emerging trends, this forum aims to highlight the transformative impact of RWE on healthcare decision-making and the collaborative efforts needed to advance this field in Latin America.
Join us to understand how the industry can contribute to generating high-quality RWE and support evidence-based policy decisions.
Moderators
Arturo Figueroa, MSc
Pharmamanagement EAS S de RL de CV, Mexico City, DF, Mexico
Speakers
Diego Guarin, MD, MPH, MA
Merck, Kenilworth, NJ, USA
Dr. Diego Guarin is the Regional Market Access Lead for Latin America at Merck (known as MSD outside USA and Canada). where he focuses on improving patient access to health innovations. Since 2008, he has been leading Regional Market Access and Evidence and Value Development (HEOR) teams at top global biopharmaceutical companies. In addition to his industry experience, he worked as consultant at multilateral organizations (PAHO, GFATM) and in a health policy think-tank.
Dr. Guarin has contributed to the development of Pharmacoeconomics and Outcomes Research in Latin America since 2007 serving as chair of two ISPOR Latin American Consortium Committees (2010-12/2014-16) and being appointed as the first representative of Latin America at the ISPOR Health Science Policy Council (2017-2019). He is also member of HTAi interest group on Developing Countries since 2009.
In addition, during the past 8 years, he has been a company representative at FIFARMA, the Latin American Federation of the Pharmaceutical Industry, serving as co-Chair of to working groups (Value and Access to Innovation 2017-20/2021-22 and Health Policies 2022-to date).
Dr. Guarin graduated as Medical Doctor from Universidad del Rosario-1653 (Colombia) and holds master’s in Public Health in Developing Countries from the London School of Hygiene and Tropical Medicine (UK), Health Economics and Pharmacoeconomics from Universitat Pompeu Fabra (Spain) and in Health Economics Policy and Management from the London School of Economics and Political Science (UK).
Patricia Herrera, Eng
MSD Mexico, Mexico, EM, Mexico
Marcos Santos, MD, PhD
UNESCO Chair of Bioethics - Brasilia University, Sao Paulo, SP, Brazil
Radiation-Oncologist with Master in High Technologies in Radiation-Oncology, at the University of Murcia, Murcia, Spain; MBA in Pharmacoeconomics at the Paris-Descartes University, in Paris, France and PhD in Medical Ethics at the UNESCO Chair in Bioethics, Brasília University, Brasília/DF. Member of the Quality-of-Life Study Group in Head and Neck Cancer – EORTC (European Organization for Research and Treatment of Cancer). Vice-President of the ISPOR Brazilian Chapter.
16:15 - 17:45
ISPOR Ukraine-Hungary-Poland Chapters Meeting
Come and connect with the ISPOR Hungary, Poland and Ukraine chapters during this joint meeting, open to all interested in learning about current trends in the Central and Eastern Europe region. The chapters will also share perspectives on priority topics in their respective countries. This is a great opportunity to network with colleagues and discover how to actively engage with these chapters.
17:00 - 18:00
Podium Sessions
Real-World Analyses of Electronic Health Records
Digital Conference Pass
This session will feature research advanced methods to curate and analyze electronic health records.
Moderator
Isaac Corro Ramos, PhD
Institute for Medical Technology Assessment, Roterdam, ZH, Netherlands
Isaac Corro Ramos, PhD obtained his Master’s degree in Mathematics (option Statistics and Operations Research) from the University of Sevilla in June 2001. Between June 2001 and July 2005 Isaac had several jobs in different working areas and countries. He worked as a high-school Mathematics teacher in Sevilla (Spain), as a software programmer in Madrid (Spain) and Vienna (Austria), and as a researcher in Rome (Italy). In July 2005 he started his Ph.D. at the Department of Mathematics and Computer Science of the Eindhoven University of Technology working on the STRESS (Statistical Testing and Reliability Estimation of Software Systems) project. He defended his thesis on December 15, 2009. Since August 2009 Isaac works as a scientific researcher at the Institute for Medical Technology Assessment (iMTA) in Rotterdam, The Netherlands. Since he enrolled iMTA he has worked in several research projects whose subjects include probabilistic modelling, cost-effectiveness analysis of health care technologies, value of information analysis and discrete event simulation.
P47: Evaluation of Natural Language Processing (NLP) on Electronic Medical Records: A Proof of Concept on Chronic Graft Versus Host Disease (cGVHD) in France
5:30PM - 5:45PM
Colas S1, Gilles V1, Chantepie S2, Daguindau E3, Huynh A4, Loschi M5, Robin M6, Raus N7, Requillard C8, Chuttoo L8, Poplu A8, Allali N1, Kiprijanovski D1, Leouay F8, Jeanbat V9, Kirion J9, Cottin J9, Buchbinder N10, François S11, Villate A12, Bouee S13
1Sanofi, Paris, France, 2CHU de Caen, Caen, France, 3CHU de Besançon, Besançon, France, 4Oncopole of Toulouse, Toulouse, France, 5CHU de Nice, Nice, France, 6APHP, Paris, France, 7SFGM-TC, Paris, France, 8LIFEN, Paris, France, 9CEMKA, Bourg-la-Reine, France, 10CHU de Rouen, Rouen, France, 11CHU Angers, Angers, France, 12CHU de Tours, Tours, France, 13CEMKA, BOURG LA REINE, France
OBJECTIVES: Manual chart review used to generate real-world data can be long and expensive. We used an innovative approach based on an artificial intelligence natural language processing (AI-NLP) method to structure data into a cohort of patients affected by chronic Graft Versus Host Disease (cGVHD) following hematopoietic stem-cell transplantation (alloHSCT).
We aimed to demonstrate this approach effectiveness in terms of data quality, along with related time saving.
METHODS: The NLP scanned all electronic medical records, identified eligible patients and used relevant information to derive and infer the variables.
To ensure completeness and accuracy of the NLP derived data, a three-step quality check was performed: 1) review of variables derivation by clinical research associate (CRA) 2) their comparison with the European Bone Marrow Transplant (EBMT) registry 3) their validation by investigator experts.
RESULTS: From all alloHSCT patients’ medical files (N=306), 20 met the selection criteria, whose data were extracted with NLP. This comprehensive process took 30 hours by a data scientist and 0.5 hour of investigator expert time per center.
The completeness of data derived by NLP varied according to nature of the variables: sociodemographics and cGVHD treatments (100%), graft characteristics (>75%), alcoholism (20%), mostly depending on the information documented in the medical records. The review by CRA did not lead to discordance except for the cGVHD treatments, for which some adjustments were needed. The quality checked against EBMT registry showed high accuracy of the NLP derived data for all 20 patients. Concordance rates were: stem cell source, donor gender and type (100%), alloHSCT indication (94%), alloHSCT date (89%), CMV positivity (87%), conditioning and prophylactic treatment of cGVHD (78%). Validation by investigator experts is still ongoing.
CONCLUSIONS: This innovative AI-NLP process allows to generate a comprehensive, robust and accurate structured dataset, from scattered information of all eligible patients, in medical records, with minimal human intervention.
P48: Primary Care Resource Utilization and Costs of Imminent Subsequent Fractures in Postmenopausal Women: A Distributed Network Analysis Using Data Mapped to OMOP Common Data Model From Five European Countries
5:45PM - 6:00PM
Fabiano G1, Chen X1, Rashod-Mistry T1, Delmestri A1, Moayyeri A2, Warden J2, Reyes C3, Fernandez Bertolin S3, Brash JT4, Verhamme K5, Tan EH1, Pinedo-Villanueva R1
1University of Oxford, Oxford, OXF, UK, 2UCB Pharma, Slough, UK, 3Institut Universitari d'investigació en Atenció Primària "Jordi Gol", Barcelona, Spain, 4IQVIA, Brighton, UK, 5Erasmus MC, Rotterdam, Netherlands
OBJECTIVES: To describe primary care resource utilisation and costs of index and imminent subsequent fragility fractures in postmenopausal women using electronic medical records from the United Kingdom (CPRD-Aurum), the Netherlands (IPCI), Spain (SIDIAP), Germany (IQVIA-DA), and France (IQVIA-LPD) mapped to the OMOP-CDM.
METHODS: A time-stratified propensity-score-matched study identified three cohorts of women aged ≥50 in 2010-2018: imminent subsequent fracture within two years of their index fracture (F2); index fracture with no fracture two years prior (F1); and without fractures (F0). Consultation counts by staff roles (CPRD), specialty (SIDIAP, IQVIA-DA and IQVIA-LPD), and visit types (IPCI) were extracted and costs estimated based on local tariffs. The impact of first (F1 vs. F0) and subsequent fractures (F2 vs. F1) was described between the matched cohorts.
RESULTS: For F1 vs. F0, data from 343,573 and 1,096,675 women, respectively, were used across all databases. For F2 vs F1, samples comprised a total of 97,750 and 316,451.
Number of consultations per woman per year was consistently higher for F1 compared to matched-F0, with ratios of the medians being 1.21(CPRD), 1.28(IPCI), 1.30(SIDIAP), 3.20(IQVIA-DA), and 2.80(IQVIA-LPD). Mean costs per woman per year were higher for F1, with increases ranging from €27 (IQVIA-LPD, €95 vs. €68) to €243 (IQVIA-DA, €628 vs. €385). Number of consultations for F2 were higher than matched-F1, with ratios of the medians being 1.08(CPRD), 1.17(IPCI), 1.08(SIDIAP), 1.35(IQVIA-DA), and 1.39(IQVIA-LPD). Mean costs were higher for F2, except for IQVIA-LPD: between -€3 per woman per year (IQVIA-LPD €95 vs. €91) and €72 (IQVIA-DA, €708 vs. €636).
CONCLUSIONS: Fragility fractures are associated with an increased number of primary care consultations and associated costs, with imminent subsequent fractures generally furthering this burden. This is the first multi-country European study describing resource utilisation and costs amongst postmenopausal women with fractures using data mapped to OMOP-CDM.
P46: Comparing Data Collection Methods: Health Care Professional Survey-Based Chart Audit Versus Automated Electronic Health Record Databases in Multicountry Drug Utilization Studies
5:15PM - 5:30PM
Jouaville S1, Drouot D2
1IQVIA, La Defense, 92, France, 2IQVIA, La Defense, Hauts-de-Seine, France
OBJECTIVES: When automated electronic health record (EHR) databases are insufficient or unavailable, Health Care Professional survey-based chart audit (HSCA) offer a valuable alternative for health outcomes research studies. However, limited information exists regarding the comparison between HSCA data and EHR data.
METHODS: This was a secondary analysis of 2 multi-country post-authorization safety studies, one HSCA and an HER databases study, both aiming to collect drug utilization data to assess the effectiveness of risk minimization measures related to the use of an antiepileptic drug among female patients. Patient data from 6 HER databases (general practitioners (GP) panel in France, UK, Germany and Spain; neurologists/psychiatrists (NP) panel in Germany and Spain) were compared to HSCA data collected during the same period over the same physician specialties in four countries. Two age groups (females of child-bearing potential (CBP), and non-CBP) and 3 possible indications (epilepsy, bipolar disorder, migraine) were defined. Distribution of age groups and indications in both study populations was compared using EHR data as reference.
RESULTS: The non-CBP age group was predominant in all panels (ranging from 50.5% in UK GP HSCA to 67.3% in Germany GP EHR) except in Spain NP panel (45.5% in EHR and 46.6% in HSCA). Epilepsy was the first indication recorded in all panels (ranging from 58.7% in Spain GP HSCA to 96,3% in Germany GP EHR) except in Spain NP panel (29.9% in HER and 18.8% in HSCA). These was no significant differences for age group distribution between the 2 sources in France GP, Germany and Spain NP panels. Proportion estimates for indications differed between the 2 sources in the majority of the panels.
CONCLUSIONS: Differences in data collection methodology can explain the discrepancies observed between the 2 sources, however, the use of HSCA can generate valuable insights on routine clinical practice.
P45: Optimizing Electronic Medical Records Data Completeness With On-Premise Artificial Intelligence: A Study on Large Language Models Enhancing Medication Documentation
5:00PM - 5:15PM
Shaked O, Gruzman D, Kustin T, Tish G, Haron Y, Galperin G, Sadetzki S
Briya Labs, Tel Aviv, Israel
OBJECTIVES: The study assesses the contribution of using free-text from electronic medical records (EMR) by large language models (LLMs), to the completeness and validity of information on medication-use. We present an example of data on aspirin intake during pregnancy.
METHODS: Data on medication use (Acetylsalicylic Acid, “Aspirin”) was derived from available EMR sources, coded and free-text fields in several hospitals within Briya’s network. Medication listed in coded fields was identified using ATC codes. Free-text analysis of EMR from the documenting departments (emergency room, delivery room, high-risk and post-delivery wards) was performed using on-premise open-source LLM (LLama 3-8B). Frequency of aspirin use was described by source of data, department and hospital. Sample of notes were reviewed by human experts for validation of LLM and F1 score, precision and recall (sensitivity) were calculated.
RESULTS: The study population included 16,122 women who gave birth to 19,185 infants (18,836 deliveries) between 03.2020- 05.2024. 1,142 women (6.1% of deliveries) used aspirin during pregnancy. Positive aspirin use was documented 1,596 times, 1,111 in free-text (range 1-6 notes/women). Compared to experts, LLM validation metrics were: F1=0.9; precision=0.88, recall=0.92). About 66% of patients who received the medication (n=755) were identified exclusively via free-text, another third (n=356) were identified by both free-text and coded fields, resulting in free-text coverage of 97.3%. The remaining 2.7% of aspirin uptake was identified exclusively in coded fields. Comparison by documenting department and hospitals, dosage, and treatment duration will be presented.
CONCLUSIONS: The use of AI for free-text data abstraction could capture the vast majority of data on medication use. LLMs demonstrate excellent zero-shot performance without task-specific training, and deployed on-premise without GPUs, optimizing hospital resources. Completeness and quality of medication-use are highly dependent on source of data.
Addressing Challenges With Health-Related Quality of Life Measurement
Digital Conference Pass
Health-Related Quality of Life (HRQoL) measurement is a cornerstone of health economics and outcomes research, playing a crucial role in healthcare decision-making and resource allocation. However, accurately capturing and interpreting HRQoL data presents numerous methodological challenges. This podium session brings together four studies that discuss the complexities of HRQoL measurement and utility estimation across diverse contexts and populations. By examining mapping functions, measurement tool comparisons, temporal considerations, and proxy ratings, this session aims to enhance our understanding of HRQoL measurement challenges and inspire innovative approaches to overcome them.
Moderator
Irina Kinchin, PhD, MSc
Trinity College Dublin, the University of Dublin, Dublin, D, Ireland
Dr Irina Kinchin is a Research Assistant Professor at Trinity College Dublin's Centre for Health Policy and Management, specializing in impact evaluation and outcomes research. Her work focuses on developing innovative approaches to measuring and valuing health and wellbeing outcomes, particularly in complex and progressive conditions such as dementia. A methodologist for the NIHR Programme Grants committee and Associate Editor for both the International Journal of Technology Assessment in Health Care and Quality of Life Research, she leads several major research initiatives funded by HRB, IRC, EuroQol Foundation, and the Alzheimer's Association. Dr Kinchin was recognized as a Top Scholar Worldwide in Economic Impact Analysis in 2024.
P42: EQ-5D-5L Mapping Function Recently Recommended by NICE Is Likely to Generate Higher Utility Values
5:15PM - 5:30PM
Maervoet J1, Bergemann R2
1Parexel International, Wavre, Belgium, 2Parexel International, Basel, Switzerland
OBJECTIVES: In the absence of an accepted local EQ-5D-5L tariff, NICE requires EQ-5D-5L responses to be mapped onto the long-standing EQ-5D-3L value set for the UK. Their 2022 Manual recommends using the mapping function based on the EEPRU dataset recently developed by the NICE Decision Support Unit (DSU), instead of the van Hout crosswalk that was previously endorsed. Our aim was to compare utility values derived using these two mapping methods.
METHODS: For all 3,125 unique health states existing in the EQ-5D-5L system, utility estimates were calculated using both mapping functions. Absolute differences in resulting values were calculated and a scatterplot was generated to identify outliers and allow visual comparison. Differences in mean utility values were evaluated in subsets of health states, grouped by the level of severity of reported health issues. Simulations in hypothetical patient populations were conducted to evaluate the potential impact of the mapping functions on utility values derived from EQ-5D-5L clinical trial data.
RESULTS: For 1,898 (61%) health states spread across the severity spectrum, the DSU EEPRU mapping function generates a higher utility value than the van Hout Crosswalk. The mean difference was 0.062 (± 0.139 standard deviation), ranging from +0.619 to -0.198 for individual health states. Our analyses and simulations suggest that mean utility values for a patient population estimated with the DSU mapping function would typically lie 0.050 to 0.100 higher than those obtained with the van Hout Crosswalk.
CONCLUSIONS: Our study shows that not only the choice of the EQ-5D instrument, but also the mapping approach used can have a substantial impact on utility values and, hence, estimated health gains and cost-effectiveness ratios. The DSU EEPRU mapping function tends to shift UK utility values further upwards, potentially making it harder for new technologies to demonstrate utility gains and obtain NICE recommendation.
P43: Health-Related Quality of Life in People Living With Dementia: Over and Underestimation of Proxy-Ratings
5:30PM - 5:45PM
Buchholz M1, Engel L2, Pfaff M1, Michalowsky B1
1German Center for Neurodegenerative Diseases (DZNE), Greifswald, MV, Germany, 2Monash University, Melbourne, Australia
OBJECTIVES: Proxy ratings primarily provided by informal caregivers are usually administered if people living with dementia (PlwD) are cognitively unable to rate health independently. Typically it is reported, that proxies underestimate PlwD health. However, analyses of self- and proxy-rated discrepancies in individual responses that focus on HRQoL dimensions are currently lacking. Therefore, we examined discrepancies between self- and proxy-rated health on an individual response level.
METHODS: We analyzed EQ-5D-5L baseline data of a cluster-randomized, controlled intervention trial, administered as a self- and proxy-proxy-assessment of n=174 dyads (PlwD and their caregivers). Initially, we calculated agreement statistics (weighted kappa) for each dimension of the EQ-5D. For individual response levels of each dimension, we analyzed the degree of inconsistencies between self- and proxy ratings ranging from 0 (identical responses in all dimensions) to 5 (different responses in all five dimensions). Further, we grouped the PlwD into “no problems” and “problems” depending on the EQ-5D-5L dimensions to compare the response distances between PlwD and caregivers.
RESULTS: PlwD had a mean age of 80.1 years (49.1% female) and caregivers a mean age of 67.9 years (67.8% female). Every third PlwD lived alone (62%), and 90% of PlwD were mild to moderate cognitively impaired (MMSE mean score 19.2). In general, PlwD rated their health better than proxies, with lowest kappa coefficients for usual activity (0.23) and highest for pain/discomfort (0.40). Nine dyads (5%) showed identical ratings. Among the grouped PlwD without and with problems, proxies estimated the PlwD as either worse (underestimation) or better (overestimation), coupled with more frequent mid-response options by proxies. Over- and underestimation was more present in mobility, usual activities, and self-care.
CONCLUSIONS: The two trends of contrary response behavior (over- and underestimation) of both rater types underline the need for further research when using HRQoL measures as proxy instruments and examining influencing factors in that context.
P44: Examining the Prediction Properties of a Visual Analogue Scale for Utility Measurement in End-Stage Liver Disease Pre and Post Transplantation
5:45PM - 6:00PM
Canton P1, Burns R2, Mulrennan K2, McCarthy E3
1Atlantic Technological University, Sligo, Ireland, 2Atlantic Technological University, Sligo, Sligo, Ireland, 3Atlantic Technological University, Galway, Galway, Ireland
OBJECTIVES: This study aims to investigate the predictive properties between a Visual Analogue Scale (VAS) and the EuroQol EQ-5D-5L utility scores in End-Stage Liver Disease (ESLD) patients across European countries as part of the EU COPE transplantation trial.
METHODS: Complete case data was extracted from the EU COPE liver transplantation trial (n=79) across three time points. Country-specific EQ-5D-5L normative values were used to calculate utility scores. Pearson’s correlation coefficient assessed relationships between utility scores and the VAS scores. Age was the predictor for two linear regression models that explored its influence on the VAS and utility scores. A pooled analysis of all time points evaluated the predictive power of the VAS on utility scores.
RESULTS: In the UK, correlations ranged from 0.55 to 0.62 (p < 0.001). Spain and Belgium showed correlations above 0.70 (p < 0.05), while Germany had weaker correlations, especially at 30 days post-transplant (p = 0.054). Age significantly affects VAS scores in the UK, Spain, and Belgium. Utility scores demonstrated significant age effects in the UK and Belgium, but not in Germany and Spain. When assessing model fit, R2 values range from 34.6% to 41.5%, which signifies moderate predictive model given limitations on available controls and confounding factors. Therefore, it suggests that utility scores explain a moderate amount of the variability in EQ-VAS overall.
CONCLUSIONS: The study found that utility and VAS scores are associated, though the correlation strength varies across countries. Age significantly affects both measures, contributing to variation, which suggests age-related stratification is necessary for ESLD quality of life modelling. The moderate and variable predictive power of utility scores may indicate that the two techniques cannot be considered as interchangeable. Further research is warranted in understanding computational approaches addressing the impact of clinical heterogeneity for assessing quality of life.
P41: The Influence of Recall and Timing of Assessment on the Estimation of the QALY When Health Fluctuates Recurrently
5:00PM - 5:15PM
Sanghera S1, Coast J2, Walther A3, Peters T4
1University of Bristol, Bristol, BST, UK, 2University of Bristol, Bristol, UK, 3University Hospitals Bristol, Bristol, Bristol, UK, 4University of Bristol, Bristol, Bristol, UK
OBJECTIVES: When health fluctuates recurrently, obtaining meaningful quality-of-life estimates is challenging. Quality-of-life changes may be missed or over/underestimated due to measures’ recall periods and timing. Using chemotherapy as a case study, we compare responses using different recall periods and timing to inform how/when to capture quality-of-life during predictable recurrent fluctuations in health; and consider the implications for other contexts with fluctuations.
METHODS: During one 3-weekly cycle of chemotherapy for cancer, patients were randomly assigned to complete:
(1) EQ-5D-5L daily with a daily recall period, weekly with a weekly timeframe, and at 3 weeks with a 3-week timeframe (2) SF-12v2 daily with a daily recall period, weekly with a weekly timeframe, and at 3 weeks with a 3-week timeframe; (3) SF-12v2 daily with a 3-week recall period; Utilities (values anchored at 1 (full-health) and 0 (dead)) were generated and repeated measures ANOVA, t-tests and effect sizes calculated.
RESULTS: 503 patients consented; all 21 daily questionnaires were completed by 84(50%), 67(40%), 72(43%) in the groups respectively.
Both measures captured fluctuations in quality-of-life suggesting differences are due to recall effects. Mean daily scores were greater than scores for the past week at days 7, 14 and 21 (p<0.0001), which were greater than the past 3 weeks (p<0.0001). By capturing quality-of-life with a ‘past week’ recall at days 7, 14 or 21, utility was overestimated by 0.0782 to 0.0266, with the comparison at day 7 reaching a minimally important difference. Capturing quality-of-life with ‘past 3 weeks’ overestimated utility by 0.0746 and 0.0310 for EQ-5D and SF-6D, heavily skewed by the first treatment week.
CONCLUSIONS: Current practice of using a single estimate with a daily (EQ-5D) or longer (SF-12/SF-36) recall could bias cost-effectiveness estimates. Quality-of-life should be captured frequently with short recall when fluctuations are likely, with longer recall less frequently in more stable periods.
Breakouts: IP, WS, & OBS
Are There Any Mutual Learnings on Health Policy Among CEE Countries?
Digital Conference Pass
Level: Introductory
ISSUE:
Central and Eastern European (CEE) countries share common historical, economic, and social backgrounds, which present unique challenges and opportunities in their healthcare systems. This panel will explore the potential for mutual learning and collaboration on health policy among them, aiming to identify whether shared experiences can foster improved health outcomes and system efficiencies across the region. We will examine whether CEE countries look to each other for guidance on pressing health policy topics.
OVERVIEW:
The discussion will delve into the implementation of multi-criteria decision analysis and value-based approach, with a particular focus on its role in supporting reimbursement decisions for highly innovative and orphan drugs. We will also explore the inclusion of real-world data in decision-making processes, not only for newly invented health technologies but across a broader spectrum of healthcare interventions. Another key area of focus will be the experience on the assessment and reimbursement of digital health technologies as well as medical devices, examining how CEE countries can develop robust frameworks to support the integration of these innovations. Additionally, we will consider the implementation efforts related to the EU regulation on HTA, exploring how collaboration in these areas can streamline processes and contribute to better decision-making. The panel will also share insights related to the methods of health technology assessment and the reimbursement of medical devices. This discussion will provide a comprehensive overview of the opportunities and challenges faced by CEE countries in their health policymaking, highlighting areas where mutual learning and collaboration can lead to significant improvements in healthcare delivery and outcomes across the region. By drawing on the expertise and experiences of a range of governmental representatives, we aim to determine whether CEE countries truly look to one another when making health policy decisions and if yes, understand the criteria they use in doing so.
Moderators
Malwina Holownia-Voloskova, MSc, PhD
Certara, Cracow, n/a, Poland
Malwina is Associate Director HEOR&MA in Certara and her area of responsibility includes projects related to Central and Eastern Europe (CEE). She has 12+ years of experience in the field of drug market access and health technology assessment in various fields of medicine. In the past she was the head of the HTA department, which she founded within the scientific institute of the Moscow City Health Department. She has also actively participated in organizing the international meetings of ISPOR branches for CEE region. Her doctoral thesis was focused on health-related quality of life, measured by the EQ-5D-5L questionnaire; the topic of her master's thesis was comparison drug reimbursement systems in Great Britain, Germany, and Russia, both diplomas were defended at the Medical University of Warsaw. Her achievements also include co-authoring 60+ scientific publications.
Panelists
Gergo Meresz, Ph.D.
MediConcept Ltd., Budapest, PE, Hungary
Gergő completed his Ph.D. in health technology assessment. The understanding of methods used for health technology assessment is accompanied with an emerging portfolio of leadership and consensus building skills.
After his departure from governmental positions, Gergő is serving as the lead of international affairs at MediConcept Ltd., a life sciences consulting firm from Budapest, Hungary.
Oresta Piniazhko, PhD
State Expert Centre of the Ministry of Health of Ukraine, Kyiv, Ukraine, Kyiv, Ukraine
Oresta Piniazhko, PhD, Director of HTA Department at State Expert Centre of Ministry of Health, Ukraine.
Oresta is an experienced expert in HTA, pharmaceutical policy and implementation practitioner. She holds a PhD degree in Pharmacoeconomics and is currently holding a position of Director of HTA Department at the State Expert Center of the Ministry of Health of Ukraine, ensuring management and implementation of the best international practices of HTA into health care system of Ukraine since February 2019. Being a dynamic communicator she is also a President of Ukraine ISPOR Chapter since 2017 and before ISPOR Ukraine Students Network (2015-2017). Oresta is visiting lecturer at The Institute of Business Education of Vadym Hetman Kyiv National Economics University and senior lecturer at Danylo Halytskyi Lviv National Medical University.
Eva Turk, PhD, MBA
Ministry of Health, Ljubljana, Slovenia
Eva has 15 years' experience in health systems reseach, currently working as senior researcher at the Centre for Digital Health and Social Innovation at the University of Applied Sciences, as well as she is responsible for HTA and value-based healthcare in Slovenian Ministers' Health Office. She holds an MBA in Healthcare Management (WU Wien) and a PhD in Health Sciences (University of Oulu).
Expanding the Definition of Value to Encompass Whole Health Through Standardized Patient-Centered Outcomes Measurement for Obesity
Digital Conference Pass
Level: Intermediate
ISSUE:
The global prevalence of obesity has nearly tripled over the past decades, reaching epidemic proportions, making its effective clinical management a global priority. Despite substantial research, the complex and relapsing nature of obesity, driven by various clinical, socioeconomic, genetic, and environmental factors, continues to hinder progressUtilizing a holistic view of health is essential for HEOR analyses of obesity, requiring expanded definitions of healthcare value.This issue panel will explore how ICHOM's standardized outcome measures for obesity aligns with ISPOR’s Strategic Plan 2030, and from a practical standpoint, how the measurement set can be applied to HEOR analyses and obesity policy. Our panelists, including a clinician, researchers, and patient, will discuss research implications, patient perspectives, and policy impacts of these measures, providing a comprehensive overview of their potential to improve obesity management. OVERVIEW:
The session will begin with an overview provided by Jennifer Bright, who will cover the unique aspects of ICHOM’s development of standardized outcome sets for obesity, their global track record of use, and the key debate topics. Despite the high global prevalence and huge healthcare burden of obesity, its effective clinical management remains a challenge. ICHOM’s standardized patient-centered outcome measure may offer a promising solution to address this challenge. The session will include: - Introduction (10 minutes): Ms. Bright on ICHOM’s obesity set development.
- Panelist Presentations (7 minutes each):
- Dr. Patton: Standardised outcome measurement implications for direct patient care, research, and advocacy
- Dr. Pizzi: Linkage between obesity and whole health which is a strategic priority for ISPOR and by extension HEOR professionals
- Dr. Blanchette: applied experience in HEOR analyses of obesity drugs (GLP-1 inhibitors) and utility of the ICHOM measurement set for this work
- Moderated Debate (20 minutes): Discussion on overcoming challenges and optimizing outcomes/care.
- Interactive Q&A (9 minutes)
Moderators
Spencer Connell, DAT
ICHOM, Indianapolis, IN, USA
Spencer comes from a decade of serving as an Athletic Trainer in the occupational health space before joining the International Consortium for Health Outcomes Measurement (ICHOM) as an Outcomes Research Project Manager. He was responsible for leading the international, multidisciplinary working group responsible for defining the Set of Patient-Centred Outcome Measures for Adults living with Obesity, and he is overseeing the initiation of the first Obesity Learning Collaborative where providers will be implementing the IHCHOM Adult Obesity Set in order to engage in shared learning, share best practice, and drive clinical quality improvement across sites.
Panelists
Christopher Blanchette, PhD, MBA
Novo Nordisk, Plainsboro, NJ, USA
Dr. Blanchette is a health economist leading clinical and HEOR research at Novo Nordisk (US).
Laura Pizzi, PharmD, MPH
ISPOR, Lawrence Township, NJ, USA
Dr. Laura Pizzi is Chief Science Officer for ISPOR and Research Professor at Rutgers University. For nearly 25 years, she has led interdisciplinary teams of methodologists, statisticians, and clinicians to design and conduct economic and outcomes analyses on healthcare interventions and is a frequent author, speaker, and mentor on the topic.
At ISPOR, she leads the organization’s scientific strategy and initiatives in collaboration with ISPOR's global members and stakeholders.
Morgan Salmon, .
AHP-Obesidad, Copenhagen, Denmark
Interplay Between Budget Impact Analysis (BIA) and Cost-Effectiveness Analysis (CEA): Theory versus Practice
Digital Conference Pass
Level: Intermediate
ISSUE: The pharmacoeconomic evidence provided by the BIA and CEA plays an important role in decision-making within healthcare systems. Despite these two instruments, systems do not always use both analyses or have different interpretations and criteria. To what extent this impacts the access to innovative medicines and how it can support their clinical assessment remains challenging. Efforts have been made to synergize the use of both BIA and CEA, for example, in a “three-dimensional pharmacoeconomic model” or “lock,” while some question the need for CEA. In the Netherlands, drugs or treatments with a high price or financial risk are placed in the lock, meaning the national authorities require both analyses for a decision. Are then BIA and CEA sufficient to address innovative medicines?
As the importance and performance of BIA and CEA increase, the need for consistency in combined use and interpretation becomes more apparent. Therefore, discussing the need to standardize the rules for a logical interplay between BIA and CEA might help industry, academia and health authorities to write and read the same language.
OVERVIEW: This panel will discuss the importance and the need to standardize the rules for a logical interplay between BIA and CEA. The moderator, Cornelis Boersma, will reflect on the situation (5min) and provoke debate based on several questions (15min): Is CEA always the appropriate instrument, and how far that depends on the BIA?; Could the development of a tool linking BIA and CEA support the decision-making of innovative drugs in the phase of their clinical assessment? How can industry, academia, and health authority contribute and benefit from such a tool? The panelists Josephine Mauskopf, Jaime Caro, Thea van Asselt, will address the perspectives mentioned in 10 min presentation per panelist. The audience will actively engage in the debate via online polls and open questions (10min).
Moderators
Cornelis Boersma, PhD
University of Groningen, Department of Health Sciences, UMCG; Open University, Heerlen, Department of Management Sciences and Health-Ecore Ltd, Zeist, The Netherlands, Zeist, UT, Netherlands
Prof. Dr. Cornelis Boersma (1978) has a master’s degree in pharmacy and a PhD in pharmaco-epidemiology, health economics and health policy (University of Groningen). He has ~20 years of experience in healthcare from various roles such as scientific researcher, teacher, consultant and the positions he held at the pharmaceutical industry. Cornelis is an independent consultant (Health-Ecore), entrepreneur (Digital Health Link, SensUR Health and PITTS). Cornelis is Professor ‘Sustainable Health and Innovation’ at the Open University and health economist at the University of Groningen/University Medical Center Groningen. His research focuses on health-economic and epidemiological studies as well as health policy/system research. He is supervisor of over 10 PhD-students and has over 50 national and international scientific publications (H-index: 17). In everything he does, he wants to contribute to most optimal, accessible and affordable health(care) for everyone. It is his ambition to invest – in collaboration with private and public parties – in sustainable health(care).
Panelists
J. Jaime Caro, MDCM, FACP, FRCPC
McGill University, London School of Economics, Evidera, Lincoln, MA, USA
J. Caro develops and applies novel techniques in modeling, health economics, comparative effectiveness, epidemiology, and outcomes research. To provide a better alternative to the well-known QALY, he is working on the BADI, a broader approach to valuing health benefits. He continues to develop DICE, the unified approach to health economic modeling that he created. Working with health technology assessment agencies and academic groups, he is formalizing this innovation to enable rapid, standardized and less error-prone development of decision-analytic models. Dr. Caro has adapted an engineering technique – discrete event simulation – to model diseases and their treatment and extended it to simulate the design of clinical trials and observational studies. He applied this technique to provide comparative effectiveness information in the absence of head-to-head trials in a new method called Simulated Treatment Comparisons and is applying simulation to make registries more feasible and efficient – an approach called SAVES. He proposed an innovative approach to the assessment of health technologies involving the efficiency frontier and is helping address the growing problem of judicialization of health care system decisions.
Josephine Mauskopf, PhD, MHA, MA
RTI International, Research Triangle Park, NC, USA
Josephine Mauskopf, PhD, MHA, is Distinguished Fellow, Emeritus, of Health Economics at RTI-HS. She has extensive experience both as a consultant and within the pharmaceutical industry. She has designed pharmacoeconomic research programs for drugs for bacterial infections, viral infections, psychiatric illness, and neurologic diseases. Dr. Mauskopf has estimated budget impacts for new products for schizophrenia, multiple sclerosis, bipolar disease, breast cancer, and HIV infection as well as for new vaccination programs. She has cochaired two ISPOR Task Forces one for Guidelines for Budget Impact Analysis and one for Economic Evaluation Methods for Vaccination Programs.
Thea van Asselt, PhD
University Medical Center Groningen/ University of Groningen/ National Healthcare Institut in Netherlands/ The Netherlands Organisation for Health Research and Development, Groningen, GR, Netherlands
Thea van Asselt (PhD) is a Health Economist at the departments of Epidemiology and Health Sciences of the University Medical Center Groningen (UMCG), The Netherlands. She is experienced in trial-based and model-based economic evaluations, covering a wide range of clinical areas. After obtaining a PhD degree from Maastricht University (2008) she continued to work in health economics research and moved to Groningen in 2014. She has many years of experience in performing cost-effectiveness analyses in national and international projects. These range from several Dutch studies into the cost-effectiveness of mental health interventions, innovative medicines to an ongoing H2020 project on real-time monitoring of COVID-patients in the ICU. Being a health economic lead in several NICE appraisals over the years she has also gained knowledge on the specifics of the appraisal and decision process in an international setting.
Dr. van Asselt is the co-coordinator of the course ‘economic evaluation in healthcare’ in the MSc BA Health of the faculty of Economics and Business at the University of Groningen. She is also a member of the 'Rational Pharmacotherapy' committees of the Netherlands Organisation for Health Research and Development (ZonMw) and of the Scientific Advisory Board of the Dutch Healthcare Institute that advises the Dutch Ministry of Health on reimbursement of pharmaceutical care.
Re-Using and Sharing Systematic Review Data between Competing Companies and HTA Stakeholders; Is It Feasible and How Can Challenges be Overcome?
Level: Intermediate
ISSUE:
This panel will debate Health Technology Assessment (HTA) r
esearch waste associated with systematic literature reviews (SLRs)
conducted by multiple companies for different assets launching in the same indication. The extent to which waste can be reduced through SLR data reuse between companies and HTA bodies will be discussed. The potential solution of living systematic reviews (LSRs) and shared libraries will be debated; opportunities and challenges will be considered to determine the feasibility of this approach.
OVERVIEW:
HTAs evaluate the value of new treatments relative to standard of care. Submissions require the development of costly and time-consuming SLRs. Particularly in disease areas with rapidly emerging new treatments, research waste can occur, with multiple companies conducting similar SLRs. Thilo Schaufler will provide an overview of research waste in HTA and the impact on patients. Thilo will
propose that LSRs could allow reuse and expansion of data by companies and HTA stakeholders. [8 minutes] Helene Vioix will offer a company perspective in favor of LSRs, arguing that static SLRs are outdated. Helene will describe challenges of rapidly emerging evidence, with companies producing SLRs to support multiple activities. [9 minutes] Sue Harnan will raise considerations from the HTA perspective, as an Evidence Assessment Group member. Sue will consider how LSRs could improve transparency and consistency, noting potential pitfalls, methodological considerations, and challenges. [9 minutes] Vicki Young
will describe practical barriers that must be overcome to share LSRs between stakeholders. Vicki will cover LSR commissioning, competitive interests, and stakeholder sacrifices required to gain from LSRs. Thilo will facilitate debate on overcoming shared LSR challenges. The impact on HTA methods and funding will be considered. Audience members will have an opportunity to comment on each challenge. Finally, each panelist will summarize the feasibility of LSRs in HTA from their perspective. [20 minutes]
Moderators
Thilo Schaufler, Ph.D.
CSL Vifor, Glattbrugg, ZH, Switzerland
Thilo holds a PhD in health economics and has 18 years’ experience in different Market Access roles of increasing responsibility in the pharmaceutical industry. He has extensively published in scientific journals and at international conferences.
Panelists
Sue Harnan, MSc
The University of Sheffield, SHEFFIELD, United Kingdom
Sue has been working in HTA as a member of an EAG (SCHARR-TAG) for the National Institute for Health and Care Excellence (NICE) for over 15 years. Her background is in Systematic Reviews and Evidence Synthesis.
Helene Vioix, PharmD, MSc
Evidence and Value Development, Merck Healthcare KGaA, Darmstadt, Germany
Helene is a global leader in the pharmaceutical industry. She has a background in pharmacy and health economics and 14 years' industry experience.
Vicki Young, PhD
Cytel Statistical Services Ltd, London, LON, United Kingdom
Vicki has an academic background (MSc, PhD) and 14 years’ experience in Health Economics and Outcomes Research consultancy.
Do the Challenges With Assessment of PROs in Oncology Trials Really Outweigh the Benefit?
Digital Conference Pass
Level: Intermediate
PURPOSE:
Whereas patient-reported outcomes (PROs) are seldom included in early phase oncology studies and are often included in later phase studies, the measurement, and data capture strategy of PROs in oncology clinical trials has frequently come under criticism. This workshop will examine the challenges associated with the assessment of PROs throughout oncology drug development, and taking account of recent guidance from regulators, will also provide workable solutions.
DESCRIPTION:
Workshop attendees will gain a working knowledge of the benefits of using PROs across oncology drug development programs. Drs. Byrom, Sen and Gnanasakthy will explore approaches to assessing tolerability and aiding optimal dose selection with newer targeted therapies in cancer drug development, particularly in early phase studies. Through case studies, they will illustrate the increasing importance of PRO data in better characterizing the dose-tolerability relationship during the early development phase of oncology drug development. Inviting audience contributions, they will discuss pragmatic approaches to collecting symptomatic PRO data in early phase studies, especially where there is little prior knowledge of the treatment's side effect profile. For later phase studies, exploring the implementation of the FDA draft guidance on core PRO measures in cancer trials, Drs. Byrom, Sen and Gnanasakthy will examine unanswered questions crucial to the success of this approach. Inviting audience commentary, they will debate whether implementing subscales of larger measures independently might alter the context of question administration and affect the measurement properties of the standalone subscales. Using published examples, the audience will be invited to provide their views on the impact of subscale context. Finally, they will examine the possible burden on patients of more frequent at-home PROM assessments—what data do we have to evaluate the impact of more frequent assessments, and what gaps remain in our understanding of what drives unrealistic burden?
Discussion Leaders
Bill Byrom, B.Sc., Ph.D.
Signant Health, Nottingham, NTT, United Kingdom
Bill Byrom, PhD, serves as a principal COA scientist at Signant Health. He has worked in the Pharmaceutical industry for 30+ years, first within clinical development and, latterly, focused on the development and application of clinical trial technologies. Bill is a recognized industry leader in eClinical product strategy, electronic clinical outcome assessments (eCOA), and decentralized clinical trials. He is an experienced scientific expert and the author of 80+ publications, and two industry textbooks on electronic patient-reported outcomes (ePRO). He serves on an ISPOR expert task force on Performance Outcomes, is a member of the Critical Path Institute’s (C-Path) working group on Chronic Heart Failure developing a COA for CHF research, and serves on the Executive Advisory Board of the EU-funded Learning Network for Advanced Behavioural Data Analysis (LABDA) research program researching novel methods for advanced sensor-derived movement behaviour data analysis. Bill is a member the Drug Information Association’s (DIA) working group on meaningful change, and a core contributor to the C-Path eCOA Consortium where he previously served as Industry Vice Director.
Dr. Byrom graduated in mathematics and statistics from Nottingham University, UK, and was awarded a PhD for his work on mathematical modelling of tropical disease at Strathclyde University, Glasgow, UK. Bill is a Senior Visiting Professor within Nottingham Trent University's Medical Engineering Design Research Group.
Discussants
Ari Gnanasakthy, MSc, MBA
RTI Health Solutions, Succasunna, NJ, USA
Ari Gnanasakthy is a Principal Scientist at RTI Health Solutions. Prior to his appointment seven years ago at RTI-HS Ari was an Executive Director at Novartis Pharmaceuticals, and headed the Patient Reported Outcomes Center of Excellence. He has been in the pharmaceutical industry for almost 35 years. Within Novartis he has been in various functions including Biostatistics, Health Economics, Pricing, and Outcomes Research. Ari has extensively published in topics related PROs.
Rohini Sen, PhD
AbbVie, Madison, NJ, USA
Rohini Sen, PhD, is a patient experience data enthusiast and healthcare professional with over a decade of experience in patient-centered outcomes research and strategic implementation, specializing in oncology and rare diseases. She currently serves as the Director of Patient Centered Outcomes Research (PCOR) Oncology HEOR at AbbVie, where she leads cross-functional teams in integrating patient perspectives into drug development processes. Recently, Dr. Sen was honored with the prestigious AbbVie CAN Impact Award for her dedication to putting patients first and successfully integrating Patient Experience Data (PED) early in oncology trials for the AbbVie oncology pipeline. She has a proven track record in developing innovative clinical outcome assessments (COAs) that enhance patient engagement and contribute to successful FDA and EMA regulatory communications. Dr. Sen has held significant positions at Takeda Pharmaceuticals, including Associate Director of Global Evidence and Outcomes for Rare Diseases and Neuroscience. She holds a Ph.D. in Psychometrics and an M.S. in Statistics from the University of Connecticut, along with a B.S. in Mathematics from Bryn Mawr College. Her research interests include sensor-based digital endpoints, psychometrics, observational research, real-world evidence, and instrument design. Dr. Sen is committed to advancing patient experience data generation in line with industry standards and trends.
Real-World Evidence Policy: Is Harmonization Between Regulatory and HTA a Help or Hindrance?
Digital Conference Pass
Level: Intermediate
PURPOSE: To debate whether new and emerging policies and guidance on the use of real-world evidence (RWE), should be focusing on harmonisation between regulatory and Health Technology Assessment (HTA). Through interactive discussions and real-time polling, we seek to identify the benefits and opportunities of harmonisation, but also the challenges and hindrances this could pose in regard to future policy development.
DESCRIPTION: In recent years, there has been an increasing number of policies on the use of RWE within regulatory and HTA healthcare decision-making. As more policies emerge with differing methods and recommendations, it raises the question of whether there is a greater need for harmonization between policy for regulators and HTA? Differing organizational remits of regulators and HTA agencies will likely place some limits on harmonization, but to what extent should there be a drive to harmonize other elements including between medicines and MedTech? Does harmonization support future RWE policy development or will it hinder efforts to develop policy by encouraging guidance that is too broad and non-specific?
In this workshop, Rita Peeters will introduce the topic by discussing the evolution of RWE policies across regulatory/HTA organisations (10 minutes). Katharine Cresswell will discuss the context of the Innovative Health Initiative - Integration of Heterogeneous Data and Evidence towards Regulatory and HTA Acceptance (IHI-IDERHA) project and what degree of harmonization was found in their recent global review of these policies (10 minutes). Patrice Verpillat will provide the regulatory perspective discussing perceived gaps and opportunities for future development and harmonization (10 minutes). Niklas Hedberg will discuss the HTA perspective and where harmonization may be challenging (10minutes).
A 20-minute interactive audience discussion, facilitated by real-time polling will allow discussion of critical arguments for and against harmonisation, and priority areas for future policy. Poll results will be displayed, allowing participants to see points of consensus and differentiation.
Discussion Leaders
Rita Peeters, PhD, MBA
J&J MedTech, Machelen, Belgium
Rita Peeters is since June 2016, Senior Director Regulatory Affairs Policy and Intelligence EMEA at Johnson & Johnson. In May 2011, she came back to JnJ as the Head of Regulatory Affairs for the Medical Devices & Diagnostics Marketing Operation companies in EMEA. Prior to joining J&J, she was Director of Clinical Research, Cardiac Rhythm Disease Management at Medtronic.
She started her career at the research department of Terumo Europe. During the following years, she was responsible for Clinical Affairs, Regulatory Affairs, Quality Affairs and Process Excellence at CR Bard, and later, for the Electrophysiology division of Johnson & Johnson - Biosense Webster. Before joining Medtronic, she was Director Health Economics and Medical Affairs for the Sorin Group CRDM division in Paris.
Rita graduated as a Pharmacist from the Catholic University of Leuven and obtained also a degree in Industrial Pharmacy. She holds a PhD in Pharmaceutical Sciences, a MBA from EHSAL European University College Brussels and a post-graduate in Health Economics from the University of York, UK.
Discussants
Katharine Cresswell, BSc (Int), MPH
National Institute for Health and Care Excelllence, Manchester, LAN, United Kingdom
Niklas Hedberg, MSc
Dental and Pharmaceuticals Benefits Agency (TLV), Stockholm, Sweden
Niklas Hedberg is the Chief Pharmacist
at the Swedish governmental authority,
the Dental and Pharmaceuticals Benefits
Agency (TLV).
Niklas is the co-chair for medicinal products in the Member States Coordinaton Group on HTA.
Niklas was the chair of the EUnetHTA JA3 and EUnetHTA21 Executive Board from 2018 to 2023.
Niklas has been involved in the national
pricing and reimbursement decision
making for pharmaceutical products
since 2001, and for medical devices
since 2009. He has a broad experience in
different aspects of value-based
evaluation.
Patrice Verpillat, MD, MPH, PhD
European Medicines Agency (EMA), Amsterdam, Netherlands
Dr. Patrice Verpillat (MD, MPH, PhD) is the Head of the Real World Evidence (RWE) Workstream at the European Medicines Agency (EMA). He is a medical doctor, specialist in epidemiology. Before joining the EMA, he has worked during 20 years in the pharmaceutical industry where he had positions in several international companies, always dealing with real world data (RWD) and non-interventional studies (NIS) in order to bring RWE into research, access and life-cycle product management.
Dr. Verpillat has published over 70 articles in Medline referenced journals. He has been involved in many organisations such as ENCePP, ICH M14 working group, European pharma association (efpia) and ISPE
Contextualizing Uncertainty in EU HTA by Coming to Terms With Imperfect Data: Can We Use Novel Analytic Methods to Quantify the Data Imperfections and Biases?
Digital Conference Pass
Level: Intermediate
PURPOSE:
The cross-European Health Technology Assessment Regulation is now a reality. EU methodological guidelines published the minimum acceptable methodological standards to guide sponsors on designing the quantitative evidence synthesis to support comparative effectiveness results. The acceptability bar for these analyses has been set so high as to almost exclusively require findings from randomized trials with high quality, internal validity and statistical precision. In circumstances where such evidence generation is unfeasible, such as in rare diseases or complex evidence pathways, this leaves limited room for non-randomized evidence and detailed exploration of uncertainty in joint clinical assessments (JCAs). Statistical uncertainty was over-emphasized over the importance of the “shifted null hypothesis”.
DESCRIPTION:
This workshop will involve a role play, interactive, fictional EU JCA submission and assessment. The discussion leader will consider predictive PICOs for a hypothetical technology in a targeted population with high unmet need and complex evidence generation needs (5 minutes). Dr. Hernandez will take the industry perspective exploring the challenges in producing strong evidence for such technologies. Dr Kent will discuss the role of non-randomised studies in the context of JCAs and present as an example results of the Q-BASEL study, an assessment of quantitative bias analysis methods for external control arms in lung cancer. Dr Pedder will discuss whether bias-adjustment and other information sharing synthesis methods can be used to strengthen inference where high quality randomized evidence is limited. (15 minutes each). Then, the audience will help inform the company’s submission evidence package and hypothetically exploring what would be the impact in JCA if bias adjustment methods were explored. An HTAG discussion will be mirrored with emphasis on issues around internal, external validity and precision of comparative effects. An interactive discussion including live polling questions will conclude the workshop (10 minutes). Statisticians, epidemiologists, payers, and Industry and patient representatives will benefit.
Discussion Leaders
Grammati Sarri, PhD, MSc, DiDS
Cytel, London, LON, United Kingdom
Grammati has 15+ years’ experience in epidemiology studies, evidence synthesis and decision making from her prior roles in academia, health technology assessment (NICE, UK), WHO and private health consultancies. Grammati has strong methodological experience in generating impactful evidence from different sources (clinical trials, real-world evidence, economic studies), clearly communicating findings in an impactful way and using complex analytical methods for health care-decision making. Grammati currently holds several positions in international organizations (ISPE), invited speaker to PhD training programmes in pharmacoepidemiology and current Advisory Board member to a EU funded trial on AI in breast cancer surgery (CINDARELLA). Grammati is working on EU JCA developments (produced blogs, delivered webinars, methods trainings) and topics around uncertainty, bias in both applications of non-randomized evidence and evidence synthesis. Publications and presentations related to RWE innovation: driving outputs on building knowledge around RWE methods guides, reliable RWE methods (framework for combining RWE with RCTs for decision-making [ISPE-endorsed], RWE quality assessment, statistical methods, data transferability, external controls for single-arm trials), and driving innovation in the use of RWE around topics such as patient experience, the lifecycle or living decision-making, health inequalities, data transferability across countries, specific challenges for RWE use in single-arm trials for rare and very rare diseases.
Discussants
Luis G. Hernandez, PhD, MPH, MSc
Takeda Pharmaceuticals America, Inc., Westford, MA, USA
Luis Hernandez, PhD MPH MSc. I am the Head of Global Health Economics and of US HEOR at Takeda Oncology. I lead the teams responsible for developing the Global and US HEOR strategy and evidence generation to demonstrate the value of our innovative medicines for patients, payers, strategic accounts, health systems and society to accelerate and sustain access across the world; for supporting value-based pricing; and for shaping the environment in HEOR and real-world evidence areas that may impact market access.
I have more than 15 years of experience in health economics and outcomes research, evidence generation planning, market access, and cross functional team leadership and management. I have led and co-authored multiple peer-reviewed publications and presentations at scientific conferences, and have been part of several expert panels in HEOR, real-world evidence and market access.
Prior to joining Takeda, I spent 13 years at Evidera (a business within Pharmaceutical Product Development, LLC, [PPD] a leading global contract research organization [CRO]) where I spearheaded health economics evidence generation and dissemination to support the optimal value proposition for pipeline, launch, and lifecycle assets. During my tenue, I held several leadership roles including Senior Director and Senior Scientist heading Evidera’s health economics modeling and simulation in Waltham, Massachusetts, USA.
Between 2009 and 2010, I was a full time professor of Probability, Statistics, Discrete Event Simulation, and Decision Analysis for undergraduate and graduate students at Universidad de los Andes (Bogota, Colombia) for the Industrial Engineering Department and the Executive MBA program.
I hold a PhD in Health Economics from the University of Groningen (the Netherlands), a Master’s in Public Health from Tufts University (Boston, Massachusetts, USA), and Master’s and Bachelor’s degrees in Industrial Engineering from Universidad de los Andes (Bogota, Colombia).
At ASCO 2024, I received the 40Under40 in Cancer Award, which honors top professionals who are making groundbreaking contributions in the field of oncology.
Seamus Kent, PhD
Erasmus University Rotterdam, Rotterdam, ZH, Netherlands
Seamus is Assistant Professor in HTA at Erasmus University Rotterdam. Prior to joining Erasmus Seamus was a Senior Adviser in Data & Analytics at NICE where he led the development of NICE's Real-world evidence framework and a Senior Adviser in HTA at Flatiron Health a RWD company in oncology.
Hugo Pedder, PhD, MSc, BSc
University of Bristol, Bristol, BST, United Kingdom
Hugo is a Research Fellow at the University of Bristol, primarily developing methods for evidence synthesis. He sits on a NICE HTA Committee and Co-Director of the NICE Technical Support Unit, providing technical expertise to support NICE Clinical Guideline development. Hugo is a Statistical Ambassador for the Royal Statistical Society and is passionate about communicating research and data literacy.
Challenges and Opportunities for Modeling the Impact of Cell and Gene Therapies in the Context of Evolving Regulatory Environments
Digital Conference Pass
Level: Intermediate
PURPOSE: Developing cost-effectiveness models (CEMs) and budget impact models (BIMs) for cell and gene therapies (C>s) is inherently challenging due to the lack of consistent control arms, small sample sizes in registrational trials, difficulty in defining durable clinical cures using surrogate endpoints, defining treatment-eligible populations, and high upfront costs. Ongoing changes to the regulatory environments in the EU introduce additional uncertainties. This workshop discusses some of the relevant methodological and organizational challenges in developing value dossiers for C>s from multiple perspectives and approaches to addressing these challenges in the context of evolving regulatory environments. DESCRIPTION: Dr. Steuten (12 minutes) will present a framework for conceptualizing CEMs that incorporates multiple requirements of various EU countries vis-à-vis PICO criteria, building scenarios in the core model to meet these expectations (12 minutes). Mr. Ektare will discuss the framework for constructing simplified BIMs tailored for both national and local policymakers, emphasizing the need for scenarios that account for alternative payment models, such as annuity-based payments, and the integration of Real-World Evidence (RWE) for monitoring treatment effectiveness (12 minutes). Dr. Fameli will discuss what readiness looks like for manufacturers, how manufacturers adapt to the changing landscape of regulations, and what opportunities and critical challenges are faced internally. Finally, Dr. Ghabri will discuss how the current HTA environments can streamline market access for these life-saving treatments by promoting evidence-based decision-making and collaboration between countries, such as opportunities for harmonized modeling practices and designing and sharing registry data and models (12 minutes). The workshop will conclude with a 12-minute Q&A session. This workshop is designed for health economists, policymakers, industry analysts, and other stakeholders in developing and assessing economic models for innovative therapies. The audience will be polled for the most pressing challenges and the types of methods and data they want to see to overcome them.
Discussion Leaders
Lotte Steuten, PhD
Office of Health Economics, London, LON, United Kingdom
Lotte Steuten, PhD, is Deputy Chief Executive of the not-for-profit Office of Health Economics (OHE) and the scientific and business lead for its international research-led work program—maintaining its reputation for objective, innovative, and high-quality research, and meeting its charitable goals. She is also an Honorary Visiting Professor at City, University of London (UK) contributing to its teaching programmes in health economics.
For more than 20 years, Lotte has worked in academic, research, and consulting HEOR roles with one focus — improving healthcare decision-making through high-quality research and analysis. To achieve that, she has collaborated with HTA authorities, pharmaceutical and medical device companies, patients, payers, policymakers, academic researchers, clinical specialists, and venture capitalists. She has gained broad HEOR expertise of various technologies: precision medicine, oncology, vaccines, antibiotics, diagnostics, digital health, and medical devices.
Lotte’s career has been based in the United States, the Netherlands, and the UK. Collaborations with HEOR experts in Southeast Asia, Africa, and Latin America have provided her with deep insights into the differences and commonalities between the role of HEOR in different healthcare systems, cultures, and societies. With this background, she has: led diverse international teams; developed strategies and delivered on program and organisational missions, values, and objectives; acquired research funding and managed budgets; and been responsible for executive decision-making as well as legal and fiduciary matters.
As an active ISPOR member since 2004, Lotte has served on various Taskforces and Committees, delivered Short Courses, was co-Chair of ISPOR EU 2021, and is currently a member of the Board of Directors, and an Associate Editor for Value in Health. She aims to be a role model for diversity and played an active role in the 'Women in HEOR' initiative.
Before joining OHE, Lotte worked in Seattle (US) as an Associate Professor at the University of Washington and at the Fred Hutch Cancer Research Center. She graduated cum laude with her PhD from Maastricht University (NL) and then worked at Brunel University (UK) and Twente University (NL).
Discussants
Indranil Bagchi, PhD
GSK, Philadelphia, PA, USA
Dr. Indranil Bagchi is the Global Head of Pricing & Market Access at GSK. In this role, Indranil drives the overall strategy on value demonstration and market access across all product areas in the GSK portfolio, to secure access to medicines for our patients and to maximize the value of our portfolio.
Indranil has more than two decades of experience in market access across several major companies in the pharmaceutical industry. In 2014, Indranil received the ‘Outstanding 50 Asian Americans in Business’ award and in 2010, Indranil was recognized in Pharmaceutical Executive magazine’s annual roster of Emerging Leaders, “The New Breed of Leadership.” Indranil is a frequent speaker and contributor to forums, articles and conferences addressing issues related to access to medicines.
Prior to GSK, Indranil was Senior Vice President and Worldwide Head of Value and Access at Novartis Oncology. Previously at Pfizer, he was Vice President and Global Head of Payer Insights and Access and prior to that, he was with GlaxoSmithKline in Health Economics and Outcomes Research. Dr. Bagchi has an undergraduate degree in Pharmacy, a master’s degree in Pharmacy and Healthcare Administration and a doctoral degree in Pharmaceutical Socioeconomics.
Varun Ektare, MPh
Indence Health, Thane West, MH, India
Salah Ghabri, PhD, HDR
French National Authority for Health (HAS), Paris, 75, France
Senior Health economist Researcher and scientific referent in economic evaluation - Department of Medical Evaluation - French National Authority for Health (HAS) since 2013. My research interests revolve around economic evaluation of health technologies and health programs, analysis of uncertainty and health economic modelling and budget impact analysis.
Use of Machine Learning Techniques to Create External Control Arms: Guidance for Submissions to Health Technologic Assessment
Digital Conference Pass
Level: Intermediate
PURPOSE:
External control arms (ECAs) are increasingly used to contextualize single-arm trial evidence. In the absence of randomization to balance the treatment groups to be compared, clinical profiles from trial patients are typically matched to external controls using propensity score matching before outcome analyses are performed. Alternative
analytical approaches based on machine learning (ML) predictions, such as G-computation and Doubly Debiased Machine Learning, are being considered for inferring efficacy outcomes. The use of such advanced techniques is guided by data sparsity and high-dimensionality. For this reason, most health technology assessment (HTA) bodies do not provide specific recommendations on the methods for matching clinical trials to observational data in their guidelines. The workshop aims to present the benefits and challenges of using matching methods based on machine learning techniques for creating ECAs in clinical trials. Our speakers will engage in real-time polling upfront to understand the audience (5 minutes). The workshop will conclude with an interactive audience discussion (10 minutes).
DESCRIPTION:
Dr. Guelfucci will introduce the topic to the audience and introduce the speakers (5 minutes).
Dr. Guelfucci will provide a comprehensive description of the methods used to compare with external controls in the most recent ECAs submitted to HTA agencies and their appraisals (10 minutes).
Dr. Mayer will delve into the context in which ML-based methods for ECAs are relevant and discuss their data requirements, inference benefits and challenges (15 minutes). Lastly,
Dr. Kreif will address recent developments and the necessary requirements to ensure their suitability for submissions to HTA bodies. She will also explore potential reporting checklists that can assist in meeting these requirements (15 minutes). The session will close with a Q&A session with the audience on the use and acceptability of ML-based matching methods for external control arms (10 minutes).
Discussion Leaders
Florent Guelfucci, MSc, PhD
Syneos Health, RENNES, 35, France
With 15+ years of experience in epidemiology in Consulting firms' HEOR and RWE practices in Europe, Dr. Guelfucci has acquired a strong and wide-ranging experience in conducting RWE & HEOR studies, leading analysts and guiding clients on the use of real-world data to reach their objectives. He has conducted numerous health economic (assessment of healthcare expenditures, economic burden) and epidemiological research (prevalence/incidence, treatment patterns, comparative effectiveness) utilizing secondary data (registries, claim data, EMR data). His expertise also includes feasibility assessments of secondary database studies and external control arms, evaluating the quality and fit-for-purpose of identified data sources. He has authored roughly 30 publications.
He holds a post-graduate degree in Econometrics (Rennes 1), a master's degree in Health economics (Paris 1-Panthéon Sorbonne) and a PhD degree in statistics and cognitive science.
Discussants
Noemi Kreif, MA, MS, PhD
University of Washington, Seattle, WA, USA
Dr Kreif’s research is centred around incorporating methodological innovations from statistics, causal inference and machine learning into health economics and health policy research. Her most recent methodological research project developed a causal machine learning framework to estimate treatment effect heterogeneity of health policies, and linked these estimates with decision models to provide optimal treatment recommendations. Dr Kreif was part of the ISPOR task force which authored the “PALISADE Checklist” and Good Practice Report on Machine Learning Methods in Health Economics and Outcomes Research. Prior to joining the CHOICE Institute at the University of Washington, she was a senior research fellow at the Centre for Health Economics, and a lecturer at the London School of Hygiene and Tropical Medicine, where she previously received her PhD (2013). Previously, she worked at an international pharmacoeconomics consulting company. Dr Kreif has published widely in leading health economics and statistics journals, including Health Economics, Journal of Health Economics, American Journal of Epidemiology and Biometrics.
Imke Mayer, PhD
Owkin, London, LON, United Kingdom
Imke holds PhD in Mathematics and Computer Science from EHESS (Paris) and Inria Saclay and a MSc degree in Applied Mathematics from ENS Paris-Saclay (MVA). During her PhD she developed methods for causal inference with missing values and integrative analyses of experimental and observational data. She applied these methods on clinical data from a French national registry and on data from multicentric international clinical trials (CRASH-2 & CRASH-3) on major trauma patients. During her post-doc at Charité University Medicine (Berlin), she worked on cluster-randomized trial designs and analysis in the context of public health research and health economic evaluations.
18:00 - 19:00
Woman in HEOR Reception
Join Us for the Women in HEOR Reception!
Continue the inspiring conversations from the Women in HEOR session at ISPOR Europe in Barcelona! Join us for networking and collaboration with fellow professionals who share your passion for advancing women’s roles in health economics and outcomes research. This is a unique opportunity to connect, share ideas, and foster relationships that can lead to impactful initiatives within our field. All attendees are welcome!
Networking Reception
End the second day of the conference at the Networking Reception where connections are made, ideas are sparked, and collaborations begin to flourish.
Wed 20 Nov
7:00 - 8:00
Coffee and Connect
It may be the last day, but there is still so much to learn! Head to the breakout rooms for the final session banks and an opportunity to share your thoughts with fellow attendees. Provided by ISPOR
7:00 - 13:00
Registration Hours
8:00 - 9:00
Breakouts: IP, WS, & OBS
Patient-Centered Research: Using Patient Experience Data Generated During Drug Development to Inform Healthcare Decision-Making: What Researchers Need to Know to Generate Fit-for-Purpose Data While Considering the Global Picture
Digital Conference Pass
Level: Intermediate
ISSUE:
The utility of patient experience data to support drug development remains unclear. The panel including a regulator, a patient, an HTA and an industry representative, will discuss how to build a common framework based on present and evolving notions of fit-for-purpose data.
OVERVIEW:
The Pharmaceutical Industry is increasingly including patients’ views in all stages of drug development. Some regulatory authorities and HTA bodies even go the extra-mile with for example, EMA including patients’ representatives in its committees and decision making processes. As shown with the IMI PREFER’s qualification, both Industry and Regulators are now taking this a step further by leveraging scientific methodologies that enable a more structured patient's input so that patient preferences and outcomes that are meaningful to patients can be systematically considered and captured during drug development. Last year, the ISPOR Task Force on Using Patient Preferences to Inform Decision Making issued a Good Practice report. This session while involving a regulator, an HTA, a patient and an industry representative, will thus provide a forum to debate, from a variety of different perspectives and experience, whose responsibility is to generate patient experience data, and to discuss potential mechanisms to foster constructive dialogue between stakeholders when generating and assessing such data. The moderator will provide an overview of the activities, opportunities and challenges associated with patients’ experience and preference data generation (10 minutes). The panel will then present their perspectives (10 minutes each), and using case examples will discuss what the future could look like, and whether guidance will be released while also considering the global picture. The moderator will then not only facilitate a debate on areas for harmonization and more operationalized recommendations, but will also oversee a debate between the panellists and incorporate questions/ comments from the audience (10 minutes).
Moderators
Solange Corriol-Rohou, MD, PhD
Pharmaceutical Company: AstraZeneca, GMD, Paris, 75, France
Solange Corriol-Rohou, a pulmonologist/immuno-allergist by training, joined AstraZeneca R&D in 2004 and is currently Sr. Regulatory Affairs & Global Policy Director, with responsibilities in the Respiratory/Infection and Vaccine/Immune franchises. Over the past 20 years, moving from the French Medicines Agency/EMA and academia to the pharmaceutical industry, she has gained strategic experience in drug development. She is quite active within EFPIA, ICH and IMI/IHI, and passionate about paediatric drug development, rare/ultra-rare diseases and drug development optimisation.
Panelists
Rosa Gonzalez-Quevedo, PharmD, PhD
European Medicines Agency (EMA), Amsterdam, Netherlands
Niklas Hedberg, MSc
TLV, Tandvårds- och läkemedelsförmånsverket / Dental and Pharmaceutical Benefits Agency, Stockholm, Sweden
Niklas Hedberg is the Chief Pharmacist
at the Swedish governmental authority,
the Dental and Pharmaceuticals Benefits
Agency (TLV).
Niklas is the co-chair for medicinal products in the Member States Coordinaton Group on HTA.
Niklas was the chair of the EUnetHTA JA3 and EUnetHTA21 Executive Board from 2018 to 2023.
Niklas has been involved in the national
pricing and reimbursement decision
making for pharmaceutical products
since 2001, and for medical devices
since 2009. He has a broad experience in
different aspects of value-based
evaluation.
Julian Isla
Microsoft, Foundation 29, Madrid, Spain
Julian Isla is the Chief Scientific Officer of the European Dravet Syndrome Federation, and European organization of patients with Dravet Syndrome. Julian Isla founded Dravet Syndrome Foundation seven years ago. Dravet Syndrome Foundation is committed to find new treatment for Dravet Syndrome, an epileptic encephalopathy having long lasting seizures refractory to treatment as severe developmental delay as main symptoms. Julian is the father of Sergio, a young boy six years old who has Dravet Syndrome. Julian is software engineer by training and he works for Microsoft as full time employee. Despite of not having a neuroscience or medical background he gained the skills to be part of the Orphan Drug Committee at European Medicines Agency (EMA) as patient representative. Julian is also part of the Therapeutic Advisory Group for Eurordis, the biggest organization of rare diseases in Europe. In Spain he is ambassador of the Spanish Rare Diseases Federation and member of Ciberer (Spanish Network for research on rare diseases) scientific advisory group”
Ending the Struggle: How Can We Put Outcome-Based Risk Sharing Agreements Out of Their Misery?
Digital Conference Pass
Level: Intermediate
ISSUE:
Outcome-Based Risk Sharing Agreements (OBRSA), such as outcomes-based or leasing agreements, hold promise to address both financial risk and clinical uncertainties in market access of innovative products. OBRSA so far have primarily been implemented on a case-by-case basis with varying success. Yet, systematic evaluation of these cases has been limited. W
ithout a standardized approach, it remains difficult to assess past experiences, and formulate best-practices. To move forward, decision makers and technology developers need information on the specific
settings and circumstances in which OBRSAs are appropriate and when they should be avoided.
OVERVIEW:
This Issue Panel will debate whether OBRSA have a role in supporting market access of innovative products and, if so, what types of products, and what should the agreements look like?
Dr. Elisabeth Fenwick will moderate this session in which David Thomson will start with a brief overview of OBRSAs – what they are and how they have been used. He will set the scene by presenting their strengths and weaknesses, and expectations around two upcoming UK OBRSA pilots.
Dr. Saskia Knies will reflect on past experiences from the perspective of an HTA-organization and HTx-consortium. She will defend that OBRSAs
appear promising in theory, but in practice have not yet delivered the desired results. Alternative arrangements such as simple price discounts can achieve similar outcomes with greater simplicity. Dr. Renske ten Ham will
argue that OBRSAs can support market access of innovative drugs. However, better understanding is needed of prerequisites for successful implementation, which include disease and product characteristics. She will present an approach to assess impact of OBRSAs on population health
using a gene therapy-case study. Before moving to an interactive discussion, Dr. Fenwick will outline recent work exploring circumstances where OBRSAs might be an option to address uncertainty by both the technology developer, ánd the payer.
Moderators
Elisabeth Fenwick, PhD
OPEN Health Communications, London, LON, United Kingdom
Elisabeth Fenwick is Chief Scientific Officer for HEOR & Market Access at Open Health, based in Oxford in the UK.
Liz provides scientific and strategic support to HE projects globally. She has extensive experience in economic evaluation and health economic modeling having worked in the field for over 20 years. She has worked on a variety of projects in a wide range of disease areas including oncology, respiratory, infectious diseases, cardiology, ophthalmology, and orphan diseases.
Liz has also contributed to methods in the field, in particular relating to decision analytic modeling and simulation methods, probabilistic decision analytic modeling and value of information analysis. Liz was a member of the ISPOR joint task force on good research practices in modeling and a co-author on the joint taskforce paper on uncertainty and co-chaired/co-authored the recent ISPOR task force assessing emerging good practice in value of information analysis for research decisions.
Liz has a PhD and MSc in Health Economics as well as an MSc in Operations Research and joined Open Health from ICON plc where she led the modeling team for the global HE group. Prior to her consultancy career, Liz spent over 15 years as an academic working at University of York, McMaster University, and most recently University of Glasgow.
Panelists
Saskia Knies, PhD
Zorginstituut Nederland, Diemen, NH, Netherlands
Saskia Knies is a coordinating advisor at the National Health Care Institute (Zorginstituut Nederland, ZIN) in the Netherlands. She is responsible for the broadening and improving the HTA tasks at ZIN and leader of WP 2 on Implementation of SUSTAIN-HTA. In addition, she was involved in the recent update of the health economics guidance that was published in January 2024. Saskia began her career in health economics/HTA in 2007 during her PhD research at Maastricht University. Since 2012 she is working as an advisor at the National Health Care Institute.
Renske Ten Ham, PhD, PharmD, MSc
University Medical Center Utrecht, Utrecht, Netherlands
Renske ten Ham is an assistant professor at the UMC Utrecht in the Netherlands and specializes in health economics & health technology assessment (HTA) of regenerative medicines, including gene and cell-based therapies. Trained as a pharmacist and with an MSc in HTA, she holds a PhD in Drug Innovation titled: “Development, market authorization and market access of gene- and cell-based therapies”.
At the Julius Center Renske is team lead of the HTA regenerative medicines (RM) team in which she strives to increase translation of RM and (academic) gene and cell-based therapies towards implementation in healthcare services. In doing so her research focusses on payment models, development cost, (early) economic evaluations and business models.
David Thomson, BSc(Hons), MSc
National Institute for Health and Care Excellence, Manchester, United Kingdom
Changing With the Times: Keeping Up With Advancements in Comparative Efficacy Techniques While Maintaining the Path of HTA Informativeness
Digital Conference Pass
Level: Experienced
ISSUE:
Advancements in therapies with novel mechanisms of action drive our need to consider emerging, time-varying network meta-analysis (NMA) approaches during HTA submissions. These methods, relaxing the proportional hazards (PH) assumption, can appropriately and maximally capture benefits over legacy treatments or placebo. However, detailed guidance including precedent use of time-varying NMA methods supporting HTAs, and their subsequent impact on cost-effectiveness analysis (CEA), is limited.
OVERVIEW:
Our panel addresses a recent NICE technology appraisal (TA858) in advanced renal cell carcinoma. Due to PH violations in the network, time-varying NMA were performed. However, their results were not applied for lack of interpretability and an inability to judge clinical plausibility for the trial and long-term survival extrapolation. In response, we present an enriched scenario of the MTA, demonstrating the advantages, limitations and differential impact on a CEA applying different NMA methods: PH, fractional polynomial, restricted cubic spline and parametric. Increasing needs to account for time-varying relative efficacy are reflected in both NICE HTA requirements and the European Union (EU) Joint Clinical Assessment (JCA) guidelines. While crucial to accurately capture changes in comparative effectiveness, the complexity of associated methods compounds familiar challenges such as study and population heterogeneity in the network. This compounded project scope has implications both for the JCA and CEA-based HTA systems such as NICE. Within the EU HTA setting, a crucial unknown is the individual member states’ receptivity of novel comparative efficacy methods. As the current role of HTA is very different per country, there may be differences between member states’ levels of preparedness to assess their application. Thus, exploration and reporting on how these methods can reduce uncertainties, what uncertainties remain, and how they impact CEA is key, so that some level of uniformity across member states may be achieved.
Moderators
Julie Roiz, MSc
Lumanity, London, DBY, United Kingdom
Julie Roiz holds an MSc from the French National School of Statistics (ENSAI, Rennes, France) with a major in health economics. Her two main areas of expertise are decision modeling and methods associated with meta-analysis and indirect comparison, which she taught at ENSAI for four years. Julie has experience in multiple types of models (including cohort, patient-level simulations, and dynamic transmission models), meeting various objectives, such as health impact, cost-consequence, cost-effectiveness, and budgetary impact. She has worked in multiple indications, with a particular interest in infectious diseases and economic evaluation of vaccination programs, including influenza, varicella-zoster virus, pertussis, cytomegalovirus, hepatitis A and B, and sexually-transmitted diseases.
Panelists
Maiwenn Al, PhD
Erasmus School of Health Policy & Management, Rotterdam, Netherlands
Dr. Maiwenn Al is associate professor of Modelling in HTA at Erasmus School of Health Policy & Management, Rotterdam, the Netherlands.
Her research focus is on modelling studies and methods for dealing with uncertainty in economic evaluations. Together with Ben van Hout, Maiwenn developed the cost-effectiveness acceptability curve, now widely used to describe uncertainty around ICERs. She was the 2006 recipient of the ISPOR Health Economics Outcomes and Research - Methodology Award, for the paper she wrote with Jan Oostenbrink on the analysis of incomplete cost data due to dropout.
From 2012=2016, Maiwenn was co-editor of Value in Health. Until recently, Maiwenn was a member of the Scientific Advisory Board of the National Health Care Institute (ZIN) in the Netherlands. She was also on the committee for the revision of the Dutch guidelines for economic evaluations in healthcare.
Maiwenn has been health economic lead to many technology appraisals for NICE in the UK. Besides this work for NICE and various teaching activities, her current work targets the use of real-world evidence for the health economic assessment of medical devices (H2020 project COMED).
Miranda Cooper, MSc
AstraZeneca, Sheffield, DBY, United Kingdom
Statistician with a demonstrated history of working in and alongside the pharmaceutical industry with an interest in indirect treatment comparison methods and survival analysis. Strong mathematics background with a Bachelor of Science (BSc) focused in Mathematics from Durham University and a Master of Science (MSc) in Statistics from the University of Sheffield.
Tracy Westley, MScPH
Lumanity, Dundas, NB, Canada
Leading expert and passionate contributor of timely and impactful HEOR statistical workstreams; supporting the larger messaging
Integrating the Patient Voice Into Economic Assessments of Health Technologies
Digital Conference Pass
Level: Intermediate
ISSUE:
The ISPOR Patient-Centered SIG aims to facilitate engagement and partnership of patients in all stages of health-related research and care delivery. Economic evaluation of health technologies is an important aspect in determining patient access to new technologies. Cost-effectiveness analyses (CEAs) are used in several countries to support coverage decision-making. To enhance inclusion of patient perspectives, preferences, and values, it is imperative to engage patients when designing CEAs and healthcare decision-making. However, there is a lack of guidance available on the appropriate approach to engaging patients in health economic research, which may be attributed to the inherent complexity of cost-effectiveness analyses. To address these concerns, the panel discussion organized by the SIG invites panelists to offer their insights on patient engagement in the economic evaluation of health technologies from HTA, economic modeling, and patient perspectives.
OVERVIEW:
Nan Qiao will give an overview of the background information, highlighting that despite the EU JCA implementation, many member states still conduct economic assessments to inform coverage and pricing decisions. Patient engagement in the economic assessment improves the patient-relevance of value assessment. However, determining the most appropriate approaches for patient engagement is essential given the complexity of economic assessment.
Anke Peggy Holtorf will outline the broader values and possible methods of involving patients in HTA or the economic assessment as an HTA expert.
Andrew Briggs will provide his insights from the perspective of a modeling expert, discussing the best approaches to engage patients in the design, implementation, and dissemination of cost-effectiveness analyses.
Bettina Ryll will contribute insights from the patient perspective, emphasizing the value of patient engagement in the economic assessment and addressing the opportunities and challenges associated with patient engagement. The panel will be open for audience questions and suggestions to explore strategies for enhancing the relevance of health economic analysis from the patient perspective.
Moderators
Nan Qiao, PhD, MPH, MSc, MBBS
Merck, Lansdale, PA, USA
Nan Qiao currently serves as the Chair of the ISPOR Patient-Centered Special Interest Group and having been a member of the program committee of the ISPOR Patient-Centered Research Summit. In her role as Medical Affairs Director at Merck Sharp & Dohme (MSD), she develops oncology HEOR content to facilitate scientific exchange and engage key decision-makers. She led economic evaluations of pembrolizumab as a treatment for early-stage and metastatic lung cancer and provided support for various HTA submissions during her prior position as an economic and data scientist at MSD.
Panelists
Andrew Briggs, DPhil, MSc, MSc, BA
London School of Hygiene and Tropical Medicine, London, London, United Kingdom
Andrew Briggs is Professor of Health Economics at the London School of Hygiene & Tropical Medicine, and co-Director of the Global Health Economics at the School. He has authored over 300 peer reviewed publications and is a Clarivate 'highly-cited' researcher. He has co-authored two textbooks, one published by OUP entitled Decision Modelling for Health Economic Evaluation, and the other published by Wiley entitled Statistical Methods for Cost-Effectiveness Analysis.
A link to his GooGle Scholar page: https://bit.ly/GS-briggs
Anke-Peggy Holtorf, PhD, MBA
PCIG @ HTAi.org, Basel, Switzerland
Anke-Peggy is the founder of the Health Outcomes Strategies GmbH consulting agency (www.health-os.com) with focus on value of healthcare products and related policy decisions for the in- and off-patent sector, reimbursement strategies, outcomes research and health economics, health technology assessment processes, payer interactions, product / device / service synergies.
Anke-Peggy acted as evaluator at the EU-commission on multiple occasions and is faculty member at the University of Utah College of Pharmacy where she reads in International Health Policy. She is engaged in workgroups for Precision Medicines Value (ISPOR), for Digital Health (ISPOR) and for Patient and Citizen Involvement (PCI) in HTA (as project coordinator for PCIG at HTAi) and leads projects on PCI in Low-and Middle-Income Countries and on PCI processes in HTA.
Bettina Ryll, MD, PhD
Melanoma Patient Network Europe, and Past Chair of the Patient Advocates Working Group, European Society for Medical Oncology, Uppsala, Sweden
Dr. Ryll holds a medical degree from the Free University of Berlin, Germany, and a PhD in Biomedical Sciences from University College London, UK. After losing her husband to Melanoma, she founded the Melanoma Patient Network Europe and developed a special interest in patient-centric clinical research and innovation, innovative trial designs and novel drug development concepts. Between 2019 and 2021, she was member of the first EU Cancer Mission Board where her particular interest has been how to best leverage the potential of personalized medicine for patients and society through novel forms of collaboration and the support of health policy and governance. Currently, she works as strategist at SIR, Stockholm School of Economics' Research Institute for Mission Zero Cancer, an innovation ecosystem in health financed by Vinnova, Sweden's innovation agency.
EU HTA Regulation: Ready for 2025 EU Joint Clinical Assessment (JCA)
Digital Conference Pass
Level: Intermediate
ISSUE:
The EU HTA Regulation (EU 2021/2282) represents a significant shift in how new health technologies are evaluated and accessed across Europe. Implemented in January 2022, the regulation comes into full effect in January 2025. While regulatory decisions are centralized in the EU, most countries have their own Health Technology Assessment (HTA) process. HTA methods and processes vary by country leading to differences in ratings, access to medicines and delay in time for patients in the EU. The HTA Regulation aims to improve and accelerate patients’ access to new health technologies in the EU by harmonizing clinical assessment across Member States. As the EU HTA Joint Clinical Assessment (JCA) will parallel the review and approval process by Regulators, time for preparation of both dossiers, review and decision making and alignment with following national reimbursement are anticipated to provide new challenges for the involved stakeholders. It can also be anticipated that interference of the content discussion cannot be avoided as data analysis will continue during the review phase. This panel discussion will dive into how the implementation of this process may impact drug development for Europe in general in future, (e.g impact on Scientific Advice procedures, on development program design and on outcome of the reviews) and underline the key elements which need to be put in place to ensure a speedy, future-proof preparedness from both sponsors, but also health authorities.
OVERVIEW: Timeframe 45 min: moderator and panelists introducing their perspective in a motivational speech (5 minutes each), 15 minutes discussion on selected topics and 10 minutes on questions from auditorium
Moderators
Stephanie Rosenfeld, Dr. med.
Bayer AG, Berlin, Germany
Dr. Stephanie Rosenfeld is a trained physician and has earned a master’s degree in health business administration. She works in Market Access as global Head of Cell & gene therapy and early portfolio strategy at Bayer AG. Stephanie has a longstanding history in HEOR, Evidence Based Medicine and Market Access with previous roles as Head of Market Access and Head of Evidence Based Medicine, Sanofi Germany. Stephanie is passionate about translating policy into HTA and access and has been a longstanding and active member of vfa, BPI and Efpia, representing her respective companies.
Panelists
Michael Berntgen, PharmD, PhD
European Medicines Agency (EMA), Amsterdam, Netherlands
Michael Berntgen is Head of the Scientific Evidence Generation Department at the European Medicines Agency (EMA), Amsterdam. This department supports the development of medicines to ensure generation of robust and relevant scientific evidence, also in collaboration with other stakeholders (e.g. patients, HTAs). Activities include the provision of scientific advice and methodology qualification, management of the PRIME scheme, support to medicines for the paediatric population and for orphan diseases, as well as provision of expertise and support in translational sciences. Furthermore, Michael coordinates across the Agency the collaboration with HTA bodies and payers, to foster timely access to medicines.
Inka Heikkinen, MSc, MBA
Lundbeck Pharma, Copenhagen, Copenhagen, Denmark
Lead of the EFPIA regulatory-HTA interaction and Joint Scientific Consultation workstream. Leads R&D and regulatory policy strategy and operational excellence at Lundbeck. Master degrees on health economics and in policy, an Executive MBA degree with specialisation in finance.
Anne Willemsen, MSc
Zorginstituut Nederland (ZIN), Diemen, Netherlands
Anne Willemsen holds double Masters Degrees in Health Sciences, specialising in both Health Technology Assessment and Health Policy. This led her through research and project management roles within the Dutch National Healthcare Institute (Zorginstituut Nederland) over the past years. Between 2016 and 2023 she has been working for EUnetHTA and has been coordinating Joint Clinical Assessments (JCA). Since April 2023, she was elected as the co-chair of the HTA Coordination Group JCA subgroup under the HTA Regulation.
Giving Voice to Rare Diseases During Joint Clinical Assessment (JCA)
Digital Conference Pass
Level: Intermediate
ISSUE: JCA will roll out in 2025, starting with all new oncology products. This will expand to include products with orphan designation in 2028. Assessment of rare diseases will face additional challenges compared with non-orphan products in terms of clinical trial design, low patient numbers, heterogenous populations, and differing standards of care across the 27 European Union member states. It is important that companies start preparing now to ensure studies are designed appropriately and to understand the PICOs (population, intervention, comparator, and outcomes) that will apply. It may be appropriate to use pragmatic approaches to reduce the research questions to be appraised and innovative methodology to consider the available evidence, both from clinical trials and real-world data. Understanding the potential options to efficiently consider all relevant populations and to ensure that a voice is given to patients across these populations within rare diseases will be vital. All stakeholders will need to collaborate to balance challenges due to the constraints of the time and data available with the needs of patients.
OVERVIEW: This panel discussion will convene experts in rare diseases, patient advocacy, and statistics to share their insights and experiences regarding JCA requirements. Caroline Ling will moderate the session, introducing the topic and the panellists. Abigail Stevenson will explore the considerations and potential challenges associated with evidence generation across all 27 European Union member states for rare diseases. Emma Hawe will consider efficient approaches to evidence generation from the perspective of literature reviews and indirect comparisons where multiple PICOs exist. The patient advocate will articulate the significance of giving a voice to specific populations in rare diseases, emphasizing the urgency of access to new treatments and importance of patient-relevant outcomes. The panel presentations will span 45 minutes, followed by a 15-minute question-and-answer session, allowing the audience to engage with the panellists.
Moderators
Caroline Ling, PhD
RTI Health Solutions, Manchester, United Kingdom
Caroline Ling PhD is Executive Director in the Value & Access team at RTI-HS. She has over 20 years of experience in market access / HEOR consultancy with a particular focus on HTA. She has successfully led and managed projects for a variety of different clients in a range of disease areas.
Panelists
Emma Hawe, M.Sc.
RTI Health Solutions, Manchester, United Kingdom
Emma Hawe, MSc, is Head of Evidence Synthesis & Statistics at RTI-HS. She has over 20 years of experience as a statistician within consulting, regulatory, and academic environments. Ms. Hawe is experienced in the application of novel and standard statistical methodologies to large medical/biological data sets in diverse therapeutic areas. Her role at RTI-HS includes planning, executing, performing, and interpreting the analysis of a variety of studies, including systematic literature reviews, meta-analysis, epidemiology, health economics, and clinical trials. Prior to joining RTI-HS, Ms. Hawe was Head of Statistics at the Office of Health Economics, where she managed a variety of different projects, including burden-of-illness studies, multicriteria decision analysis, and policy-orientated projects for pharmaceutical companies, pharmaceutical trade associations, and overseas governments. Ms. Hawe has conducted many types of statistical analyses, including network meta analysis, survival analysis, factor analysis, analysis of utilities including mapping, multivariate modeling, and database analysis including the analysis of Hospital Episode Statistics (HES). She has successfully led and managed projects for a variety of different clients and contributed to many more. In addition, Ms. Hawe has experience with a variety of different statistical packages and programming languages, including R, STATA, SAS, and SQL. Ms. Hawe is author of the Office of Health Economics guide to UK health and health care statistics, a comprehensive guide to health statistics in the UK, and author of more than 75 publications in peer-reviewed journals. Previous positions have involved the statistical analysis of the combined effects of genetics and the environment on cardiovascular disease, and the study of births and infant mortality data in England and Wales over a 20-year period.
Abigail Stevenson, DPhil
Chiesi, Manchester, LAN, United Kingdom
Abi Stevenson is a Senior Manager in the Global HEOR team at Chiesi, leading economic evidence generation for rare disease therapies for Europe/International markets. The team’s work proves the value of Chiesi’s products to healthcare decision-makers to help secure patient access to life-changing treatments.
Abi is a PhD-qualified Market Access professional with >10 years of HTA experience, leading >20 successful HTA submissions in oncology and rare disease across the UK, Ireland and the Nordics. Prior to joining Chiesi, she was Associate Director of Market Access at RTI Health Solutions and a Technical Analyst at NICE, evaluating medical devices and diagnostics.
Martin Visnansky, PharmD, MBA, PhD, MSc (HTA)
Department of Public Economics, Faculty of Economics and Administration, Masaryk University in Brno, Brno, Czech Republic
Equity in HTA: Exploring Methods and Their Role in Decision-Making
Digital Conference Pass
Level: Introductory
PURPOSE: Equity continues to be a significant area of HEOR research. This workshop will describe what methods are available to address equity in health technology assessment (HTA) and discuss how and to what extent HTA agencies should consider equity. It will also explore the opportunities and challenges of incorporating health equity in HTA.
DESCRIPTION: Although there is an ever-growing body of research on equity, including methods such as distributional cost-effectiveness analysis (DCEA), the incorporation of equity in HTA is inconsistent and is not well defined. How HTA agencies consider equity is a shifting landscape, and is one that will likely evolve in the coming years.
In this workshop, Ms. Cresswell will introduce the topic of equity in HTA, including how NICE was created to end the ‘post-code lottery’ and its work to date around equity (10 minutes). Dr. Jansen (methodologist perspective) will introduce the concept of DCEA as a quantitative method to quantify the health equity impact of a technology using an illustrative model-based example, and will highlight potential implementation challenges (10 minutes). Dr. Whittington (HTA perspective) will discuss the practical and ethical challenges of conducting a DCEA within a health technology assessment, and will describe other non-DCEA opportunities to incorporate equity within HTA (10 minutes). Dr. Gsteiger (industry perspective) will explore the application of equity considerations and DCEA in clinical development programs, and will discuss current challenges (including collecting the data needed for DCEA), opportunities, and prerequisites for a more widespread use in practice (10 minutes). The workshop will allocate 20 minutes for an interactive audience discussion, including question and answer sessions. Audience polling will be employed throughout to encourage active participation and engagement. The workshop will culminate by asking the audience how they think equity should be considered in HTA.
Discussion Leaders
Katharine Cresswell, BSc (Int), MPH
National Institute for Health and Care Excelllence, Manchester, LAN, United Kingdom
Discussants
Sandro Gsteiger, PhD
F. Hoffmann-La Roche Ltd, Basel, BS, Switzerland
Sandro Gsteiger holds the position of Access Evidence Lead in the Global Access Department at F. Hoffmann-La Roche Ltd. In this role, he contributes to access evidence generation, strategy development, and the development of novel HTA methods. He is a member of the EFPIA HTA Methods WG, and of the PSI HTA SIG. He holds an MSc in mathematics (University of Fribourg, Switzerland), a PhD in statistics (Swiss Federal Institute of Technology in Lausanne), and an MSc in HTA from the University of Glasgow, UK.
Jeroen Jansen, PhD
University of California – San Francisco, San Francisco, CA, USA
Jeroen P Jansen PhD is a methodologist working at the intersection of evidence synthesis, biostatistics, and health economics.
He is an Associate Professor in the Department of Clinical Pharmacy in the School of Pharmacy at the University of California, San Francisco and Chief Scientist − Health Economics & Outcomes Research at the Precision Medicine Group.
For the past 15 years, Dr. Jansen has worked on research to understand the clinical and economic value of healthcare interventions. His research has frequently been conducted in the context of health technology assessment (HTA) with a focus on comparative effectiveness and cost-effectiveness. Prompted by the challenges encountered in applied research projects, he has performed methodological research. Notable contributions are the development of novel statistical methods to overcome the typical challenges in model-based cost-effectiveness evaluations characterized by gaps in the evidence base and complex evidence structures. Furthermore, Dr. Jansen led initiatives to develop guidance for consumers and producers of network meta-analysis studies. He has promoted a more transparent and credible approach to model-based health economic evaluations and led the development of open-source simulation models to illustrate its feasibility. Furthermore, Dr. Jansen has been involved in the ongoing development of an R software package to develop simulation models for health economic evaluations.
His current research interests are the clinical and economic value of precision medicine, incorporating health disparities in health economic modeling studies, and statistical methods for evidence synthesis.
He has published extensively in his areas of expertise and is widely cited. He is co-author of a textbook on network meta-analysis for decision-making and was associate editor for the Journal for Research Synthesis Methods. Dr. Jansen has a PhD in epidemiology from the Erasmus University in the Netherlands.
Melanie Whittington, PhD, MS
Tufts Medical Center (CEVR), Boston, MA, USA
Dr. Melanie Whittington is the Managing Director and Head of the Leerink Center for Pharmacoeconomics where she leads pharmacoeconomic evaluations of in-development and recently approved pharmaceuticals. She is also a Senior Fellow at the Center for the Evaluation of Value and Risk in Health (CEVR) at Tufts Medical Center where she works to develop novel methods for value assessment. She was previously the Director of Health Economics for the Institute for Clinical Economic Review (ICER) where she led the cost-effectiveness analyses and budget impact analyses within ICER reviews. She is a leader in the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) and was the recipient of the ISPOR Bernie J. O’Brien New Investigator Award. She has a PhD in Health Services Research with a concentration in Health Economics and Outcomes Research.
Going Beyond Health-Related Quality of Life: Towards a Broader QALY Measure for Use Across Sectors: Introducing the EQ Health and Wellbeing Short (EQ-HWB-S)
Digital Conference Pass
Level: Introductory
PURPOSE: ‘Value measurement: Assessing Value and QALY Alternatives’ remains a key trend in health economics and outcomes research. The goal of this workshop is to raise awareness of the EQ-HWB (EQ Health and Wellbeing instrument). The workshop will also explore the opportunities and strengths perceived by the speakers and audience on using the EQ-HWB-S, both from the perspective of health technology assessment (HTA) agencies and industry.
DESCRIPTION: The EQ-HWB has been designed as a standardised measure of broader aspects of quality of life related to health, social care and informal carers enabling assessment of interventions that are delivered in health, including public health, and social care. A short version, the EQ-HWB-S, can be used to generate QALYs for HTA. The instrument has experimental status which has enabled collaborators and researchers to assess its performance in diverse populations. ZIN, the HTA agency for the Netherlands, has named it as a potential measure for use when evaluating interventions in chronic conditions. Understanding the views of those who will use the tool and appraise it are important to consider.
In this workshop, Mr. Dietz will discuss the NICE reference case in relation to where deviations from the EQ-5D are allowed (10 minutes). Mr Finch will introduce the EQ-HWB and how it was developed and emerging evidence on its performance (10 minutes). Dr Efe will talk about ZIN’s decision to include the EQ-HWB in their methods document, ‘Guideline for economic evaluations in healthcare (2024 version)’ (10 minutes). Mr Lloyd will discuss their plans to use the EQ-HWB and provide an industry perspective (10 minutes). The workshop will allocate 20 minutes for an interactive audience discussion, including question and answer sessions. Audience views of the instrument and its potential use will be sought using polling to encourage active participation and engagement.
Discussion Leaders
Aureliano Paolo Finch, PhD
The University of Economic Studies, Amsterdam, NH, Netherlands
Aureliano Finch is a Senior scientist at the EuroQol Research Foundation, a member of the Descriptive System working group and of the EQ-HWB working group. His research mainly relates to the development and testing of preference based instruments of health and wellbeing. He applies both quantitative and qualitative methods for this purpose.
Discussants
Jeremy Dietz, MSc
National Institute for Health and Care Excelllence, Bristol, BST, United Kingdom
Scientific Adviser, Science Policy & Research Programme at NICE (National Institute for Health and Care Excellence)
Reva Efe, MD, MSc
Zorginstituut Nederland, Diemen, NH, Netherlands
Andrew Lloyd, DPhil
Acaster Lloyd, London, London, United Kingdom
Andrew Lloyd is co-founder and director of Acaster Lloyd Consulting Ltd. His background is in patient-reported outcomes research, with a focus on patient preferences / utilities and the application of PRO techniques within economics and outcomes research. Andrew has held senior positions at ICON PLC, Oxford Outcomes Ltd. and United BioSource Corporation. He has also held research posts at several universities within the UK and has supervised several students at masters, and doctoral level. Andrew is an honorary research fellow in the Department of Health Services Research & Policy at the London School of Hygiene and Tropical Medicine.
His research work is focused on developing and testing assessments of health-related quality-of-life with a particular focus on utility measurement. Andrew has undertaken work in a broad range of therapeutic areas including oncology, autoimmune disease, and many rare diseases. Much of the consulting work that he undertakes is used to support NICE submissions. He is an active member of ISPOR, where he teaches on their short course programs. He is a former co-editor of Value in Health and sits on the International Advisory Board for Quality of Life Research. He is a board member of the EuroQol Research Foundation.
8:30 - 10:00
Breakfast Bites and Networking Delights
Kick off the final day of the conference in the Poster and Exhibit Hall with a light breakfast – use this time to connect with exhibitors and ignite conversations. Provided by ISPOR
8:30 - 11:30
Exhibit Hall Open
8:45 - 9:45
Podium Sessions
Expanding the Definition of Value Through Research
Digital Conference Pass
Studies that involve broadening assessments of value by incorporating equity, environment, and disease-specific considerations.
Moderator
Ramiro Gilardino, MD, MHS, MSc
MSD, Dubendorf, ZH, Switzerland
Ramiro Gilardino is an impact-driven healthcare executive with over 15 years of leadership in Global Market Access, Health Policy, and HEOR. He has successfully led strategies that improved patient access, drove reimbursement success, and shaped health policy in pharmaceutical, biotech, and global organizations. An expert in advancing HTA frameworks and forging stakeholder partnerships, he is dedicated to promoting health equity and delivering value-based healthcare solutions globally.
P51: The State of the Art in Antimicrobial Resistance Economic Modelling: Is the STEDI Framework Being Used to Capture Value?
9:15AM - 9:30AM
Sadler S1, Lee R2, Kaproulia A3, Khaouiry A3, Sutton A4
1Cytel, London, LON, UK, 2Cytel, Calgary, AB, Canada, 3Cytel, Rotterdam, ZH, Netherlands, 4Cytel, Edmonton, AB, Canada
OBJECTIVES: Antimicrobial resistance (AMR) is increasingly becoming a global public health priority. Payers have traditionally relied on cost-effectiveness analysis (CEA) to value novel interventions, but standard CEA methods often fail to capture key value elements for novel antimicrobials. Some of these value elements were proposed in the 2021 AMR-specific STEDI (spectrum, transmission, enablement, diversity and insurance) framework.
To capture an updated state of the art in CEA modelling methods we reviewed economic studies in AMR since the publication of the STEDI framework, and compared their approaches with the STEDI value elements.
METHODS: We searched for economic studies published between January 2021 and April 2024 which included AMR, where the intervention was a pharmaceutical intervention or a related test. Public health, policy or stewardship interventions were excluded. Modelling methods and approaches were extracted and assessed against each value element within the STEDI framework.
RESULTS: 6,692 studies were screened and 19 studies included (seven disease transmission models, five decision tree models, two Markov models, two agent-based models, two economic evaluations alongside trials and one theoretical economic model).
Comparing to the STEDI framework value elements, seven studies included one value element (one insurance value; three transmission; three diversity), three studies included both transmission and diversity. One study included three elements (transmission, enablement and diversity). No studies included four or all five value elements. Only three studies specifically referred to the STEDI framework when discussing their considerations.
CONCLUSIONS: Our review suggests that awareness of the STEDI framework and its value elements remains low. Few authors have attempted to include multiple value elements within any economic modelling structure. Despite some attempts to include multiple STEDI value elements, there remains a considerable gap for an economic modelling approach which can capture all five value domains within the STEDI framework.
P50: Technical Validation of an Environmental Model of Aurora EV-ICD: Recommendations to Guide Environmental Criteria in Health Technology Assessment
9:00AM - 9:15AM
Pegg M1, Hallas N2
1York Health Economics Consortium, York, UK, 2Medtronic Ltd, Watford, UK
OBJECTIVES: Climate breakdown is affecting human health globally. The National Health Service (NHS) generates 26 million tonnes of CO2e per annum equivalent to the size of Croatia’s annual emissions. Healthcare suppliers possess a sizeable opportunity to support health technology environmental sustainability (HTES) underpinned by leaner pathways and resource optimization. HTA is developing approaches to include environmental sustainability (ES). Reporting a broad range of environmental outcomes is important, but there is a lack of published guidance. This study aims to develop recommendations for decision makers on how broader environmental criteria should be included in HTA, based on the technical validation of an environmental model of Aurora EV-ICD by Medtronic.
METHODS: The Medtronic environmental model estimates the impact of the device on CO2e, water usage and waste volumes over a 10-year time horizon. A multi-step process was used to internally validate the model, using a specifically designed checklist for HTES models being reported as an information conduit. Recommendations were provided to support appropriate reporting of the data to the NHS.
RESULTS: The model structure was deemed appropriate and suitable for submission to the NHS. Recommendations for reporting ES to the NHS include: 1) using and referencing multiple environmental management guidelines for reporting CO2e to ensure transparency and reproducibility; 2) applying the same principles to quantify other environmental outcomes that will enable a more holistic evaluation of unintended consequences, including human health impact, resource use and biodiversity loss; 3) using life cycle assessment software (such as OpenLCA) to overcome data challenges and to facilitate reporting appropriate and comprehensive environmental endpoint categories.
CONCLUSIONS: This multi-perspective collaboration between industry and researchers supports ES in HTA framework development. These recommendations can be used by decision makers to aid the inclusion of environmental models that supports reporting a broader range of environmental outcomes over a long-term time horizon.
P49: Cost-Effectiveness (CE) of a Multi-Cancer Early Detection (MCED) Test Varies With Population Cancer Risk
8:45AM - 9:00AM
Raoof S1, Kansal A2, Tafazzoli A2, Ye W3, Zou D3, Fendrick AM4
1Memorial Sloan Kettering Cancer Center, New York, NY, USA, 2GRAIL, LLC, Menlo Park, CA, USA, 3Evidera, Bethesda, MD, USA, 4University of Michigan, Ann Arbor, MI, USA
OBJECTIVES: An MCED test has been projected to be cost-effective in the population aged ≥50 years. Prognostic factors such as family history, comorbidities, and lifestyle can define a range of subpopulations with varying cancer incidence, which each cover a subset of cancer burden. This study evaluates how cost-effectiveness relates to cancer risk in representative subpopulations of varying prevalence.
METHODS: A Markov model compared the outcomes of annual MCED testing plus standard of care (SoC) versus SoC alone in US adults aged 50 to 79 years, tailored for various high-risk groups. Analyses were conducted in the overall population aged ≥50 years and several subpopulations: first degree family history (1.38 incidence rate ratio vs general population, 55% prevalence), obesity (1.14, 38.7%), diabetes (1.12, 24%), current smoking (1.57, 16.9%), cancer survivor (1.69, 11%), heavy alcohol use (1.26, 9.4%), solid organ transplant recipients (SOT) (2.9, 2%), hereditary cancer syndromes (HCS) (1.86, 2.2%). Subpopulations were defined by cancer incidence and excess competing mortality. Survival, costs, and quality of life were calculated over a lifetime horizon.
RESULTS: MCED testing demonstrated lower incremental cost-effectiveness ratios (ICERs) for subpopulations with elevated cancer risk versus the overall population aged ≥50 years ($66,043/QALY). ICERs were: diabetes ($66,010), obesity ($60,245), family history ($40,155), cancer survivor ($36,774), heavy alcohol use ($35,515), current smokers ($26,850), SOT ($18,013), HCS ($544). Several high-prevalence subpopulations had ICERs <$50,000/QALY, while the lowest ICERs were predicted in smaller subpopulations.
CONCLUSIONS: While MCED testing has been projected to be cost-effective for the general population aged ≥50 years, prioritizing higher cancer risk groups allows targeting more favorable ICERs at the cost of addressing a smaller fraction of the total population burden.
P52: Incorporating Equity Into UK Decision-Making: The Need for Better Equity Data for Distributional Cost-Effectiveness Analysis
9:30AM - 9:45AM
Dhanji N1, Edwards H2, Kettle J1, Treharne C1, Ramagopalan S1, Clifton-Brown E3, Pearson-Stuttard J1
1Lane Clark & Peacock, London, UK, 2Pfizer Ltd, Tadworth, Surrey, UK, 3Pfizer Ltd, Tadworth, UK
OBJECTIVES: Inequalities in breast cancer exist by deprivation (mortality) and ethnicity (incidence and mortality). Recent NICE appraisals have highlighted inequalities in breast cancer according to ethnicity but have not quantified these. Distributional cost-effectiveness analysis (DCEA) evaluates the cost-effectiveness of health interventions within different demographic groups. A key data requirement of DCEA is quality-adjusted life expectancy (QALE) by subgroup; these data are currently unavailable by ethnicity in the UK. This study therefore aimed to conduct an aggregate DCEA in breast cancer with Index of Multiple Deprivation (IMD) as the domain of equity.
METHODS: Published data enabled the development of an aggregate DCEA, requiring stratified incidence data only, with IMD as equity subgroup. Population subgroups, baseline health distributions and opportunity costs were defined by IMD quintile. Representative incremental costs and quality-adjusted life years (QALYs) for a hypothetical treatment in breast cancer were used. The Atkinson social welfare function quantified trade-offs between health maximization and equity.
RESULTS: At a £30,000/QALY threshold and an Atkinson parameter of 10.95, the incremental population net health benefit was 15.8 QALYs, and the incremental Equal Distribution Equivalent Health (EDEH) was 4.2, suggesting an improvement in population health, but a worsening of health inequalities according to IMD.
CONCLUSIONS: This study highlights challenges in quantifying how new technologies may impact health inequalities in the UK. There are known inequalities according to ethnicity in breast cancer, however, we were unable to assess that inequality due to data insufficiency, and a DCEA in breast cancer found potentially worsening inequalities by IMD. There is a need for better data across all domains of equity, particularly those with published inequalities in unmet need such as ethnicity. This would enable a better assessment of treatment impact on inequalities to inform healthcare investment. A full DCEA, considering inequalities in both incidence and mortality, would provide a more comprehensive assessment.
9:00 - 11:30
Poster Session 5
Live
9:15 - 9:45
Exhibit Hall Theater
Finally It's Out! What the NICE Position on the AI Use in Evidence Generation and Synthesis Means for HTA Submissions – the Do’s and Don’ts
Digital Conference Pass
Level: Intermediate
The much-anticipated NICE position statement on the potential AI use in evidence generation and reporting of evidence to be submitted to the agency is finally published. This statement, from a world-class HTA body, opens the door for the wider and more systematic integration of AI tools in evidence development and generation for health technologies submissions. While the agency recognized the potential of these tools in different phases of the evidence preparation (systematic review and evidence synthesis, comparative effectiveness, and real-world analytics), there are many unanswered questions and challenges for manufactures to navigate such as the acceptability bar of integrating such tools in their submissions and assess the level of investment by formally integrating such tools in their preparatory activities.
The moderator will introduce the main points in the NICE AI statement and present some background HEOR research that may have mobilized the agency to publish such statement. The other panelists will present specific opportunities and challenges in translating the NICE position on AI during conducting systematic literature reviews, comparative clinical and cost-effectiveness analysis and using RWE in data analytics. Finally, the moderator will close by presenting a roadmap (checklist) to be used when AI tools are considered in NICE submissions and will open the floor for discussion with the audience.
Sponsor: Cytel
Sponsor
Cytel
Moderators
Grammati Sarri, PhD, MSc, DiDS
Cytel, London, United Kingdom
Grammati has 15+ years’ experience in epidemiology studies, evidence synthesis and decision making from her prior roles in academia, health technology assessment (NICE, UK), WHO and private health consultancies. Grammati has strong methodological experience in generating impactful evidence from different sources (clinical trials, real-world evidence, economic studies), clearly communicating findings in an impactful way and using complex analytical methods for health care-decision making. Grammati currently holds several positions in international organizations (ISPE), invited speaker to PhD training programmes in pharmacoepidemiology and current Advisory Board member to a EU funded trial on AI in breast cancer surgery (CINDARELLA). Grammati is working on EU JCA developments (produced blogs, delivered webinars, methods trainings) and topics around uncertainty, bias in both applications of non-randomized evidence and evidence synthesis. Publications and presentations related to RWE innovation: driving outputs on building knowledge around RWE methods guides, reliable RWE methods (framework for combining RWE with RCTs for decision-making [ISPE-endorsed], RWE quality assessment, statistical methods, data transferability, external controls for single-arm trials), and driving innovation in the use of RWE around topics such as patient experience, the lifecycle or living decision-making, health inequalities, data transferability across countries, specific challenges for RWE use in single-arm trials for rare and very rare diseases.
Speakers
Grace Hsu, MSc
Cytel, Waltham, MA, USA
Grace is Director of Real-World Evidence at Cytel providing statistical consulting and guiding project strategy for the application of advanced analytics to clinical and RWE generation. She also develops statistical communications and training for non-statisticians. Examples of her peer-reviewed publications include work on COVID-19, synthetic/external control arm comparative effectiveness analysis, quantitative bias analysis, Bayesian borrowing and other methods for comparative studies for both pharmaceutical research and HTA/regulatory submissions.
Grace holds a MSc in Statistics from SFU and BSc in Mathematics from UBC. Prior to joining Cytel, Grace was a senior statistician at MTEK Sciences (acquired by Cytel in 2019), a boutique analytics firm, where she worked with the Bill and Melinda Gates Foundation (BMGF), providing simulations and adaptive trial design for large-scale BMGF-funded clinical trials and analyzing national-level registries.
Sophia van Beekhuizen, MSc, MPhil
Cytel, Rotterdam, Netherlands
Sophia is Director & Research Principal at Cytel. She has over a decade of experience in consulting with focus on ITC and HTA submissions across Europe
Link to her profile: https://www.linkedin.com/in/sophie-van-beekhuizen-97727854/
10:00 - 11:00
Spotlight Session
The Dawn of a New Era: Cross-Border Collaborations—Regional, Pan-European, and Transcontinental—How Will They Shape the Future of Access?
Digital Conference Pass
Level: Intermediate
ISSUE: In the last decade, we have seen a proliferation of regional partnerships around Europe that have worked together on horizon scanning, HTA, pricing and joint purchasing. Against the backdrop of joint clinical assessment and the revision of the EU general pharmaceutical legislation, the European Commission is keen to increase knowledge sharing—and potentially joint procurement—among all Member States. New alliances have emerged beyond the borders of the EU—often featuring the UK National Institute of Health and Care Excellence (NICE)—that involve organisations from different continents. How do these disparate collaborations compare in their objectives and methods? Where will they focus their activity? What long-term impact will they have on access environments? This spotlight session will appeal to policymakers, HTA agencies, payers and pharmaceutical companies.
OVERVIEW: Neil Grubert (moderator) will begin with an overview of the key developments in cross-border collaboration in recent years. He will identify the key features of the leading regional, pan-European and transcontinental initiatives.
Dr Roisin Adams will reflect on the challenges and opportunities for regional partnerships, such as the Beneluxa Initiative and the International Horizon Scanning Initiative, and consider how they may evolve. She will also examine how the Heads of HTA Agencies will support voluntary cooperation on HTA.
Johan Potén will share insights on how the Nordic region has become a pioneer in cross-border collaboration, bringing together HTA and procurement to accelerate access to promising new treatments.
Dr Meindert Boysen will discuss the role the Novel Medicines Platform could play in improving access to innovative health technologies across Europe and beyond, and what other regions might learn. He will also consider how mature HTA agencies from different continents are working together in alliances such as the AUS-CAN-NZ-UK Collaboration Arrangement, and how they are helping less experienced countries to develop HTA systems.
Q&A
Moderators
Neil Grubert, MA
Independent, Basildon, ESS, United Kingdom
Neil Grubert is an international market access and policy consultant and trainer with 30 years of experience tracking global drug markets. Prior to his current role, he spearheaded the Decision Resources’ internal market access business, most recently as Vice President, Global Market Access Insights. He formed the Future of Pharmaceutical Market Access forum and posts on topical market access issues on LinkedIn on a daily basis.
Panelists
Roisin Adams, MPharm, MSc., PhD
National Centre for Pharmacoeconomics, Dublin, Ireland & Discipline of Pharmacology and Therapeutics, Trinity College Dublin, Dublin, Ireland
Meindert Boysen, PharmD, MSc
Boysen Consulting International, London, England, United Kingdom
Meindert was responsible at NICE for international partnership working, developing NICE’s role in international fora and representing NICE at key international meetings, further sustaining and developing NICE’s international reputation. Until recently, Meindert was the director at NICE responsible for delivery of the programmes that together form the centre for health technology evaluation. Including the technology appraisals, highly specialised technologies, medical technologies evaluation, diagnostic assessment and interventional procedures programmes. After qualifying as a pharmacist from Utrecht University, he worked in a hospital in the Netherlands, and held positions in health outcomes and sales in the pharmaceutical industry. Meindert completed the MSc in Health Policy Planning and Financing at the London School of Hygiene and Tropical Medicine and the London School of Economics & Political Sciences and briefly worked for the King’s Fund before starting at NICE in 2004. Meindert was a member of the Board of NICE and a director of the board of the International Society for Pharmacoeconomics and Outcomes Research (2017-2020).
Johan Pontén, MA
Medicine Evaluation Committee (MEDEV), Stockholm, Sweden
Johan Pontén is Senior Manager of International Affairs at The Dental and Pharmaceutical Benefits Agency in Sweden since 2014. He is since 2021 co-chair of the Medical Evaluation Committee, MEDEV, a network of 22 national authorities from 18 Member States and Norway bringing together all the relevant institutions (national HTA agencies and social health insurers-payers) responsible for the assessment, pricing and reimbursement of medicines in Europe. He has also supported the FINOSE collaboration between Nordic HTA agencies from the start in 2017. He has had a career in the Swedish Public Service and holds a Master in Pedagogic Leadership.
Breakouts: IP, WS, & OBS
Take My Word for It: Can Patient-Centric Social Listening Enrich RWE?
Digital Conference Pass
Level: Introductory
ISSUE: In our increasingly connected world, patients find information and support from their peers online, via social media and forums. These platforms provide a rich tapestry of lived patient experiences, capturing potentially valuable insights on topics such as disease burden, quality of life, and treatment outcomes. Until recently, the information within social media platforms remained under-utilised; however, capturing these data via social media listening (SML) has garnered attention as an assessable, low-burden, real-time source of real-world data (RWD). Historically, barriers to the wider adoption of SML have included lack of acceptance/guidance from key stakeholders, challenges with handling large unstructured data, and issues regarding bias, user privacy and content reliability. However, SML is now regaining attention with the advent of new evidence standard frameworks for digital health technologies and a growing interest in leveraging artificial intelligence to analyse big data, making it timely to re-examine its capacity in enhancing patient-centric real-world evidence. This session examines the promises and limitations of SML as a medium of RWD, sitting at the crossroads of many “hot topics” in the pharmaceutical industry.
OVERVIEW: Siddharth will open with a 5-minute balanced overview of SML as a medium of RWD. Next, Jackie will discuss the mechanics, value, and impact of SML and the use of AI to bring efficiencies to SML analysis. Following this, Mary Jo will examine challenges encountered in the use of AI from a clinical development/operations perspective and illustrate use cases of AI/SML informing adverse event reporting. Finally, Jeanette will provide a decision-maker’s perspective on the evidence standards that SML would need to meet to be useful for health technology assessment and acceptable use cases. Speakers are each allotted 10-minutes, followed by 20-minutes discussion time with audience participation encouraged throughout. Stakeholders from academia, industry and patient advisory groups would benefit from attending.
Moderators
Siddharth Ramanan, PhD
Costello Medical, Cambridge, CAM, United Kingdom
Siddharth Ramanan, PhD is a Senior Analyst in Real-World Evidence at Costello Medical Ltd., Cambridge, United Kingdom. Siddharth has more than ten years of experience in real-world evidence generation (particularly database analyses) and development and execution of research strategy for RWE projects. Prior to joining Costello Medical, Siddharth worked as a scientist at the University of Cambridge, UK where he led RWE projects in Alzheimer's disease. Siddharth holds a PhD from the University of Sydney, Australia and has published extensively in the field of RWE in dementia.
Panelists
Jackie Cuyvers, MSc
Convosphere, London, United Kingdom
Jackie Cuyvers is a recognized leader in health-focused social listening and behavioural research, with over 20 years of experience dedicated to advancing healthcare outcomes through innovative insights. As the CEO and Co-Founder of Convosphere, a global social listening consultancy, Jackie has built and led diverse teams of specialists across multiple languages and regions, pioneering new methods in digital research that directly impact public health strategies. Her expertise spans the development and implementation of social listening programs that have shaped global initiatives, particularly in the pharmaceutical and healthcare sectors.
Jackie holds an MBA in Marketing and a Master’s from Oxford in the Social Science of the Internet, grounding her practical expertise in a strong academic foundation. Her work is characterized by a unique ability to translate complex data into actionable strategies, making her a sought-after speaker at industry conferences worldwide. As an advocate for the application of social intelligence in healthcare, Jackie is committed to driving behavioural change through research, ensuring that healthcare organizations are equipped to meet the challenges of today’s digital landscape.
Jackie’s deep understanding of the pharmaceutical industry, combined with her operational acumen and intellectual curiosity, positions her as a pivotal figure in advancing the use of social listening to improve patient outcomes and support the strategic goals of global health organizations.
Jeanette Kusel, MSci, MSc
National Institute for Health and Care Excellence, London, United Kingdom
Jeanette has led the scientific advice, now NICE Advice, business unit at NICE since 2018. Jeanette has led many of the digital health initiatives at NICE and spend a period of time as acting Medtech Director. Prior to NICE, she spent 9 years working with consultancy providing HTA and Health Economics services. She has a Masters in Health Economics for Healthcare Professionals at the University of York.
Mary Lamberti, PhD
Tufts University, Boston, MA, USA
Mary Jo Lamberti, PhD is a Research Associate Professor at Tufts University School of Medicine and Director of Sponsored Research at the Tufts Center for the Study of Drug Development (Tufts CSDD) in Boston. She has extensive experience benchmarking drug development operating practices. Her research focuses on a variety of areas including patient recruitment and retention in clinical trials, investigative site initiation and management, and the impact of technologies and digital solutions in clinical trials. Dr. Lamberti has been a frequent speaker at global industry conferences and has published extensively. She holds a B.A. from Wellesley College and a Ph.D. from Boston University.
Can Greater Transparency on Public Funding Received by Pharmaceutical Companies to Develop Medicines Make New Medicines More Affordable?
Digital Conference Pass
Level: Introductory
ISSUE: The EC proposed pharmaceutical directive will require developers to be transparent regarding any direct financial support received from public bodies for R&D of medicines. This issue panel explores the implications and reactions of academia, industry, and payers. How should companies report the public funding received? How can payers use this information in HTA and price negotiations? How does it affect incentives to finance and conduct R&D?
OVERVIEW:
Jaime Espin will introduce the issue and panelists (5 minutes) David Epstein will discuss academic scholarship providing insight into what public funding of R&D achieves, and whether such investments should be recovered in lower prices for medicines (15 minutes) Yannis Natsis will argue that transparency into public funding streams can contribute to tackling the information asymmetry that fuels that power imbalance between companies & payers. Public funding is only one aspect of the multilayered public support that goes into biomedical R&D. The ultimate objective is to provide equitable & affordable access for all patients but also steer innovation. (15 minutes) Carole Longson will argue that the interplay between public and private R+D is complex especially when considering the value attribution of transferring innovation from academia into industry. Many scientists move between academia and industry during their careers, bringing with them their knowledge, skills and intellectual property. Greater transparency in understanding the financial flows involved in bringing innovation to patients is important and useful. However, a framework to apportion costs and value across the public/private interface is challenging. A key element to be discussed will be how to develop and implement the right incentives that allow each actor in this complex public/private innovation system to be rewarded appropriately. (15 minutes) The moderator will ask the audience vote on their preferred approach in a poll (2 minutes) and to submit questions for the panel (10 minutes)
Moderators
Jaime Espin, PhD
Andalusian School of Public Health, Granada, GR, Spain
Jaime Espín has a PhD in Economics (University of Granada), a Bachelor Degree in Law (University of Granada) and a Master in Health Economics (Andalusian School of Public Health). He has been Visiting Graduate Fellow in the Department of Economics in the University of California (UCSB); Visiting Professor in Duke University (Fuqua Business School), Columbia University (Mailman School of Public Health) and Harvard University (Department of Population Medicine at Harvard Medical School and Harvard Pilgrim Health Care Institute); and Visiting Researcher in The University of Chicago.
Prof. Espín is an expert in European pharmaceutical policies and has been external advisor for the European Commission during the High Level Pharmaceutical Forum (Working Group on Pricing). He has been member of the Experts Committee on Pharmaceutical Policies of the Pan American Health Organization (PAHO/WHO), member of the Pricing Policy Group of the WHO/HAI Project on Medicines Prices and Availability and Expert of the WHO Guideline Panel on Country Pharmaceutical Pricing Policies. He has participated in several commissioned reports for a HTA Agency in Spain (AETSA),
He is co-author of the several European Commission publications as “Differences and Commonalities in pricing and reimbursement systems in Europe” and co-editor of the Pan America Health Organization (WHO/PAHO) publications as “The access to high cost medicines in the Americas” and “Guideline for implementing generic strategies in Latin America and the Caribbean as a mechanism to improve access to medicines”.
Panelists
David Epstein, Phd
University of Granada, Granada, Spain
DAVID EPSTEIN is Professor at the Department of Applied Economics, University of Granada, Spain. His research centres on the economic evaluation of health care technology. He was special economic advisor to the Ministry of Health in Spain from 2021 - 2023 and participates as observer in the EU Joint Scientific Consultation subgroup
Carole Longson, MBE
Carole Longson Consultant, Manchester, United Kingdom
Carole is a respected leader in health technology assessment with considerable achievements in research, public and private sectors. She has unparalleled insight into the health technology innovation pipeline having pioneered the development of NICE’s health technology evaluation programmes. After a career in drug discovery at GSK, Carole was an Executive Director at NICE from 2000-2018 and Chief Scientific Officer at the Association of the British Pharmaceutical Industry from 2018-2020. She was formerly President of Health Technology Assessment International and now is an independent advisor including Life Science Advisor at NICE.
Carole is currently vice chair of the Medicines Discovery Catapult in the UK, was previously on the Scientific Advisory Committee for the EU Innovative Medicines Initiative (IMI) and holds non-Executive Director and advisory roles in the UK and abroad. Throughout her career, she has championed the public and private sector collaborating to ensure medical innovation benefits patients in a way that is sustainable for healthcare systems.
Yannis Natsis, MA, BA
European Social Insurance Platform (ESIP), Brussels, Belgium
Yannis Natsis is the Director of the European Social Insurance Platform (ESIP), the umbrella organisation bringing together 45 national statutory social security institutions from 18 countries. ESIP is the voice of social protection and security in Europe or as Yannis puts it one of Europe’s truest treasures.
He has more than 10 years of experience in EU advocacy and policymaking. Prior to joining ESIP in February 2022, he led the advocacy for better and affordable medicines at the European Public Health Alliance (EPHA). In May 2019, he was appointed by EU Member States to the Management Board of the European Medicines Agency (EMA), a position he held until December 2021. Additionally, Yannis has been a Board member of the European Health Forum Gastein (EHFG), the leading EU health policy platform since 2018.
Yannis previously worked for the TransAtlantic Consumer Dialogue (TACD) focusing on health and pharmaceutical policies. From 2006-2010, he was an investigative reporter for Greece's award-winning TV news programme "Fakeli" and a contributor to one of Greece’s most respected dailies “Kathimerini”.
He has a Master's degree in International Conflict Analysis from the University of Kent, UK and a Bachelor's degree in European Studies from Pantion University of Social and Political Sciences, Athens, Greece. A Greek national, he is fluent in Greek, English and French.
Incorporating Life Cycle Drug Pricing in Cost-Effectiveness Models: What Are the Opportunities and Risks for Health Systems and Manufacturers?
Digital Conference Pass
Level: Intermediate
ISSUE:
Generic and biosimilar medicines provide health benefits at low costs long after the patent expiration of the brand-name products they replace. However, most cost-effectiveness analyses of new products do not account for this process of drug genericization. This has led to concerns that current cost-effectiveness analyses may overestimate the long-term costs of pharmaceuticals, leading to inappropriate pricing and access decisions for new drugs. In this panel we will debate whether drug genericization should be reflected within cost-effectiveness analysis of new pharmaceuticals.
OVERVIEW:
Mark Sculpher will provide an accessible overview of the issue (5 minutes), this will be followed by a short presentation by each panelist (12 minutes) and an open debate on the topic. Peter Neumann will argue that it is time for economic evaluations of new pharmaceuticals to use broader methods that account for downstream, generic pricing. Beth Woods will argue that including drug genericization within cost-effectiveness analysis would substantively increase prices of new medicines, and that this methods shift would need to be accompanied by a change to the principles of value-based pricing. Dan Ollendorf will discuss the approaches currently taken by HTA agencies like ICER to life-cycle pricing, and the practicality and acceptability of the types of changes proposed by Peter and Beth. This presentation will be of interest to a wide range of stakeholders with interests in economic evaluation of pharmaceuticals, and pharmaceutical pricing and reimbursement policy.
Moderators
Mark Sculpher, PhD
University of York, York, United Kingdom
Mark Sculpher is Professor of Health Economics and Head of Department of the Centre for Health Economics, University of York. He is also Co-Director of the Policy Research Unit in Economic Evaluation of Health and Care Interventions, a programme of research for the UK Department of Health and Social Care funded by the National Institute for Health Research (NIHR).
Panelists
Peter Neumann, ScD
Tufts Medical Center, Boston, MA, USA
Peter J. Neumann, Sc.D., is Director of the Center for the Evaluation of Value and Risk in Health (CEVR) at the Institute for Clinical Research and Health Policy Studies at Tufts Medical Center, and Professor of Medicine at Tufts University School of Medicine. He is the founder and director of the Cost-Effectiveness Registry, a comprehensive database of cost-effectiveness analyses in health care. Dr. Neumann has written widely on the role of clinical and economic evidence in pharmaceutical decision making and on regulatory and reimbursement issues in health care. He served as co-chair of the 2nd Panel on Cost-Effectiveness in Health and Medicine. He is the author or co-author of over 300 papers in the medical literature, and the author or co-author of 3 books: Using Cost-Effectiveness Analysis to Improve Health Care (Oxford University Press, 2005) ; Cost-Effectiveness in Health and Medicine, 2nd Edition (Oxford University Press, 2017); The Right Price: A Value-Based Prescription for Drug Costs (Oxford University Press, 2021). Dr. Neumann has served as President of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR). He is a member of the editorial advisory board of Health Affairs and the health policy advisory board for the Congressional Budget Office. He has also held several policy positions in Washington, including Special Assistant to the Administrator at the Health Care Financing Administration. He received his doctorate in health policy and management from Harvard University.
Dan Ollendorf, PhD
Institute for Clinical and Economic Review, Boston, MA, USA
Dan Ollendorf is Chief Scientific Officer and Director of Health Technology Assessment (HTA) Methods and Engagement at the Institute for Clinical and Economic Review (ICER), with responsibility for managing the organization’s internal health economics capacity and program of external collaboration, as well as collaboration with international HTA bodies and other stakeholders on key process and methods topics.
From 2007-2018, Dr. Ollendorf was Chief Scientific Officer for ICER, where he oversaw the broader HTA process and managed relationships with multiple stakeholders and research collaborators. From 2018-2023, Dr. Ollendorf served as the Director of Value Measurement & Global Health Initiatives at the Center for the Evaluation of Value and Risk in Health (CEVR) at Tufts Medical Center, where he focused on value assessment methods, drug pricing policy, and building international capacity for and interest in HTA. In addition to his current role at ICER, Dr. Ollendorf maintains a faculty appointment at CEVR and continues to teach in the Center’s graduate certificate program as well as participate in ongoing research.
Dr. Ollendorf holds a PhD in clinical epidemiology from the University of Amsterdam and an MPH from Boston University. He is the current Chair of the Health Technology Assessment International Global Policy Forum as well as a non-resident Fellow at the Center for Global Development, and served on the US Medicare Evidence Development and Coverage Advisory Committee (MEDCAC) from 2015-2019. Dr. Ollendorf has authored over 125 articles in peer-review journals and is co-author of The Right Price: A Value-Based Prescription for Drug Costs (Oxford University Press).
Beth Woods, BA, MSc
University of York, York, United Kingdom
Beth is a Senior Research Fellow in the Team for Economic Evaluation and Health Technology Assessment (TEEHTA) and has been based at the University of York since 2014. Beth holds a BA in Economics from the University of Cambridge and an MSc in Economic Evaluation in Healthcare from City University.
Prior to joining CHE Beth was a Director in the Health Economics team at Oxford Outcomes, a private consulting firm, where she specialised in applied economic evaluation from 2006-2013.
Beth has worked on economic evaluations of a range of technologies, including the application of advanced statistical and decision modelling methods. Beth has also contributed to methods in the field, in particular relating to model structuring in oncology, evaluation of pharmaceutical pricing policy, and evaluation and pricing of technologies to address antimicrobial resistance.
Beth is a member of the NIHR HTA Clinical Evaluation and Trials Board and sits on the Board of Directors for the York Health Economics Consortium.
Re-using and Sharing Systematic Review Data Between Competing Companies and HTA Stakeholders: Is It Feasible and How Can Challenges Be Overcome?
Digital Conference Pass
Level: Intermediate
ISSUE:
This panel will debate Health Technology Assessment (HTA) r
esearch waste associated with systematic literature reviews (SLRs)
conducted by multiple companies for different assets launching in the same indication. The extent to which waste can be reduced through SLR data reuse between companies and HTA bodies will be discussed. The potential solution of living systematic reviews (LSRs) and shared libraries will be debated; opportunities and challenges will be considered to determine the feasibility of this approach.
OVERVIEW:
HTAs evaluate the value of new treatments relative to standard of care. Submissions require the development of costly and time-consuming SLRs. Particularly in disease areas with rapidly emerging new treatments, research waste can occur, with multiple companies conducting similar SLRs. Thilo Schaufler will provide an overview of research waste in HTA and the impact on patients. Thilo will
propose that LSRs could allow reuse and expansion of data by companies and HTA stakeholders. [8 minutes] Helene Vioix will offer a company perspective in favor of LSRs, arguing that static SLRs are outdated. Helene will describe challenges of rapidly emerging evidence, with companies producing SLRs to support multiple activities. [9 minutes] Sue Harnan will raise considerations from the HTA perspective, as an Evidence Assessment Group member. Sue will consider how LSRs could improve transparency and consistency, noting potential pitfalls, methodological considerations, and challenges. [9 minutes] Vicki Young
will describe practical barriers that must be overcome to share LSRs between stakeholders. Vicki will cover LSR commissioning, competitive interests, and stakeholder sacrifices required to gain from LSRs. Thilo will facilitate debate on overcoming shared LSR challenges. The impact on HTA methods and funding will be considered. Audience members will have an opportunity to comment on each challenge. Finally, each panelist will summarize the feasibility of LSRs in HTA from their perspective. [20 minutes]
Moderators
Thilo Schaufler, Ph.D.
CSL Vifor, Glattbrugg, ZH, Switzerland
Thilo holds a PhD in health economics and has 18 years’ experience in different Market Access roles of increasing responsibility in the pharmaceutical industry. He has extensively published in scientific journals and at international conferences.
Panelists
Sue Harnan, MSc
The University of Sheffield, SHEFFIELD, United Kingdom
Sue has been working in HTA as a member of an EAG (SCHARR-TAG) for the National Institute for Health and Care Excellence (NICE) for over 15 years. Her background is in Systematic Reviews and Evidence Synthesis.
Helene Vioix, PharmD, MSc
Evidence and Value Development, Merck Healthcare KGaA, Bad Homburg, HE, Germany
Helene is a global leader in the pharmaceutical industry. She has a background in pharmacy and health economics and 14 years' industry experience.
Vicki Young, PhD
Cytel Statistical Services Ltd, London, LON, United Kingdom
Vicki has an academic background (MSc, PhD) and 14 years’ experience in Health Economics and Outcomes Research consultancy.
Bridging Nations, Diverging Views: Exploring the Global Landscape of Indirect Treatment Comparisons Acceptance by Health Technology Assessment Bodies
Digital Conference Pass
Level: Intermediate
ISSUE:
In the absence of head-to-head randomized controlled trials, indirect treatment comparisons (ITCs) can be used to derive comparative effectiveness between therapies. However, there is significant variation in global health technology assessment (HTA) agencies’ acceptance of ITC methodologies, resulting in inconsistent decisions on drug reimbursements, and hindering timely patient access. As the global landscape of the role of ITCs in HTA submissions continues to evolve, particularly with EU’s joint clinical assessment (JCA), it is critical to discuss the key barriers to ITC acceptance and to identify appropriate methodological approaches to increase the value and transparency of ITCs for decision-making.
OVERVIEW:
This panel seeks to dissect the challenges and disparate viewpoints surrounding the utilization of ITCs in HTA submissions and their acceptance by global HTA agencies. Moderated by Dr. Chris Cameron, this panel will delve into the key barriers to acceptance of ITCs, potential strategies to consider in addressing these concerns, and emerging trends shaping the acceptance landscape. The panel discussion will be 50 minutes in length, plus 10 minutes for audience questions. Key questions include:
- What do you see as the key barriers to acceptance of ITCs by HTA agencies?
- What do you believe specifically needs to be done by industry sponsors to overcome these barriers when submitting ITCs to HTA agencies?
- How will EU JCA improve the consistency and acceptance of ITCs used in HTA submissions across Europe?
The panel will feature insights from experts representing HTA agencies, industry sponsors, and academia. Attendees, including HTA professionals, regulators, academics, and industry stakeholders, will gain valuable perspectives on navigating the intricate landscape of ITC acceptance in HTA submissions.
Moderators
Chris Cameron, MSc, PhD
EVERSANA, Sydney, NS, Canada
Dr. Chris Cameron is Chief Scientific Officer at EVERSANA. He is a global thought leader in health economics and outcomes research with over 15 years of experience. Prior to joining EVERSANA, Chris was a partner at Cornerstone Research Group Inc., and lead of Health Economics at CADTH. He has also been a consultant decision scientist for ICER, a member of the Economic Guidance Panel at pCODR, and served on PMPRB Technical Panel on drug pricing guidelines in Canada. Chris is an adjunct professor at Dalhousie University, and has authored over 180 peer-reviewed publications in many of the world’s top journals, including JAMA, Lancet, and BMJ. His research has been featured in the Globe and Mail and New York Times. He was recently named one of the most prolific authors in the world on indirect treatment comparisons and was a co-author on the PRISMA NMA reporting guidelines. Chris holds a bachelor’s degree in mathematics and an MSc and PhD in Epidemiology where he was a Vanier Canada Graduate Scholar. He also completed a fellowship on real-world data analytics at Harvard University and FDA’s Sentinel Initiative.
Panelists
Nicholas Adlard, MA MSc MBA
Novartis Pharmaceuticals UK Ltd, Busserach, SO, Switzerland
With a career spanning many years in the Pharma Industry, I have spent the last 15 years focusing on Access and HEOR specialisms, supported by an MBA and MSC in Health Economics and Health Policy. I have been associated with 10 peer reviewed manuscripts on indirect comparison topics.
Stefan Lange, MD
Dierks+Company, Berlin, BE, Germany
1989 to 1993 Junior doctor and assistant doctor at the Medical Clinic of the Ferdinand Sauerbruch Hospital, Wuppertal.
1993 to 2004 Research Associate, Department of Medical Informatics, Biometry and Epidemiology, Ruhr University, Bochum.
1994 Doctorate in Medicine, Heinrich Heine University, Düsseldorf.
2003 Habilitation and venia legendi (award of title of Privatdozent) in Medical Biometry and Clinical Epidemiology, Ruhr University, Bochum.
2004 to 2024 Institute for Quality and Efficiency in Health Care, Cologne. Deputy Director since 2005.
Since 08/2024 Chief Scientific and Medical Officer at Dierks+Company, Berlin.
Olivia Wu, PhD
University of Glasgow, Glasgow, United Kingdom
Olivia holds the William R Lindsay Chair of Health Economics and is co-Director of Health Economics and Health Technology Assessment (HEHTA) at the University of Glasgow. Her expertise is in the adaptation and application of HTA methodologies in context. Her research is multidisciplinary in nature, and spans across a wide range of clinical areas and different types of health interventions (e.g. pharmacological treatments, medical devices, diagnostic tests, and digital health interventions). She has led numerous health economic evaluations alongside clinical evaluations and trials, which work has informed clinical guidelines and health policy decisions, both at national and international levels. Olivia also has a body of work on novel and effective use of evidence synthesis approaches to maximise the value of existing data for informing clinical and economic evaluations. Currently, Olivia leads the Complex Reviews Synthesis Unit (CRSU) which hosts one of the nine Evidence Synthesis Groups funded by the National Institute for Health and Care Research (NIHR). Olivia is a long-standing advisor to HTA agencies within and out with the UK. She has previously served as member of the NICE Technology Appraisal Committee for 10 years.
Olivia has a strong commitment to developing an inclusive environment for the next generation of researchers within and out with the UK. She co-leads the ISPOR Women in Health Economics and Outcomes Research Initiative and is a steering member of the NIHR Race Equity and Diversity in Careers Incubator. Olivia is a strong advocate for mentoring and provides mentorship to academic and government agency researchers both at national and international levels.
Taking the “Greener” Pill: A Case Study for Incorporating Carbon Footprint in Health Technology Assessment
Digital Conference Pass
Level: Introductory
PURPOSE: To test the feasibility of accounting for carbon emissions in HTA and go beyond theoretical considerations by showing and discussing worked examples for carbon footprint (CF) assessment inspired by methods used in clinical trial conduct.
DESCRIPTION: Previous discussions at ISPOR addressed the normative aspect of incorporating environmental impacts in HTA, but they stopped short of practical consideration of how this could be implemented. HEOR is not the only field grappling with methodological and data availability challenges related to assessing greenhouse gas (GHG) emissions, and we can learn from initiatives in other parts of the industry, such as clinical trial conduct. This session will focus on practical examples of methods to incorporation of carbon footprint assessment into HTA.
This session will start with a discussion of types of incentives available to encourage industry to improve the carbon efficiency profile of products (regulatory, reputational, market-based), followed by outlining how HTA bodies have approached this debate to date, and summarising the progress made (15min, Hampson). A state-of-the art method for assessing CF via a full or partial lifecycle analysis and synergies between data generation for carbon emission assessment in trials and for HTAs will be discussed. An example of an approach to CF assessment of a clinical trial will be shown, alongside challenges encountered and solutions implemented related to methodology and data availability (15min, Cohen). Concepts of “parallel environmental evaluation” and “evaluation integrated into HTA” will be demonstrated via a case study. Aspects of the methodology will be discussed including direct/indirect impact on carbon emissions; potential ways of presenting CF alongside traditional cost-effectiveness results; converting GHG emissions into monetary values using alternative concepts; an exploratory method for calculating carbon emission adjusted economically justifiable price (15min, Marczell). Active participation of the audience will be encouraged via polling (15min).
Discussion Leaders
Ruth Chapman, PhD
Evidera Ltd, London, LON, United Kingdom
Epidemic Modeler
Discussants
Michael Cohen, MSc, MBA, SEA
PPD, Ft Lauderdale, FL, USA
Michael Cohen, MSc, MBA, SEP, is the Senior Director and Lead of Environmental Sustainability at PPD, part of Thermo Fisher Scientific. Michael leads PPD's environmental sustainability strategy focused on models, decarbonization and carbon accounting. Michael did his undergraduate studies at University of California, Berkeley and graduate work at Northwestern University, as well as completing an MBA in Finance from Louisiana State University, Shreveport. Recently, Michael also passed the International Society of Sustainability Professionals “Sustainable Excellence Professional” credentials.
Grace Hampson, MSc
Office of Health Economics, London, United Kingdom
Grace is an economist with expertise in the methods and processes of value assessment, reimbursement and healthcare decision making. Her current research interests include interactions between health technology appraisal and novel payment mechanisms, particularly in relation to innovative technologies such as cell and gene therapies and new drugs to tackle Antimicrobial Resistance. Grace leads OHE’s Policy Organisation and Incentives in Health Systems research team.
Kinga Marczell, PhD
Evidera, London, United Kingdom
Adjusting Survival Estimates for Treatment Switching: New Guidance and the Role of Real-World Evidence
Digital Conference Pass
Level: Intermediate
PURPOSE:
NICE's Decision Support Unit has published a new technical support document about treatment switching (when trial participants receiving the control treatment crossover to the experimental treatment). The
TSD 24 (April, 2024) offers comprehensive reporting guidelines and specific recommendations. We review the various crossover adjustment methods recommended (e.g., rank-preserving structural failure time models (RPSFT), inverse probability of censoring weighting (IPCW), two-stage estimation (TSE)) and highlight recent statistical developments. We then explain the reasoning behind the TSD's recommendations and review key takeaways from a review of recent NICE submissions. Given that real-world evidence (RWE) is increasingly used to support applications for regulatory and HTA approvals, the TSD 24 discusses the potential of using external data for crossover adjustment. We review examples of how external data has been used for crossover adjustment in a number of recent studies (e.g., used to validate different crossover adjustment methods, and used to create an external control arm without any crossover contamination). Finally, we discuss a newly proposed method to estimate counterfactual survival by leveraging RWE to supplement RCT data in an efficient and unbiased manner. This approach may prove particularly valuable given the increasing availability of routine electronic health record (EHR) data.
DESCRIPTION:
Workshop attendees will be asked (real-time polling) to consider the impact of the latest NICE guidance, if RWE has potential to improve cross-over adjustment, and if different sources of bias can be adequately addressed. Ms. Cope will chair the session and begin by summarizing the various statistical methodologies often used for cross-over adjustment (10 min). Dr. Latimer will then review the recently published TSD 24 guidance (12 min). Dr. Jansen will review specific examples of how RWE has been considered for cross-over adjustment (12 min) and Dr. Campbell will conclude by considering newly proposed methods for integrating external data (12 min).
Discussion Leaders
Shannon Cope, MSc
Precision AQ, VANCOUVER, Canada
Shannon Cope, MSc, is a Senior Vice President with Precision AQ. Shannon has over ten years of experience consulting for pharmaceutical industry regarding evidence synthesis and economic evaluations to assess the value of new interventions.
Discussants
Harlan Campbell, PhD
University of British Columbia, Vancouver, BC, Canada
Harlan Campbell is a statistician serving as Adjunct Professor at the University of British Columbia and as Principal Scientist/Senior Methodologist at Precision AQ. He completed his PhD in Statistics at UBC, researching methods for hypothesis testing and causal inference. His applied research focusses on applications in evidence synthesis, epidemiology and clinical trials.
Jeroen Jansen, PhD
University of California – San Francisco, San Francisco, CA, USA
Jeroen P Jansen PhD is a methodologist working at the intersection of evidence synthesis, biostatistics, and health economics.
He is an Associate Professor in the Department of Clinical Pharmacy in the School of Pharmacy at the University of California, San Francisco and Chief Scientist − Health Economics & Outcomes Research at the Precision Medicine Group.
For the past 15 years, Dr. Jansen has worked on research to understand the clinical and economic value of healthcare interventions. His research has frequently been conducted in the context of health technology assessment (HTA) with a focus on comparative effectiveness and cost-effectiveness. Prompted by the challenges encountered in applied research projects, he has performed methodological research. Notable contributions are the development of novel statistical methods to overcome the typical challenges in model-based cost-effectiveness evaluations characterized by gaps in the evidence base and complex evidence structures. Furthermore, Dr. Jansen led initiatives to develop guidance for consumers and producers of network meta-analysis studies. He has promoted a more transparent and credible approach to model-based health economic evaluations and led the development of open-source simulation models to illustrate its feasibility. Furthermore, Dr. Jansen has been involved in the ongoing development of an R software package to develop simulation models for health economic evaluations.
His current research interests are the clinical and economic value of precision medicine, incorporating health disparities in health economic modeling studies, and statistical methods for evidence synthesis.
He has published extensively in his areas of expertise and is widely cited. He is co-author of a textbook on network meta-analysis for decision-making and was associate editor for the Journal for Research Synthesis Methods. Dr. Jansen has a PhD in epidemiology from the Erasmus University in the Netherlands.
Nicholas Latimer, PhD, MSc
Delta Hat Ltd, Nottingham, UK; University of Sheffield, Sheffield, Great Britain
Nick is a Professor of Health Economics at the University of Sheffield, a Yorkshire Cancer Research Senior Fellow, and an Analyst for Delta Hat Ltd. He was a member of NICE Technology Appraisal Committee B for 5 years and is a member of the NICE Decision Support Unit (DSU). Nick’s research focuses on survival analysis in economic evaluations. He has authored four DSU technical support documents related to survival analysis, treatment switching, and partitioned survival models. His current work involves investigating the use of cancer registry datasets to estimate the comparative effectiveness of cancer treatments used in clinical practice.
Podium Sessions
Health Technology Assessment in Action
Digital Conference Pass
Examinations of health technology assessment reforms, measures, and outcomes.
Moderator
Gihan Elsisi, PhD
The American University in Cairo, cairo, C, Egypt
Gihan Hamdy Elsisi, Msc, PhD is Assistant Prof, American University in Cairo. She is the founder and former Head of Pharmacoeconomic Unit at the Egyptian drug Authority (EDA), Egyptian Ministry of Health and consultant at USAID and WHO EMRO. Elsisi received PhD in Pharmacoeconomics /pharmaceutical sciences from Ain Shams University and certified in Health Economics (HE) and Outcomes Research, University of Washington. Through the EDA, Elsisi was able to successfully incorporate Pharmacoeconomic and Outcomes Research into the coverage decisions of drugs in Egypt and build HE capabilities in different Middle East countries. On international level, she is the Health Science Policy Council Advisor, at International Society for Pharmacoeconomic and Outcomes Research (ISPOR). She is the principal author of both Health Care Systems Roadmap for Pharmaceutical pricing and reimbursement and the Pharmacoeconomic Guidelines for Egypt, ISPOR. She is an executive member in HE Diploma Committee, American University in Cairo. Elsisi represented Egypt in the First and Second Middle East Africa Pricing & Reimbursement Future Trends Workshop in Muscat 2012 and in Dubai 2013. She has several publications in HE and also contributed to a number of clinical research and surveys. She is the treasurer of ISPOR Egypt Chapter. She is a special guest speaker at many international companies for achieving market access and maximizing commercial performance in North Africa & Middle East.
P53: Budget Impact Analysis of a Possible Extension of Public Funding for the Treatment of Disease-Related Malnutrition in Spain
10:00AM - 10:15AM
Lifschitz E1, Breton I2, Álvarez-Hernández J3, León Sanz M3, Botella-Romero F2
1Hiris Care, Madrid, Madrid, Spain, 2Sociedad Española de Endocrinología y Nutrición (SEEN), Madrid, Madrid, Spain, 3Sociedad Española de Nutrición Clínica y Metabolismo (SENPE), Madrid, Madrid, Spain
OBJECTIVES: Although the treatment of Disease-Related Malnutrition (DRM) reduces rehospitalizations by 27.5% and the average length of stay (ALOS) by 26.1%, the establishment of nutritional treatment at home in Spain is limited by an outdated regulatory framework. The aim of this research is to estimate the financial burden that a possible extension of DRM treatment at home would entail after hospital discharge.
METHODS: A group of chronic diseases/clinical conditions that nutrition experts propose to include in the financing of Home Enteral Nutrition (HEN) was analyzed, when they are associated with severe DRM: severe COPD, New York Heart Association Class III-IV Heart Failure, Child-Pugh C Chronic Liver Disease, Chronic Kidney Disease stage IV, and Pressure ulcers stage III-IV. Target population was estimated from a systematic literature review, prioritizing studies on the Spanish population, and data from official sources. The cost of HEN (Oral Nutritional Supplementation: 400 mL/day for 12 weeks) and hospitalizations was included in the calculation of the net cost per patient. A dissemination rate of 24% (expert opinion) and a time horizon of one year were assumed, from the perspective of the National Health System (NHS).
RESULTS: Per year, 14,096 patients are discharged with these chronic diseases/conditions and severe DRM from Spanish public hospitals. The average cost of the HEN is €742.40/patient, and the cost of hospitalization is €900.56/day. The reduction in rehospitalizations and ALOS in those receiving HEN allows us to estimate a net cost per patient treated with HEN of -€3,240.69. The financing of 24% of these patients would generate savings of almost eleven million euros for the NHS.
CONCLUSIONS: The financing of the HEN in these diseases would have an impact on significant savings for the NHS, even without considering other savings due to a reduction in non-hospital care resources (other medicines, home care).
P55: Navigating Change: A Comparative Analysis of Health Technology Assessment Reforms Across Agencies and Their Interdependencies
10:30AM - 10:45AM
Cubi-Molla P1, Kumar G2, Radu P3, Garau M1, Bell E4, Pan J5, Gilardino R6, van Bavel J7, Brandtmuller A8, Nelson K9, Goodall M10
1Office of Health Economics, London, LON, UK, 2Office of Health Economics, London, Lon, UK, 3Office of Health Economics, London, UK, 4UKHSA, London, LON, UK, 5Adelphi Values PROVE, Bollington, Cheshire, UK, 6MSD, Dubendorf, ZH, Switzerland, 7MSD, Sydney, NSW, Australia, 8MSD Pharma Hungary Ltd, Budapest, Hungary, 9Drexel University, Philadelphia, PA, USA, 10Goodall HTA Consulting, Manchester, Manchester, UK
OBJECTIVES: Changes in Methods and Processes (M&P) of Health Technology Assessment (HTA) agencies can affect time and degree of patient access to treatments. Our study investigates HTA reforms and explores interdependencies and proactivity in implementing M&P reforms by 14 HTA agencies in Europe, Asia-Pacific, and North America. The study explores how the agencies' positions on modifiers, real-world evidence (RWE), patient involvement, surrogate endpoints and discounting have evolved.
METHODS: We conducted a targeted literature review on M&P guidelines and subsequent changes implemented by 14 HTA agencies from 2010 to 2023. We supplemented and validated initial findings with 29 semi-structured interviews with country-specific experts. We used analytical tools to create interdependency networks and a framework to group HTA agencies based on proactivity and influence.
RESULTS: We classified PBAC(Australia), CADTH(Canada), NICE(England), IQWiG(Germany), and ZIN(the Netherlands) as catalysts of HTA reforms and internationally influential; HAS(France), TLV(Sweden), and KCE(Belgium) as traditionalists; DMC(Denmark), AIFA(Italy), INFARMED(Portugal), ACE(Singapore), AEMPS(Spain), and CDE(Taiwan) as observers.
All HTA agencies appear to use modifiers in their decision-making, whether through an explicit framework or implicitly with an element of discretion. Recent years have seen an increase in guidance or clarification on opportunities for patient involvement in HTA. Most of the HTA agencies consider RWE and use of surrogate endpoints to some extent in decision making, with the caveat that final outcomes are preferred. Base case discount rates have remained mostly unchanged over time, and all agencies have permitted sensitivity analyses on rates for historical or international comparison.
CONCLUSIONS: Our work provides an overview of past HTA M&P reforms internationally. While some HTA agencies have evolved and formalised their positions on certain aspects, others lack an explicit stance. International collaborations represent a valuable route to accelerate changes by encouraging consistency and providing leadership. To be fully successful, collaborations should ensure wide involvement of stakeholders at an early stage.
P56: The Value of NICE Scientific Advice
10:45AM - 11:00AM
Wang Y1, Mccracken F1, Landels H1, Kusel J2
1National Institute for Health and Care Excellence, Manchester, UK, 2National Institute for Health and Care Excellence, London, UK
OBJECTIVES: The National Institute for Health and Care Excellence (NICE) launched its advice service, NICE Scientific Advice, in 2009. A previous study reported that medicinal products that received scientific advice had a reduced time for NICE appraisal than those that did not. To identify the recent impact of scientific advice on time for NICE appraisal, we conducted a study on medicinal products that sought scientific advice from 2020 to 2023.
METHODS: This study examined NICE Technology Appraisal (TA) and Highly Specialised Technologies (HST) guidance published from June 2020 to May 2023. It identified technologies that received scientific advice from the NICE Advice database and matched the intended conditions to the published guidance. The study compared the time from marketing authorisation to guidance publication for technologies that received scientific advice verses those that did not.
RESULTS: 230 TA and HST guidance documents were published during the study period and were included in the analysis.
Of the 230 technologies analysed, 51 of these had received scientific advice. For these products, it took a median of 312 days from marketing authorisation to the publication of NICE guidance, compared with 400 days for those that did not receive scientific advice. It is important to note that various other factors may impact time to guidance publication, but these would apply to both technologies that have and have not received scientific advice, hence these were not measured as part of this study.
CONCLUSIONS: The results of this study support previous findings, showing the benefits of NICE scientific advice for medicinal technologies. Products that received scientific advice had a shorter time from marketing authorisation to the publication of the NICE TA or HST guidance.
P54: An Insight Into Real-World Evidence (RWE) As a Component of Submission to NICE & CADTH in the Last 3 Years
10:15AM - 10:30AM
Heinz S1, Kumari C2, Ataide J2, Bourakkadi M2
1Ipsos GmbH, London, LON, UK, 2Ipsos UK, London, London, UK
OBJECTIVES: The use of RWE as a component of Health Technology Assessment (HTA) submission has seen a marked increase in recent years. This study aims to understand this increasing trend of RWE submission to NICE and CADTH in the last 3 years as well as the impact of recent RWE guidance published by these HTA agencies.
METHODS: A search was conducted on NICE and CADTH websites for HTAs published between January 2021 and December 2023. After excluding non-relevant HTAs, selected appraisals with RWE were further analysed for study type, data sources, and their type of use in the submission e.g. utility cost, and efficacy.
RESULTS: A total of 539 and 256 appraisals, were identified on NICE and CADTH websites, respectively. Among included appraisals, around 60% in the UK and 37% in Canada included RWE. Most RWE studies in both countries were observational, primarily utilizing retrospective cohorts, and the predominant sources of data were patient & disease registries, or electronic health records. Overall, the most common use of RWE included in the submissions was related to efficacy, patient characteristics and healthcare resource utilization. Of all the CADTH and NICE appraisals incorporating RWE, around 50% were in oncology. A trend of increasing RWE submissions was observed in both countries, rising from 51% in 2021 to 66% in 2023 in the UK and from 24% to 47% in Canada.
CONCLUSIONS: NICE and CADTH lean towards increased importance and thoroughness given to RWE in the context of the HTA processes as shown by this analysis and recent guidelines. Nonetheless, HTA in general still lacks clarity about which type of RWE study and data sources can be used and when. There is great potential to drive mutual understanding of RWE’s impact further, establish acceptance thresholds, and ensure transparency in decision-making related to RWE
Research on Health Inequities
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A series of research studies focusing on equity and neglected populations, including innovation in methodological approaches on how to measure health inequality.
Moderator
Iñaki Imaz-Iglesia, MD, PhD, MPH
Institute of Health “Carlos III” (AETS-ISCIII), Madrid, M, Spain
Scientific Researcher and Director of the Spanish National School of Public Health (ENS) of the Institute for Health Carlos III (ISCIII) in Madrid, Spain. He is Medical Degree and Specialist in Medicine Preventive and Public Health. PhD on Public Health in Zaragoza University (Spain) with a work about the use of real world evidence in HTA in Spain. He has led and collaborated in many research projects and scientific publications, at national and international level, about Health Technology Assessment, Economic Evaluation and Health Services Research.
P59: Measuring Health Inequality Aversion in Canada: An Equity-Efficiency Trade-Off Experiment
10:30AM - 10:45AM
Iragorri N1, Ali S2, Mishra S3, Sander B4
1University of Toronto, Toronto, ON, Canada, 2Western University, London, ON, Canada, 3Unity Health, Toronto, ON, Canada, 4University Health Network, Toronto, ON, Canada
OBJECTIVES: To estimate the extent to which Canadians are averse to health inequalities, a critical component for equity-informative economic evaluations but lacking in the Canadian context.
METHODS: We conducted three experiments among representative samples of adult Canadians to elicit value judgements about reducing socioeconomic-related health inequality (i.e. the gap in healthy life expectancy between the highest and lowest household income quintiles) vs. improving overall population health. Each experiment compared two programs: (i) universal and tailored vaccination; (ii) non-specific prevention programs (universal prevention vs. tailored prevention interventions); (iii) generic health care programs (program A vs. program B). Tailored programs and program B had a more equitable distribution of additional life years, while universal programs and program A were more efficient. We used benefit trade-off analysis to estimate the Atkinson health inequality aversion index.
RESULTS: We recruited 3,000 adult Canadians (1,000/experiment). Preferences for the vaccination, prevention, and generic interventions were distributed as follows: minimizing inequalities (i.e., egalitarians; Atkinson Index undefined): 54%, 55%, and 57%, respectively; maximizing the health of the population with the highest income (i.e., pro rich; Atkinson Index<0): 31%, 22%, and 16% respectively; willingness to trade some health to reduce inequalities (i.e., weighted prioritarians; Atkinson Index>0): 13%, 19%, and 22% respectively; improving the health of the individuals with the lowest income (i.e., maximins; Atkinson Index=∞): 0%, 1%, and 3%, respectively; and maximizing total health (i.e., health maximizers; Atkinson Index=0): 2%, 3%, and 2%, respectively. The median response reflected a preference for minimizing health inequalities across the three experiments. A stronger aversion to health inequality was observed among females, younger respondents (18-40 years old), and respondents with lower income (<$50,000 household income per year).
CONCLUSIONS: Our findings suggest a strong aversion to health inequality among Canadians with over half of respondents consistently willing to minimize health inequalities regardless of the cost to efficiency.
P58: Consideration of Asylum Seekers, Refugees, and Recent Migrants When Evaluating Health Equity: Towards Leaving No One behind
10:15AM - 10:30AM
Chepynoga K1, Gregg M2, Vanderpuye-Orgle J3
1Parexel International, Hørsholm, 85, Denmark, 2Parexel, Austin, TX, USA, 3Parexel International, Billerica, MA, USA
OBJECTIVES: Health equity (HE) ensures equitable healthcare access for all, regardless of socio-economic status, identity, or background, and is a key WHO global health priority. Asylum seekers, refugees, and recent migrants in the European Union (EU) are often excluded from HE evaluations due to their temporary residence status, though many settle locally. This study examines inclusion of asylum seekers, refugees, and recent migrants hosted by the EU in data collection used for HE evaluation.
METHODS: Measuring health disparities among population subgroups is a standard approach for assessing HE. Characteristics such as socio-economic status and ethnicity are used to classify populations into subgroups, while unmet health needs is a metric measuring access to healthcare across defined subgroups. Data is often collected through household surveys that track expenditures on goods and services (including healthcare).
Methodologies and questionnaires for four surveys (household budget survey, EU statistics on income and living conditions survey, EU labor force survey, and European health interview survey) were reviewed to identify inclusion of asylum seekers, refugees, and recent migrants. Asylum seekers and refugees’ headcount was defined from Agency for Asylum and European Commission while unmet health needs data was collected from published literature.
RESULTS: Despite hosting 1.1 million asylum seekers and 7.1 million refugees in the EU in 2023, all surveys excluded these populations in national statistics on expenditures and healthcare services consumption. Primary reason for exclusion was an anticipated non-response rate due to linguistic barriers and misunderstanding of survey objectives. Only migrants with longer-term residence were included whose unmet health needs in Germany, Greece, Spain, and France in 2019 was 11.6%, while all residents in these countries experienced unmet needs ranging from 0.2% (Spain) to 8.1% (Greece).
CONCLUSIONS: Exclusion of vulnerable populations when measuring access to health may result in underestimation of health equity metrics which may be misleading for policy makers.
P60: Socioeconomic Burden of Major Diseases in Eight Latin American Countries
10:45AM - 11:00AM
Hernandez-Villafuerte K1, Müller M2, Ostwald D1
1WifOR Institute, Darmstadt, HE, Germany, 2WifOR, Darmstadt, HE, Germany
OBJECTIVES: Health investments impact labor supply by affecting workforce health and capabilities. We introduce a novel methodology combining value chain effects and health economics to show how health investments drive economic development. Using the concept of human capital— abilities, skills, knowledge, and motivation—we estimate the monetary value of the socioeconomic (SoC) burden of disease. It reflects how disease reduces labor supply by impairing individuals' capital utilization. The method is applied to the Brazilian case for cardiovascular, neoplasms, ischemic heart disease, lower respiratory infections, breast cancer, type 2 diabetes, and migraine.
METHODS: We assumed that years lost due to disability or mortality are non-productive, where Years Lived with Disability (YLDs) captures presenteeism and absenteeism, and Years of Life Lost (YLLs) premature mortality. Two approaches are considered: the Human Capital Approach (HCA), which assumes an irreplaceable loss of productivity upon premature death, and the Friction Cost Approach (FCA), which assumes that within a year, the deceased's tasks are redistributed, absorbed, or adapted within the production process. Moreover, using Input-Output matrices, we estimate the direct economic effects and calculate spillover effects in adjacent economic sectors and induced effects along value chains. Additionally, we addressed both paid and unpaid work.
RESULTS: In 2022, Brazil's GDP loss due to diseases was 4.1% ($77.1 billion) under the FCA and 7.1% ($135.9 billion) under the HCA. Migraines and type 2 diabetes were the top contributors under the FCA. Under the HCA, cardiovascular disease and migraines have the highest burden.
CONCLUSIONS: Decreases in labor supply not only hinder economic growth but also exacerbate inequalities and adversely affect vulnerable populations, as seen in Brazil. Key questions arise: What is the socioeconomic value of investing in health? Which healthcare strategies promote economic development? By measuring the SoC burden, we bring light to these questions and help policymakers prioritize budget allocations.
P57: Quantifying Inequalities Across the Breast Cancer Pathway and Their Impact on Health Outcomes in England
10:00AM - 10:15AM
Russell R1, Ward A1, Taylor K1, Sloan R2, Bray BD3, Giles C4, Tsang C1, Clifton-Brown E1
1Pfizer Ltd, Tadworth, UK, 2Lane Clark & Peacock LLP, London, UK, 3LCP Health Analytics, London, UK, 4Shine Cancer Support, Poole, UK
OBJECTIVES: Reducing health inequalities is a policy priority for the National Health Service (NHS) in England. Breast cancer is the most common cancer in women in the UK; despite premature mortality rates declining, inequalities in care remain. The aim of this study was to model the potential impact of levelling up inequalities in the breast cancer care pathway for women in England.
METHODS: A targeted literature review and analysis of publicly available NHS data sources (2016-2022) were used to describe variation in the breast cancer pathway for women in England by socioeconomic deprivation, geography, and ethnicity. The potential additional life years (PALYs) saved if variations in stage at diagnosis were addressed was modelled using a parametric survival model to extrapolate NHS 5-year survival data.
RESULTS: Women living in the most deprived communities, and black women, experience significant inequalities across the pathway. Diagnosis via screening across socioeconomic groups and ethnicity was lowest for black women (23%) and women living in the most deprived communities (29%) compared to white women (34%) and women in the least deprived communities (36%). 22% of black women were diagnosed with late-stage cancer compared to 14% of white women. Women in more deprived communities had the highest incidence of late-stage cancer compared to women in less deprived communities. Levelling up inequalities in stage at diagnosis could result in black women in England gaining 6 additional months of life, and women in the most socioeconomically deprived communities gaining an increase of 3 months.
CONCLUSIONS: Addressing inequalities in the diagnosis and treatment pathway could improve survival outcomes for women with breast cancer in England. There are opportunities for health systems and stakeholders to work collaboratively to uncover the root causes of breast cancer inequalities to identify solutions to tackle these.
11:00 - 11:30
Break
11:30 - 12:45
Plenary Session
ISPOR CEO and CSO Updates
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Welcome & CEO's UpdatesRob Abbott, CEO & Executive Director
ISPOR - The Professional Society for Health Economics and Outcomes Research
Science & Health Policy Updates
Laura Pizzi, Chief Science Officer
ISPOR - The Professional Society for Health Economics and Outcomes Research
Speaker
Rob Abbott
ISPOR, Lawrenceville, NJ, USA
Mr Abbott is a highly accomplished, visionary leader with more than 20 years’ experience providing executive-level strategic and operational leadership in businesses, nongovernmental organizations (NGOs), government environments, as well as professional societies and associations. Most recently, he served as executive director and CEO of Health Technology Assessment International, a global nonprofit society focused on health technology assessment (HTA). He is recognized for his knowledge in HTA, strategy, and stakeholder relations. He has successfully guided a number of organizations through their transformation and growth. He is also a pioneering social responsibility thinker and advisor who has authored 2 books and numerous articles on the nature of business and society. Mr Abbott holds an undergraduate honors degree from the University of Victoria and graduate degrees from the University of Toronto. Additionally, he holds professional designations as both a management consultant and an ICF-accredited coach.
Laura Pizzi, PharmD, MPH
ISPOR, Lawrence Township, NJ, USA
Dr. Laura Pizzi is Chief Science Officer for ISPOR and Research Professor at Rutgers University. For nearly 25 years, she has led interdisciplinary teams of methodologists, statisticians, and clinicians to design and conduct economic and outcomes analyses on healthcare interventions and is a frequent author, speaker, and mentor on the topic.
At ISPOR, she leads the organization’s scientific strategy and initiatives in collaboration with ISPOR's global members and stakeholders.
Reality Check: Are We Bridging the Evidence Gaps for Patients?
Digital Conference Pass
Welcome & CEO's UpdatesRob Abbott, CEO & Executive DirectorISPOR - The Professional Society for Health Economics and Outcomes Research
Science & Health Policy Updates
Laura Pizzi, Chief Science Officer
ISPOR - The Professional Society for Health Economics and Outcomes Research
Plenary Address
This plenary session will take place after the two others that will address first the payers’ perspective and then the HTA’s perspective. This last session will focus on the regulatory step and discuss how the needs from all the stakeholders could be better anticipated, considering the discussions that would have taken place during the two other sessions.
We, regulators, HTA agencies, payers and pharmaceutical industry, are all working closely with patients to ensure that innovative drugs can benefit patients as early as possible. This collaboration is guided by the ambition to enable and use the generation of reliable and valid evidence that will address and answer different questions, e.g. for benefit/risk assessment, for relative effectiveness assessment... We are relying on the totality of evidence generated, either from randomized controlled trials, or from other designs such as studies using real-world data. Even if differences in scope may exist, there are overlapping commonalities in what constitutes meaningful evidence generation. We also must deal with (remaining) uncertainties at different stages of decision making. How can we limit these uncertainties, and ensure that at the end of the overall approval process, all stakeholders get the needed evidence to take an informed decision and ensure an innovative drug reach the patients if appropriate?
Moderators
Patrice Verpillat, MD, MPH, PhD
European Medicines Agency (EMA), Amsterdam, Netherlands
Dr. Patrice Verpillat (MD, MPH, PhD) is the Head of the Real World Evidence (RWE) Workstream at the European Medicines Agency (EMA). He is a medical doctor, specialist in epidemiology. Before joining the EMA, he has worked during 20 years in the pharmaceutical industry where he had positions in several international companies, always dealing with real world data (RWD) and non-interventional studies (NIS) in order to bring RWE into research, access and life-cycle product management.
Dr. Verpillat has published over 70 articles in Medline referenced journals. He has been involved in many organisations such as ENCePP, ICH M14 working group, European pharma association (efpia) and ISPE
Speakers
Nikos Dedes, BA
Greek Patients' Association, Athens, Attika, Greece
Peter Mol, PhD
College ter Beoordeling van Geneesmiddelen, Utrecht, Netherlands
Peter Mol is professor of drug regulatory science at the University Medical Center Groningen. His research focus is on developing new tools to support regulatory decision-making and the exchange of knowledge between regulatory authorities, health care professionals and lay people. He is currently involved in projects around personalized medicine, new data sources (RWE), patient-centric ways to weigh drug benefit-risk; e.g., using Patient Relevant Outcomes, Quality of Life, and Patient Preference information, and on risk communication (Direct Healthcare Provider Communication). He is Principal Investigator of the HORIZON More-EUROPA project (More-Europa - Research (umcgresearch.org)).
He is also the Committee for Medicinal Products for Human Use (CHMP) member for the Dutch Medicines Evaluation Board. He was from 2012 to 2023 member (vice chair 2016-2022) of EMA’s Scientific Advice Working Party. He has coordinated over 300 EMA and national scientific advice procedures for drug development programs for cardiometabolic, gynecology and hematology products. He was chair of the EMA Cross-Committee Task force on Registries (2016-2023).
Yannis Natsis, MA, BA
European Social Insurance Platform (ESIP), Brussels, Belgium
Yannis Natsis is the Director of the European Social Insurance Platform (ESIP), the umbrella organisation bringing together 45 national statutory social security institutions from 18 countries. ESIP is the voice of social protection and security in Europe or as Yannis puts it one of Europe’s truest treasures.
He has more than 10 years of experience in EU advocacy and policymaking. Prior to joining ESIP in February 2022, he led the advocacy for better and affordable medicines at the European Public Health Alliance (EPHA). In May 2019, he was appointed by EU Member States to the Management Board of the European Medicines Agency (EMA), a position he held until December 2021. Additionally, Yannis has been a Board member of the European Health Forum Gastein (EHFG), the leading EU health policy platform since 2018.
Yannis previously worked for the TransAtlantic Consumer Dialogue (TACD) focusing on health and pharmaceutical policies. From 2006-2010, he was an investigative reporter for Greece's award-winning TV news programme "Fakeli" and a contributor to one of Greece’s most respected dailies “Kathimerini”.
He has a Master's degree in International Conflict Analysis from the University of Kent, UK and a Bachelor's degree in European Studies from Pantion University of Social and Political Sciences, Athens, Greece. A Greek national, he is fluent in Greek, English and French.
Almath Spooner, .
Abbvie, Dublin, Ireland
Anne Willemsen, MSc
Dutch National Healthcare Institute, Diemen, Netherlands
Anne Willemsen holds double Masters Degrees in Health Sciences, specialising in both Health Technology Assessment and Health Policy. This led her through research and project management roles within the Dutch National Healthcare Institute (Zorginstituut Nederland) over the past years. Between 2016 and 2023 she has been working for EUnetHTA and has been coordinating Joint Clinical Assessments (JCA). Since April 2023, she was elected as the co-chair of the HTA Coordination Group JCA subgroup under the HTA Regulation.
