Program

* Program subject to change

Sun 17 Nov7:00 - 16:20Registration Hours12:30 - 12:35Welcome RemarksSpeakers:

Eberechukwu Onukwugha, PhD, University of Maryland School of Pharmacy, Baltimore, MD, USA

Shirley Wang, PhD, Brigham & Women's Hospital, Harvard Medical School, Boston, MA, USA

12:35 - 13:35Session 1Unleashing the Latent Power of Real-World Evidence in Decision-MakingLevel: Introductory

In recent years, there has been a proliferation of guidance, demonstration projects, and use cases involving the use of real-world evidence (RWE) to inform regulatory, payer, and health technology assessment (HTA) decisions, but what have we learned? This session will provide a balanced overview of recent successes and challenges for HTA Experts will showcase success stories where RWE has been pivotal in assessing value for reimbursement decisions. This will be followed by an exploration of ongoing challenges, including transportability of evidence across jurisdictions, data quality concerns, and methodological hurdles that limited the use of RWE in HTA decision-making.

Moderators

Shirley Wang, PhD

Brigham & Women's Hospital, Harvard Medical School, Boston, MA, USA

Dr. Wang is an associate professor at Brigham and Women’s Hospital, Harvard Medical School, lead epidemiologist for the Food and Drug Administration’s (FDA) Sentinel Innovation Center, and member of the PCORI Methodology Committee. She leads the Meta-Research in Pharmacoepidemiology program, with recent projects aimed at improving the transparency, reproducibility and robustness of evidence from healthcare databases (www.repeatinitiative.org) and informing when and how real-world evidence studies can draw causal conclusions to inform regulatory or other healthcare decision-making (www.rctduplicate.org). She is currently PI on multiple NIH R01s and is also funded by FDA. Her methods work has received multiple awards from international societies.

Speakers

Ashley Jaksa, MPH

Aetion, Inc, Boston, MA, USA

Ashley Jaksa is VP, Scientific Strategy & Partnerships at Aetion. She focuses on collaborating with decision-makers to enhance their use of RWE and setting methodological guidance for RWE generation. She also works with Aetion’s biopharma clients on incorporating RWE in their global market access strategy. She has presented her research at numerous international conferences including ISPOR, HTAi, and AMCP, and published her findings in various peer-reviewed publications. She holds a bachelor's degree from the University of Michigan-Ann Arbor and a MPH from Yale University.

Maria Kamusheva, PhD

Sofia Medical University, Sofia, Bulgaria

Maria Stefanova Kamusheva holds a master’s degree in Pharmacy from the Faculty of Pharmacy, Medical University-Sofia in 2011. In 2014, she defended her Ph.D. thesis “Pharmacoeconomics of the Treatment of Rare Diseases”. Since 2014, she started working at the department “Organization and economics of pharmacy” in Faculty of Pharmacy at Medical University of Sofia. She has 2 nonclinical specialties: “Organization and economics of pharmacy and wholesaling practice” and “Hospital pharmacy”. She is conducting Practicals and Seminars, supervision of master in the mandatory and free eligible subjects taught in the department: Social pharmacy and pharmaceutical legislation, Pharmacoeconomics, Pharmaceutical care, Medical devices, Pharmaceutical business and marketing, Biostatistics in Bulgarian and English language etc. The scientific and research work of Maria Kamusheva is focused on several areas of modern pharmaceutical science and practice, such as Pharmacoeconomics and Pharmaceutical legislation. Author and co-author of 78 scientific papers, 4 textbooks, 434 citations in the peer reviewed and indexed journals in Scopus (h index=12). Expert in the field of pharmacoeconomics and pharmaceutical legislation. Leading scientist and participant in 10 scientific projects, funded by Medical university of Sofia, European Union’s Horizon 2020; COST (European Cooperation in Science and Technology; European Commission ERASMUS+. She is a reviewer for scientific journals such as Frontiers in Public Health, International Journal of Clinical Pharmacy, European Journal of Hospital Pharmacy, and Pharmacia Prof. Kamusheva is a member of ISPOR, Bulgarian Pharmaceutical Union, Bulgarian Pharmaceutical Science Society, and Bulgarian Association of Drug Information. She is a member of expert groups assisting the National Council on Prices and Reimbursement of Medicinal Products and National Evaluation and Accreditation Agency in Bulgaria.

Steve Williamson, MSc

National Institute for Health and Care Excellence (NICE), United Kingdom

Steve Williamson is NICE’s Associate Director for Managed Access which includes the Cancer Drugs Fund and Innovative Medicines Fund. Steve’s Team is responsible for all aspects of the development, planning and delivery of the Managed Access, working collaboratively with NHS England colleagues. Steve is a key opinion leader on NICE’s activities relating to managed access and evidence generation. Steve is a pharmacist whose background includes oncology and a payer role as well as a long-standing involvement in HTA implementation of cancer medicines from a system perspective. Before joining NICE Steve was England’s National Lead Cancer pharmacist for 5 years having moved from Consultant Cancer Pharmacist role in the North East of England. Steve is a past Chair of the British Oncology Pharmacy Association authoring many of their Guidelines and National Standards. Steve teaches on the Oncology MSc degree course at Newcastle University and has published and undertaken research on various topics on cancer medicines and chemotherapy service delivery.

13:35 - 13:50Networking Break13:50 - 14:50Session 2Methods for Causal Inference Using Real-World DataLevel: Introductory

For the use of RWE in HTA and health policy decision making, it is crucial to use valid estimates of the benefits, harms and costs of the interventions of interest. In this session, methodological approaches and frameworks for causal inference and their applicability in light of data availability and quality are presented and discussed.

Barbra Dickerman will detail the design of the target trial emulation framework and describe applications of the approach to reduce bias (e.g., immortal time bias, time-varying confounding) and to generate actionable evidence to inform decision-making. Nicholas Latimer will present the use of hypothetical estimands in clinical trials when standard intention-to-treat (ITT) analysis does not yield an answer to the decision problem, and relate this issue to the analysis of real-world data and cost-effectiveness. Sebastian Schneeweiss will focus on dealing with differential measurement issues frequently encountered in external control arm (ECA) analyses via calibration and hybrid designs.

The goal of this session is to sensitize all stakeholders to potential biases in the use of RWE and to discuss solutions for providing high-quality RWE for HTA bodies and health policy decision making.

Moderators

Uwe Siebert, MD, MPH, MSc, ScD

UMIT TIROL - University for Health Sciences and Technology, Hall in Tirol, Austria and Harvard Chan School of Public Health Harvard University, Hall in Tirol, 7, Austria

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.

Speakers

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.

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.

Sebastian Schneeweiss, MD, ScD

Brigham and Women’s Hospital, Harvard Medical School, 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.

14:50 - 15:05Networking Break15:05 - 16:05Session 3Embracing Diversity and Tackling Heterogeneity in Data, Methods, and JurisdictionsLevel: Introductory

Real-world data (RWD), gathered from everyday clinical practice creates real-world evidence (RWE) that supports decision-making for regulatory bodies, health technology assessment (HTA) bodies, and payers. To enhance the value of RWD for decision making, diverse data and methods across various jurisdictions are employed for a more accurate outcome assessment. This diversity introduces variability or heterogeneity in study outcomes, posing interpretation. This session delves into the sources of heterogeneity and strategies to address it. Speakers will discuss how differences in RWD and data from different healthcare settings influence variation between estimates and explore designs and statistical methods to assess and minimize this variability.

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

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®)

Chantal Quinten, PhD

European Medicines Agency (EMA), AMSTELVEEN, NH, Netherlands

Chantal Quinten is an expert in health data analytics and real-world data (RWD). She holds an advanced degree in Biostatistics and Biomedical Sciences. In her current role at the European Medicines Agency (EMA), she leads key initiatives to harness real-world data for regulatory decision-making, advancing evidence-based approaches and supporting methodological development. Chantal has also held pivotal roles at the European Centre for Disease Prevention and Control (ECDC) and the European Organisation for Research and Treatment of Cancer (EORTC), where she contributed to enhancing disease surveillance as well as clinical and patient-reported data.

Daniel Rosenberg, PhD

Johnson & Johnson - Innovative Medicine, Basel, Switzerland

Daniel has worked as a pharmacoepidemiologist in the pharmaceutical industry in both large and small companies for over 20 years. Daniel started his career with a Post Doctoral fellowship under the direction of Prof. Dr. Harry Guess in Pharmacoepidemiology at University of North Carolina, followed by nearly a decade at GlaxoSmithKline. Daniel founded the Epidemiology group at Actelion Pharmaceuticals Ltd. and led this group until Johnson and Johnson’s acquisition of Actelion in 2017. In his present position at J&J within the Global Epidemiology department, Daniel leads a Cross Therapy Area Group of different Disease Area Epidemiology Teams and the Rare Disease/Small and Special populations Epicenter team. Daniel continues his work to shape and develop the use of RWE in the regulatory setting including the identification of relevant & reliable, diverse fit-for-use real-world data sources. Daniel serves as the EFPIA lead representative to the ICH M14 Expert Working Group who are developing a harmonized guidance on the use of real-world data in pharmacoepidemiology studies for safety assessment. Currently, Daniel chairs the Rare Disease Special interest group (SIG) within the International Society of Pharmacoepidemiology.

16:05 - 16:20Networking Break16:20 - 17:20Session 4Overcoming Obstacles: Charting the Path AheadLevel: Introductory

During this session, based on the work of RWE4Decisions, new actions for all stakeholder groups will be presented and documented and those for HTA, Industry and Analytics Groups will be discussed, emphasizing the evolving policy and system developments supporting the actions in the EU. François Meyer will facilitate a conversation among the panelists and the audience about the applicability of these actions in their day-to-day work and the implications for JSCs and JCAs in EU HTA. The goal of the session is to provide concrete actions that each stakeholder group can take to improve the accessibility and quality of RWD and improve the use of high-quality RWE studies in HTA decision-making.

RWE4Decisions is a multistakeholder learning network that facilitates stakeholder dialogues over the life cycle of highly innovative medicines. It seeks to identify the real-world evidence needed to inform HTA/Payer decisions, clarifying how RWE will be assessed and supporting effective implementation of Post-Launch Evidence Generation. RWE4Decisions used a mixed methods approach involving HTA/Payers, industry, clinicians, patient groups, analytics experts and registry holders in the EU and Canada over the past year to develop a set of actions to build better RWE for HTA (updating actions from a previous 2020 publication.)

Moderators

Francois Meyer, MD

FIPRA, Paris, 75, 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.

Speakers

Niklas Hedberg, MSc

Dental and Pharmaceuticals Benefits Agency (TLV), Stockholm, AB, 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.

Jana Hlaváčová, MA

European Multiple Sclerosis Platform, Prague, Czech Republic

Jana Hlaváčová is a patient advocate, external policy advisor to the European Multiple Sclerosis Platform and a co-founder of a patient involvement agenda at the Czech Ministry of Health. As a law graduate, living with multiple sclerosis since 2012, she is dedicated to enhancing patient participation in healthcare decision-making, where she specialises in HTA and use of patient experience data. Representing EMSP, she contributes to RWE projects like MS Data Alliance and more-EUROPA. At the Ministry, she was responsible for legally and practically integrating patient input into the reimbursement process for orphan medicinal products as part of evolving patient involvement agenda.

Piia Rannanheimo, MSc

Finnish Medicines Agency (FIMEA), Kuopio, Finland

Piia Rannanheimo, Chief Specialist at the Finnish Medicines Agency's Development and Information Section, has over a decade of experience in health technology assessment. In her current role, she is involved in developing the secondary use of health and social care data in Finland, advancing pharmaceutical product and market data management, and promoting data-driven decision-making for the rational use of medicines. Among other things, Piia is a member of the RWE4Decisions Steering Group.

Conor Teljeur, PhD

Health Information and Quality Authority (HIQA), Dublin, Ireland

Conor has worked in health technology assessment at the Health Information & Quality Authority in Ireland since 2010. He is mainly involved in data analysis in terms of epidemiology and economic evaluation, but has contributed across all domains of HTA. The HTA topics he has worked on include a diverse range of public health interventions. Conor was closely involved in writing a number of national methodological guidelines, as well as international guidelines as part of the EUnetHTA collaboration. Before stumbling into HTA, Conor worked as an academic researcher with an interest in spatial analysis and health inequalities.

Massoud Toussi, MBA, MSc, PhD, MD

Cytel, Paris, France

Massoud Toussi is Vice President, Global Head of Real-World Evidence in Cytel. He has more than 20 years of experience in leading research teams in the design, development and conduct of studies in the field of real-world evidence, pharmacoepidemiology, outcomes research and data science. He is a member of ISPE-ISPOR RWE Steering Committee, past Chair of HTAi Interest group on RWE-AI and co-author of the ENCePP Guide on Methodological Standards. He is the co-author of THE PASS Book, and several methodological and research papers on the conduct of real world studies with a focus on PASS studies. Massoud is a medical doctor (M.D) and holds a PhD in medical informatics from the University of Sorbonne Paris Nord, a MBA from The University of Quebec in Montreal, and a MSc on international management form the University of Paris Dauphine.

17:20 - 17:30Closing RemarksSpeaker:

Massoud Toussi, MBA, MSc, PhD, MD, Cytel, Paris, France

17:30 - 18:30Networking Reception

ISPOR Real-World Evidence Summit 2024

17 November

Program

ISPOR–The Professional Society for
Health Economics and Outcomes Research