Mon May 17
10:00 AM - 4:00 PM
Poster & Exhibit Viewing
Live
Stop in to learn about the latest technology, services, and devices and to connect one-on-one with exhibitors and sponsors during our Exhibit Viewing Hours. Be sure to reserve some time to view the latest research in HEOR through our virtual poster gallery. Browse, comment, and discuss our posters at any time while our virtual platform is active.
11:45 AM - 12:45 PM
Breakout Session 1
COVID-19 Vaccines: How to Overcome Hesitancy and Inequities with People at the Center
Live
ISSUE:
On December 11, the USFDA issued emergency use authorization for the first COVID-19 vaccine. While the vaccine is now launched and the first shots already given, not everyone will get access at once. Moreover, as access and availability open up, it will be increasingly critical to overcome vaccine hesitancy overall, especially among high-risk populations. Historically there has been high vaccine hesitancy among minority populations. In a Pew Research Center survey conducted in 2020, only 54% of Black adults said they definitely or probably would get a COVID-19 vaccine compared with 74% of White and Hispanic adults. In addition, an underrepresentation of minority groups in COVID-19 clinical trials presents a challenge, when the disease’s disproportionate impact on minority communities is widely documented.
OVERVIEW:
This panel will explore the link between COVID-19 vaccine education and the broader vaccine hesitancy and health equity goals. Susan Garfield will be monitoring the panel and will give an overview of vaccine hesitancy and acknowledge the reasons for historical mistrust among racial minorities. The panel will discuss the impact of hesitancy on achieving herd immunity and the importance of the influence of cultural awareness, education, transparency and trusted voices in the community. How can we enable an integrated vaccine advocacy approach to patient engagement, with a focus on at-risk, minority and vulnerable populations? How can we quantify the challenge and use demand planning tools to pinpoint where to focus? Five years into the vaccination experiment – we will discuss the outcomes of various public and private programs to overcome vaccine hesitancy- bringing proprietary data from state and national surveys of sentiment data to inform the conversation. Lastly, the panel will debate and highlight the issues related to return to work and explore the employers role in the health and wellness of their employees.
Moderators
Susan Garfield, DrPH
Ernst & Young, Boston, MA, USA
Panelists
Alethia Jackson, JD
Walgreen Co., Washington, DC, USA
Heidi Steinecker, MA
Ernst & Young, Sacramento, CA, USA
Canada: The New Drugstore for America or Entering the Deep Freeze?
Live
ISSUE:
While many US payers and patient associations support the introduction of a US drug re-importation plan, it has been faced with opposition from drug makers and providers. Jill Crich will provide an overview of the issue and moderate the panel discussion. Andras Incze will deliver a critical view of the evidence surrounding this issue, including how pharmaceutical companies currently manage parallel exports amongst European countries to protect local drug supply and avoid margin loss. Lou Garrison will argue the effect this plan will have on Canadian and US drug manufacturers and prescription medication costs in the US, and whether Canada has the capacity to meet the demands of the US market. John Adams will discuss the impact on patient access to treatment concerning drug supply and pricing, and consider how the interim order will protect Canadian patients. Discussion time will be included, and audience participation encouraged throughout.
OVERVIEW:
In the US, increased prescription drug expenditures have created concern for consumers and have prompted escalated attempts to import drugs from Canada, where prices of prescription medicines can be less than half the price in the US. For over a decade, federal and state lawmakers have advocated for legislation allowing for the importation of prescription drugs, which is only legal under defined, limited circumstances. In September 2020, the Trump Administration proposed two new pathways which would authorize states, territories, and Indian tribes to implement programs for the importation of prescription drugs from Canada. On November 27, 2020, the Canadian government issued an interim order to block the exportation of drugs that are subject to shortage reporting. Will the approval of a US drug re-importation plan lead to a measurable reduction in prescription medication costs for US consumers, and if so, will this lead to drug shortages in Canada?
Moderators
Jill Crich, BSc, MSL
Costello Medical, Boston, MA, USA
Panelists
John Adams, BA
Can PKU and Allied Disorders Inc, Toronto, ON, Canada
Louis P. Garrison, PhD
University of Washington, Seattle, WA, USA
Lou Garrison, PhD, is professor emeritus in The Comparative Health Outcomes, Policy, and Economics Institute in the School of Pharmacy at the University of Washington, where he joined the faculty in 2004.
For the first 13 years of his career, Dr. Garrison worked in non-profit health policy at Battelle and then the Project HOPE Center for Health Affairs, where he was the Director from 1989-1992. Following this, he worked as an economist in the pharmaceutical industry for 12 years. From 2002-2004, he was vice president and head of Health Economics & Strategic Pricing in Roche Pharmaceuticals, based in Basel, Switzerland.
Dr. Garrison received a BA in Economics from Indiana University, and a PhD in Economics from Stanford University. He has more than 150 publications in peer-reviewed journals. His research interests include national and international health policy issues related to personalized medicine, benefit-risk analysis, and other topics, as well as the economic evaluation of pharmaceuticals, diagnostics, and other technologies.
Dr. Garrison was elected as ISPOR President for July 2016-June 2017, following other leadership roles since 2005. He recently co-chaired the ISPOR Special Task Force on US Value Frameworks. He was selected in 2017 by PharmaVOICE as being among “100 of the Most Inspiring People” in the industry. He recently received the PhRMA Foundation and Personalized Medicine Coalition 2018 Value Assessment Challenge First-Prize Award as lead author on a paper on “A Strategy to Support the Efficient Development and Use of Innovations in Personalized and Precision Medicine.”
Andras Incze, PhD, MBA
Baden-Wuerttemberg Cooperative State University Germany & Akceso Advisors AG, Basel, BS, Switzerland
Spotlight Session
COVID-19 Vaccine Access – What Were the Goals, and Are They Being Met?
Live
This Spotlight session will explore the HEOR issues that have arisen in the prioritization and effectuation of access to COVID-19 vaccines. Considerations will include short-term and long-term risk minimization, disparity reduction, behavioral modification, and economic benefit, and HEOR can inform these sometimes competing goals.
Moderators
Maarten J. Postma, PhD
University of Groningen, University Medical Center Groningen, Groningen, Netherlands
Speakers
David Bishai, MD, PhD
Johns Hopkins Bloomberg School of Public Health, Baltimore, MD, USA
Brahim Bookhart, MBA, MPH
Johnson & Johnson, Philadelphia, PA, USA
Ruth Waitzberg
Myers-JDC-Brookdale Institute, Jerusalem, Israel
Ruth Waitzberg is a researcher scholar since 2010 at the Health Policy Team, the Myers-JDC-Brookdale Institute. She is also a researcher at the Department of Healthcare Management at the Technical University of Berlin and is involved in projects with the European Observatory on Health Systems and Policies. Ruth has worked in collaboration with Israeli policymakers and has won competitive grants for independent studies on healthcare policy and economics.
Ruth holds a BA in sociology, anthropology and political science and an MA in public policy, both from the Hebrew University of Jerusalem, and is finalizing her joint PhD at the Technical University of Berlin and Ben Gurion University of the Negev.
Breakout Session 1
What Do You Do with Your Clinical Outcome Assessments (COAs) in a Pandemic?
Live
PURPOSE:
Clinical outcome assessments (COAs) are often used in clinical trials to assess treatment benefit. A face-to-face element is generally involved during COA development or administration. However, the COVID-19 pandemic has created an altered landscape for use of COAs in clinical trials. The purpose of this workshop is to discuss ideas and best practices for the development, validation, and administration of COAs in clinical trials during a pandemic while considering trial participant safety. Participants will gain an overview of the challenges of administering COAs during a pandemic and hear discussion about addressing these challenges.
DESCRIPTION:
A primary challenge with COVID-19 restrictions has been the prevention of in-person data collection, which can be a key part of COA development and administration. Some COAs may be administered virtually with little modification. Other COAs such as certain clinician-reported (ClinRO) and performance outcome (PerfO) measures may be more difficult to administer remotely. These challenges need to be fully considered to ensure that the mode and/or method of administration is suitable and appropriate for the COA and measurement objectives. Changing the administration method during a study also has implications which need to be addressed. Additionally, the potential impact of the pandemic on the concepts being captured requires consideration. Potential issues and proposed solutions will be explored during this workshop, drawing on the presenters’ experiences working on multiple projects during the COVID-19 pandemic in regulatory, industry and consultancy contexts. The implications for future clinical trial design will also be discussed (e.g. remote visits, decentralized trials).
Discussion Leader
Elizabeth (Nicki) Bush, MHS
Eli Lilly and Company, Indianapolis, IN, USA
Elizabeth (Nicki) Bush is the director and global head of the Patient-Focused Outcomes Center of Expertise at Eli Lilly and Company, responsible for the design and implementation of patient-centered measurement strategies to support drug development efforts across Lilly’s portfolio. In this capacity, she and her team advise drug development teams in the selection, modification, and development of clinical outcome assessments (COAs) for use in clinical trials and other research studies, and utilize mixed methods approaches to evaluate the measurement properties of COAs and aide in interpretation of the resultant data.
Nicki has held research and leadership positions in the patient-centered measurement field in both consultancy and industry for over ten years. During this time, she has collaborated with partners in industry, regulatory agencies, and academia and held the position of industry co-director of the PRO Consortium from 2016-2018. Her previous experience includes behavioral research and program evaluation. Ms. Bush earned her BA in the liberal arts (philosophy and history of mathematics and science) from St. John’s College and her master’s degree in epidemiology from the Johns Hopkins Bloomberg School of Public Health.
Selena Daniels, PharmD, MS
US Food and Drug Administration, Silver Spring, MD, USA
Jason Randall, PhD, MSc
Clinical Outcomes Solutions, Folkestone, KEN, United Kingdom
Tara Symonds, PhD
Clinical Outcomes Solutions Ltd, Folkestone, United Kingdom
Tara Symonds is the Clinical Outcomes Assessment (COA) strategic lead & chief science officer at Clinical Outcomes Solutions providing advice on COA strategy from development to dissemination. Tara has more than 25 years of experience in the development and implementation of COAs for use in clinical research. Her areas of interest and expertise are in qualitative research, ePRO/BYOD (Bring Your Own Device), and application of PRO measures in comparative effectiveness research by incorporation into electronic medical records and registries. Many of the questionnaires and screening tools she has worked on, can be accessed at: www.pfizerpatientreportedoutcomes.com. She has worked across numerous therapeutic areas and has extensive expertise in sexual health, women’s health, pain, respiratory and rare diseases. She worked in industry for 16 years, her final assignment was to set-up and lead the PRO Center of Excellence at Pfizer. Tara has much experience in negotiating COA labelling at both FDA and EMA and in formulating best positioning of COA data for maximizing reimbursement with EU and US payers. Tara has also previously worked in academia teaching undergraduate and postgraduate psychology. She has published more than 80 peer reviewed articles and is co-author of the following book: “Cappelleri et al (2014) Patient Reported Outcomes: Measurement, Implementation and Interpretation, CRC Press.” Tara holds a Doctorate in Health Psychology from the University of Huddersfield, UK.
Using Probabilistic Quantitative Bias Analysis to Account for Unmeasured Confounders When Estimating Treatment Effects in Real-World Data.
Live
PURPOSE:
To provide an introduction and applied examples of how probabilistic quantitative bias analysis (QBA) can be used to measure uncertainty in treatment effect estimates derived from real-world data (RWD) in the presence of unmeasured confounding, measurement error and selection bias.
DESCRIPTION:
Analyses of RWD are increasingly being used to support HTA and regulatory submissions. Last year the FDA provided guidance for its use in submissions to the agency. The effective use of RWD to support regulatory or HTA submissions relies on being able to generate unbiased estimates of treatment effects. However, decision makers are often concerned with possible sources of bias in evidence generated from RWD including selection bias, measurement error and confounding. Statistical adjustments abate some of these concerns but the completeness of RWD with respect to variables that would allow for the measurement of bias remains a concern. QBA is a method that allows for the quantitative measurement of selection bias, unmeasured confounding, and measurement error on the direction and magnitude of an estimated treatment effect. The workshop will first provide an overview of the methods underlying the approach (10 minutes). We will then present an application of QBA by describing analyses of the Flatiron Health Analytic Database for treatment of non-small cell lung cancer (20 minutes). Finally, other worked examples will be presented based on common criticism encountered in FDA reviews of evidence from RWD (15 minutes). The workshop will also review approaches for combining QBA with methods to handle missing data, two problems commonly encountered together in RWD. Statisticians, and regulatory or HTA reviewers will benefits from attending this workshop gaining exposure to a technique that will become more commonly applied as access and use of RWD in submissions increases.
Discussion Leader
Paul Arora, PhD
University of Toronto, Toronto, ON, Canada
Alind Gupta, PhD
Cytel, Toronto, ON, Canada
Samantha Wilkinson, PhD
Roche, Welwyn Garden City, United Kingdom
Qualitative and Quantitative Applications of Incorporating the Patient Voice into Value Assessments
Live
PURPOSE:
This workshop will describe recent and novel applications where patients have been engaged to provide qualitative and quantitative data to inform value assessments.
DESCRIPTION:
Value assessment is emerging as a key policy lever for pharmaceutical coverage and reimbursement decision-making in the United States. Value frameworks largely rely on traditional value assessment methods such as comparative effectiveness research (CER), literature-based cost-effectiveness analyses (CEAs), and appraisal committees that deliberate the relative value of pharmaceutical interventions. A common critique of these traditional value assessment methods is that they do not represent the full impact of pharmaceutical interventions on patients. Value assessment developers look to health economists for rigorous and appropriate methods to enhance the traditional approaches. However, health economists have not reached a consensus on how to measure and incorporate additional criteria important to patients and broader society into value assessment applications. This workshop will review recent and novel efforts in patient engagement and the elicitation of patient-centered data to qualitatively and quantitatively inform value assessment. Dr. McQueen will introduce the topic and present recent research from the University of Colorado’s pValue Center on eliciting value criteria from patients with rare diseases. Dr. Oehrlein will provide a background on the National Health Council’s patient engagement experience, including patient group surveys to inform health economic models. Dr. Slejko will present recent research from the University of Maryland’s PAVE Center on disease-agnostic patient-informed value elements among chronic disease patients. Finally, Dr. Whittington will provide an overview of the Institute for Clinical and Economic Review’s growing experience with patient engagement and how patient-centered value criteria may inform health economic models. Each discussant will have 10-15 minutes to address their portion of the workshop. Using ISPOR polling software, audience members will respond to feasibility of approaches presented, along with their own patient engagement experiences for value assessment.
Discussion Leader
Robert McQueen, PhD
University of Colorado Anschutz Medical Campus, Denver, CO, USA
R. Brett McQueen is an Assistant Professor at the University of Colorado (CU) Skaggs School of Pharmacy and Pharmaceutical Sciences, and member in the Center for Pharmaceutical Outcomes Research. His research interests include decision-analytic modeling applications and methodology, applied microeconometrics in health, and novel value assessment methods. Brett has current funding in micro-costing health interventions, evaluating performance-based risk sharing agreements, estimating patient and payer preferences for various pharmaceuticals, and novel value assessment methods. He is the course director for “Pharmaceutical Economics and Policy Analysis” in the Pharmaceutical Outcomes Research PhD program at CU.
Elisabeth Oehrlein, PhD, MS
National Health Council, Washington, DC, USA
Julia F. Slejko, PhD
University of Maryland, Baltimore, MD, USA
Julia F. Slejko, PhD is an Associate Professor of Pharmaceutical Health Services 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. 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 is co-lead of ISPOR’s Women in HEOR initiative and currently Co-Chair Elect of the ISPOR Faculty Advisor Council.
Melanie Whittington, PhD
Institute for Clinical and Economic Review, Boston, MA, USA
12:45 PM - 3:00 PM
Poster Presentations and Exhibitor Meet Ups
Live
Interact with the authors of the latest research discoveries in HEOR through our virtual poster presentations and gallery. In between poster discussions, connect one-on-one with our exhibitors and sponsors. Engage with potential solution, service, and resource providers.
Poster Schedule
1:15 PM - 2:45 PM
Educational Symposium
Real-world data and COVID-19: How will your evidence generation strategy change in a post-pandemic era?
Live
The impacts of the COVID-19 pandemic on the healthcare system are undeniable. Disruptions in healthcare delivery stemming from the prioritization of COVID-19 cases have led to notable repercussions for both preventative and acute care. These shifts in utilization patterns have significant implications for real-world evidence generation, as data collected during the pandemic will require special considerations in order to deal with reactive changes to healthcare provision and accessibility during this period. Further, selected trends in care developed during the pandemic, such as the increased reliance on telehealth and expansion of community based care by large chain pharmacies may be incorporated into long-term care strategies moving forward. Lastly there is the potential to accelerate giving patients control over their health data, a trend that was emerging pre-covid which is a potential game-changer in both the clinical trials and RWE arenas. With the uncertainty that lies ahead, how can life sciences’ professionals continue leveraging real-world data to answer their organization’s most pressing research questions? Join experts from IBM Watson Health and Chiesi USA for a discussion on the impact of a pandemic on healthcare utilization, data collection, and real-world evidence generation. In this session, presenters will discuss:
The impact of the pandemic on overall healthcare utilization/cost trends, and the implication of these changing trends for your research How and when your research approach may need to change Potential longer term impacts of the COVID-19 pandemic, such as learnings related to healthcare accessibility and equity
Sponsor
IBM Watson Health
Moderators
Tina Moen, PharmD
IBM Watson Health, Denver, CO, USA
Speakers
Brenna Brady, PhD
IBM Watson Health, Laurel, MD, USA
Martin Marciniak, RPh, PhD, MPP
Chiesi USA, Inc., Cary, NC, USA
Bill Saunders, MPH, PhD
Chiesi USA, Inc., Cary, USA
Joseph Tkacz, MS
IBM Watson Health, Bethesda, MD, USA
Beyond Cancer: Moving to High-Quality Real-World Data in Chronic Diseases
Despite representing a huge percentage of healthcare spend, chronic conditions receive significantly less focus on real-world data (RWD) development and usage than cancer. This two-part session explores how RWD is being developed and used in areas such as dermatology, rheumatology, cardiovascular and behavioral health. Panelists will explore:
Part 1: The evolution of registries to data networks, standards for quality, advances in machine learning and natural language processing (NLP).
Part 2: Use cases for accelerating research, measuring outcomes, and personalizing care.
Sponsor
OM1
Moderators
Gary Curhan, MD, ScD
OM1, Boston, MA, USA
Speakers
Terrie Livingston, PharmD
EMD Serono, Wayland, MA, USA
Kathryn Starzyk, ScM
OM1, Boston, MA, USA
Marta Van Beek, MD, MPH
University of Iowa Hospitals and Clinics, Iowa City, IA, USA
3:00 PM - 4:00 PM
Social Hour
Live
Hosted by ISPOR 2021 Co-Chairs J. Jaime Caro, PhD, MDCM, FRCPC, FACP, John Hernandez, PhD, and Petra Wilson, PhD, meet and chat with your fellow peers in HEOR and ISPOR leaders during our nightly social hours. Discuss the sessions of the day, what you are looking forward to for the rest of the week, and the future of HEOR with our Co-Chairs. Attendance is limited, so be sure to join early to ensure you have a spot in our Social Hours!
Student Network Session
Student Network Roundtable Event
The ISPOR Student Roundtable Event offers opportunities for student attendees to hear from experts on a wide array of subject manner ranging from career advice to scientific topics. When the event was in person, the event format was to have 4 presenters each discussing a different topic with attendees rotating from one table to the next. In the virtual format, we would like to explore accomplishing "rotating" between tables by having Breakout rooms set up where attendees will be moved from one topic to the next after 10 minutes. At the completion of having attendees visit all 4 speakers there will be 10 minutes at the end of the event for open discussion for all speakers.
Speaker
Dalia Dawoud, PhD
National Institute for Health and Care Excellence, London, LON, United Kingdom
Dalia Dawoud, PhD, is Senior Scientific Adviser at the National Institute for Health and Care Excellence (NICE). She holds MSc in Economic Evaluation in Health Care from City University London and PhD in pharmaceutical policy and economics from King’s College London.
She has long experience in using economic evaluation in clinical guidelines development and health technology assessment (HTA), gained through working on NICE Clinical Guidelines as well as technology appraisals. Dalia’s research interests are focused on the advanced methods of evidence synthesis and use in economic models and the use of real-world evidence to inform drug development and health care decision making. Dalia currently has overall responsibility of overseeing the delivery of NICE allocated tasks on a portfolio of IMI and Horizon 2020 funded research projects including EHDEN and HTx. She is widely published in the field of pharmaceutical policy and pharmacoeconomics. She also serves as Associate Editor for ISPOR journal Value in Health and as Associate Editor for Pharmacoeconomics and Outcomes Research for Elsevier’s journal Research in Social and Administrative Pharmacy. Dalia also holds adjunct position as Associate Professor at the Faculty of Pharmacy, Cairo University.
Victoria Divino, BA
IQVIA, Arlington, VA, USA
Christopher St. Clair, PharmD
FDA, Silver Spring, MD, USA
Zafar Zafari, PhD
University of Maryland School of Pharmacy, Baltimore, MD, USA
8:00 PM - 9:00 PM
Pre-Released Session
Value Assessment Gives Us Estimates – but Is It Answering the Right Questions?
Live
ISSUE: In the US, value assessment is rapidly becoming a cornerstone of decision making within contracting and payment negotiation, even as debate continues about whether we have the right tools and methods to support the increasing demand for quantifiable estimates of value. These value assessments give us numbers – but are they answering all of the right questions? And if not, what additional elements and frameworks are needed?
OVERVIEW: This panel will explore the current capabilities and limitations of value assessment methods, consider the most important unmet needs in discerning high and low-value care, and debate the best way forward to answering questions that matter to all stakeholders. Samuel Nussbaum will moderate and provide insight on payers’ needs when making value-driven decisions. Steven Pearson will provide the value assessor’s perspective and describe current real-world use. Annie Kennedy will provide the patient community perspective on the alignment between value assessments and value delivered to patients. Bruce Sherman will provide insights from the perspective of the employer community. Panelists will provide brief remarks, reserving the majority of the session for panel discussion and audience questions.
Moderators
Samuel Nussbaum, MD
Innovation and Value Initiative, Clayton, MO, USA
Panelists
Annie Kennedy, BS
EveryLife Foundation for Rare Diseases, Washington, DC, USA
Steven Pearson, MD, MSc
Institute for Clinical and Economic Review, Boston, MA, USA
Bruce Sherman, MD, FCCP, FACOEM
North Carolina Business Group on Health, Greensboro, NC, USA
Risks and Rewards of Tokenization: Clinical Trial Data Meets the Real World
ISSUE
:
The efficacy of new drugs is usually established by clinical trials (CTs) that are conducted with a finite follow-up period At the end of the CT, patients are often no longer followed Data tokenization allows for patients in a CT to be followed using RWD, which in turn will facilitate accurate understanding of post-clinical trial treatment patterns and healthcare resource utilization, as well as providing information on long-term effectiveness and safety These benefits would be specifically of importance when: It may not be feasible or ethical to run large and lengthy CTs (as often occurs in oncology and rare disease) A situation warrants urgency, such as during the COVID-19 pandemic At the same time, merging data from various sources in a secure manner is challenging due to: Lack of interoperability among existing healthcare systems Concerns regarding privacy and potential vulnerability of patients’ personal and health-related information. An urgent need therefore exists to explore this topic from the perspectives of relevant stakeholders OVERVIEW
:
The panel will discuss the opportunities and challenges associated with combining the data of patients enrolled in the clinical trial with real-world data sources.
Moderators
Arnaub Chatterjee, MHA, MPA
Acorn AI at Medidata, a Dassault Systèmes company, Boston, MA, USA
Panelists
John Concato, MD, MS, MPH, BEng
FDA, Silver Spring, MD, USA
Najat Khan, PhD
Johnson & Johnson, New York, NY, USA
Andrew Kress, BA
HealthVerity, Philadelphia, PA, USA
Recruitment for Stated Preference Surveys. Do Repsondents with Self-Reported Versus Physician-Confirmed Diagnoses YIELD Similar Results?
Live
ISSUE: Patients with a physician-confirmed diagnosis are the ideal respondents for stated-preference research in healthcare/regulatory evaluation. However, there are logistical challenges associated with recruiting patients from clinical sites. Recruitment of patients with a physician-confirmed diagnosis may be associated with longer study duration and greater cost. It has been suggested that national panels of patients provide direct access to willing patient participants which can translate to lower expenses and shorter study timeframes. It is not clear if preference results elicited from a self-reporting cohort are similar to preferences from patients recruited directly from the clinic setting. Are panels a suitable substitute for clinical enrollment to elicit preferences? The answer to this methodological question would potentially lead to greater efficiencies and may encourage the conduct of health preference studies to inform regulatory decisions.
OVERVIEW: This panel will debate the pros and cons of a utilizing a self - reporting cohort to elicit patient preferences. Dr. Tarver will moderate the panel. She will pose questions for the panelists to discuss. Some questions are: Do preference surveys administered to respondents via panels and clinically diagnosed patients deliver similar results? Are samples similar in terms of demographics, disease severity, health literacy, numeracy, and other factors? Does this influence the results? How do vendors who manage panels request information on members’ medical conditions? What are recruitment rates for panel-derived vs. physician-recruited samples? What’s the recruitment duration difference (panel vs. physician diagnosed)? What measures can be taken to optimize the reliability of a self-reported diagnosis? Dr. Reed will discuss methodological considerations in comparing preferences between samples. Olufemi Babalola will present an example from the CDRH that compares preference results from a panel (self-reported) to a clinically diagnosed cohort in the case of fibroids. Lisa Kaiser will highlight best practices around the recruitment into and management of their panel.
Moderators
Michelle Tarver, MD, PhD
U.S. Food and Drug Administration, Silver Spring, MD, USA
Panelists
Olufemi Babalola, MHS, MSc
Food and Drug Administration, Silver Spring, MD, USA
Lisa Kaiser, BS
Dynata Healthcare Solutions, Furlong, PA, USA
Shelby Reed, PhD, RPh
Duke Clinical Research Institute, Durham, NC, USA
Shelby D. Reed, PhD, RPh, is professor in the Departments of Population Health Sciences and Medicine and the Duke-Margolis Center for Health Policy at Duke University. Dr. Reed has 20 years of experience in economic evaluation, health services research, and health policy. Dr. Reed has extensive expertise in designing and conducting trial-based and model-based cost-effectiveness analyses of medical diagnostics and interventions in numerous therapeutic areas. She was a member of the ISPOR Task Forces that published recommendations for Good Research Practices for Cost-Effectiveness Analysis Alongside Clinical Trials in 2005 and 2015. She has led a wide range of economic and epidemiological studies using secondary data from healthcare claims, clinical trials, surveys, and disease registries. In her evaluations of health policy issues, she has developed computer models to analyze the potential economic impact of trends in clinical trial design, changes in reimbursement policies, financial incentives and the regulatory process in the development of orphan drugs, and the societal value of alternative approaches to identifying drug safety problems. Dr. Reed currently leads the Center for Informing Health Decision at the Duke Clinical Research Institute. Dr. Reed received her pharmacy and doctoral degrees from the School of Pharmacy at the University of Maryland and completed her training at the University of Washington.
Improving the Odds of a Successful Contract Research Project – Results from a Sponsor-Vendor Collaboration
ISSUE
: The pharmaceutical industry funds thousands of research projects each year. Most projects run smoothly but problems do arise. This panel will identify a set of common issues and recommend some best practices for successful engagements.
OVERVIEW
: In 2020, the ISPOR Institutional Council (IC) undertook a study to better understand sponsor-vendor interactions. A qualitative survey of IC members was conducted, results of which were utilized to develop a quantitative survey questionnaire. The questionnaire was completed by 158 ISPOR members who were either sponsors or vendors and involved in contract research. Four major issues were identified: 1) managing changes to scope and time, 2) achieving the quality expected for the project, 3) prompt/ timely reply as issues arise, and 4) stability of staffing/leadership (and managing turnover).OVERVIEW A working group and then the full IC identified project management strategies that could minimize the potential impact of each of these issues. This issue panel will present the result of this project and solicit comments and suggestions to further enhance the probability of successful sponsored HEOR projects.
Moderators
Bill Marder, PhD
IBM Watson Health, Winchester, MA, USA
Panelists
Ami Buikema, MPH
Optum, Eden Prairie, MN, USA
Khalid Kamal, MPharm, PhD
West Virginia University, Morgantown, WV, USA
Khalid M. Kamal, M. Pharm., Ph.D. is a Professor and Chair of the Department of Pharmaceutical Systems and Policy at West Virginia University School of Pharmacy, Morgantown, WV. Dr. Kamal's primary research and teaching interests have been pharmacoeconomics, patient-reported outcomes research, research methods, and improving quality of care using real-world data sources such as electronic medical records and specialty pharmacy data. He has been a Visiting Professor teaching pharmacoeconomics and decision modeling courses at institutions such as Kobe Gakuin University in Kobe, Japan and King Saud University in Riyadh, Saudi Arabia. Dr. Kamal serves as the Chair (2019-2023) of the Faculty Advisory Council within ISPOR.
David L. Van Brunt, PhD, MS, BA
AbbVie, Chicago, IL, USA
When Patients and Clinicians Talk, How Are We Listening? A Discussion of How Patient and Clinician Experience Shapes HTA Recommendations
Live
ISSUE
: Innovative therapies are continually being developed, and Health Technology Assessments (HTA) agencies are evolving to keep up with the pace of innovation. A key tenant of HTA is the engagement of clinicians and patients who provide context and experience with the disease and therapy under review. Are we optimizing the perspectives of these key stakeholders into the HTA processes as novel therapies continue to enter the health system?
OVERVIEW
: HTAs are designed to inform decision making through a multi-stakeholder approach considering many perspectives. For many years, the health care system has committed to focusing on patient-centred care, and that priority should also apply to HTA. The Canadian Agency for Drugs and Technologies in Health (CADTH) has grown to become a leader in patient engagement in HTA, where now there is a call for patient and clinician input for every review. Yet today’s treatment paradigms have been transformed by innovation and novel clinical trial designs, and the pace of new drug discovery continues to increase, putting pressure on the HTA process to further evolve. Real-world insights and the treatment values that patients and clinicians share can be enhanced. For this session, a diverse group of panelists will examine the strengths and opportunities that exist to meaningfully incorporate the experiences that patients and clinicians into HTA. The session will begin with each panelist describing the strengths and opportunities in patient and clinician engagement in HTA using examples to highlight their perspective (10 minutes each). Following these presentations, a moderated discussion among the panelists will focus on the merits and feasibility of the opportunities outlined by the panelists (30 minutes), and will include an opportunity for the audience to participate through questions and discussion.
Moderators
Alexandra Chambers, BSc, MA
Novartis, Dorval, QC, Canada
Panelists
Sarah Berglas, BSc, BA
CADTH, Ottawa, ON, Canada
Christina Sit, BSc
Lung Cancer Canada, Toronto, ON, Canada
Paul Wheatley-Price, BSc, MBChB, FRCP (UK), MD
Ottawa Hospital Cancer Centre, Ottawa, ON, Canada
What Is Research-Grade Real-World Evidence and Why Does It Matter?
ISSUE: Increasingly, real-world evidence (RWE) is used to make clinical assertions and influence the standard of care through regulatory, reimbursement, and clinical decision-making. High validity RWE is increasingly desirable and required. But, the industry has not yet reached consensus on what it means to produce “research-grade” evidence using “fit-for-purpose” data. As physicians, insurance, and regulators increasingly scrutinize RWE for quality, it is now an industry imperative to understand issues of data quality and design a strategy to produce high-validity evidence.
OVERVIEW: This panel will seek to unpack this complex issue. Dan Riskin, the panel moderator, will set the stage for discussion by highlighting key questions, including: What are the differences between traditional and advanced RWE? What factors influence credibility of RWE? Where are there still gaps in data accuracy and population generalizability? Tina Boussard will describe scientific efforts to use advanced data sources and how this influences data quality. Keri Monda will address how the pharmaceutical industry is innovating in advanced RWE while maintaining and even growing efforts in traditional RWE. Nancy Dreyer will comment on advances in evidence validity and outline current thinking on research-grade RWE in the era of the 21st Century Cures Act. The moderator will ensure that at least 15 minutes of the session is devoted to soliciting audience interaction and feedback on the competing viewpoints.
Moderators
Dan Riskin, MD, MBA
Verantos, Menlo Park, CA, USA
Panelists
Nancy Dreyer, MPH, PhD
IQVIA, Cambridge, MA, USA
Nancy Dreyer is Senior Vice President and Chief Scientific Officer for Real World Solutions at IQVIA, and Adjunct Professor of Epidemiology at the University of North Carolina at Chapel Hill. She leads the IQVIA Center for Advanced Evidence Generation. A fellow of both the International Society for Pharmacoepidemiology and DIA, her current work is focused on COVID-19 along with other issues of regulatory and public health importance. Recent recognition includes the Red Jacket honor from PharmaVOICE in 2020 and DIA’s Global Inspire Award for Author of the Year in 2019. She is also celebrating recent publication of the 4th edition of the popular book “Registries for Evaluating Patient Outcomes: a User’s Guide.”
Tina Hernandez-Boussard, M.P.H, Ph.D., M.S.
Stanford University, Stanford, CA, USA
Keri Monda, PhD
Amgen Inc, Thousand Oaks, CA, USA
What Can Be Gained from Payer/HTA Involvement in the Multi-Stakeholder Consensus Development of Core Outcome Sets for Late Phase Clinical Trials?
Live
ISSUE: Panelists will explore the potential value of payer and health technology assessor (HTA) participation in consensus-based core outcome set (COS) development for drug clinical trials, discuss hurdles to their participation and debate the potential benefits (or drawbacks) of COS to coverage and payment decision-making.
OVERVIEW: Gene therapies and biologics promise paradigm shifts for many diseases, though at considerably higher prices. Decision makers assessing the added value of these agents for patients, in relation to their high costs, encounter major challenges: a) heterogenous outcomes hinder comparisons with traditional therapies, or b) effective new therapies impact standards of care such that traditional outcomes lose relevance. COS development may address these and other challenges by bringing together multiple stakeholder perspectives to deliberate and reach consensus on critical outcomes for decision-making. Through this lens, payers and HTA are key stakeholders in these initiatives.
Attendees will hear from moderator Donna Messner, PhD, President and CEO of Center for Medical Technology Policy. Dr. Messner and her team have developed multi-stakeholder consensus-based COS for late phase clinical trials under their Green Park Collaborative program. She will describe COS objectives and methodology, highlighting ways COS have reflected payer/HTA concerns. The panelists provided payer and HTA perspectives as participants in one of two recent COS initiatives: coreSCD (sickle cell disease) and coreASTHMA. Each panelist had her own motivations for participating and a unique experience during the consensus process. Panelists will be asked to present on the following:
What were you able to bring to the [multi-stakeholder consensus] process? What were you able to take away? Should more payers/HTA stakeholders get involved in COS development, why or why not? What is the potential impact of greater development and uptake of COS for future evidence-based policy-making? Dr. Messner will then facilitate an interactive discussion and take questions from the audience.
Moderators
Donna Messner, PhD
Center for Medical Technology Policy, Baltimore, MD, USA
Panelists
Foluso O Agboola, MBBS, MPH
Institute for Clinical and Economic Review, Boston, MA, USA
Jill Morrow-Gorton, MD, MBA
University of Pittsburgh (UPMC) Health Plan, Germantown, MD, USA
Emily Tsiao, PharmD
Premera Blue Cross, Mountlake Terrace, WA, USA
Can RWE Observational Research Replicate RCT Results and Should We Care?
Live
ISSUE: There are many studies and reviews focused on the ability of real world evidence studies to replicate and reproduce randomized clinical trials. However, we often use RWE to answer questions that an RCT is not designed to answer. Can RWE replicate RCTs and if they do does that really give us comfort that RWE is credible and reliable?
OVERVIEW: RCT replications studies using secondary data sources are currently producing much awaited data to see how well RWE can produce ‘accurate’ results as defined by replicating RCT findings. DUPLICATE is sponsored by the FDA and intends to replicate 30 completed Phase III or IV trials and to predict the results of seven ongoing Phase IV trials using Medicare and commercial claims data. OPERAND conducted by Brown University and Harvard Pilgrim Health Care Institute (funded by unrestricted grants from a consortia of pharmaceutical companies) intends to replicate the ROCKET-AF trial for atrial fibrillation and the LEAD-2 trial for Type 2 diabetes control using claims from commercial and Medicare Advantage plans and electronic medical record data from OptumLabs Data Warehouse. This panel will explore viewpoints from both RWE experts involved in these duplication studies and as RWE experts, and from the experts who understand the strength of evidence that is needed in order to make causal inference or comparative effectiveness/efficacy claims. The moderator will open the session with a 5 minute introduction to the issue and each speaker will have 10 minutes to discuss their view point. There will be interactive use of polling through the zoom platform and at least 20 minutes for audience Q&A or for the panelists to query each other on their viewpoints if on-demand. This session will be of interest to HEOR professionals in life sciences industry, academia, regulatory or payer space who are interested in RWE.
Moderators
Lucinda Orsini, DPM, MPH
COMPASS Pathways, Skillman, NJ, USA
Panelists
Barbara Bierer, MD
Brigham and Women's Hospital, Harvard University, Cambridge, MA, USA
Jeroen Jansen, PhD
PRECISIONheor, San Anselmo, CA, USA
David Thompson, PhD
Syneos Health, Manchester, MA, USA
Should We Be Talking "Embedded" rather than "Pragmatic" for Real-World Clinical Trials?
ISSUE: Since first being described more than 50 years ago, the pragmatic clinical trial (PCT) has been considered the most scientifically rigorous of all real-world research designs. Yet, despite its promise, the PCT design has been relatively underutilized in comparison to other approaches for real-world evidence (RWE) generation, such as prospective observational research and retrospective studies of electronic health records (EHRs) and administrative claims databases. More recently, a variety of organizations have focused attention on the potential to embed clinical trials in real-world practice settings as a means of involving practicing physicians, recruiting real-world patients, and leveraging existing EHR systems for data capture. Is this approach better suited to RWE generation? Should we be talking “embedded” rather than “pragmatic” for real-world clinical trials?
OVERVIEW: In its RWE Program Framework, the US Food & Drug Administration highlighted the benefits of integrating clinical trials into real-world practice settings, indicating that real-world data systems can be leveraged in this fashion to generate regulatory-grade evidence. Other organizations, including the NIH Collaboratory, the Clinical Trials Transformation Initiative (CTTI) and the Australian Clinical Trials Alliance, have workstreams devoted to advancing these objectives. Nirosha Lederer, the panel moderator, will set the stage for the discussion by outlining the key questions, including: What are the defining characteristics of embedded clinical trials? In what ways are embedded trials similar or different from pragmatic trials? Is embedding clinical trials in real-world practice a more practical way of improving their generalizability? David Thompson will highlight similarities and differences between embedded and pragmatic trials. Lindsay Kehoe will describe CTTI’s progress to date to identify practical issues associated with embedded clinical trials. Lou Fiori will provide an overview of case studies of embedded clinical trials undertaken in the US Veterans Administration system. The moderator will solicit audience interaction and feedback on the alternative viewpoints.
Moderators
Nirosha Lederer, PhD
Aetion Inc, Boston, MA, USA
Panelists
Louis Fiore, MD
VA System, NEWTON, MA, USA
Lindsay Kehoe, MS, CGC
Clinical Trials Transformation Initiative, Durham, NC, USA
David Thompson, PhD
Syneos Health, Manchester, MA, USA
Are Value Frameworks in Latin America Fit for Purpose When Assessing COVID-19 Health Technologies?
Live
ISSUE: The SARS-CoV-2 infection (COVID-19) has had profound health, social and economic effects across the world. By mid-December 2020, four countries in Latin America (Argentina, Brazil, Colombia and Mexico) had more than 1 million cases and were among the top 10 countries in the world in number of deaths due to COVID-19. Health innovations to tackle the pandemic, ranging from vaccines and antivirals to medical devices and diagnostics are advancing at incredible speed adding pressure to provide rapid and broad access to these technologies for those who need in order to manage the pandemic. Health Technology Agencies in Latin America follow explicit value frameworks to assess health technologies. These frameworks have a narrow definition of value, mainly focused in elements like health benefits (e.g. LYG, QALYs) and costs, disregarding other elements. COVID-19-related health technologies illustrate challenges and opportunities to advance the definition of what is value and which other elements should be considered based on international recommendations.
OVERVIEW: This panel will address challenges faced by decision makers to assess the value of COVID-19-related health innovations under the established value frameworks in three countries with established HTA agencies (Brazil, Colombia and Mexico). Experts from health technology producers including pharmaceuticals, medical devices, and diagnostics, will discuss the challenges and opportunities faced in the region highlighting those value elements that are not being considered yet.
Moderators
Homero Monsanto, PhD
Merck & Co., Inc., Cidra, PR, USA
Panelists
Claudia Brabata, MSc
Medtronic, USA, Miami, FL, USA
Diego Guarin, MD, MPH, MA
Merck, Kenilworth , NJ, USA
Jose Y. Santiago, PhD
Abbott, USA, Des Plaines, IL, USA
The Future and Evolution of Advanced Modeling Techniques to Support the Development of COVID-19 Mitigation and Prevention Policies
Live
ISSUE: The COVID-19 global pandemic is an unprecedented public health challenge requiring significant resources to evaluate the problem and determine the best solutions from a health and economic perspective. Much of the decision making related to COVID-19 have been centered around prevention, which requires forecasting. This issue panel will discuss the data and modeling needs for policy makers, health care decision makers, and the average US resident for the COVID-19 pandemic, specifically, with blue sky considerations for a non-specific future pandemic.
OVERVIEW: This panel will discuss the modeling and real-time data needs to support good decision making during the COVID-19 global pandemic. The panel will consider past challenges in data collection and forecasting, and the solutions that were used, as well as remaining challenges. The panel will also debate the role of the scientist in communicating critical data to decision makers at all levels of government and health system. Ms. Chou will moderate and provide an overview of the pandemic from a US perspective as well as a framework for thinking through the data and modeling challenges for COVID-19. Dr. Karaca Mandic will describe the challenges with real-time data collection and interpretation with the University of Minnesota COVID-19 Hospitalization Tracking Project. Dr. Singh will review advanced modeling techniques that have been or could be implemented in supporting COVID-19 decision making. Finally, Dr. Waller will describe his efforts relating to assessing inequity in spatial access to testing sites between racial and ethnic groups in Atlanta, Georgia. After a 6 minute introduction from the moderator, each panelist will present for approximately 8 minutes, reserving 30 minutes for a moderated Q/A discussion, with the option for attendees to submit questions to the panel.
Moderators
Jacki Chou, MPP, MPL
Precision Health Economics, Los Angeles, CA, USA
Panelists
Romilla Batra, MD, MBA
SCAN Health Plan, Long Beach , CA, USA
Pinar Karaca Mandic, PhD
University of Minnesota, Carlson School of Management, Minneapolis, MN, USA
Lance Waller, PhD
Emory University, Atlanta, GA, USA
Tue May 18
10:00 AM - 11:30 AM
Plenary Session
Public Health…Economics?
The COVID-19 pandemic has highlighted major methodological issues regarding the assessment of value, the trade-offs involved, and the approaches to estimating the public health and economic consequences. The public health concern is to decrease transmission, sustain hospital capacity, and reduce mortality, by imposing various measures on the population; the trade-offs include increased unemployment, diminished GDP, substantial rescue packages, and massive damage to some industries. Today, much as it was 50 years ago in our field, these expanded trade-offs are mostly made informally by various authorities, but newer methods of “public health economics” are emerging to help decision makers with quantitative understanding of the consequences. This session will explore an expanded perspective on HEOR scope and application and examine, in turn, possible lessons for our consideration of value and its assessment.
Moderators
J. Jaime Caro, MDCM, FACP, FRCPC
Evidera, McGill University, London School of Economics, Lincoln, MA, USA
Jaime Caro, Chief Scientist at Evidera and Professor of Epidemiology and of Medicine at McGill University and Professor in Practice at London School of Economics. He pioneered the use of DES, developed the Simulated Treatment Comparison approach and proposed the efficiency frontier as an alternative to cost/QALY. Recently, he has developed a new modelling technique, DICE simulation, tailored to problems in Health Technology Assessment.
Speakers
Mairin Ryan, BSc, Pharm, PhD
Health Information and Quality Authority (HIQA), Dublin, Ireland
Máirín Ryan is the Director of HTA at HIQA, Ireland. Previously, Máirín was the Chief 1 Pharmacist at the National Centre for Pharmacoeconomics and before that worked as a clinical pharmacist. She is an Assistant Professor in Pharmacoeconomics at Trinity College Dublin.
She leads a team who undertake HTAs of drugs, devices, diagnostics and public health programs. The team also provides evidence synthesis to support National Clinical Guidelines and national health policy, most recently supporting the national public health response to COVID-19.
Máirín currently serves as a member of the Executive Board of EUnetHTA. She has previously served as Chair and before that Vice-Chair of the EUnetHTA Assembly.
Anders Tegnell
Public Health Agency of Sweden, Solna, Sweden
Dr. Anders Tegnell is the State Epidemiologist of Sweden, the Deputy Director General as well as the Head of the Department of Public Health Analysis and Data Management at the Public Health Agency. He is a medical doctor with a PhD in infectious diseases and a Master in Epidemiology from the London School of Hygiene and Tropical Medicine. Dr. Tegnell has extensive experience of leading different Swedish government agencies at different levels in the field of public health, disease control and preparedness for health threats, and has also experience from international work, for example from Laos in 1990-1993 (WHO) and from DRC during the Ebola outbreak in 1995.
Meaghan Thumath, MSc PH, BSN, RN
WHO, University of British Columbia, Vancouver, BC, Canada
Meaghan is a registered nurse and senior practice leader at the BC Centre for Disease Control, where she started her career as a street nurse and has moved into policy and leadership roles in the overdose and HIV response. She holds advance practice nursing certification in sexual and reproductive health as well as an addiction nursing fellowship and advanced training in HIV and public health. She is currently working on her PhD part-time as a Trudeau Scholar at the University of Oxford's Centre for Evidence Based Intervention researching the impact of child welfare policy on marginalized women's health.
10:00 AM - 4:00 PM
Poster & Exhibit Viewing
Live
Stop in to learn about the latest technology, services, and devices and to connect one-on-one with exhibitors and sponsors during our Exhibit Viewing Hours. Be sure to reserve some time to view the latest research in HEOR through our virtual poster gallery. Browse, comment, and discuss our posters at any time while our virtual platform is active.
11:30 AM - 1:45 PM
Poster Presentations and Exhibitor Meet Ups
Live
Interact with the authors of the latest research discoveries in HEOR through our virtual poster presentations and gallery. In between poster discussions, connect one-on-one with our exhibitors and sponsors. Engage with potential solution, service, and resource providers.
Poster Schedule
11:45 AM - 12:45 PM
Women in HEOR Session
Lost in Translation: How to Optimize Communications in the COVID Era
Effective communication has always been key for successful dialogue, healthy relationships, and professional growth. The COVID-19 pandemic, leading to mandatory quarantine and minimal face-to-face interactions, has had a critical impact on our daily communication skills. Further, COVID-19 has put a spotlight on how the research community needs to effectively communicate with a wider audience, beyond our peers. This panel will cover communication challenges related to new modes of communications (virtual) and to a different/wider audience. Amy Glass, an experienced speaker on Communications in the Age of COVID, will share tactics, skill-building techniques, and lessons learned for communicating effectively in both venues. The forum will feature considerable discussion and interaction among the keynote speaker and leaders of the Women in HEOR initiative and will include audience Q&A. Pre-conference polling will be used to seed specific discussion points to expand upon in this session. The session is open to all Virtual ISPOR 2021 registrants – women and men in HEOR.
Speaker
Amy Glass, .
BRODY Professional Development, Jenkintown, PA, USA
Amy Glass is chief executive officer of BRODY Professional Development. Amy believes that a company’s most valuable asset is its people and she is driven to help employees to grow and succeed. Since 1998, she has helped clients leverage their strengths and improve competencies. Amy’s ability to explain challenging concepts in a clear and relatable way helps her participants to absorb new information and fast-track growth. Specializing in areas such as leadership presence, communication, presentation skills, and influence; Amy presents at conferences globally and her articles are regularly published in magazines and newspapers. She has worked with many Fortune 100 companies and is co-author of the book Leadership Presence: The Influential Intangible. Amy earned her bachelor’s degree in Communications from Boston University. When not coaching or presenting, Amy enjoys traveling, spending time with family and friends, and can often be found at baseball fields or basketball courts with her husband and two sons.
Julia F. Slejko, PhD
University of Maryland, Baltimore, MD, USA
Julia F. Slejko, PhD is an Associate Professor of Pharmaceutical Health Services 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. 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 is co-lead of ISPOR’s Women in HEOR initiative and currently Co-Chair Elect of the ISPOR Faculty Advisor Council.
Olivia Wu, PhD
University of Glasgow, Glasgow, United Kingdom
Olivia Wu, PhD is director of the Health Economics and Health Technology Assessment (HEHTA) Research Unit and William R Lindsay chair of Health Economics, at the University of Glasgow. She is also director of the Complex Reviews Support Unit, a national methods support unit for evidence synthesis funded by the UK National Institute for Health Research (NIHR). Olivia has expertise in a broad range of health technology assessment (HTA) methodologies and is interested in adapting and applying HTA methodologies in context. She has a particular interest in evidence synthesis and economic evaluations. Her research spans across a wide range of clinical areas and different types of health technologies (eg, pharmacological treatments, medical devices and diagnostic tests). Her work has informed clinical guidelines and health policy decisions, both at national and international levels. In addition to her research, Olivia has been a long-standing member of the NICE Technology Appraisal Committee and advisor to Healthcare Improvement Scotland. She has also been advisor to HTA agencies in Brazil, China, Taiwan and Thailand.
11:45 AM - 1:15 PM
Educational Symposium
Gene Therapies: Reimbursement Challenges and Pricing Opportunities in the US
Live
Economic evaluation studies may underestimate the value of innovative cell and gene therapies in rare diseases, impacting on Health Technology Assessment (HTA) frameworks, market access and pricing decisions. This symposium will explore the challenges faced by HTA and other reimbursement bodies, as they consider a wide range of cell and gene therapies. Panelists will discuss drug value evidence-based challenges, the coverage policies that global payers have used to address these challenges, and their impact on value assessment frameworks, contracting and affordability.
Sponsor
Novartis Gene Therapies
Moderators
Omar Dabbous, MD, MPH
Novartis Gene Therapies, Bannockburn, IL, USA
Speakers
Michael Drummond, MCom, DPhil
University of York, York, YOR, United Kingdom
Michael Drummond, BSc, MCom, DPhil is professor 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 650 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. He is currently 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.
Lou Garrison, PhD
University of Washington,The Comparative Health Outcomes, Policy, and Economics Institute, Seattle, USA
Lou Garrison, PhD, is professor emeritus in The Comparative Health Outcomes, Policy, and Economics Institute in the School of Pharmacy at the University of Washington, where he joined the faculty in 2004.
For the first 13 years of his career, Dr. Garrison worked in non-profit health policy at Battelle and then the Project HOPE Center for Health Affairs, where he was the Director from 1989-1992. Following this, he worked as an economist in the pharmaceutical industry for 12 years. From 2002-2004, he was vice president and head of Health Economics & Strategic Pricing in Roche Pharmaceuticals, based in Basel, Switzerland.
Dr. Garrison received a BA in Economics from Indiana University, and a PhD in Economics from Stanford University. He has more than 150 publications in peer-reviewed journals. His research interests include national and international health policy issues related to personalized medicine, benefit-risk analysis, and other topics, as well as the economic evaluation of pharmaceuticals, diagnostics, and other technologies.
Dr. Garrison was elected as ISPOR President for July 2016-June 2017, following other leadership roles since 2005. He recently co-chaired the ISPOR Special Task Force on US Value Frameworks. He was selected in 2017 by PharmaVOICE as being among “100 of the Most Inspiring People” in the industry. He recently received the PhRMA Foundation and Personalized Medicine Coalition 2018 Value Assessment Challenge First-Prize Award as lead author on a paper on “A Strategy to Support the Efficient Development and Use of Innovations in Personalized and Precision Medicine.”
Peter Neumann, ScD
Tufts Medical Center, Boston, MA, USA
Access to Oncology Combinations: the “Combination Challenge”
Live
While combination therapies are not new in oncology, in recent years there have been significant advances in treatment with the introduction of targeted therapies and immunotherapies. Combinations of these new, innovative oncology therapies increasingly represent the preferred treatment option for many patients, delivering significant benefits and prolonged survival compared to monotherapies. However, access to branded combinations poses several challenges, particularly where each constituent is produced by different manufacturers and present in multiple indications. These difficulties ultimately lead to the “Combination Challenge”; where patients would benefit from the launch of better therapeutic options, manufacturers would see their volume increase and payers would value the combination however there may be barriers which prevent patients from having access to new combination. The session will explain the various Access challenges and discuss potential Policy solutions to address these.
Sponsor
MSD
Moderators
Duane Schulthess, BA, MBA
Vital Transformation, Wezembeek Oppem, VBR, Belgium
Speakers
Berit Eberhardt
International Kidney Cancer Coalition, Duivendrecht, NH, Netherlands
Christopher Henshall, PhD
Brunel University, London, United Kingdom
Luca Morlotti, ,
MSD, Lucerne, Switzerland
Tim Wilsdon, Msc
Charles Rivers Associates, London, United Kingdom
1:45 PM - 2:45 PM
Breakout Session 2
Network Meta-Analysis in HTA Submissions: Methodological Advances and Impact on HTA Methods Guidance
Live
PURPOSE:
To describe the current status and latest methodological advances in the use and conduct of network meta-analysis (NMA) and indirect treatment comparison (ITC) for the purpose of submission to health technology assessment (HTA) agencies.
DESCRIPTION:
The use of ITC and NMA for estimating relative effectiveness and safety of treatments in absence of direct, head-to-head randomized evidence has been expanding over the last decade in HTA submissions, as well as in clinical guidelines. Methodological progress in this area has been fast and steady and HTA agencies and drug developers have embraced new methods, but it is challenging to keep up to date with the pace of these advances. In this workshop, Sofia Dias will describe the latest methodological advances and modelling approaches used in conducting ITC and NMA and their application in different contexts and for different types of technologies and outcomes of interest (12 minutes). Dalia Dawoud will describe the current status of using ITC and NMA in HTA submissions and the latest trends observed in terms of the methods used. She will also outline how NICE considered the latest methodological advances in its methods’ review (12 minutes). Jeroen Jansen will highlight how some of these methodological advances were informed by actual evidence synthesis challenges experienced while preparing HTA submissions. Furthermore, he will outline the challenges that pharmaceutical and consultancy companies can face in adjusting to new methodological advances and facilitating the uptake of these methods (12 minutes). Daan Reid will discuss some currently available software tools for undertaking ITC and NMA, showing how they build on open-source implementations to drive adoption of new methods and improve efficiency, transparency and user-friendliness of the evidence synthesis process (12 minutes). The workshop is relevant to attendees from various backgrounds including HTA agencies, academics, consultancies and pharmaceutical companies.
Discussion Leader
Dalia Dawoud, PhD
National Institute for Health and Care Excellence, London, LON, United Kingdom
Dalia Dawoud, PhD, is Senior Scientific Adviser at the National Institute for Health and Care Excellence (NICE). She holds MSc in Economic Evaluation in Health Care from City University London and PhD in pharmaceutical policy and economics from King’s College London.
She has long experience in using economic evaluation in clinical guidelines development and health technology assessment (HTA), gained through working on NICE Clinical Guidelines as well as technology appraisals. Dalia’s research interests are focused on the advanced methods of evidence synthesis and use in economic models and the use of real-world evidence to inform drug development and health care decision making. Dalia currently has overall responsibility of overseeing the delivery of NICE allocated tasks on a portfolio of IMI and Horizon 2020 funded research projects including EHDEN and HTx. She is widely published in the field of pharmaceutical policy and pharmacoeconomics. She also serves as Associate Editor for ISPOR journal Value in Health and as Associate Editor for Pharmacoeconomics and Outcomes Research for Elsevier’s journal Research in Social and Administrative Pharmacy. Dalia also holds adjunct position as Associate Professor at the Faculty of Pharmacy, Cairo University.
Sofia Dias, PhD
CRD, University of York, York, YOR, United Kingdom
Jeroen P Jansen, PhD
School of Pharmacy, University of California San Francisco, San Francisco, CA, USA, and PRECISIONheor, Oakland, CA, USA
Daan Reid, MSc
, University Medical Centre Groningen, Groningen, GR, Netherlands
Lost in Transmission: Analytic Applications of Public Health Data to Inform Evaluations of the Efficacy, Effectiveness, and Value of Interventions for COVID-19
Live
PURPOSE:
To visualize and interactively explore how public health data can illustrate trends in the incidence and severity of COVID-19 across jurisdictions; To help participants understand current disease burden and transmission parameters; To understand how these epidemiologic parameters can inform the effectiveness of COVID-19 prevention strategies and feed in to economic models to predict the value of upcoming interventions
DESCRIPTION:
Routine public health data on COVID-19 presents opportunities to not only monitor trends in infections, but also to calculate transmission parameters for epidemiologic models and for informing economic modeling of potential interventions. This workshop will review the types of data typically reported to describe COVID-19 burden, and how these vary across jurisdictions. We will explore how these data are used to inform epidemiologic models developed to understand SARS-COV2 transmission and COVID-19 burden, using real-world examples from three North American jurisdictions with differing population sizes, densities, and intervention strategies. We will present dynamic visualizations with which audience members can interact to observe how manipulating input parameters (e.g., number of new cases, disease duration, mortality, and recoveries) affects factors such as the size of the affected population and R0 (or its real-time approximation, Rt). Our workshop will also demonstrate how surveillance data and epidemiologic parameters can feed into economic models of COVID-19. Given the multi-faceted nature of COVID-19 interventions and their widespread impact, for economic models to be useful they will need to robustly address societal costs and benefits. We will explore how graphical visualizations and epidemiologic measures, such as Rt, may inform key steps in the economic evaluation of upcoming interventions like vaccines. The costs of such interventions can be contextualized by the implications on the spread of disease. This component will be framed as a Q&A session with economic modelers, and tips and pitfalls for COVID-19 modeling will be explored.
Discussion Leader
Scott Emerson, MSc
Broadstreet Health Economics & Outcomes Research, Vancouver, BC, Canada
Karissa Johnston, PhD
Broadstreet Health Economics & Outcomes Research, Vancouver, BC, Canada
John Schneider, PhD
Avalon Health Economics, Morristown, NJ, USA
Shelagh Szabo, MSc
Broadstreet Health Economics & Outcomes Research, Vancouver, BC, Canada
Three Preference Studies Sponsored By FDA CBER to Inform Benefit-Risk Assessment for Biologics
Live
PURPOSE:
Understand how patient preference information (PPI) informs benefit-risk assessment at the US Food and Drug Administration (FDA) Center for Biologics Evaluation and Research (CBER) Learn the methodological lessons from three PPI studies sponsored by CBER and how to apply them to PPI studies for regulatory application Learn how to report the findings from these studies and discuss their potential impact on regulatory decision-making DESCRIPTION:
CBER is committed to better listening and capturing patients’ voices by advancing the science of measuring patient inputs to inform benefit‐risk assessments. CBER has sponsored three PPI studies to address key considerations of applying PPI research in regulatory contexts. These studies have elicited patients’ quantitative benefit-risk tradeoff preferences for biological products that intend to treat sickle-cell disease (SCD), osteoarthrosis of the knee (KOA), and hard-to-control type 1 diabetes mellitus (T1DM). A speaker from the FDA will provide context on how PPI is used in the regulatory decision-making process, how regulatory considerations impacted the studies’ designs, and how the results can inform regulatory decision-making. Individual investigators of these studies will present for 10-minutes and share key findings, discuss lessons learned and show patients’ evaluation of the preference-elicitation instruments. The presentation will include:
Adaptation of a PROM as PPI study attributes in two different ways Recruitment of participants by self-reported diagnosis vs. physician confirmed diagnoses and whether these impact patient preferences Use of hypothetical baseline severity in patients with mild vs. those with actual severe SCD status Impact of patient symptom severity on their tolerance of SCD treatment-related risks Preferences and preference heterogeneity for islet cell therapy in patients with hard-to-control T1DM
Discussion Leader
Juan Gonzalez, PhD
Duke Clinical Research Institute, Cary, NC, USA
Dr. Gonzalez is an Assistant Professor in the Department of Population Health Sciences at the Duke University School of Medicine. He is an expert in the design of stated-preference survey instruments and the use of advanced statistical tools to analyze stated-preference data. His research has focused on two main areas: 1) transparency in benefit-risk evaluations of medical interventions, and 2) elicitation of health preferences from multiple stakeholders to support shared decision making.
Dr. Gonzalez Co-led the first FDA-sponsored preference study. The study was highlighted in FDA’s recent precedent-setting guidance for submitting patient-preference evidence to inform regulatory benefit-risk evaluations of new medical devices. More recently, Dr. Gonzalez collaborated with the Medical Devices Innovation Consortium (MDIC) to prepare the first catalog of preference-elicitation methods suitable for benefit-risk assessments of medical devices. The catalog was part of the Patient-Centered Benefit-Risk Assessment Framework developed by MDIC. As a core group member of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Conjoint Analysis Task Force, Dr. Gonzalez helped draft good-practice recommendations for statistical analysis, interpretation, and reporting of health preference data. Dr. Gonzalez is the current Chair of the ISPOR special interest group on stated-preference research.
Martin Ho, MS
Center for Biologics Evaluation and Research, U.S. Food and Drug Administration, Fulton, MD, USA
Carol Mansfield, PhD
RTI Health Solutions, Research Triangle Park, NC, USA
Leslie Wilson, Ph.D.
University of California, San Francisco, Hillsborough, CA, USA
Spotlight Session
Findings from the Payer Summit – Managed Entry Agreements
Live
One of the biggest challenges payers face today is the impact of innovative, new therapies for diseases with unmet needs (oncology & rare diseases). These new therapies show promise but there is often significant uncertainty regarding their added value to patients and cost to the health care system. In other cases, the new therapies front load most of the cost to the beginning of care. This has created strain on healthcare system budgets, raising affordability and equity issues, and creating a great deal of uncertainty for assessment bodies and payers order to address the high upfront costs of curative therapies. A potential solution to address these concerns is the development of performance-based managed entry agreements.
ISPOR will host the ISPOR Payer Summit on April 22, 2021. This multi-stakeholder working meeting includes payers, HTA bodies, manufacturers, CROs, academia, and patient representatives. This spotlight session will summarize the findings from the summit and explore the challenges and benefits of performance based MEAs. Hearing different perspectives from the stakeholders defining the points of convergence (or agreement) and working to align the differences related MEAs is critical to achieve optimum patient access.
Moderators
Mark Trusheim, MS, BS
Massachusetts Institute of Technology, Cambridge, MA, USA
Mark Trusheim, MS, BS, Strategic Director, NEWDIGS and Visiting Scientist MIT, Boston, MA
Mark Trusheim is Strategic Director, MIT NEWDIGS where he also co-leads the Financing and reimbursement of Cures in the US (FoCUS) Project; and a Visiting Scientist at the MIT Sloan School of Management. Through MIT he has also served as a Special Government Employee for the FDA’s Office of the Commissioner.
Mark’s research focuses on the economics of biomedical innovation, especially precision financing for patient access, precision medicine, adaptive pathways, platform trials and digital health advances.
Prior to MIT, his career spanned big data at Kenan Systems, marketing at Searle Pharmaceuticals, eHealth as Vice President of Monsanto Health Solutions, genomics as President of Cereon Genomics, and policy as the President of the Massachusetts Biotechnology Council.
He holds degrees in Chemistry from Stanford University and Management from MIT.
Speakers
Jessica Daw, PharmD
Sentara Health Plans, Virginia Beach, VA, USA
Jessica Daw, PharmD, MBA currently serves as the vice president, Pharmacy at Optima Health and has over 15 years of managed care experience in multiple lines of business, including Commercial, Medicaid, Medicare, and Exchange. She has experience focusing in quality, medication therapy management, care management programs, formulary and utilization management, value-based contracting, physician risk sharing arrangements, compliance, and PBM oversight. Jessica is currently on the AMCP Board of Directors. She is also the co-chair of the North American Health Technology Assessment (HTA) Roundtable for ISPOR. Jessica earned her PharmD from Duquesne University and her Master of Business Administration from the University of Pittsburgh.
Hemant Phatak, PhD
Tamir Singer
NHS England, London, United Kingdom
Breakout Session 2
Can We All Afford a Cure? The Greatest Challenges Now Facing Payers
Live
ISSUE:
The cost of gene therapies is unlike anything the healthcare industry has witnessed before. In 2019, Novartis’ spinal muscular atrophy gene therapy, Zolgensma, was priced at a record $2.125 million, with the Institute for Clinical and Economic Review commenting that this price is justified by the value provided. The industry has now set a precedent, demonstrating that governments and health insurers are willing to pay premium prices. With several curative therapies launching on an annual basis, it is important to consider how payers are responding to the funding and access challenges posed by these therapies, and what are the remaining enduring challenges that must be tackled? These include access inequities between private and public healthcare plans, implementation of value-based contracting, and issues with portability between private healthcare plans.
OVERVIEW:
Kate Hanman will moderate and introduce the key challenges currently facing payers providing coverage for gene therapies. Debra Miller (patient panelist) will outline the patient perspective of access to curative therapies and explore the inequities between private and public insurance plans. Ramesh Arjunji (industry panelist) will debate what types of arrangements manufacturers should anticipate making with government healthcare providers to ensure gene therapies are not excluded from coverage. Michael Sherman (payer panelist) will discuss how drug manufacturers can best work with insurance providers to manage the financial risks associated with increasing numbers of marketed gene therapies, including addressing issues around portability when patients switch healthcare insurers.
Moderators
Kate Hanman, BSc, MSc
Costello Medical, London, LON, United Kingdom
Panelists
Ramesh Arjunji, PhD
AvroBio, Cambridge, MA, USA
Debra Miller, ,
CureDuchenne, Newport Beach, CA, USA
Michael Sherman, MD, MBA, MS
Harvard Pilgrim/Tufts Health Care, Wellesley, MA, USA
Uncertainty in Uncertainty: How Much Can We Really Reduce the Influence of Unmeasured Confounders in Real-World Data?
Live
ISSUE:
Is it even possible to account for unmeasured confounding in a way that makes comparative studies relevant for decision making? How do payers factor in uncertainty in their decision-making? In cases where important clinical or policy questions have not been addressed through randomized trials, retrospective data are used to enhance the totality of evidence to make decisions, yet a limitation is the possibility that unmeasured factors can influence treatment choice and thus influence outcomes. While multiple techniques have been proposed to minimize unmeasured confounding and responsibly infer causality, there is no clear consensus on an appropriate approach.
OVERVIEW:
This interactive panel will debate methods to help identify and reduce the influence of unmeasured confounders in retrospective analyses and whether or not they are ready for serious consideration by regulatory and HTA bodies. Some potential solutions to move forward may include: pre-specification of proposed methodology for adjustments to reduce bias, interim assessment of baseline data without the knowledge of outcome to assess comparability of real world cohort, incorporation of "estmands" approach to address "intercurrent" events such as differential dropouts. The moderator will frame the problem (3 min), speakers will have 8 minutes each to present their views (25 min). Next, the moderator describes a case study in oncology with unmeasured confounders, asking panelists to describe how he or she would design a study to minimize risk of unmeasured confounding and communicate uncertainty (10 min). Audience voting and Q&A concludes (15 min).
Moderators
Scott Ramsey, MD, PhD
Hutchinson Institute for Cancer Outcomes Research, Fred Hutchinson Cancer Research Center, Seattle, WA, USA
Dr. Ramsey is a general internist and health economist. He is a Full Member in the Cancer Prevention Program, Public Health Sciences Division at the Fred Hutchinson Cancer Research Center, where he directs Hutchinson Institute for Cancer Outcomes Research, a multidisciplinary team devoted to clinical and economic evaluations of new and existing cancer prevention, screening and treatment technologies. In addition, Dr. Ramsey is a Professor in the Schools of Medicine and Pharmacy at the University of Washington. Trained in Medicine and economics, Dr. Ramsey’s research focuses on economic evaluations in cancer. He has published widely on patterns of care, costs, and cost-effectiveness of treatments for lung, colorectal, and prostate cancer. His research portfolio and interests include: large scale SEER-Medicare/Cancer Registry data linkages, patient reported outcomes, economic modeling of health care interventions, cost-effectiveness analysis, quality of life assessment, patterns of care, health care utilization, economic burden of disease for patients and society, pragmatic trial design, early technology assessment, and stakeholder engagement. Dr. Ramsey is co-Chair of the Outcomes and Comparative Effectiveness Committee of the Southwest Oncology Group, past President of the International Society of Pharmacoeconomics and Outcomes Research (ISPOR), and has served on the IOM Cancer Policy Forum.
Panelists
Blythe Adamson, PhD, MPH
Flatiron Health, New York, NY, USA
Blythe Adamson is a Senior Quantitative Scientist at Flatiron Health. She uses health economics, math, epidemiology, and data science to research and identify high-value medicines in development. Her research has included dynamic transmission modeling of infectious diseases, novel microsimulation modeling methods in oncology, and cost-effectiveness studies to inform policy. Dr. Adamson received her PhD in Pharmacoeconomics and Masters in Public Health in Epidemiology from the University of Washington in Seattle. Before joining Flatiron, she worked on the development of HIV vaccines at Fred Hutchinson Cancer Research Center and informed Gates Foundation investing decisions with the Institute for Disease Modeling at Global Good.
Anirban Basu, PhD
University of Washington, Seattle, WA, USA
Anirban Basu is a Professor of Health Economics and the Stergachis Family Endowed Director of The CHOICE Institute at the University of Washington, Seattle. He holds joint appointments with the Departments of Health Services and Economics at UW, is a Faculty Research Fellow at the US National Bureau of Economic Research, and an elected Fellow of the American Statistical Association. His work sits at the intersection of microeconomics, statistics, and health policy. His research focuses on understanding the economic value of health care through scientific disciplines of applied economic theory, comparative and cost-effectiveness analyses, causal inference methods, program evaluation, and outcomes research. He served on the Second Panel on Cost-effectiveness Analysis in Health and Medicine and serves on the Editorial Advisory Board for Value in Health Journal. He received his master's in Biostatistics from UNC-Chapel Hill and a PhD in Public Policy Studies from the University of Chicago.
Jennifer Malin, MD, PhD
UnitedHealth Group, Santa Monica, CA, USA
3:00 PM - 4:00 PM
ISPOR Forums
Exploring New Frameworks for Real-World Evidence (RWE) Best Practices in Asia Pacific: the REALISE Project
Real-World Evidence (RWE) is increasingly considered globally as a useful resource to support timely patient access to innovative technologies. Many countries in the Asia Pacific have begun incorporating RWE into healthcare decisions, although the pace and extent of adopting RWE vary from country to country. However, generating reliable and robust RWE depends largely on how to collect, analyze, and incorporate real-world data (RWD) in a consistent and efficient manner. In light of this consideration and recent international initiatives about RWD, global experts in 11 Asian health systems initiated the REALISE Project to develop a guidance document on the use of RWD/RWE to inform HTA for decision making in Asia. This session will feature an expert group of international speakers including key members of the REALISE Project to talk about the current landscape of RWD/RWE for HTA in Asia, how RWE has evolved, and where it is headed next. They will also discuss major challenges encountered by Asia Pacific HTA agencies in the use of RWD/RWE and share their experiences on how to use RWE to support HTA and reimbursement decision making by case studies.
Moderators
Jeonghoon Ahn, PhD
Ewha Womans University, Seoul, Korea, Republic of (South)
Speakers
Amanda Adler, MD, PhD, FRCP
National Institute for Health & Care Excellence (NICE), Fowlmere, 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
Jeonghoon Ahn, PhD
Ewha Womans University, Seoul, Korea, Republic of (South)
Hwee-Lin Wee, PhD
National University of Singapore, Singapore, Singapore
ISPOR Women in HEOR Social Hour
Live
Meet and continue the conversation with today’s Women in HEOR Keynote Speaker, Amy Glass of BRODY Professional Development, and leaders of the Women in HEOR Initiative (Julia F. Slejko, PhD, University of Maryland School of Pharmacy and Olivia Wu, PhD, University of Glasgow) at this Social Hour. Network with your fellow peers and those who support the advancement of women in the field of HEOR. This social hour is hosted by the ISPOR Women in HEOR Initiative and is intended to be lively, interactive, and open to both men and women.
Student Network Session
Student Research Spotlight
5 Student Presenters were chosen based on receiving the highest scored abstracts for Virtual ISPOR 2021. Students will present a 5-minute elevator pitch about their research. After each presenter finishes there will be 5 minute Q&A with moderators Dr. Zeba Khan and Dr. Khalid Kamal.
Moderators
Khalid Kamal, MPharm, PhD
West Virginia University, Morgantown, WV, USA
Khalid M. Kamal, M. Pharm., Ph.D. is a Professor and Chair of the Department of Pharmaceutical Systems and Policy at West Virginia University School of Pharmacy, Morgantown, WV. Dr. Kamal's primary research and teaching interests have been pharmacoeconomics, patient-reported outcomes research, research methods, and improving quality of care using real-world data sources such as electronic medical records and specialty pharmacy data. He has been a Visiting Professor teaching pharmacoeconomics and decision modeling courses at institutions such as Kobe Gakuin University in Kobe, Japan and King Saud University in Riyadh, Saudi Arabia. Dr. Kamal serves as the Chair (2019-2023) of the Faculty Advisory Council within ISPOR.
Zeba Khan, RPh, PhD
ISPOR, Malvern, PA, USA
Speakers
Shu Huang, MPH, MS
University of Florida, Gainesville, FL, USA
My name is Shu Huang, and I am a 2nd year PhD student at the University of Florida College of Pharmacy majoring in Pharmaceutical Outcomes and Policy. Before joining the program, I have achieved an MS degree in Biotechnology from Johns Hopkins University, and an MPH degree in Biostatistics from Yale school of public health. My current research interests are in suicide prediction modeling, chronic pain, opioid overdose, and machine learning.
Gilbert Ko, PharmD, MBA
University of Washington, Bellevue, WA, USA
Woojung Lee, PharmD
Genentech Inc., SEATTLE, WA, USA
Woojung Lee is a 4th year PhD candidate at the Comparative Health Outcomes, Policy, and Economics (CHOICE) Institute at the University of Washington. Before entering the PhD program, she received her PharmD from Seoul National University in Seoul, South Korea, in 2016 and BS in biology from Yonsei University in 2012. Woojung has worked on health economics and outcomes projects using real-world data analysis (e.g., electronic medical records and claims data), disease modeling, and systematic review. Her research has been around the use of real-world evidence in economic assessment of drugs, the value of cancer drugs and diagnostic testing, novel applications of machine learning in HEOR, medication experience in older adults and dementia prevention strategies. She is currently working on her dissertation on the challenges and the value of older adult-specific clinical trials, which is supported by the PhRMA Foundation.
Natalie Ourhaan, M.B.A, B.S
University of Florida, Gainesville, FL, USA
Sohul Shuvo, M.S., M.B.A.
University of Tennessee Health Science Center, Memphis, TN, USA
8:00 PM - 9:00 PM
Pre-Released Session
Modeling and Simulations for Healthcare Decision Maker: Battling COVID-19 and Other Biomedical Challenges
Live
PURPOSE:
This workshop will focus on advanced modeling tools and how they might be used to inform healthcare decision making, including COVID-19 intervention polices as a recent example. Workshop participants will understand the foundational constructs of simulation and modeling techniques and the reliance of these models on the quality of the data used to inform inputs and assumptions. Participants of this workshop will be able to understand the basics for key types of selected statistical and mathematical models as well as what types of questions these models are suited to answer in health evaluations.
DESCRIPTION:
Health economic evaluation is used to support decision makers in areas including public health, epidemiology, infectious diseases and pharmacology. This workshop aims to provide some background and overview on cutting edge modeling and simulation methods to address continuously changing healthcare problems and data. Ms. Jacquelyn Chou will provide an introduction and overview for the workshop, as well as basic principles and objectives for simulations and models (5 minutes). Dr. Bhramar Mukherjee will provide an overview of the basics for statistical models alongside key considerations and limitations when there is delay or underreporting, as in the case of COVID-19. (15 minutes). Dr. Zhilan Feng will walk-through the fundamentals and considerations for key types of mathematical dynamic models including complex agent based models and infectious disease transmission models (15 minutes). Finally, Dr. Mubayi will review the applications of these models for health economics via methods such as decision trees and microsimulation models. He and Ms. Chou will also facilitate an interactive session where participants can help sort research questions into the type of model and data that is best suited to answer that question via polling, as well as submit their own research questions to be sorted into model types by fellow participants via polling (30 minutes).
Discussion Leader
Jacki Chou, MPP, MPL
Precision Health Economics, Los Angeles, CA, USA
Zhilan Feng, PhD
Purdue University, West Lafayette, IN, USA
Anuj Mubayi, PhD
PRECISIONheor, Los Angeles, CA, USA
Bhramar Mukherjee, PhD
University of Michigan, Ann Arbor, Ann Arbor, MI, USA
Improving Validity and Relevance of Indirect Comparisons and Network Meta-Analysis: Implications for HTA Submissions
PURPOSE:
Indirect comparisons and network meta-analysis (NMA) estimate relative treatment effects between competing health technologies, however these methods may lack validity if there are differences in patient populations between included studies. Furthermore, in Health Technology Assessment (HTA) the estimates need to be relevant to the target population for the decision. This workshop will provide an overview of methods for indirect comparisons and NMA that aim to account for population differences. Advantages and disadvantages of the different methods and implications for future HTA submissions will be discussed.
DESCRIPTION:
This workshop will begin with an overview of different approaches to population adjustment for indirect comparisons and NMA. If individual patient data (IPD) are available for all studies then IPD-level network meta-regression is the gold standard approach. However, in HTA IPD are typically only available for one or a subset of studies. Available methods in this case include matching-adjusted indirect comparison (MAIC), simulated treatment comparison (STC), network meta-regression, and the recently developed multilevel network meta-regression (ML-NMR). The methods will be compared as to their: validity; flexibility to synthesize networks of any size and availability of IPD; and ability to produce estimates in a given target population of interest. Their performance in simulation studies will be presented. Recommendations on when to use these methods for future HTA submissions will then be discussed based on hypothetical yet representative case-studies characterized by different evidence scenarios. Dr. Jansen will introduce current methods for population adjustment in HTA. Dr. Phillippo will then describe ML-NMR and compare the methods. Finally, Prof Welton will discuss recommendations for HTA. Presentations will last 40 minutes, followed by an interactive Q&A session, providing the audience an extended opportunity to discuss the issues raised with the panel. This workshop will benefit those involved in preparing or critiquing HTA submissions that utilize population adjustment methods.
Discussion Leader
Jeroen P Jansen, PhD
School of Pharmacy, University of California San Francisco, San Francisco, CA, USA, and PRECISIONheor, Oakland, CA, USA
David Phillippo, PhD
University of Bristol, Bristol, BST, United Kingdom
Nicky Welton, PhD
University of Bristol, Bristol, United Kingdom
Professor, Statistical and Health Economic Modelling, Director, NICE Guidelines Technical Support Unit
How the Time-Drive Activity-Based Costing Contributes to Increasing Value in Health Care? the Example of the Brazilian Stroke Care Pathway
Live
PURPOSE:
This workshop intends to share with the audience how by applying the value-based health care (VBHC) framework, the Brazilian Institute for health technology assessment (IATS) is producing contributions for the scientific community and the Brazilian stroke care pathway. The Time-driven Activity-based Costing methodology is one of the gold-standards recommendations of the value-agenda. However, its application in a real-world setting is still emergent and frequently registers methodological heterogeneity. Motivated by that, the TDABC in healthcare Consortium (
www.tdabcconsortium.com ) was created to improve the quality of projects that apply TDABC. The community already involves members from all continents, and in Brazil is working with the national stroke network in a research to evaluate value for the stroke-care pathway. By attending this workshop, the audience will learn why the TDABC is the gold-standard recommendation in the context of VBHC, how it is possible to explore the method to identify and measure cost-saving opportunities and consequently increase value, and how TDABC in addition to the stroke ICHOM standard set is being used to evaluate and increase value for the stroke care pathway.
DESCRIPTION:
The speakers will introduce the TDABC in the context of VBHC (20 min), explain the sequence of eight steps to apply it using real examples (20 min), and present the value analysis for the Brazilian stroke care pathway. The stroke case discussion will explore how the value-analysis performed can guide waste reduction for the stroke care pathway and identify opportunities to increase the quality of care (20 min).
Discussion Leader
Ana Paula Etges, PhD, Eng.
National Health Technology Assessment Institute, Porto Alegre, RS, Brazil
Sheila Ourique Martins, PhD, MD
Hospital Moinhos de Vento, Porto Alegre, Brazil
Carisi Anne Polanczyk, ScD, MD
National Health Technology Assessment Institute, Porto Alegre, Brazil
Building the Real-World Ecosystem for Safety Signal Management: It Takes a Village!
Live
PURPOSE
: In the world of medical device surveillance, the processes of signal management (detection, discernment and evaluation) are complex and driven by dynamic convergence of device-, provider-, setting- and patient- related characteristics. This workshop will focus on identification of actionable factors for building the (1) capacity of device ecosystem to ascertain and characterize the signal; (2) efforts to utilize novel methods including artificial intelligence/machine learning (AI/ML) and blockchain; (3) transparent and timely communication between stakeholders; and (4) value of patient-centric, interdisciplinary approaches that integrate the medical product ecosystem.
DESCRIPTION
: Varying levels of unfamiliarity with real-world data (RWD), study designs, and analytic approaches appropriate for signal management has resulted different levels of trust across the medical device ecosystem, underscoring the need for education and training. This workshop will engage workshop participants in building an ecosystem-driven model for signal management, using examples from paclitaxel-coated devices for the treatment of peripheral arterial disease and diagnostics for identification of biomarkers for oncology treatments. Dr. Richey will discuss methodological capacity and identify needs, some early successes and introduce early- and long-term opportunities. Dr. Marinac-Dabic will present the integration of digital health solutions including blockchain and AI/ML algorithms to advance the value of RWD in active surveillance. Dr. Tieche will highlight the need for responsible transparency and communication across health care sectors and stakeholder perspectives. Dr. Wamble will detail the importance of characterizing new and ongoing risk-benefit balance from a market access perspective. The workshop participants will work interactively in four groups to identify tactical approaches in each of the four domains discussed (i.e., methodologic capacity, integration of digital solutions, communications, and interdisciplinary approach). Interested participants will then also be invited to join the post-workshop mirror stakeholder group to engage in further discussions on this topic.
Discussion Leader
Danica Marinac-Dabic, MD, PhD, MMSc, FISPE
U.S. Food and Drug Administration, Silver Spring, MD, USA
Mary Beth Ritchey, PhD, FISPE
Med Tech Epi, LLC, Philadelphia, PA, USA
Christopher Tieche, PhD
Medtronic, Winnetka, IL, USA
David Wamble, PhD
Bristol Myers Squibb, Lawrence Township, NJ, USA
Successfully Selecting, Developing, and Deploying Digital Clinical Measures of Health
Live
PURPOSE:
To support the audience in selecting, developing, and deploying fit-for-purpose, meaningful digital clinical measures across clinical research, patient care, and public health.
DESCRIPTION:
The global COVID-19 pandemic has brought new urgency to the drive toward devising a common framework for the development and deployment of high quality, patient centric, digital clinical measures. Multiple digital technologies for symptom tracking and contact tracing are currently in use and/or development, and clinical researchers are now designing new decentralized clinical trial strategies using telemedicine and remote patient monitoring to minimize pandemic-related disruption of the clinical research enterprise. Adoption of a comprehensive "how-to" roadmap to synthesize best practices in the digital health field is essential for guiding these efforts and safeguarding public health. The Digital Measures Playbook (The Playbook) synthesizes best practices from the digital health field into one comprehensive "how-to" document. A first draft, built by collaborators from the Digital Medicine Society, Elektra Labs, Genentech, a member of the Roche Group, Koneksa, Myokardia, Sage Bionetworks, Scripps Research, and the U.S. Food and Drug Administration. The Playbook is now being advanced for broad adoption by these organizations and new collaborators from ActiGraph; Activinsights; American Pharmacists Association; BlackThorne Rx; Center for Digital Health at Brown University; Covance; Digital.Health; Duke University; Eli Lilly and Company; the European Medicines Agency; Evidation Health; Merck and Co., Inc.; Open mHealth; Pfizer; physIQ; Savvy Coop; Takeda; VivoSense; and Winterlight Labs. This session will be delivered as an interactive workshop, providing participants with the foundational elements of selecting, developing, and deploying high quality digital clinical measures that cut across use cases. Speakers are co-creators of The Playbook with extensive experience and expertise in measurement science, HEOR, the science of patient engagement, regulatory science, and -- perhaps most importantly -- education. This speaker panel demonstrates disciplinary diversity as well as effective pre-competitive collaboration.
Discussion Leader
Elizabeth (Nicki) Bush, MHS
Eli Lilly and Company, Indianapolis, IN, USA
Elizabeth (Nicki) Bush is the director and global head of the Patient-Focused Outcomes Center of Expertise at Eli Lilly and Company, responsible for the design and implementation of patient-centered measurement strategies to support drug development efforts across Lilly’s portfolio. In this capacity, she and her team advise drug development teams in the selection, modification, and development of clinical outcome assessments (COAs) for use in clinical trials and other research studies, and utilize mixed methods approaches to evaluate the measurement properties of COAs and aide in interpretation of the resultant data.
Nicki has held research and leadership positions in the patient-centered measurement field in both consultancy and industry for over ten years. During this time, she has collaborated with partners in industry, regulatory agencies, and academia and held the position of industry co-director of the PRO Consortium from 2016-2018. Her previous experience includes behavioral research and program evaluation. Ms. Bush earned her BA in the liberal arts (philosophy and history of mathematics and science) from St. John’s College and her master’s degree in epidemiology from the Johns Hopkins Bloomberg School of Public Health.
Jennifer Goldsack, MS, MBA
DiMe, Boston, MA, USA
Bray Patrick-Lake, MFS
Evidation Health, San Mateo, CA, USA
Betsy Tschosik, PhD
Genentech, South San Francisco, CA, USA
Addressing Health Inequity in the U.S.: How Can Value Assessment (VA) Contribute to More Equitable Healthcare Resource Allocation?
Live
PURPOSE:
The purpose of this workshop is to explore challenges in addressing health inequity as an objective in value assessment (VA), and to demonstrate and discuss the applications of potential methods in the US context using real-world model examples.
DESCRIPTION:
There is system-wide consensus and emphasis on the importance of tackling the widening health disparities in our society. Various promising modeling approaches, most notably the distributional cost-effectiveness analysis (DCEA), have been proposed to support more equitable resource allocation in health care. However, the extent to which these methods can be applied in the pluralistic, fragmented US healthcare system is unclear. In this workshop, the speakers will first discuss concepts and measures of health equity, and key challenges in addressing equitable resource allocation using existing methods (~10 minutes). To tackle these challenges, ensuring data representations from the underserved subpopulations is the necessary first step. Using the IVI’s model focusing addressing major depressive disorder (IVI-MDD) as an example, researchers will share strategies on how to recruit individuals of color and lower socioeconomic status in a component patient preference survey (~10 minutes). Discussions on available modeling will include:
how to build a Medicaid perspective in a VA model (~10 minutes); how to leverage US data to conduct DCEA through a case study on the impact of funding inpatient COVID-19 treatments on underlying health equality. (~10 minutes). Insights from the workshop are applicable across all stakeholders: researchers will gain insight into available sources and methods for US equity assessment; innovators and patient groups can learn about how to encourage the traditionally underrepresented communities to participate in the data generation and collection process for health data; and payers and US HTA bodies can learn about data availability and suggested next steps for integration of equity assessment into decision-making.
Discussion Leader
Susan Dosreis, PhD
University of Maryland School of Pharmacy, Baltimore, MD, USA
Susan dosReis, PhD is a professor and Vice Chair of Research in the Department of Pharmaceutical Health Services Research at the University of Maryland School of Pharmacy. She has expertise in stated preference methods to evaluate trade-offs of medication benefits and risks. As Director of the Patient-Driven Values in Healthcare Evaluation (PAVE) Center, she is applying novel methods to better understand treatment decision-making from a variety of patient groups, and in particular the underserved communities.
Louis P. Garrison, PhD
University of Washington, Seattle, WA, USA
Lou Garrison, PhD, is professor emeritus in The Comparative Health Outcomes, Policy, and Economics Institute in the School of Pharmacy at the University of Washington, where he joined the faculty in 2004.
For the first 13 years of his career, Dr. Garrison worked in non-profit health policy at Battelle and then the Project HOPE Center for Health Affairs, where he was the Director from 1989-1992. Following this, he worked as an economist in the pharmaceutical industry for 12 years. From 2002-2004, he was vice president and head of Health Economics & Strategic Pricing in Roche Pharmaceuticals, based in Basel, Switzerland.
Dr. Garrison received a BA in Economics from Indiana University, and a PhD in Economics from Stanford University. He has more than 150 publications in peer-reviewed journals. His research interests include national and international health policy issues related to personalized medicine, benefit-risk analysis, and other topics, as well as the economic evaluation of pharmaceuticals, diagnostics, and other technologies.
Dr. Garrison was elected as ISPOR President for July 2016-June 2017, following other leadership roles since 2005. He recently co-chaired the ISPOR Special Task Force on US Value Frameworks. He was selected in 2017 by PharmaVOICE as being among “100 of the Most Inspiring People” in the industry. He recently received the PhRMA Foundation and Personalized Medicine Coalition 2018 Value Assessment Challenge First-Prize Award as lead author on a paper on “A Strategy to Support the Efficient Development and Use of Innovations in Personalized and Precision Medicine.”
Stacey Kowal, BS, MSc
Genentech, Alameda, CA, USA
Stacey Kowal currently serves as a principal researcher in Genentech’s Health Policy and Systems Research team where her work focuses on identifying and testing new methods to assess the impact of new healthcare technologies. Her background includes both US and global work to inform health technology assessment and reimbursement as well as design of public health interventions, drawing on disciplines of applied mathematics, economics, and international health policy. Stacey’s current research aims to increase focus on equity effects in health economics and outcomes research (HEOR), including integration of distributional elements into healthcare decision-making. Stacey is a Udall scholar, a Truman scholar and a Marshall scholar. She holds a BS in Mathematics from Alma College, an MSc in public health from the London School of Hygiene and Tropical Medicine and an MSc in International Health Policy and Health Economics from the London School of Economics.
Richard Xie, PhD
The Innovation and Value Initiative, Newton, MA, USA
Identifying Health Innovation Gaps: A Landscape Analysis
Live
PURPOSE:
Funding and activity in health innovation may not be aligned with the areas of highest public health burden and costs to the system. This workshop will focus on an attempt to develop of a reliable and valid model that helps to forecast, identify, and prioritize critical health and innovation gaps.
DESCRIPTION:
The Department of Health and Human Services (HHS) and FasterCures convened a group of health experts and data scientists from public and private sectors to develop an objective, data-based methodology to identify critical health innovation gaps in order to inform future programs and policies. The multidisciplinary group aimed to develop a reliable and valid model to help identify critical health innovation gaps as measured by three domains: public health impact, healthcare costs, and innovation activity. The workshop will outline the process for developing the metrics from data sources and methodology for an analytic model and data analyses. Results from the pilot study will also be presented. The first discussion leader will present in 15 minutes the overarching goal of the project and the methodologies employed by each of the subgroups. The second presenter will then present for 10 minutes the detailed methodology used within the public health impact subgroup to identify conditions with the highest public health impact for the pilot and calculate research disparities. Workshop participants will then contend with issues in choosing data sources and developing metrics to measure public health burden and contribute feedback on methods of the pilot. Finally, how the data from all three domains were brought together in a multicriteria decision modeling will be presented. The final model can be leveraged to identify the areas that have relatively low innovation activity compared to public health burden and costs and allow the HHS to consider prioritization of resources to these important areas.
Discussion Leader
Elise Berliner, PhD
Agency for Healthcare Research and Quality, Rockville, MD, USA
Laura Gressler, B.A., M.S.
University of Maryland, Baltimore, MD, USA
Adaptive Reimbursement: Innovating for Patient Access and Better Outcomes for All
Live
PURPOSE:
Based on MIT NEWDIGS LEAPS Project experience, inspire participants to explore Adaptive Reimbursement opportunities to accelerate adoption of RWE-based patient regimen optimization to simultaneously improve quality-of-life and economic outcomes.
DESCRIPTION:
Scientific, clinical development and regulatory advancements are fueling unprecedented biopharmaceutical innovation from gene therapies to immunotherapies. These upstream innovations have not been matched by patient-centered downstream innovations that employ payment and other incentive systems to implement patient specific regimen improvements informed by leveraging real-world evidence. Adaptive reimbursement combines downstream innovations that:
Creatively cover and finance therapies to ensure timely, appropriate access, enhance healthcare system sustainability and reward upstream innovation; and Reward optimized treatment regimens for each patient through the generation of real-world evidence that reduces uncertainties regarding biopharmaceutical product performance. This Panel will explore current Adaptive Reimbursement examples from the MIT NEWDIGS LEAPS project, discuss developing future innovations, and describe approaches to overcome the significant implementation challenges. Hear how:
Durable cell and gene therapy precision financing models could apply to other therapeutic areas Adaptive reimbursement mechanisms might fund RWE production platforms (indirectly or directly) by creating financial demand for their results Data needed to administer adaptive reimbursement contracts can also be used to de-risk market access, identify unmet medical needs, and monitor patient benefits from therapies RWE platforms could fuel the design and implementation of innovative adaptive reimbursement models
Discussion Leader
Angela Banks, MBA, MA
UnitedHealth Group, Washington, DC, USA
Ron Potts, MD
Kaiser Permanente, Portland, OR, USA
David Strutton, PhD, MPH
Merck, North Wales, PA, USA
Mark Trusheim, MS, BS
Massachusetts Institute of Technology, Cambridge, MA, USA
Mark Trusheim, MS, BS, Strategic Director, NEWDIGS and Visiting Scientist MIT, Boston, MA
Mark Trusheim is Strategic Director, MIT NEWDIGS where he also co-leads the Financing and reimbursement of Cures in the US (FoCUS) Project; and a Visiting Scientist at the MIT Sloan School of Management. Through MIT he has also served as a Special Government Employee for the FDA’s Office of the Commissioner.
Mark’s research focuses on the economics of biomedical innovation, especially precision financing for patient access, precision medicine, adaptive pathways, platform trials and digital health advances.
Prior to MIT, his career spanned big data at Kenan Systems, marketing at Searle Pharmaceuticals, eHealth as Vice President of Monsanto Health Solutions, genomics as President of Cereon Genomics, and policy as the President of the Massachusetts Biotechnology Council.
He holds degrees in Chemistry from Stanford University and Management from MIT.
VALUE Assessment of Biosimilars: Challenges and Considerations
Live
PURPOSE:
To provide guidance to and raise awareness of workshop participants regarding methodological issues surrounding the value assessment of biosimilars
DESCRIPTION:
The use of biosimilars faces significant gaps and challenges in terms of their health technology assessment, which have not yet received sufficient attention in the health economic literature and in guidelines issued by many health technology assessment agencies. This workshop will explain the following methodological issues in the value assessment of biosimilars:
the choice of the appropriate technique of economic evaluation (cost-minimization or other) and of the appropriate comparator. the approach to filling the clinical evidence gap when the marketing authorization of the biosimilar has been granted on the basis of extrapolation (and no phase 3 clinical trial data are available). the approach to value assessment of biosimilars in treatment naïve patients and in patients previously treated with biologics/biosimilars. whether value assessment of biosimilars should account for the potential ‘nocebo’ effect and how. the management of uncertainty and barriers to uptake related to biosimilars and the role of managed entry agreements. the valuation of value-added services. the societal valuation of expanding access to treatment in terms of capturing QALY gains at population level. The workshop explores these issues based on a systematic literature review conducted by the ISPOR Biosimilar SIG (presented by Steven Simoens and Evelien Moorkens) and discusses these issues from the perspective of health technology assessment agencies (presented by Dalia Dawoud) and of pharmaceutical industry (presented by Delphine Courmier).
Discussion Leader
Delphine Courmier, PhD, MBA
AMGEN, Thousand Oaks, CA, USA
Dalia Dawoud, PhD
National Institute for Health and Care Excellence, London, LON, United Kingdom
Dalia Dawoud, PhD, is Senior Scientific Adviser at the National Institute for Health and Care Excellence (NICE). She holds MSc in Economic Evaluation in Health Care from City University London and PhD in pharmaceutical policy and economics from King’s College London.
She has long experience in using economic evaluation in clinical guidelines development and health technology assessment (HTA), gained through working on NICE Clinical Guidelines as well as technology appraisals. Dalia’s research interests are focused on the advanced methods of evidence synthesis and use in economic models and the use of real-world evidence to inform drug development and health care decision making. Dalia currently has overall responsibility of overseeing the delivery of NICE allocated tasks on a portfolio of IMI and Horizon 2020 funded research projects including EHDEN and HTx. She is widely published in the field of pharmaceutical policy and pharmacoeconomics. She also serves as Associate Editor for ISPOR journal Value in Health and as Associate Editor for Pharmacoeconomics and Outcomes Research for Elsevier’s journal Research in Social and Administrative Pharmacy. Dalia also holds adjunct position as Associate Professor at the Faculty of Pharmacy, Cairo University.
Evelien Moorkens, MSc PhD
KU Leuven, Leuven, Belgium
Steven Simoens, MSc, PhD
KU Leuven, Leuven, VBR, Belgium
Mixed Methods Research: Using Embedded Qualitative Interviews to Enhance Interpretation of Clinical Trial Outcomes
Live
PURPOSE:
Conducting embedded interviews within clinical trials is a new research paradigm that can provide a variety of benefits, including understanding treatment benefit and safety/tolerability from the patient perspective, and estimating thresholds for meaningful change. Using a mixed method approach, embedded interview data can enrich our understanding of the patients’ experience to illuminate and complement the PRO and biomedical outcomes collected in the trial. This workshop will illustrate how embedded interviews can be utilized to generate in-depth and meaningful patient input through the drug development process.
DESCRIPTION:
After a brief introduction of the purpose of this type of research and value for different stakeholders, the speakers will present examples of embedded interview use within clinical trials and address resulting contributions to drug development research in each case study.
(10 minutes) Introduction to description, uses and value of mixed methods embedded interview research to stakeholders. (Miriam Kimel) (10 minutes) Overview of value-added contributions of mixed method embedded interviews to support regulatory inquiries. (Naomi Knoble) (10 minutes) Case study on use of mixed methods assessment of patient treatment experience, including disease changes and tolerability in oncology. (Laurie Eliason) (10 minutes) Case study on use of longitudinal mixed methods approach to assess treatment benefit-risk assessment in a rare disease. (Carla Dias Barbosa) Audience Interactive element
(20 minutes) SMALL GROUP DISCUSSION The audience will be invited to further the knowledge gained by developing a list of potential applications for embedded interview research through the drug develop process, with stated objective(s) and possible challenges for each example. At the end of the workshop, participants will be able to:
Identify at potential benefits that might be realized from incorporating a mixed methods design within their clinical trials Identify key challenges to embedding qualitative interviews into clinical trials Describe methods for meeting these challenges
Discussion Leader
Carla Dias Barbosa, MSc
Evidera, London , LON, United Kingdom
Laurie Eliason, MPH
GlaxoSmithKline, Upper Providence , PA, USA
Miriam Kimel, PhD
Evidera, Bethesda, MD, USA
Naomi Knoble, PhD
Food and Drug Administration, Silver Spring, MD, USA
We Have the Technology: Building Better Comparative Effectiveness Methods for the Future
Live
PURPOSE:
To present recently proposed methods for comparative effectiveness including the application of machine learning through frequentist model averaging (fMA) and doubly robust methods. Along with novel methods for estimation, we will discuss advances in evaluating the robustness of analyses to the potential for unmeasured confounding.
DESCRIPTION:
Evidence from real world data (RWE) is increasingly becoming a vital component of decision health care decision making. However, limitations inherent with causal inference are challenging. One challenge is the need for selecting the ‘right’ statistical models both for treatment selection and outcome analysis. For treatment selection models, propensity scores estimated from logistic regression are the gold standard. However, new approaches using tree-based methods, balancing algorithms, and double scores have been proposed. Penalized regression has become popular for building outcome models and doubly robust methods bring the promise of more robust analyses. There is no consensus on the best approach and simulations suggest there is no single best method for all scenarios. Speakers will cover the above issues as well as future directions of including machine learning for causal inference using targeted maximum likelihood methods (TLME) and fMA. With fMA researchers specify many potential methods and models into the analysis and cross validation determines the better approaches in a data driven fashion. Another challenge in comparative effectiveness is addressing bias due to unmeasured confounding. In recent years a suite of methodologies has emerged ranging from methods quantifying the robustness to confounding to methods using external data to adjust the estimated causal effect. The speakers will facilitate an understanding of unmeasured confounding options by presenting different statistical procedures within a unified framework. Tools for selecting and implementing, along with simulations and real data examples will be discussed.
Discussion Leader
Alan Brnabic, MS
Eli Lilly and Company, Croydon, NSW, Australia
Hana Lee, PhD
FDA, Silver Spring, MD, USA
David Madigan, PhD
Columbia University, Brookline, MA, USA
Anthony Zagar, PhD
Eli Lilly and Company, Indianapolis, IN, USA
Xiang Zhang, PhD
CSL Behring, Philadelphia, PA, USA
Advancing Employer Engagement on VALUE Assessment
Live
PURPOSE:
This workshop will provide lessons learned from three projects focused on improving employer evidence needs and engagement in value assessment (VA). These findings will inform how current methods can better account for health plan attributes that matter to employers.
DESCRIPTION:
Employers are key healthcare purchasers in the United States. They have a vested interest in ensuring employees have access to timely care that is most likely to meet individual employee and/or family needs. As healthcare costs rise, employers are doubling down on achieving improved value for their dollar. Health care value, from the employers’ perspective, is broader than that considered by many stakeholders and includes the relative impact on clinical outcomes, employee productivity, and organizational value proposition. However, employers have not traditionally been included in discussions about VA. As VA frameworks are increasingly used to inform health care decision-making, it is important that employers and VA bodies engage in meaningful discussions with one another on what is important to them, discuss alignment in perspectives and needs, and identify strategies to improve current approaches. This workshop is relevant for VA bodies, researchers, patients, students, healthcare purchasers, payers and policymakers. Ms. Rehayem will provide an introduction to the US employer and healthcare purchaser communities, including an overview of US medical directors on gaps in current value frameworks (10-minutes). Dr. Oehrlein will describe tangible actions for enhancing value assessment derived from a series of patient-employer roundtables on VA (10-minutes). Ms. Westrich will review survey results on employer and payer perspectives on the importance of incorporating productivity, caregiver burden, and other indirect benefits in VA (10-minutes). Ms. Bright will describe employer contributions to a major depressive disorder model (10-minutes). We will then have an audience Q/A discussion (20-minutes).
Discussion Leader
Jennifer Bright, MPA
Innovation and Value Initiative, Alexandria, VA, USA
Elisabeth Oehrlein, PhD, MS
National Health Council, Washington, DC, USA
Margaret Rehayem, MA
National Alliance of Healthcare Purchaser Coalitions, Washington DC, DC, USA
Kimberly Westrich, MA
National Pharmaceutical Council, Washington, DC, USA
Can Performance-based Risk Sharing Arrangements (PBRSAs) for Medtech Address Procurement and Market Access Challenges? Scanning the Current Horizon and a View to the Future.
Live
PURPOSE:
This panel seeks to discuss performance-based risk-sharing agreements (PBRSA) for medical technologies (MedTech); to identify whether taxonomies of PBRSAs for pharmaceuticals are applicable for MedTech; and, through discussion, develop a nascent set of best practices for device-specific PBRSA. Mark Sculpher will present a taxonomy based on the the nature of the decisions taken by health systems and the type of evidence needed to support these. Susan Garfield, a consultant active in the area of PBRSA, will bring the industry perspective and provide insights the challenges of planning, execution, and evolution of these plans, and Payam Abrashimi will serve as a representative of a payer perspective into how PBRSA can address the needs of payers and procurement bodies; Richard Charter will moderate and provide an overview of PBRSA.
DESCRIPTION:
Procurement bodies have increasingly sought rigorous evidence before accepting premium price products. Recent pandemic challenges have accelerated these trends of more cautious evaluation of MedTech prior to procurement. Yet, regulatory agencies in many instances allow for the commercialization of devices without rigorous trials providing the evidence sought by procurement. In the absence of such data, evidence development through performance-based risk-sharing agreements (PBRSA) have recently generated increased interest, development, and emergent use. This panel seeks to discuss recent years’ growing interest in PBRSA for MedTech and the roles, opportunities, and challenges within this landscape of procurement bodies, governments, health technology assessment (HTA) entities, and payers. Topics for discussion include how to deliver PBRSA, what is the role of health economics and outcomes research in reducing the clinical and economic uncertainty, and the role of real-world evidence in risk-share agreements. Each speaker will speak for approximately 15 minutes. The audience is encouraged to bring their experience to the discussion; open questions highlighted during the panel will be tested with audience polling.
Discussion Leader
Payam Abrishami, PhD
Dutch National Health Care Institute, Diemen, Netherlands
Giuditta Callea, PhD
SDA Bocconi School of Management, ROMA, RM, Italy
Richard Charter, MSc
Alira Health, Basel, BS, Switzerland
Mark Sculpher, PhD
University of York, York, YOR, United Kingdom
Mark Sculpher is professor of Health Economics at the Centre for Health Economics, University of York, UK where he leads the Centre’s Programme on Economic Evaluation and Health Technology Assessment. He is also co-director of the Policy Research Unit in Economic Evaluation of Health and Care Interventions, a seven-year programme, run collaboratively with the University of Sheffield and funded by the UK Department of Health.
Mark has worked in the field of economic evaluation and health technology assessment for over 30 years. He has researched in a range of clinical areas including heart disease, cancer, diagnostics, and public health. He has also contributed to methods in the field, in particular relating to decision analytic modeling and techniques to handle uncertainty, heterogeneity, and generalisability. He has over 250 peer-reviewed publications and is a co-author of two major text books in the area: Methods for the Economic Evaluation of Health Care Programmes (OUP, 2015 with Drummond, Claxton, Torrance and Stoddart) and Decision Modeling for Health Economic Evaluation (OUP, 2006 with Briggs and Claxton).
Mark is an emeritus member of the UK National Institute of Health Research (NIHR) College of Senior Investigators. He has also been a member of the National Institute for Health and Clinical Excellence (NICE) Technology Appraisal Committee and the NICE Public Health Interventions Advisory Committee. He currently sits on NICE’s Diagnostics Advisory Committee. He chaired NICE's 2004 Task Group on methods guidance for economic evaluation and advised the Methods Working Party for the 2008 update of this guidance. He has also advised health systems internationally on HTA methods including those in France, Ireland, Japan, Singapore, Germany, Portugal, and New Zealand. He has been a member of the Commissioning Board for the UK NHS Health Technology Assessment Programme, the UK NIHR /Medical Research Council’s Methodology Research Panel, and the UK Department of Health’s Policy Research Programme’s Commissioning Panel. He served as President of ISPOR for the 2011-12 term.
Preparing for a Policy Sandbox: Engaging Stakeholders to Advance the Use of Rwd in HTA
Live
PURPOSE:
This workshop introduces the sandbox approach and explains its possible applications in health technology assessment (HTA), using methodological and policy challenges around real world data (RWD) for HTA-decisions. Attendees should walk away with insight in why it would be beneficial and how it might work to engage all stakeholders in such a complex environment to test emerging methods, policies and processes.
DESCRIPTION:
In 2022, a policy sandbox will be organized in the Horizon2020 funded project HTx (Next Generation Health Technology Assessment) to test new methods to make better use of RWD. There is a growing recognition of the value of RWD in addition to efficacy data from RCTs. However, some hurdles remain in benefiting from the use of RWD. A policy sandbox is a safe environment where the outcomes of the project can be translated into HTA practice while engaging stakeholders. We have seen that it is crucial for the acceptance and uptake of any new method that stakeholders are engaged during development. Despite its potential, this is the first time that a sandbox will be applied in HTA-setting. Milou Hogervorst will give a background on the willingness to use RWD for HTA decisions in challenging circumstances (8 min.). Secondly, Johan Pontén will elaborate on the identified challenges of using RWD in HTA (10 min.). Thirdly, Ayla Lokhorst will outline useful change models for engaging stakeholders (10 min.). Finally, Dalia Dawoud will present the concept of policy sandboxes and its use as a controlled environment to test new HTA methods (13 min.). Q&A time will be given at the end and questions will be asked on other examples/situations where it would be useful to apply the policy sandbox format (19 min.). This workshop will be very relevant for attendees from various backgrounds including academics, HTA agencies and pharmaceutical companies.
Discussion Leader
Dalia Dawoud, PhD
National Institute for Health and Care Excellence, London, LON, United Kingdom
Dalia Dawoud, PhD, is Senior Scientific Adviser at the National Institute for Health and Care Excellence (NICE). She holds MSc in Economic Evaluation in Health Care from City University London and PhD in pharmaceutical policy and economics from King’s College London.
She has long experience in using economic evaluation in clinical guidelines development and health technology assessment (HTA), gained through working on NICE Clinical Guidelines as well as technology appraisals. Dalia’s research interests are focused on the advanced methods of evidence synthesis and use in economic models and the use of real-world evidence to inform drug development and health care decision making. Dalia currently has overall responsibility of overseeing the delivery of NICE allocated tasks on a portfolio of IMI and Horizon 2020 funded research projects including EHDEN and HTx. She is widely published in the field of pharmaceutical policy and pharmacoeconomics. She also serves as Associate Editor for ISPOR journal Value in Health and as Associate Editor for Pharmacoeconomics and Outcomes Research for Elsevier’s journal Research in Social and Administrative Pharmacy. Dalia also holds adjunct position as Associate Professor at the Faculty of Pharmacy, Cairo University.
Milou Hogervorst, PhD
Utrecht University, Utrecht, Netherlands
Ayla Lokhorst, MA
Zorginstituut Nederland (ZIN), Diemen, Netherlands
Johan Pontén, MA
Dental and Pharmaceutical benefits Agency, TLV, Stockholm, Sweden
Wed May 19
10:00 AM - 11:30 AM
Plenary Session
Stepping Up to the Challenge- How Novel Collaborations and New Players are Reshaping the HEOR Landscape
New and interesting HEOR collaborations are emerging to address the extraordinary challenges many countries face today with their health systems and that also focus on health technology innovation. Traditional players—such as regulators, payers, and providers—are seeking innovative partnerships that leverage new data sources. New players—including major technology companies, digital health startups, and engineers with machine learning backgrounds—are utilizing strategic alliances to "reinvent" HEOR. This session will bring together thought leaders from government, payers, technology, and academia who are doing things differently in healthcare to share examples and thoughts on how this may reshape the world of HEOR.
Moderators
Robert Califf, MD
Verily, Google Health, South San Francisco, CA, USA
Robert M. Califf, MD, MACC, is the Head of Clinical Policy and Strategy for Verily and Google Health for Verily and Google Health. Prior to this Dr. Califf was the vice chancellor for health data science for the Duke University School of Medicine; director of Duke Forge, Duke’s center for health data science; and the Donald F. Fortin, MD, Professor of Cardiology. He served as Deputy Commissioner for Medical Products and Tobacco in the U.S. Food and Drug Administration (FDA) from 2015-2016, and as Commissioner of Food and Drugs from 2016-2017. A nationally and internationally recognized leader in cardiovascular medicine, health outcomes research, healthcare quality, and clinical research, Dr. Califf is a graduate of Duke University School of Medicine. Dr. Califf was the founding director of the Duke Clinical Research Institute and is one of the most frequently cited authors in biomedical science.
Speakers
Amy Abernethy, MD, PhD
U.S. Food and Drug Administration, Silver Spring, MD, USA
Amy P. Abernethy, M.D., Ph.D. is an oncologist and internationally recognized clinical data expert and clinical researcher. As the Principal Deputy Commissioner of Food and Drugs, Dr. Abernethy helps oversee FDA’s day-to-day functioning and directs special and high-priority cross-cutting initiatives that impact the regulation of drugs, medical devices, tobacco and food. As acting Chief Information Officer, she oversees FDA’s data and technical vision, and its execution. She has held multiple executive roles at Flatiron Health and was professor of medicine at Duke University School of Medicine, where she ran the Center for Learning Health Care and the Duke Cancer Care Research Program. Dr. Abernethy received her M.D. at Duke University, where she did her internal medicine residency, served as chief resident, and completed her hematology/oncology fellowship. She received her Ph.D. from Flinders University, her B.A. from the University of Pennsylvania and is boarded in palliative medicine.
Carolyn Bradner Jasik, MD
Omada Health, San Francisco, CA, USA
Dr. Jasik is a board-certified Clinical Informatics physician with experience in both hospital-based technology
innovation and creating digital solutions for health behavior change. At Omada Health she directs the clinical
and research teams to create and test the next generation of chronic disease programming. In the past year,
she has lead the launch of expanded programming in diabetes, cardiovascular disease, and mental health.
Prior to joining Omada she worked at the University of California, San Francisco (UCSF) where she directed
the implementation of the Epic electronic health record, integrated digital health programming into clinical
practice, and conducted clinical trials on health technology. She completed her undergraduate degree in public
policy at Princeton University, MD at the University of Chicago, and her research training at UCSF. Dr. Jasik
continues to practice at UCSF where she is an Associate Professor of Pediatrics.
Dipak Kalra, MBBS, PhD
The European Institute for Innovation through Health Data, Ghent, Belgium
Professor Dipak Kalra is President of The European Institute for Innovation through Health Data (i~HD). He plays a leading international role in research and development of Electronic Health Records, including the development of ISO standards on EHR interoperability, personal health records, EHR requirements, security and data protection. Dipak has led multiple Horizon 2020 and IMI projects in these areas, alongside pharma companies, hospitals, patient and professional organisations and ICT companies. His current projects include the generation and acceptance of real world evidence in pregnancy, the governance of patient-centric clinical trials, scaling up the quality, interoperability and the reuse of health data for research including inputs to the European Health Data Space, the readiness of hospitals to generate evidence for value based care, and an initiative to explain the value of research using health data to the public. Dipak is Professor of Health Informatics at University College London, Visiting Professor at the University of Gent, a member of CEN and ISO standardisation bodies and a former GP.
Harlan Krumholz, MD
Yale University, New Haven, CT, USA
Harlan Krumholz is a cardiologist, the Director of the Yale New Haven Hospital Center for Outcomes Research and Evaluation (CORE) and the Harold H. Hines, Jr. Professor of Medicine at Yale University. He is a leading expert in the science to evaluate and improve the quality and efficiency of care, reduce disparities, improve integrity in medical research, and avoid wasteful practices. His team has led successful national and international quality improvement initiatives, architected national quality measures and measurement strategies in partnership with CMS, promoted patient-centric approaches to research and care, created strategies and tools to enable open science and advance scientific integrity, and advanced regulatory science in partnership with the FDA. Dr. Krumholz is a co-founder of HugoHealth, a patient-centric platform to engage people as partners in research and clinical care, facilitating interoperability and the secure, permission-based movement of digital health data. He is a co-founder of medRxiv, a preprint server for clinical research. Dr. Krumholz is a member of the National Academy of Medicine and was named one of the top-cited scientists, with an h-index of almost 200. He received degrees from Yale College, Harvard Medical School and the Harvard School of Public Health.
10:00 AM - 4:00 PM
Poster & Exhibit Viewing
Live
Stop in to learn about the latest technology, services, and devices and to connect one-on-one with exhibitors and sponsors during our Exhibit Viewing Hours. Be sure to reserve some time to view the latest research in HEOR through our virtual poster gallery. Browse, comment, and discuss our posters at any time while our virtual platform is active.
11:30 AM - 1:45 PM
Poster Presentations and Exhibitor Meet Ups
Live
Interact with the authors of the latest research discoveries in HEOR through our virtual poster presentations and gallery. In between poster discussions, connect one-on-one with our exhibitors and sponsors. Engage with potential solution, service, and resource providers.
Poster Schedule
11:45 AM - 12:45 PM
ISPOR Forums
Handling Missing Data in Health Economics and Outcomes Research: State of the Art Guidance and Good Practices
Overview of topic: Whenever individual level data are used as the basis of a statistical modelling, partially or fully unobserved (often referred to as “missing”) data is likely to limit the amount of information available from the evidence which might lead to biased inferences. While health economics and outcome research (HEOR) is no exception, there are also other context-specific issues and complexities that often make the modellers work harder, with an even higher potential for biased inferences made from the data and impacting the whole decision making process that characterises HEOR. Building on previous work developed by the ISPOR Missing Data in HEOR Working Group, we have updated a comprehensive literature review to understand methodological approaches used to account for missing data in cost-effectiveness analyses. Studies were eligible only if they focused on addressing missing data for costs, Health Related Quality of Life (HRQoL) measures, or utility. We have also collected and critically reviewed current guidelines and suggestions on how to deal with missing data, specifically in HEOR. Leveraging the findings of the review we have produced a characterization of different methods available for handling missing data in HEOR and have provided key strengths and limitations of different classes of methods. Finally, we have consolidated this information into a generalized practical framework for implementing best practices focused on the reporting of missing data and methodological considerations/justification for accounting for missing data. The objective and goal of this forum will be to focus on presenting the key findings of this project, potential recommendations, and have a discussion to gain feedback on a potential framework for dealing with missing data.
Speaker
Gian Luca Di Tanna, PhD, MPhil, MSc
The George Institute for Global Health, University of New South Wales, Sydney, NSW, Australia
Rita Kristy, MS
Astellas Pharma Global Development, Northbrook, IL, USA
Kumar Mukherjee, PhD
Philadelphia College of Osteopathic Medicine, Georgia Campus, Suwanee, GA, USA
11:45 AM - 1:15 PM
Educational Symposium
Delivering on Promises: Enhancing Patient Access to (Neo)adjuvant Cancer Treatments
Live
This session’s focus will be on remaining unmet medical need in the fight against cancer; on the promises that (neo)adjuvant cancer treatments offer to patients, physicians, and societies; and on ways to overcome the challenges such treatments face in terms of value demonstration and appreciation. Four speakers - scientific leader, payer, regulator, and an industry representative - will offer their perspectives to get a better understanding of the value of early treatment. They will highlight the unmet medical needs and discuss the potential of (neo)adjuvant treatments to improve cancer treatment along the entire continuum of care (screening, treatment, social care). They will also dive-in on the hurdles to broad and rapid access of (neo)adjuvant treatments with a special view towards limitations around delivery, acceptance, matching of scientific evidence in HTA and P&R context. Finally, the discussion will focus on possible solutions for broader and more rapid access to early treatments such as evidence generation during authorization, innovative contracting, and multi-stakeholder collaboration.
Sponsor
MSD
Moderators
Duane Schulthess, BA, MBA
Vital Transformation, Wezembeek Oppem, VBR, Belgium
Speakers
Francis Arickx, .
,, Lucerne, Switzerland
Nadia Harbeck, MD, PhD
Brustzentrum der Universität München (LMU), Munich, BY, Germany
Milayna Subar, MD
Merck & Co., Kenilworth, NJ, USA
Sean Tunis, MD, MSc.
Rubix Health, Baltimore, MD, USA
Expedited Pathways to Decentralized Trials - The Transformation of the Regulatory Process and Increasing Dependence on RWE
Live
Precision medicine is redefining how we evaluate and treat disease. No longer is cancer defined by its organ of origin, tissue or cell type but rather its genotype. Over just a few decades lung cancer has been transformed from one disease affecting 100,000+ in the U.S. annually to dozens of cancers that each inflict just a few thousand. Conducting traditional 3-phased clinical trials in these orphan disease categories on a myriad of novel therapeutics while trying to expedite therapies from lab bench to bedside is forcing a transformation of both the clinical development and regulatory process. From cohort expansion to accelerated pathways to decentralized trials, this transformation has placed real-world evidence at the center of the approval process. We’ll explore how the traditional one-size-fits-all clinical development and regulatory approval paradigm has evolved to become an increasingly customizable process.
Sponsor
Cardinal Health
Moderators
Bruce Feinberg, DO
Cardinal Health Specialty Solutions, ATLANTA, GA, USA
Speakers
Jonathan Kish, PhD, MPH
Cardinal Health, Miami, FL, USA
Shibani Pokras, MPH
GlaxoSmithKline, Bryn mawr, PA, USA
Scott Swain, PhD, MPH
Cardinal Health Specialty Solutions, Dublin, OH, USA
Jacqueline Vanderpuye-Orgle, PhD
Parexel, LA VERNE, CA, USA
1:45 PM - 2:45 PM
Breakout Session 3
Race to Secure COVID-19 Vaccines - What Vaccine Attributes Should Matter Most in Low-to-Middle-Income Countries?
Live
PURPOSE:
This workshop will utilize Multi-Criteria Decision Analysis (MCDA) to compare the holistic value of different COVID-19 vaccines from the perspective of Emerging Markets, looking beyond clinical efficacy and safety to consider other factors such as societal value, affordability, storage / distribution requirements to evaluate which vaccine candidates bring most value to developing countries.
DESCRIPTION:
As of DEC 2020, four COVID-19 vaccines have received authorization for emergency use in some countries and several other vaccine candidates have reached late-stage trials, which may be an effective tool to manage the coronavirus pandemic by reducing the spread of COVID-19. However, budgetary constraints and challenges in storage and distribution cloud the outlook for many low-to-middle income economies, raising concerns around equitable access. In this workshop, MCDA will be used to compare the holistic value of late-stage vaccine candidates from the perspective of low-to-middle income economies. Results from this analysis can inform prioritization of vaccine candidates by agencies (governments, COVAXX) looking to procure COVID-19 vaccines for developing countries. An overview of the EVIDEM MCDA framework will be provided at the start of the session and the audience will use a real-time polling application to vote on the weight that should be given to different domains and criteria of the EVIDEM MCDA framework to make it applicable for COVID-19 vaccines in the context of developing countries. Then, speakers will conduct a comprehensive comparison of COVID-19 vaccines across different criteria (efficacy, safety, # shots, storage / distribution requirements). The audience will then use a real-time polling application to vote on the value that the different vaccine candidates offer as interventions for COVID-19 immunization. Speakers will close the session by presenting the results from the comparative MCDA for COVID-19 vaccines, contextualizing the implications for decision-makers in low-to-middle income countries, and opening up the floor for questions from the audience.
Discussion Leader
Valeria Boers Trilles, MPhil
CBPartners, New York, NY, USA
Manuel Antonio Espinoza, MD MSc PhD
Pontificia Universidad Catolica de Chile, Santiago, Chile
Manuel Espinoza is Associate Professor in the Department of Public Health and Chief of the Health Technology Assessment Unit, both at Pontificia Universidad Católica de Chile. He is also Visiting Honorary Fellow in the Centre for Health Economics at University of York and Consultant for the Interamerican Bank of Development. He has served as advisor and consultant for several public entities in Latin America as well as institutions such as the World Bank and World Health Organization. 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 has been focused on the development of methods and processes for prioritization in health care as well as applied research in health economic evaluation.
Diego Guarin, MD, MPH, MA
Merck, Kenilworth , NJ, USA
Miguel Martin De Bustamante, BA
CBPartners, San Francisco, CA, USA
Directed Acyclic Graphs in Practice: Opportunities and Challenges
Live
PURPOSE:
To discuss how to utilize the potential of directed acyclic graphs to estimate causal effects to inform health technology assessment (HTA), and how to detect and address challenges.
DESCRIPTION:
Directed Acyclic Graphs (DAGs) are a great tool to define and visualize research questions and to understand potential confounding and selection bias. They can be used to help design analyses of observational (or “real world”) data, and of randomized studies when post-randomization confounding is suspected. DAGs may be used to help define appropriate analysis methods and data that are necessary for drawing causal conclusions, and may help identify the direction of potential biases. We will illustrate (1) the potential of DAGS for assisting in the analysis of both randomized and observational studies, (2) discuss the challenges typically encountered in practice, and (3) consider how DAGs might be used by HTA decision makers. We will promote potential practical solutions, taking into account the perspectives of academic methodologists as well as the needs of decision-makers. The workshop will consist of three short presentations, interactive tests and polls, and a concluding discussion. The first speaker (Felicitas Kuehne) will present key concepts covered in DAGs in 10 minutes (conditional independencies, confounders, colliders, mediators), followed by a short interactive test (5 minutes). Application of DAGs in both randomized studies and observational (real world) data will be illustrated by the second speaker (Michael Schomaker) by means of realistic examples, both for clear and challenging contexts (15 minutes). Next, a case study will be presented, including an interactive element to ensure participation of the audience (5-10min). Then, the third speaker (Nicholas Latimer) will offer thoughts on how DAGs might be used within the HTA decision-making process (10 minutes), giving the perspective of a NICE Appraisal Committee member. The workshop will conclude with a moderated discussion involving the audience.
Discussion Leader
Felicitas Kuehne, MSc
UMIT-University for Health Sciences, Medical Informatics and Technology, Hall in Tirol, Austria
Felicitas Kuehne is a senior scientist in decision-analytic modeling and epidemiology at the Department of Public Health, Health Services Research and Health Technology Assessment at UMIT in Hall i.T., Austria. She is leading the Program on Causal Inference in Science and is the Coordinator of the HTADS course “Causal Inference for Assessing Effectiveness in Real World Data and Clinical Trials: A Practical Hands-on Workshop”. At the Institute, she conducts decision-analytic as well as epidemiological studies in several disease areas and teaches epidemiology classes at several universities in Europe as well as at international conferences.
Felicitas completed her Master of Science in Health Policy and Management at the Harvard School of Public Health in 2001. From 2001 to 2011 she worked as a consultant for pharmaceutical companies, conducting several cost-effectiveness analyses in a variety of disease areas. She joined UMIT in 2011.
Her research interests include evaluating public health intervention by applying evidence-based quantitative methods from epidemiology, comparative effectiveness research, health services and outcomes research, economic evaluation, and decision sciences. Her current substantive research focuses on identifying synergies of causal inference and decision science. The main disease areas are cardiovascular diseases, cancer, and infectious diseases including HIV/AIDS and hepatitis C.
Her studies at Harvard were partly sponsored by complementary scholarships of the Carl Duisberg Gesellschaft (1 year) and the Boehringer Ingelheim Stiftung (2 years). In 2013, Felicitas received with her team the third German research award for general medicine of the Dr. Lothar Beyer Foundation with a shared decision-making project in the cardiovascular disease area. Felicitas Kuehne was awarded two Tyrolean Science Fund (TWF) grants in 2016 and 2020 for causal inference projects.
Nick Latimer, PhD
ScHARR - University of Sheffield, Sheffield, United Kingdom
Michael Schomaker, PhD
UMIT - University for Health Sciences, Medical Informatics and Technology, Hall in Tirol, Austria
Are Child QALYs Equivalent to Adult QALYs?
Live
ISSUE:
Decision-makers often use the mantra ‘a QALY is a QALY’: a unit of health gain has the same value, regardless of who benefits. But is that mantra appropriate, and is it followed? Recent research suggests that child health state values tend to exceed values for corresponding adult health states. This has implications for the evaluation of technologies that cover both children and adults, and for clinical trials in which participants are children at baseline and transition to adulthood by the end of the trial/model. It also raises questions about social value. Does society consider child QALYs and adult QALYs to be equivalent? If not, is it appropriate to reflect age-related preferences in value sets or to introduce QALY weights/modifiers? What should these weights be based on?
OVERVIEW:
Moderator Koonal Shah will open the session by summarizing the issues. Richard Norman will explain how the issues are being tackled in Australia, where he is leading research on the comparability of child and adult QALYs with decision-makers, patient representatives and the public. Rosie Lovett will provide the perspective of an HTA agency, describing NICE’s current decision-making approach and the research that has informed NICE’s policy. She will explore how to interpret child QALYs and use them in decision-making. Vivian Reckers-Droog will present research on age-related preferences from the Netherlands, noting how current decision-making frameworks fail to reflect these preferences. She will argue that preferences for setting priorities based on age should be disentangled from preferences that feed into QALY estimates. Following the three 10-minute presentations, the remainder of the session will be reserved for panel/audience discussion, with suggestions on how to link research with the needs of policy-makers particularly welcomed. Polling questions will be administered to seek the audience’s views on key (and in some cases controversial) questions raised by the panelists.
Moderators
Koonal Shah, PhD
PHMR Ltd, London, LON, United Kingdom
Panelists
Nancy Devlin, PhD
University of Melbourne, Melbourne, VIC, Australia
Alan Lamb, PhD
National Institute for Health and Care Excellence (NICE), Manchester, United Kingdom
Vivian Reckers-Droog, MSc
PHMR Ltd, Rotterdam, ZH, Netherlands
Real World Evidence Utilization Challenges: Building an Integrated System of Rwe Data Transparency, Stakeholder Outreach and HTA Acceptance
Live
ISSUE:
The utilization of Real World Evidence as a source of valuable scientific and medical insight continues to grow. A challenge among scientists and academics generating RWE is how to present, publish and influence behavior among stakeholders who are not experts in understanding the variances and sources of RWE. This panel will discuss the barriers and opportunities in communicating RWE via strategic publication planning across a multidisciplinary stakeholder constituency. The panel will review the ISPOR RWE Transparency Initiative as a key element in expanding the understanding and utilization of RWE and discuss solutions to expand the audience and thus the influence of RWE.
OVERVIEW:
This panel will explore the need to expand the dissemination of Real World Evidence beyond HEOR circles (academics/industry) to maximize societal health and economic benefits of ‘mainstreaming’ RWE. Utilizing RWE in health decision making continues build a body of evidence as an important resource to improve patient outcomes and enhance the optimum use of healthcare resources. Each panelist will present topline issues outlined below in a 10 minute overview. Audience polling questions will be part of the program to enhance attendee interaction and education. Following the panelist’s overview the moderator will initiate a question and answer session between the three panelists and audience participants.
Moderators
Lucinda Orsini, DPM, MPH
COMPASS Pathways, Skillman, NJ, USA
Panelists
Laura Happe, PharmD, MPH
Academy of Managed Care Pharmacy, CORNELIUS, NC, USA
Jonathan Kowalski, PharmD, MS
BresMed US, Ladera Ranch, CA, USA
Robert Matheis, Phd, MA
International Society for Medical Publication Professionals, Parsippany, NJ, USA
How Can Medical Technology Researchers Engage Underrepresented Populations during a Pandemic Such As COVID-19?
Live
ISSUE:
Engaging patients and community members from underrepresented populations in research is challenging. Pandemics may exacerbate these challenges, but patient and stakeholder engagement remain feasible when best practices and authentic approaches to partnership are employed. The panel will discuss the processes used to conduct research during the COVID-19 pandemic to elicit honest and diverse views surrounding COVID-19 testing. The project serves as an exemplar for how to engage patients in research and collaborate between academic and government researchers during a pandemic.
OVERVIEW:
The United States Food and Drug Administration’s (FDA’s) Centers of Excellence in Regulatory Science and Innovation (CERSIs) are collaborations between FDA and academic institutions to advance regulatory science. The University of Maryland CERSI research endeavor aims to understand perceptions about COVID-19 testing, and to help the FDA better understand ways to protect public health. Learnings from this study may help inform efforts for monitoring COVID-19 spread as well as help tailor health messages for underrepresented populations. Working with FDA co-investigators, The PATIENTS Program at the University of Maryland School of Pharmacy in Baltimore and University of Maryland at College Park collaborators are conducting a qualitative study using virtual focus group discussions and key informant interviews to gather information from underrepresented participants in the greater Baltimore area. The populations of interest are:
African Americans across the age spectrum Individuals whose primary language is Spanish across the age spectrum Older adults regardless of race or ethnicity People with lower literacy or health literacy People with chronic conditions Asymptomatic individuals with COVID-19 test positive household member(s) Each speaker will present for 10 minutes followed by an interactive discussion with the audience. Interested stakeholders may include academics, industry, and government agencies interested in patient-centered research during a pandemic.
Moderators
C. Daniel Mullins, PhD
University of Maryland School of Pharmacy, Baltimore, MD, USA
C. Daniel Mullins, PhD is a professor and chair of the Pharmaceutical Health Services Research Department at the University of Maryland School of Pharmacy. 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), patient-centered outcomes research (PCOR), pharmacoeconomics, and health disparities research. He directs the University of Maryland PATient-centered Involvement in Evaluating effectiveNess of TreatmentS (PATIENTS) Program, which received a University of Maryland Baltimore Champion of Excellence Award. He has received funding as a Principal Investigator from the NIH/NIA, NIH/NHLBI, AHRQ, and the Patient-Centered Outcomes Research Institute (PCORI) and was the Shared Resources Core Director for the NIH-sponsored University of Maryland Center for Health Disparities Research, Training, and Outreach. He previously served as a Regular Member of the AHRQ HSR and the NCI-J Study Sections and currently serves as a Regular Member for the AHRQ HCRT Study Section. He also has chaired PCORI Study Sections.
In addition to his work on federal grants, Professor Mullins is co-editor-in-chief for Value in Health and is author/co-author of approximately 200 peer-reviewed articles and book chapters on pharmacoeconomics, outcomes research, and pharmaceutical policy and health disparities research in journals such as The American Journal of Managed Care, The American Journal of Public Health, Cancer, Chest, Health Affairs, Health Services Research, JAMA, Journal of Clinical Oncology, Pharmacoeconomics, and Social Science & Medicine. He has received an Outstanding Service Award from the Drug Information Association (DIA) and two Service Awards from the International Society for Pharmacoeconomics and Outcomes Research (ISPOR). In 2007, he received the Dr. Patricia Sokolove Outstanding Mentor Award from the University of Maryland, Baltimore campus-wide Graduate Student Association. In 2013, he was the recipient of the Dr. Daniel D. Savage Memorial Science Award, the Association of Black Cardiologists’ most prestigious annual award. Also, in 2013, he was awarded a University System of Maryland Wilson H. Elkins Professorship. In 2014, he and Robin Newhouse were named co-researchers of the year for the University of Maryland Baltimore campus.
Panelists
Jasmine Cooper, MA, PhD
University of Maryland Baltimore, Baltimore, MD, USA
Jessica Weinberg, MPP
FDA, Silver Spring, MD, USA
Chauncey Whitehead, N/A
CDW Health and Wellness Lifestyles, LLC, Baltimore, MD, USA
Spotlight Session
What Role Should HTA Play in the US Healthcare Marketplace?
Live
ISSUE: Health technology assessment (HTA) can help achieve the policy goals of ensuring affordability and encouraging innovation. The US relies mostly on privately funded HTA. However, there is an economic and healthcare rationale to supplement private HTA activities by leveraging and expanding some funding from the private sector in a public sector initiative, and to reduce the inefficient and duplicative efforts within the current HTA ecosystem. Recently, an expert panel sponsored by the USC Schaeffer Center and the Aspen Institute developed a set of six recommendations for public funding of an advisory-only HTA in the US that—when taken together—will bolster the HTA landscape and improve decision making in the healthcare marketplace.
OVERVIEW: The panel will present six recommendations for the future of HTA in the US—including public funding of an advisory-only HTA organization—and explore the challenges associated with implementing them. Lou Garrison will moderate the panel and provide the background and rationale for why an advisory-only, publicly funded HTA organization should be considered in the US as well as pose key questions for the panelists to debate: Should the US adopt a “NICE-like” approach to HTA? Should US HTA reports use agreed-upon methods, such as cost-effectiveness analysis? Should insurers be required to use HTA reports in their coverage decisions? Darius Lakdawalla will discuss why the US can benefit from a mix of privately and publicly funded HTA, and the specific roles a publicly funded HTA organization might play. Peter Neumann will highlight the challenges associated with including economic evaluation in US HTA reports and discuss the importance of broad stakeholder engagement. Gail Wilensky will discuss the importance of conducting HTA for all technologies in the US. All panelists will weigh in on why an advisory-only HTA organization is ideal for the US and how it can have a positive impact.
Moderators
Louis P. Garrison, PhD
University of Washington, Seattle, WA, USA
Lou Garrison, PhD, is professor emeritus in The Comparative Health Outcomes, Policy, and Economics Institute in the School of Pharmacy at the University of Washington, where he joined the faculty in 2004.
For the first 13 years of his career, Dr. Garrison worked in non-profit health policy at Battelle and then the Project HOPE Center for Health Affairs, where he was the Director from 1989-1992. Following this, he worked as an economist in the pharmaceutical industry for 12 years. From 2002-2004, he was vice president and head of Health Economics & Strategic Pricing in Roche Pharmaceuticals, based in Basel, Switzerland.
Dr. Garrison received a BA in Economics from Indiana University, and a PhD in Economics from Stanford University. He has more than 150 publications in peer-reviewed journals. His research interests include national and international health policy issues related to personalized medicine, benefit-risk analysis, and other topics, as well as the economic evaluation of pharmaceuticals, diagnostics, and other technologies.
Dr. Garrison was elected as ISPOR President for July 2016-June 2017, following other leadership roles since 2005. He recently co-chaired the ISPOR Special Task Force on US Value Frameworks. He was selected in 2017 by PharmaVOICE as being among “100 of the Most Inspiring People” in the industry. He recently received the PhRMA Foundation and Personalized Medicine Coalition 2018 Value Assessment Challenge First-Prize Award as lead author on a paper on “A Strategy to Support the Efficient Development and Use of Innovations in Personalized and Precision Medicine.”
Speakers
Darius Lakdawalla, PhD
USC Leonard D. Schaeffer Center for Health Policy and Economics, Los Angeles, CA, USA
Darius Lakdawalla is a widely published, award-winning researcher and a leading authority on health economics and health policy. He holds the Quintiles Chair in Pharmaceutical Development and Regulatory Innovation at the University of Southern California, where he sits on the faculties of the School of Pharmacy, the Sol Price School of Public Policy, and the Leonard D. Schaeffer Center for Health Policy and Economics, one of the nation’s premier health policy research centers.
His research has focused primarily on the economics of risks to health, the value and determinants of medical innovation, the economics of health insurance markets, and the industrial organization of healthcare markets. His work has appeared in leading peer-reviewed journals of economics, health policy, and medicine, including the American Economic Review, Quarterly Journal of Economics, Health Affairs, the Journal of Health Economics, and the New England Journal of Medicine. In addition, his work has been featured by prominent popular press outlets, such as the Wall Street Journal, National Public Radio, Forbes, and the New York Times.
Peter Neumann, ScD
Tufts Medical Center, Boston, MA, USA
Gail R. Wilensky, PhD
Project HOPE, Bethesda, MD, USA
3:00 PM - 4:00 PM
Social Hour
Live
Meet and chat with your fellow peers in HEOR and ISPOR leaders during our nightly social hours. During this hour, we will be focusing on your career story. Hear from our three Social Hour Hosts on their journey to become the professional that they are today. We invite you to join this Social Hour to share your story and learn from others.
Attendance is limited, so be sure to join early to ensure you have a spot in our Social Hours!
ISPOR Forums
HTA’s Evolving Role for More Integrated Latin American Health Systems
The COVID-19 pandemic has had a profound impact on Latin America and is accelerating widespread changes in the region’s highly fragmented healthcare landscape. Innovative, agile and timely HTA processes will be instrumental to meet the needs of rapidly changing systems and technological innovation. Through practical cases shared by expert panelists, this session will cover key areas where HTA is evolving for the region, including with novel HTA processes for high cost therapies including gene therapies, the deployment of HTA in hospital and other care settings, and how digital transformation is bringing real world data (RWD) into greater prominence into decision-making.
Moderators
Stephen Stefani, MD, MBA
UNIMED Central RS, Porto Alegre, Brazil
Speakers
Cesar Alberto Cruz Santiago, MSc, PhD, MD
Ministry of Health, Mexico City, MEX, Mexico
Veronica Laura Sanguine, MD
RedARETS, Ciudad Autonoma de Buenos Aires, Argentina
Stephen Stefani, MD, MBA
UNIMED Central RS, Porto Alegre, Brazil
How to Critically Appraise the Quality and Risk of Bias in Systematic Reviews of Cost and Cost-Effectiveness Outcomes
Overview: Speakers will present the main criteria for critical appraisal of systematic reviews of cost and cost-effectiveness outcomes (SR-CCEOs) and introduce the ISPOR CrIteria for Cost (-Effectiveness) Review Outcomes (CiCERO) Checklist. CiCERO comprises the task force’s recommendations and 13 signaling questions to consider when evaluating the risk of bias in SR-CCEOs, as well as the quality of their conduct and reporting. Purpose: There has been a significant increase in the number of SR-CCEOs. The differences in clinical and health settings, heterogeneity in applied methods, and reporting make it challenging to interpret the findings of SR-CCEOs. An analysis of guidelines on conducting and reporting SR-CCEOs resulted in the identification of multiple disagreements in these recommendations, hence the motivation behind the ISPOR Good Practices Task Force Report. Recommendations are organized in six stages:
planning and development search in SR-CCEOs study selection and eligibility critical appraisal of the included studies data extraction and synthesis presentation and reporting. Description: This forum will give participants clear guidance and experience on using the 3 different CiCERO versions for reviews that:
summarize cost and cost-effectiveness outcomes focus on methods of cost and cost-effectiveness studies use AMSTAR-2 as the main tool. The forum will also facilitate discussion of the most controversial aspects of evaluation of quality and risk of bias in systematic reviews, such as obligatory components in SR-CCEOs’ aims, search restrictions and transferability, and approaches to synthesis in SR-CCEOs. It will include a hands-on training using the CiCERO checklist to appraise an example SR-CCEO. This presentation will benefit researchers, payers, assessors, regulators and health technology producers. Developed by the ISPOR Systematic Reviews with Cost- and Cost-effectiveness Outcomes Good Practices Task Force.
Speaker
Candyce Hamel, MPH
Canadian Association of Radiologists, Ottawa, ON, Canada
Lena Mandrik, PhD
University of Sheffield, Sheffield, YOR, United Kingdom
JL (Hans) Severens, PhD
Bristol-Myers Squibb Foundation, Rotterdam, ZH, Netherlands
Thu May 20
10:00 AM - 11:00 AM
Breakout Session 4
What Makes Real World Evidence (RWE) Believable for Regulatory Decisions? Is It Real, Chance or Confounding?
Live
PURPOSE:
To discuss considerations for improving believability of real-world evidence at the protocol stage .
DESCRIPTION:
Regulators are increasingly considering real-world evidence (RWE) of medical products to support new indications or expand labeling. FDA issues their RWE guidance in December 2021. In a 2019 Duke-Margolis workshop, a panelist suggested that health authorities seek ‘believability’ of RWE in order to make informed decisions. Foundational to believability are well-articulated objectives and study designs that are pre-specified in registered protocols. In medical product studies, design elements such as new-user design, active comparators and propensity score approaches help minimize confounding and other biases. A pre-specified analytic plan, including sensitivity analyses and bias quantification techniques, also improves believability. Importantly, less reliance on p-values, which may be driven by larger sample sizes typical in real-world data (RWD), and greater use of confidence intervals and distributions aide interpretation. Replication in other settings pre-specified at protocol stage can also enhance believability. To contribute to believability, information bias can be minimized by ensuring that RWD are complete, reliable, valid and relevant for the specific research objective (i.e., fit-for-purpose). We will discuss protocol-stage influences on believability and how larger effect sizes, fit-for-purpose data quality for the specific research question, and analytic techniques help minimize risk of various biases, thereby maximizing believability and robustness of results.
Introduction (5 min): Dr. Brodovicz Dr. Chang will give the regulatory perspective on RWE to Support Decisions for Medical Devices (10 min) Dr. Leclerc will propose real-world data quality aspects important in RWE research (10 min) Dr. Assimon will present on study designs and analytic features that minimize bias (10 min) Dr. Girman will show that effect size, study design features and data quality drive credibility and believability, recommending steps for triangulating effect estimates (10 min) Interactive panel discussion and Q&A (15 min)
Discussion Leader
Magdalene Assimon, PharmD, PhD
University of North Carolina, Durham, NC, USA
Cynthia Girman, DrPH, FISPE
CERobs Consulting, LLC, Wrightsville Beach, NC, USA
Jacinthe Leclerc, RN, PhD
Université du Québec à Trois-Rivières, Levis, QC, Canada
The Many Tentacles of Estimands: Patient-Reported Outcomes, Real-World Evidence, Health Economics and Outcomes Research, and Beyond
Live
PURPOSE:
The EMA recently adopted the ICH E9 R1 document on estimands. We discuss how to apply the estimand framework across different aspects of outcomes research, including PROs and RWE. Participants will become familiar with estimands and be able to critically evaluate and apply strategies for constructing estimands for different endpoints.
DESCRIPTION:
The newly approved ICH E9 R1 document on estimands is an effort to align trial objectives, design and analysis, improve communication, and clarify interpretation of the effect of treatment. As ICH member organizations adopt the document, understanding and constructing estimands will be critical going forward. This workshop will cover a) how concepts from causal inference can help with understanding estimands, b) designing and communicating an estimand strategy for PRO endpoints in challenging trial settings, c) a regulatory perspective on key considerations for estimands, and d) how the estimand framework can be applied for RWE. Dr. Gutman will discuss the potential outcomes framework for causal inference and show how it relates to estimands and can facilitate the interpretation of treatment effects. Dr. Roydhouse will provide an example using estimands for PRO endpoints in trials with seriously ill patients and discuss strategies for PRO analysis and communicating PRO results. Dr. Mishra-Kalyani will describe regulatory thinking regarding estimands and considerations such as treatment discontinuation. Dr. Manson will discuss the challenges for trial planning and communication using estimands in RWE and HEOR, and strategies for trial design and analysis that incorporate aspects such as crossover and immature data for secondary endpoints (e.g. survival). Each presentation will be 10 minutes to allow time for discussion. Online polling will be used encourage audiences to share their experiences and questions. The workshop will be beneficial to outcomes researchers and industry and patient stakeholders interested in understanding recent developments in regulatory evaluation, trial design, PROs and RWE.
Discussion Leader
Roee Gutman, PhD
Brown University School of Public Health, Pawtucket, RI, USA
Stephanie Manson, PhD
Novartis, East Hanover, NJ, USA
Pallavi Mishra-Kalyani, PhD
FDA, Silver Spring, MD, USA
Jessica Roydhouse, PhD
Menzies Institute for Medical Research, Hobart, TAS, Australia
Methodological Challenges in Assessing the Health Economic Implications of International Reference Pricing: Measuring its Impact on Patient Access and Pharmaceutical Innovation
Live
PURPOSE:
To demonstrate the methodological challenges and pitfalls in assessing the impact of International Reference Pricing on patients and pharmaceutical innovation, and to provide guidance for future research in the topic.
DESCRIPTION:
International Reference Pricing (IRP) is applied by a large number of countries and its use is growing: using the prices for the same product from other countries to set or negotiate initial pharmaceutical prices for price regulation at launch or their revision later. Several authors investigated the intended and unintended consequences of IRP. All were facing numerous challenges in conducting their research including: limited availability and reliability of price sources; ambiguity when trying to match the same product across all countries; normalizing the prices to the same level – e.g., ex-factory; choosing the most appropriate foreign exchange rate for conversion; picking the IRP basket and algorithm to use for so-called “soft rule” countries; assessing the patient access to the treatment; and assessing the impact on innovation. The Discussion Leaders have extensive academic, publication and practical experience in the area, from different perspectives: as author on the economic impact of IRP from the US; as former WHO advisor and leader of IRP research from a large European country; as author of IRP impact on lower-income countries; as former global industry professional. Coming from these different perspectives the Discussion Leaders will explore the methodological challenges in identifying and quantifying the consequences of IRP on patient access and on pharmaceutical innovation, including if the US were to implement such a policy. They will bring examples and cases from their own research to illustrate these challenges and propose approaches to overcome the methodological challenges inherent in such research and the complications it creates in understanding its impact.
Discussion Leader
Jaime Espin, PhD
Andalusian School of Public Health, Granada, GR, Spain
Louis P. Garrison, PhD
University of Washington, Seattle, WA, USA
Lou Garrison, PhD, is professor emeritus in The Comparative Health Outcomes, Policy, and Economics Institute in the School of Pharmacy at the University of Washington, where he joined the faculty in 2004.
For the first 13 years of his career, Dr. Garrison worked in non-profit health policy at Battelle and then the Project HOPE Center for Health Affairs, where he was the Director from 1989-1992. Following this, he worked as an economist in the pharmaceutical industry for 12 years. From 2002-2004, he was vice president and head of Health Economics & Strategic Pricing in Roche Pharmaceuticals, based in Basel, Switzerland.
Dr. Garrison received a BA in Economics from Indiana University, and a PhD in Economics from Stanford University. He has more than 150 publications in peer-reviewed journals. His research interests include national and international health policy issues related to personalized medicine, benefit-risk analysis, and other topics, as well as the economic evaluation of pharmaceuticals, diagnostics, and other technologies.
Dr. Garrison was elected as ISPOR President for July 2016-June 2017, following other leadership roles since 2005. He recently co-chaired the ISPOR Special Task Force on US Value Frameworks. He was selected in 2017 by PharmaVOICE as being among “100 of the Most Inspiring People” in the industry. He recently received the PhRMA Foundation and Personalized Medicine Coalition 2018 Value Assessment Challenge First-Prize Award as lead author on a paper on “A Strategy to Support the Efficient Development and Use of Innovations in Personalized and Precision Medicine.”
Andras Incze, PhD, MBA
Baden-Wuerttemberg Cooperative State University Germany & Akceso Advisors AG, Basel, BS, Switzerland
Zoltan Kalo, PhD
Syreon Research Institute, Budapest, PE, Hungary
Zoltán Kaló is a professor of Health Economics at the Center for Health Technology Assessment of Semmelweis University in Budapest, Hungary. Before moving to Semmelweis University in July 2019 he was the founder and co-director of an international master program in Health Policy, Planning, and Financing at Eötvös Loránd University (ELTE).
Dr. Kaló is also the founder and leader of Syreon Research Institute, an international research corporation specializing in health policy, health economic modeling, and technology assessment.
He has 25 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 and global healthcare corporations. He is a Scientific Committee member of the Innovative Medicines Initiative 2 Joint Undertaking (IMI2 JU). He was a director of ISPOR between 2012-2014, and the chair of ISPOR Central and Eastern European Network Executive Committee between 2013-2015.
Éva Kiss, MSc
Akceso Advisors AG, Basel, BS, Switzerland
Science, Art or Arbitrariness? Evaluating the Risk of Treatment Effect Waning for Novel Oncology Therapies
Live
PURPOSE:
To discuss how treatment effect waning is dealt with when modelling the cost-effectiveness of novel immune-oncology therapies in health technology assessments (HTAs) and provide recommendations regarding good practices.
DESCRIPTION:
Novel immuno-oncology therapies are demonstrating durable responses, extending survival across multiple cancer types by stimulating the body’s own immune system.
With longer term data, survival plateaus have been observed, although the evidence generation to demonstrate long-term effects is ongoing. In HTAs, the cost-effectiveness assessment of these therapies requires modelling their lifetime effect on patients, usually based on immature trial data. HTA agencies commonly request alternative hypothetical scenarios on the duration and size of the treatment effect over time, as these are key cost-effectiveness drivers. Since methodological consensus is lacking, manufacturers and assessment groups have frequently adopted non-evidence-based, arbitrary assumptions regarding treatment effect waning, while HTA agencies have considered conservative approaches in decision making. For this workshop:
Raquel Aguiar-Ibáñez will present current HTA requirements regarding treatment effect waning, and how manufacturers and HTA agencies have dealt with uncertainty around immuno-oncology therapies’ long-term effectiveness. Professor Gianluca Baio will discuss the role of supportive datasets and the advantages of adopting a Bayesian framework to account for the use of real-world evidence and clinical expert judgement to better reflect expected long-term benefits. Dawn Lee will focus on how uncertainty around long-term treatment effects might be incorporated into economic analyses depending on the modelling framework (e.g. for partitioned survival versus state transition models) and the potential to collect additional data via managed access arrangements. Nick Latimer will provide a decision-maker’s perspective on types of evidence that may be used to support long-term efficacy assumptions.
Discussion Leader
Raquel Aguiar-Ibáñez, MSc
Merck Canada Inc., Toronto, ON, Canada
Gianluca Gianluca Baio, PhD
UCL, London, United Kingdom
Nick Latimer, PhD
ScHARR - University of Sheffield, Sheffield, United Kingdom
Dawn Lee, MMath, MSc
BresMed Health Solutions Ltd., Sheffield, DBY, United Kingdom
Drop Dead: Is It Time to Remove 'Dead' from Health State Valuation?
Live
ISSUE:
By convention, values for generic preference-based measures (such as the EQ-5D) are anchored at 1 = 'full health' and 0 = 'dead'. Consequently, stated preference methods used to value health states often refer to 'death' and result in negative values for states 'worse than dead'. Evidence shows that including 'dead' in health state valuation could bias responses. The need to identify negative values creates methodological difficulties that have not been fully resolved. An alternative lower anchor (e.g. 'worst health state imaginable') could be used in place of 'dead'. However, this would constitute a dramatic shift in methodology and could have profound implications for the valuation of health states.
OVERVIEW:
The panel will debate the motion that 'dead' should be removed from health state valuation. Jacoline Bouvy will introduce the issue, describing the role of 'dead' and identifying related challenges for health technology assessment and appraisal decisions. Audience members will vote on whether they support the motion. Liv Ariane Augested will provide an overview of relevant empirical work, outlining evidence demonstrating the problems caused by the use of 'dead' as an anchor. Chris Sampson will argue in favour of the 'drop dead' motion, describing the origins of the use of 'dead', arguing that it lacks foundations in either utility theory or applied cost-effectiveness analysis. He will suggest some practical alternatives. Bram Roudijk will argue in favour of maintaining 'dead', as a practical basis for estimating quality-adjusted life years. Bram will describe the 'zero condition' and its implications for health state valuation, highlighting the value of identifying health states as being 'worse than dead'. Jacoline will summarise the arguments and outstanding questions. At the end of the session, 15 minutes will be allocated for audience discussion, including a series of polls and a vote on the overall motion to 'drop dead'.
Moderators
Jacoline Bouvy, PhD
National Institute for Health and Care Excellence, London, United Kingdom
Panelists
Mathias Barra, PhD
Akershus University Hospital HF, LØRENSKOG, Norway
Bram Roudijk, PhD
EuroQoL Research Group, Rotterdam, ZH, Netherlands
Christopher Sampson, PhD
Office of Health Economics, London, LON, United Kingdom
How Much Do Experts’ Opinions Matter When Data Is Sparse? Value of Expert Elicitation Methods in Addressing COVID-19 Pandemic Challenges
Live
ISSUE:
The COVID-19 pandemic has altered the health care system and created a “new normal” situation comprising of challenges, of which few include- need for contactless care, fear of contagion, health system capacity issues, and loss of insurance coverage. Addressing these new challenges along with existing ones, many professional organizations and societies might have to re-evaluate their strategies around treatment and management guidelines and formulary decisions. Given that COVID-19 related data is limited, expert elicitation methods including the Sheffield Expert Elicitation Framework (SHELF) and Delphi are important study methodologies that can help systematically address these challenges by eliciting expert opinions and developing consensus statements.
OVERVIEW:
The panel will debate the importance of using expert elicitation methods to address COVID-19 pandemic related challenges. Specifically, the panel will discuss the following questions:
What are the implications of the COVID-19 pandemic on existing treatment and management guidelines and formulary decisions? How and what expert elicitation methods help develop consensus statements to address these challenges? What are advantages and limitations of expert elicitation methods? Dr. Veeranki, a clinical epidemiologist with expertise in expert elicitation methods will moderate this panel. He will outline the importance of expert elicitation methods, their use in the past and highlight recent research that used these methods to address uncertainties in rare diseases. Dr. O’Hagan will discuss the development of SHELF and its application in quantifying expert opinions. Dr. Fick will describe how expert elicitation methods could be used to inform clinical decision-making and research, providing examples during COVID-19. Dr. Latimer will debate how these methods could be used to inform health technology assessments and formulary decisions during COVID-19. The panel will begin with Dr. Veeranki’s 5-minute introduction, followed by a 10-minute talk by each panelist, and a 10-minute moderated group discussion along with a 15-minute Q&A with the audience.
Moderators
Phani Veeranki, MD, DrPH
Optum Life Sciences, CYPRESS, TX, USA
Panelists
Laura Bojke, PhD
University of York, Gladstone, NJ, USA
Donna Fick, PhD, RN
Penn State College of Nursing, Boalsburg, PA, USA
Anthony O'Hagan, PhD
University of Sheffield, Nottingham, NTT, United Kingdom
10:00 AM - 12:45 PM
Poster & Exhibit Viewing
Live
Stop in to learn about the latest technology, services, and devices and to connect one-on-one with exhibitors and sponsors during our Exhibit Viewing Hours. Be sure to reserve some time to view the latest research in HEOR through our virtual poster gallery. Browse, comment, and discuss our posters at any time while our virtual platform is active.
11:30 AM - 12:30 PM
Breakout Session 5
The Patient Voice in US Drug Value Assessment
Live
ISSUE:
The Institute for Clinical and Economic Review (ICER) is becoming increasingly influential in drug pricing and reimbursement in the United States. Similarly to many HTA agencies around the world, such as NICE and CADTH, ICER has a program to encourage and empower patient organizations to participate in assessment of health technologies. Indeed, there are a number of examples where the understanding gained from patient organization input has been incorporated into ICER’s drug value assessments. However, questions have been raised about the independence of some patient organization contributions which, if true, could potentially undermine their credibility. Whether it is possible for ICER and other decision-makers, such as insurance providers, to identify and manage potentially problematic conflicts of interest without excluding genuine, valuable patient input remains under debate.
OVERVIEW:
Katie Hamilton will moderate and provide an overview of processes to incorporate patient input into health technology assessments, including how this is done in a range of healthcare systems worldwide. <Patient organization panelist> will outline the value that industry support (financial and non-financial) can bring and discuss how patient groups can ensure that such support does not compromise their advocacy efforts and engagement with decision-makers. Yvette Venable will explain how ICER views industry support of patient organizations, and how uncertainty regarding these relationships may reduce the impact of patient organization input, ultimately undermining their advocacy efforts. Finally, <insurance perspective> will explain how insurance providers can consider patient organization evidence in their decision making, including how potential conflicts of interests may be managed. Finally, the group will debate the merits of various approaches to improving how the voices of patients can be fairly incorporated into the drug value assessment and coverage process in the US.
Moderators
Katherine Hamilton, MA (Cantab) PGCert (Health Economics)
Costello Medical Consulting Ltd, Boston, MA, USA
Panelists
Sneha Dave, BS
Health Advocacy Summit, Greenwood, IN, USA
Thomas Hubbard, MPP
Network for Excellence in Health Innovation, Natick, MA, USA
Yvette Venable, BA
Institute for Clinical and Economic Review, WALTHAM, MA, USA
Accounting for Preference Heterogeneity in Stated Preference Studies: Moving from Methods to Practice to Decision Making – the Issue of Developing Useful Guidance
Live
ISSUE:
Stated-preference methods typically quantify trade-offs that patients, or other stakeholders are willing to make between treatment aspects. While many health preference studies focus on the interpretation of averages, understanding how preferences differ between subjects can be valuable for decision making. For example, the FDA guidelines on patient preference information (PPI) state: “PPI may help identify a subgroup of patients [...] who may consider the benefit-risk profile of a medical intervention favorable [...].” A range of methods has been developed to account for such preference heterogeneity. However, the increasing number of complex approaches and the technical as well as practical issues surrounding them, can be challenging for both practitioners and decision makers. This concern is supported by a recent survey conducted by ISPOR’s Health Preference Research SIG, in which 60 out of 70 (92%) surveyed preference researchers stated that more guidance is needed.
OVERVIEW:
The panel will debate the relevance of accounting for preference heterogeneity, practical considerations and challenges. The aim will be to identify how guidance can address the needs of different stakeholders, including decision makers. Sebastian Heidenreich will moderate the panel and contextualize the discussion using a recent survey conducted by a working group of the ISPOR Health Preference Research SIG. Marco Boeri will discuss the current state of practice in accounting for preference heterogeneity presenting findings from a systematic review of the health discrete choice literature that was conducted by the ISPOR working group. Ellen Janssen will outline the relevance of understanding preference heterogeneity for decision making and outline gaps in current practice. Stephane Hess will give an overview of best-practices, research needs, and challenges in analysis, interpretation and reporting of preference heterogeneity. All panelists will make clear recommendations for developing guidance that will be discussed with the audience via interactive polls and a Q&A session.
Moderators
Sebastian Heidenreich, PhD
Evidera, London, LON, United Kingdom
Panelists
Marco Boeri, PhD
RTI Health Solutions, Belfast, United Kingdom
Prof. Stephane Hess, PhD
University of Leeds, Leeds, United Kingdom
Ellen Janssen, PhD
The Janssen Pharmaceutical Companies of J&J, Baltimore, USA
One of These Years Is Not like Others: Real-World Data in the Times of COVID-19. Did the Pandemic Change the Way We Generate and Interpret Real-World Evidence?
Live
ISSUE:
The effects of COVID-19 on the health care-seeking behaviors and provisions have resulted in increased use of telemedicine, delays in routine care, prescribing behaviors changes, and variation in treatment availability and preferences. For Real-World Evidence (RWE) studies including data from 2020 and beyond, do the effects of COVID-19 have impacts on data quality, selection bias, treatment patterns, or health resource utilization? And if so, how should these challenges be mitigated?
OVERVIEW:
This panel will debate whether COVID-19 has had and will have a continued impact on how RWD is used and analyzed. Jennifer Graff will moderate the panel and introduce questions for the debate. For example, what are the effects of telehealth on the quality of data collected during the pandemic? Who is seeking care during the pandemic, and will the results be generalizable? Will we see higher rates of adverse health outcomes due to delays in seeking care or changes in access to treatments? Matt Reynolds will provide an overview of the changes observed in health resource use and outcomes. Jeffrey Brown will discuss the potential areas of concern with RWD and RWE generation prior to COVID-19 and how these remain similar amidst the COVID-19 pandemic. Jacqueline Corrigan-Curay will outline specific areas researchers should consider when using data that coincides with COVID-19. Discussion and audience questions will be encouraged.
Moderators
Jennifer Graff, PharmD
National Pharmaceutical Council, Kensington, MD, USA
Panelists
Jeffrey Brown, PhD
Harvard Pilgrim Health Care Institute and Harvard Medical School, Needham, MA, USA
John Concato, MD, MS, MPH, BEng
FDA, Silver Spring, MD, USA
Matthew Reynolds, PhD
IQVIA, Durham, NC, USA
When Is a QALY Not a QALY? Challenges in Introducing and Applying Modifiers in HTA Decision-Making.
Live
ISSUE:
HTA agencies are increasingly challenged when applying the principle of maximizing health gains, regardless of whom they accrue to, and of the innovation that delivers them. Evidence shows that health gains are not of equal societal value. For example, society might be willing to prioritize patients that are worse off. HTA agencies have introduced factors to consider in addition to cost-effectiveness, applying quantitative and qualitative approaches. In other cases, decision-making criteria remain opaque. There is a need to develop decision-making frameworks that are aligned with societal preferences, applied consistently, and transparent.
OVERVIEW:
The panel will provide an overview of modifiers currently considered in global HTA and approaches for their incorporation with cost-effectiveness evidence. The focus will be on two agencies, the Institute for Clinical and Economic Review (ICER) in the US and the National Institute for Health and Care Excellence (NICE) in England. ICER updated its value assessment framework in October 2020, with major changes to its approach to contextual considerations. NICE is currently undertaking a methods review, publishing a consultation in November 2020 on proposed changes, including introducing severity as a modifier. Adrian Towse will moderate the session and introduce the topic. Steve Pearson will discuss ICER’s experience in applying ‘other benefits, disadvantages and contextual considerations’, including how the new deliberation and voting processes have worked in practice, and lessons to date. Pilar Pinilla will present how NICE approached the topic of modifiers in its recent methods review and how the case for change was reached for modifiers, such as severity. Martina Garau will put the ICER and NICE approaches in the context of approaches proposed in the literature for capturing multiple criteria in decision-making; exploring the extent to which these are used elsewhere; exploring whether HTA can benefit from improving quality and consistency in decision making.
Moderators
Adrian Towse, MA, MPhil
Office of Health Economics, London, United Kingdom
Professor Adrian Towse is director emeritus and senior research fellow of the Office of Health Economics in the UK. Adrian’s current research includes incentives for new drugs and vaccines to tackle Antimicrobial Resistance, the use of 'risk-sharing' arrangements between healthcare payers and pharmaceutical companies, including value-based pricing approaches; the economics of pharmacogenetics for healthcare payers and the pharmaceutical industry; economic issues that affect both R&D for and access to treatments for diseases prevalent in the developing world; the economics of medical negligence; and measuring productivity in healthcare.
A visiting professor at the London School of Economics and a senior researcher at the Nuffield Department of Population Health at the University of Oxford, Adrian also has been a visiting professor at the University of York. For ten years, he served as the non-executive director of the Oxford Radcliffe Hospitals NHS Trust, one of the UK’s largest hospitals. Adrian was president of ISPOR, for the 2014-15 term.
Adrian joined the OHE in 1993 and served as director for 25 years. He holds an MA (Hons) in Politics, Philosophy and Economics from Keble College, Oxford; an MPhil in Management Studies from Nuffield College, Oxford, and the Oxford Centre for Management Studies; and is a member of the Chartered Institute of Management Accountants.
Panelists
Martina Garau, MSc
Office of Health Economics, London, LON, United Kingdom
Steven Pearson, MD, MSc
Institute for Clinical and Economic Review, Boston, MA, USA
Pilar Pinilla, MSc
National Institute for Health and Care Excellence, London, United Kingdom
Introduction to Machine Learning for Health Economic and Outcomes Research
Live
PURPOSE:
The availability of data to support health economics and outcomes research (HEOR) is increasing at an exponential rate. In the age of big data, cutting-edge machine learning algorithms, which have been tremendously successful in transportation and retail industry (e.g., Uber and Amazon) can play a promising role in HEOR. The objective of this workshop is to introduce the basics and key concepts in machine learning (ML), how machine learning can influence the field of HEOR, what are some of the potential pitfalls of machine learning and applications from health economics outcomes research that utilize machine learning.
DESCRIPTION:
In this workshop, we will introduce the current landscape in HEOR through the lens of the state-of-the-art modern machine learning techniques. In particular, we will start with reviewing basics and key concepts of modern machine learning techniques for descriptive, predictive, prescriptive analytics. We will proceed with discussing how ML differs from statistical learning and modeling and provide an overview of selected ML algorithms such as deep neural networks, and random forests. We will then illustrate state-of-the-art applications of ML to HEOR– this will include use of ML for augmenting health economic modeling for cost-effectiveness analysis, developing ML-driven Apps or online tools for running complex models in real time, and synthetic data generation using ML. We will conclude by discussing potential pitfalls of ML in health outcomes research and promising future research directions. This workshop will be primarily geared towards the interested audience from the pharmaceutical industry concerned with health economic evaluation.
Discussion Leader
Turgay Ayer, Ph.D.
Georgia Institute of Technology, Atlanta, GA, USA
Jag Chhatwal, Ph.D.
Harvard University, Boston, MA, USA
Selin Merdan, Ph.D.
Value Analytics Labs, Kennesaw, GA, USA
11:30 AM - 12:45 PM
Spotlight Session
Public Health Economics – Can We Model It?
Live
Health care and the broader economy interact of course, but in normal times we ignore these interrelationships, hoping it is safe to do so. During a pandemic, this is no longer a valid simplification but incorporating these aspects into our models is a relatively new area that presents important methodological aspects, particularly regarding the modeling approach and resulting data requirements. Should we model at an even more macro level than normal or delve into the extreme detail of agent-based simulation? Is there a reasonable middle ground? This session will present work from experts that are taking on these challenges in the context of COVID-19.
Moderators
J. Jaime Caro, MDCM, FACP, FRCPC
Evidera, McGill University, London School of Economics, Lincoln, MA, USA
Jaime Caro, Chief Scientist at Evidera and Professor of Epidemiology and of Medicine at McGill University and Professor in Practice at London School of Economics. He pioneered the use of DES, developed the Simulated Treatment Comparison approach and proposed the efficiency frontier as an alternative to cost/QALY. Recently, he has developed a new modelling technique, DICE simulation, tailored to problems in Health Technology Assessment.
Speakers
Martin Eichenbaum
Center for International Macroeconomics, Northwestern University, Evanston, IL, USA
Martin Eichenbaum is the Charles Moskos Professor of economics and the co-director of the Center for International Economics at Northwestern University. He is a fellow of the American Academy of Arts and Sciences, a fellow of the Econometric Society, a Research Associate of the NBER and an International Fellow of the C.D. Howe Institute. In addition, he is a Director of the Bank of Montreal (BMO) as well as the Aaron Institute for Economic Policy at the Interdisciplinary Center Herzlia. He is currently the co-editor of the NBER Macro Annual. He was co-editor of the American Economic Review as well as an associate editor of the Journal of Monetary Economics, the American Economic Journal - Macro, and the Journal of Money, Credit and Banking. He is currently a consultant to the Federal Reserve Bank of San Francisco, the IMF and the European Commission.
Warwick McKibbin
Australian National University (ANU), Canberra, Australia
Anna Vassall, PhD
London School of Hygiene and Tropical Medicine, London, United Kingdom
