Regulatory and Payer Evaluation of Real-World Evidence
A Roundtable Discussion
Thursday, October 30, 2025 | 9:00AM - 1:30PM ET
In-Person and Online
Joseph B. Martin Conference Center at Harvard Medical School and Zoom
Background
As methods of real-world evidence (RWE) research have rapidly developed and RWE is increasingly considered by regulators and health technology assessment (HTA) bodies, a wealth of recommendations on how to assess RWE studies have been published. Yet there remains some uncertainty on how decision-makers review the scientific validity of RWE submissions and inconsistencies in their review remain. This event will bring together regulators, HTA bodies and other stakeholders, to review the state of RWE submissions for labeling and coverage decision-making and how they are reviewed for their scientific merit.
Meeting Information
For in-person attendees: The roundtable will be held in the Pechet Family Conference Room, Joseph B. Martin Conference Center, Harvard Medical School, 77 Avenue Louis Pasteur, Boston, MA 02115. This room is located on the first floor. Breakfast will be served from 8:00-9:00AM ET, with lunch served after the event beginning at 1:30PM ET
For online attendees: The roundtable will be hosted via Zoom. Webinar details will be shared with attendees one week before the event.
In-person and online attendees are encouraged to submit questions during the event using Slido. To submit your questions during each session, please join the Q&A at slido.com, meeting code #6966426.
Please contact Shruti Belitkar or Matt Martin with registration questions or if you require any accommodations to participate fully in the event.
Pre-Reading Materials
- Candore G, Martin C, Mills MJ, et al. FRAME: Framework for Real-World Evidence Assessment to Mitigate Evidence Uncertainties for Efficacy/Effectiveness – An Evaluation of Regulatory and Health Technology Assessment Decision Making. Clin Pharmacol Ther. 2025;118(3):649-661. doi:10.1002/cpt.3713
- Jaksa A, Arena PJ, Hanisch M, Marsico M. Use of Real-World Evidence in Health Technology Reassessments Across 6 Health Technology Assessment Agencies. Value Health. 2025;28(6):898-906. doi:10.1016/j.jval.2025.02.012
- Jaksa A, Louder A, Maksymiuk C, et al. A Comparison of 7 Oncology External Control Arm Case Studies: Critiques From Regulatory and Health Technology Assessment Agencies. Value Health. 2022;25(12):1967-1976. doi:10.1016/j.jval.2022.05.016
- Bykov K, Jaksa A, Lund JL, et al. APPRAISE: A Tool for Appraising Potential for Bias in Real-World Evidence Studies on Medication Effectiveness or Safety. Value Health. Published online August 6, 2025. doi:10.1016/j.jval.2025.07.024
Agenda
All times listed below are in Eastern Time (ET).
9:00-9:10 AM
Welcome
Sebastian Schneeweiss, Professor of Medicine and Epidemiology, Harvard Medical School; Chief, Division of Pharmacoepidemiology and Pharmacoeconomics, Brigham and Women's Hospital
9:10-9:20 AM
Opening Remarks
Aaron Kesselheim, Professor of Medicine, Harvard Medical School; Director, Program On Regulation, Therapeutics, And Law (PORTAL), Division of Pharmacoepidemiology and Pharmacoeconomics, Brigham and Women's Hospital
9:20-10:30 AM
FRAME: Framework for Real-World Evidence Assessment to Mitigate Evidence Uncertainties for Efficacy / Effectiveness
Presenters:
- Mackenzie Mills, CEO and Founder, HTA-Hive; Associate Director, Medical Technology Research Group, London School of Economics and Political Science
- Gianmario Candore, Partnerships Senior Manager, Bayer AG
Discussants:
- Marie Bradley, Senior Advisor, Real-World Evidence Analytics, Office of Medical Policy, Food and Drug Administration (FDA), United States
- Karl Broich, President, Federal Institute of Drugs and Medical Devices (BfArM), Germany
- Wim Goettsch, Special Advisor in Health Technology Assessment, National Health Care Institute (ZIN), the Netherlands; Professor and Chair on HTA of Pharmaceuticals, Utrecht University
- Niklas Hedberg, Chief Pharmacist, Dental and Pharmaceutical Benefits Agency (TLV), Sweden
- Pall Jonsson, Program Director for Data and Real-World Evidence, National Institute for Health and Care Excellence (NICE), United Kingdom
- Guanqiao Li, Principal Investigator and Associate Professor, Vanke School of Public Health, Tsinghua University, China
- Álmath Spooner, Head of Europe Regulatory Policy and Intelligence, AbbVie; Vice Chair, Integrated Evidence Generation and Use (IEGU) Working Group, European Federation of Pharmaceutical Industries and Associations (EFPIA)
Moderator:
- Claire Martin, Global Policy Lead, Real World Evidence Centre of Excellence, Bayer AG
10:30-11:30 AM
Evaluating RWE Study Quality: How Internal Validity Assessments Vary by Use Case and Agency
Presenter:
- Ashley Jaksa, Principal, Research Partnerships, Target RWE
Discussants:
- Foluso Agboola, Senior Vice President of Research, Institute for Clinical and Economic Review (ICER)
- Steve Farmer, Senior Partner and Co-Owner, ABIG Health; Associate Professor of Medicine & Health Policy and Management, The George Washington University; Former Chief Strategy Officer for Coverage, Centers for Medicare and Medicaid Services (CMS), United States
- Donna Rivera, Vice President, Life Sciences, Datavant; Former Associate Director of Pharmacoepidemiology and Director, Oncology Real World Evidence Program, Food and Drug Administration (FDA), United States
- Yoshiaki Uyama, Associate Executive Director, Pharmaceuticals and Medical Devices Agency (PMDA), Japan
Moderator:
- Susana Perez-Gutthann, Senior Vice President, Global Head Epidemiology, RTI Health Solutions, an ENCePP Research Center
11:45 AM-12:15 PM
APPRAISE: A Tool for Appraising Potential for Bias in Real-world Evidence Studies
Presenter:
- Katsiaryna Bykov, Assistant Professor of Medicine, Harvard Medical School; Pharmacoepidemiologist, Division of Pharmacoepidemiology and Pharmacoeconomics, Brigham and Women's Hospital
Discussants:
- Foluso Agboola, Senior Vice President of Research, Institute for Clinical and Economic Review (ICER)
- Anne d'Andon, Independent Consultant; Former Head, Drug Evaluation Service, National Authority for Health (HAS), France
- Rimma Berenstein, Deputy Head, Pharmaceuticals Department, The Federal Joint Committee (G-BA), Germany
- Pall Jonsson, Program Director for Data and Real-World Evidence, National Institute for Health and Care Excellence (NICE), United Kingdom
Moderator:
- Mark Sculpher, Professor of Economics and Department Head, Centre for Health Economics, University of York; Co-Director, Policy Research Unit in Economic Evaluation of Health and Care Interventions (EEPRU)
12:15-1:30 PM
Recommendations and the Path Forward
Presenter:
- Mark Sculpher, Professor of Economics and Department Head, Centre for Health Economics, University of York; Co-Director, Policy Research Unit in Economic Evaluation of Health and Care Interventions (EEPRU)
Discussants:
- Foluso Agboola, Senior Vice President of Research, Institute for Clinical and Economic Review (ICER)
- Anne d'Andon, Independent Consultant; Former Head, Drug Evaluation Service, National Authority for Health (HAS), France
- Marie Bradley, Senior Advisor, Real-World Evidence Analytics, Office of Medical Policy, Food and Drug Administration (FDA), United States
- Karl Broich, President, Federal Institute of Drugs and Medical Devices (BfArM), Germany
- Steve Farmer, Senior Partner and Co-Owner, ABIG Health; Associate Professor of Medicine & Health Policy and Management, The George Washington University; Former Chief Strategy Officer for Coverage, Centers for Medicare and Medicaid Services (CMS), United States
- Niklas Hedberg, Chief Pharmacist, Dental and Pharmaceutical Benefits Agency (TLV), Sweden
- Pall Jonsson, Program Director for Data and Real-World Evidence, National Institute for Health and Care Excellence (NICE), United Kingdom
- Rebecca Nebel, Senior Director, Science and Regulatory Advocacy, Pharmaceutical Research and Manufacturers of America (PhRMA)
- Susana Perez-Gutthann, Senior Vice President, Global Head Epidemiology, RTI Health Solutions, an ENCePP Research Center
- Álmath Spooner, Head of Europe Regulatory Policy and Intelligence, AbbVie; Vice Chair, Integrated Evidence Generation and Use (IEGU) Working Group, European Federation of Pharmaceutical Industries and Associations (EFPIA)
Moderator:
- Sebastian Schneeweiss, Professor of Medicine and Epidemiology, Harvard Medical School; Chief, Division of Pharmacoepidemiology and Pharmacoeconomics, Brigham and Women's Hospital
Featured Speakers
Speaker photos and biographies will be added to this site in the coming days.
Organizer Information
This roundtable is organized by the Division of Pharmacoepidemiology and Pharmacoeconomics at Brigham and Women’s Hospital and Harvard Medical School. The event is supported by Bayer AG, who also contributed its organization. For questions about the event, please contact Shruti Belitkar or Matt Martin.
