Examining the Impact of Real-World Evidence on Medical Product Development: Proceedings of a Workshop Series (2019)
Chapter: Appendix D: Workshop Three Agenda
Appendix D
Workshop Three Agenda
Workshop Three: Application
July 17–18, 2018
National Academy of Sciences Building, Lecture Room
2101 Constitution Avenue, NW, Washington, DC 20418
The National Academies of Sciences, Engineering, and Medicine is convening a three-part workshop series, sponsored by the U.S. Food and Drug Administration, examining how real-world evidence development and uptake can enhance medical product development and evaluation. The workshops will advance discussions and common knowledge about complex issues relating to the generation and usage of real-world evidence, including fostering development and implementation of the science and technology of real-world evidence generation and usage.
- Workshop One (September 19–20, 2017) focused on how to align incentives to support collection and use of real-world evidence in health product review, payment, and delivery. Incentives need to address barriers impeding the uptake of real-world evidence, including barriers to transparency.
- Workshop Two (March 6–7, 2018) illuminated what types of data are appropriate for what specific purposes and suggested practical approaches for data collection and evidence use by developing and working through example use cases.
- Workshop Three (July 17–18, 2018) examined and suggested approaches for operationalizing the collection and use of real-world evidence through discussing “decision aids” about specific topics in study design. The decision aids are question lists developed to inform discussion around specific topics addressed at the third workshop. These discussions may help inform workshop attendees and other stakeholders about study design choices, including potential risks, costs, and reporting/transparency expectations.
DAY 1: JULY 17, 2018
| 8:00 a.m. | Breakfast Available Outside the Lecture Room |
| 8:15 a.m. | Welcome and Opening Remarks |
|
MARK MCCLELLAN, Workshop Series Co-Chair
Director Duke-Margolis Center for Health Policy |
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|
GREGORY SIMON, Workshop Series Co-Chair
Investigator Kaiser Permanente Washington Health Research Institute |
Session I: Key Considerations for Real-World Evidence Application
Session Objectives:
- Examine how some organizations are currently considering traditional and real-world evidence.
- Discuss factors that may be influencing overall cost and time investment required by traditional evidence generation.
- Consider when non-traditional data sources may be beneficial to assess outcomes.
| 8:45 a.m. | Update on the Innovative Medicines Initiative’s GetReal and View from the National Institute for Health and Care Excellence, United Kingdom |
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PALL JONSSON
Associate Director, Research and Development National Institute for Health and Care Excellence |
| 9:05 a.m. | Drivers of Expense and Delay |
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ELLIOTT LEVY
Senior Vice President, Global Development Amgen Inc. |
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| 9:25 a.m. | Patient-Collected and -Owned Data |
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KOMATHI STEM
Chief Executive Officer and Founder monARC Bionetworks |
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| 9:45 a.m. | BREAK |
Session II: When Is a Real-World Data Element Fit for Assessment of Eligibility, Treatment Exposure, or Outcomes?
Session Objectives:
- Discuss potential bias-introducing steps in evidence generation from real-world data.
- Suggest key considerations in the data collection and evidence-generation processes that influence reliability of real-world data.
- Discuss how a decision aid laying out key questions and considerations might help inform current and future studies.
| 10:05 a.m. | Introduction: A Proposed Framework for a Decision Aid |
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PALL JONSSON, Session Moderator
Associate Director, Research and Development National Institute for Health and Care Excellence |
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| 10:15 a.m. | Looking Back: How Might a Decision Aid Inform a Real-World Example? |
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JEFF ALLEN
President and Chief Executive Officer Friends of Cancer Research |
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| 10:35 a.m. |
Looking Forward: How Decision Aid Might Apply to Future Studies
Panel Discussion and Audience Q&A |
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AYLIN ALTAN
Senior Vice President of Research OptumLabs |
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ROBERT BALL
Deputy Director, Office of Surveillance and Epidemiology Center for Drug Evaluation and Research U.S. Food and Drug Administration |
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LUCA FOSCHINI
Co-Founder and Chief Data Scientist Evidation Health |
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BRANDE YAIST
Senior Director, Global Patient Outcomes and Real-World Evidence Eli Lilly and Company |
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| 12:00 p.m. | BREAK (Lunch Available Outside the Lecture Room) |
Session III: Obscuring Intervention Allocation in Trials to Generate Real-World Evidence: Why, Who, and When?
Session Objectives:
- Discuss how variability in knowledge of treatment assignment group affects:
- Provider and patient adherence and outcomes.
- Study cost and reliability.
- Suggest key factors that could affect decisions to obscure intervention allocation.
- Discuss how a decision aid laying out key questions and considerations might help inform current and future studies.
| 1:00 p.m. | Introduction: A Proposed Framework for a Decision Aid |
|
JONATHAN WATANABE, Session Moderator
Associate Professor of Clinical Pharmacy National Academy of Medicine Anniversary Fellow in Pharmacy University of California, San Diego |
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| 1:10 p.m. | Looking Back: How Might a Decision Aid Inform a Real-World Example? |
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JOHN GRAHAM
Head, Value Evidence and Outcomes GlaxoSmithKline |
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ORLY VARDENY
Minneapolis Department of Veterans Affairs Center for Chronic Disease Outcomes Research Associate Professor of Medicine University of Minnesota |
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| 1:30 p.m. |
Looking Forward: How a Decision Aid Might Apply to Future Studies
Panel Discussion and Audience Q&A |
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CATHY CRITCHLOW
Vice President, Center for Observational Research Amgen Inc. |
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NANCY DREYER
Chief Scientific Officer IQVIA |
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ALEX JOHN LONDON
Clara L. West Professor of Ethics and Philosophy Carnegie Mellon University |
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JAMES P. SMITH
Deputy Director, Division of Metabolism and Endocrinology Products Center for Drug Evaluation and Research U.S. Food and Drug Administration |
| 2:50 p.m. | BREAK |
Session IV: How Tightly Should Investigators Attempt to Control or Restrict Treatment Quality in a Pragmatic or Real-World Trial?
Session Objectives:
- Discuss how variability in treatment delivery and adherence can affect results, including
- Potential influence of variation in standard treatment practice, and
- Considerations for balancing participant autonomy and safety.
- Suggest key factors that could help determine the base comparison and level of control suited to a particular trial.
- Discuss how a decision aid laying out key questions and considerations might help inform current and future studies.
| 3:10 p.m. | Introduction: A Proposed Framework for a Decision Aid |
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JENNIFER GRAFF, Session Moderator
Vice President of Comparative Effectiveness Research National Pharmaceutical Council |
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| 3:20 p.m. | Looking Back: How Might a Decision Aid Inform a Real-World Example? |
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LARRY ALPHS
Deputy Chief Medical Officer Newron Pharmaceuticals |
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| 3:40 p.m. |
Looking Forward: How a Decision Aid Might Apply to Future Studies
Panel Discussion and Audience Q&A |
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JUDITH CARRITHERS
Director of Regulatory Services Advarra |
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W. BENJAMIN NOWELL
Director, Patient-Centered Research Global Healthy Living Foundation Co–Principal Investigator, ArthritisPower Patient Powered Research Network |
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PETER STEIN
Deputy Director, Office of New Drugs Center for Drug Evaluation and Research U.S. Food and Drug Administration |
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| 4:50 p.m. | Day 1 Wrap-Up and Concluding Thoughts/Discussion with Audience |
| 5:00 p.m. | ADJOURN WORKSHOP DAY 1 |
DAY 2: JULY 18, 2018
| 8:00 a.m. | Welcome |
|
MARK MCCLELLAN, Workshop Series Co-Chair
Director Duke-Margolis Center for Health Policy |
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GREGORY SIMON, Workshop Series Co-Chair
Investigator Kaiser Permanente Washington Health Research Institute |
Session V: How Can Bias in Observational Comparisons Be Assessed and Minimized?
Session Objectives:
- Discuss methods to assess presence of and optimally reduce bias from unmeasured confounding.
- Suggest key considerations for assessing—and communicating—uncertainty in observational studies.
- Discuss how a decision aid laying out key questions and considerations might help inform current and future studies.
| 8:10 a.m. | Introduction: A Proposed Framework for a Decision Aid |
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DAVID MARTIN
Associate Director for Real-World Evidence Analytics U.S. Food and Drug Administration |
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| 8:20 a.m. | Looking Back: How Might a Decision Aid Inform a Real-World Example? |
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HECTOR IZURIETA
Epidemiologist, Office of Biostatistics and Epidemiology Center for Biologics Evaluation and Research U.S. Food and Drug Administration |
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| 8:40 a.m. |
Looking Forward: How a Decision Aid Might Apply to Future Studies
Panel Discussion and Audience Q&A |
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GREGORY DANIEL, Session Moderator
Deputy Director Duke-Margolis Center for Health Policy |
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JESSICA FRANKLIN
Assistant Professor of Medicine Harvard Medical School |
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NICOLE GORMLEY
Team Lead, Division of Hematologic Products Office of Hematology and Oncology Products Center for Drug Evaluation and Research U.S. Food and Drug Administration |
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JAVIER JIMENEZ
Vice President and Global Head for Real-World Evidence and Clinical Outcomes Sanofi |
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HENG LI
Mathematical Statistician Center for Devices and Radiological Health U.S. Food and Drug Administration |
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MARK VAN DER LAAN
Professor, Biostatistics and Statistics University of California, Berkeley |
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| 10:00 a.m. | BREAK |
Session VI: FDA Panel
Session Objectives:
- Hear updates and perspective of current thinking about real-world evidence in Europe.
- Discuss challenges, opportunities, and remaining gaps for moving forward with real-world evidence application.
| 10:15 a.m. | A European Perspective |
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ALASDAIR BRECKENRIDGE, Session Moderator
Emeritus Professor of Clinical Pharmacology University of Liverpool |
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| 10:30 a.m. | Reflections from FDA |
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STEVEN ANDERSON
Director, Office of Biostatistics and Epidemiology, Center for Biologics Evaluation and Research U.S. Food and Drug Administration |
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JACQUELINE CORRIGAN-CURAY
Director, Office of Medical Policy, Center for Drug Evaluation and Research U.S. Food and Drug Administration |
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JEFF SHUREN
Director, Center for Devices and Radiological Health U.S. Food and Drug Administration |
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| 11:15 a.m. | Panel Discussion with Audience |
| 11:50 a.m. | Synthesis of Workshop Discussions |
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MARK MCCLELLAN, Workshop Series Co-Chair
Director Duke-Margolis Center for Health Policy |
|
|
GREGORY SIMON, Workshop Series Co-Chair
Investigator Kaiser Permanente Washington Health Research Institute |
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| 12:00 p.m. | ADJOURN WORKSHOP DAY 2 |
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