Enhancing the Application of Real-World Evidence In Regulatory Decision-Making
Washington Plaza Hotel • Washington, DC
March 3 & 4, 2016
Event Details
The last decade has seen important progress in the development of evidence from routine clinical practice, and there has been a growing interest in harnessing such real-world evidence (RWE) to fill in key information gaps for a variety of health care decision-makers. While payers, providers, 
and product sponsors have led the way in application of RWE within their respective decision-making frameworks, its use to support or enhance drug regulatory decisions by the U.S. Food and Drug Administration (FDA) has been relatively limited. Advanced RWE collection efforts and research methods, however, present timely new opportunities for improving clinical trial efficiency and aligning pre- and postmarket evidence generation activities to simultaneously meet regulatory requirements and the broader evidence needs of critical post-regulatory decision-makers
On March 3 and 4, 2016, the Duke-Margolis Center for Health Policy in cooperation with the FDA hosted a two-day public conference focused on opportunities for enhancing the use of RWE across a number of potential regulatory decisions. Through presentations and panel discussions, leading experts from across the health care ecosystem discussed the use of RWE to improve clinical trial efficiency and enhance post-market research efforts; the supporting role of observational studies in a regulatory context; potential criteria and study methods for utilizing randomization within the clinical setting to support regulatory approval decisions; and the policy, data infrastructure, and patient and provider engagement challenges that will need to be overcome to ensure success. This public conference is part of a year-long series of meetings and research activities that the Duke-Margolis Center is undertaking to both explore enhanced and more routine use of RWE within FDA’s regulatory framework and to strengthen the RWE development landscape for all stakholders.
Recording of Event
Day 1: https://www.youtube.com/watch?v=a4ydLNpz-Iw
Day 2: https://www.youtube.com/watch?v=lB05ItcPtec
Agenda- Day 1
9:00 a.m. Welcome and Introductory Remarks
Mark McClellan, Duke-Margolis Center for Health Policy
9:10 a.m. The Evidence Development Spectrum: Defining Sources and Applications of Real-World Evidence
Gregory Daniel, Duke-Margolis Center for Health Policy
9:25 a.m. FDA’s Use of Real-World Evidence to Support Decision-Making
Janet Woodcock, U.S. Food and Drug Administration
9:40 a.m. Moderated Discussion and Q&A
Speakers:
- Cliff Goodman, Lewin Group
- Bill Chin, Pharmaceutical Research and Manufacturers of America
10:25 a.m. Break
10:40 a.m. Harnessing Randomization in the Clinical Setting: Criteria for Regulatory Use Cases
Speakers:
- Bob Temple, U.S. Food and Drug Administration
- Rob Metcalf, Eli Lilly and Company
- Bill Capra, Genentech Inc.
12:00 p.m. Lunch
1:00 p.m. Harnessing Randomization in the Clinical Setting: Study Methods and Data Development Considerations for Efficient Implementation
Speakers:
- Lisa LaVange, U.S. Food and Drug Administration
- Adrian Hernandez, Duke University
- Martin Gibson, NorthWest EHealth
2:30 p.m. Break
2:45 p.m. Exploring the Use of Observational Data in Regulatory Settings
Speakers:
- Jonathan Jarow, U.S. Food and Drug Administration
- Rich Platt, Harvard University
- Marc Berger, Pfizer Inc.
- Marcus Wilson, HealthCore Inc.
3:55 p.m. Closing Remarks for Day One
Agenda – Day 2
9:00 a.m. Welcome and Recap of Day One
Mark McClellan, Duke-Margolis Center for Health Policy
9:05 a.m. Building a Robust 21st Century Evidence Development Infrastructure
Speakers:
- Melissa Robb, U.S. Food and Drug Administration
- Nancy Dreyer, Quintiles
- Sean Tunis, Center for Medical Technology Policy
- Solomon Iyasu, Merck & Co.
10:00 a.m. Incentives and Policy Options for Improving a Shared Infrastructure
Speakers:
- Ed Pezalla, Aetna Inc.
- Jennifer Graff, National Pharmaceutical Council
- Rachael Fleurence, Patient-Centered Outcomes Research Institute
10:45 a.m. Break
11:00 a.m. Meaningfully Engaging Providers: Pathways for Fully Embedding Evidence Development in Routine Care
Speakers:
- Carrie D’Andrea, University of California, San Francisco
- Mike Hogarth, University of California, Davis
11:45 a.m. Making Patients Vested Partners: Patient-Generated Data, Consent, and Data Privacy Challenges
Speakers:
- Sally Okun, PatientsLikeMe
- Marc Boutin, National Health Council
- Kim McCleary, FasterCures
12:30 p.m. Closing Remarks
Mark McClellan