Real-World Evidence Collaborative
Randomized controlled trials (RCTs) are traditionally used by the US Food and Drug Administration (FDA) to evaluate a product’s safety and effectiveness. While they are the gold-standard from a research perspective, they are resource-intensive, requiring significant financial, time, and human capital investments. Coupled with unmet clinical needs and drug access challenges, the demands of RCTs have fueled patients and legislators to issue a call to action for new ways to optimize the drug development process so that the delivery of effective drugs to the marketplace is accelerated while the public’s safety remains ensured.
Health care stakeholders are enthusiastic about exploring whether patient information that is already captured at the point of care could inform and speed the development of new drugs. Called real-world data (RWD), analyzing this burgeoning information source has the potential to provide real-world evidence (RWE), information on the use of drugs and their outcomes and to understand whether they are performing as intended in routine care.
The 21st Century Cures Act and the sixth Prescription Drug User Fee Act (PDUFA VI) set milestones for FDA to explore the use of RWE in regulatory decision-making. To inform FDA as it works to meet these milestones, the Duke-Margolis Center for Health Policy RWE Collaborative engages stakeholders to guide high-priority efforts aimed at improving the development and use of RWE. The Collaborative also strives to drive progress in the use of real-world data and evidence to improve patient treatment options and outcomes more broadly. Guided by an advisory group consisting of leaders representing medical product developers, payers, research groups, providers, patient networks, and regulators, the RWE Collaborative is interested in the following 2021 priorities:
- Embedding Practical Trials in Routine Care Delivery
- Identifying Shared Real-World Evidentiary Opportunities
- Developing Master RWE Protocols for Key Research Questions
- PGHD Pilot Implementation Case Studies
To learn more about the RWE Collaborative or to learn more about 2020 projects, please contact Morgan Romine (morgan.romine@duke.edu).
Advisory Group
Aylin Altan, Senior Vice President of Research, OptumLabs
Marc Berger, Co-Chair, Special Advisor for Real World Evidence, ISPOR
Barbara Bierer, Faculty Director, Multi-Regional Clinical Trials Center of Brigham and Women’s Hospital and Harvard
Brian Bradbury, Vice President, Center for Observational Research, Amgen
William Capra, Senior Director, Data Science, Roche Pharmaceuticals, Genentech
Adrian Cassidy, Global Head RWE, Novartis Pharmaceuticals Corporation
William Crown, Distinguished Research Scientist, The Heller School for Social Policy and Management at Brandeis University
Riad Dirani, Vice President, Global Health Economics and Outcomes Research, Teva Pharmaceuticals
Nancy Dreyer, Chief Scientific Officer & Senior Vice President, IQVIA
Andrew Emmett, FDA Liaison, Global Regulatory Policy & Intelligence, Pfizer
Carlos Garner, Vice President, Global Regulatory Affairs, Eli Lilly and Company
John Graham, Senior Vice President, Medical Engagement and Value Evidence & Outcomes, GlaxoSmithKline
Solomon Iyasu, Vice President, Head of Pharmacoepidemiology, Center for Observational and Real World Evidence, Merck & Co.
Ryan Kilpatrick, Head, Global Epidemiology, AbbVie
Christina Mack, RWE Task Force, ISPE
Eileen Mack Thorley, Senior Research Scientist, PatientsLikeMe
Irene Nunes, Vice President, Regulatory Affairs, Flatiron Health
Sally Okun, SallyOkun360
Bray Patrick-Lake, Director, Strategic Partnerships, Evidation
Eleanor Perfetto, Senior Vice President, Strategic Initiatives, National Health Council
Richard Platt, Executive Director, Harvard Pilgrim Health Care Institute and Chair, Department of Population Medicine, Harvard Medical School
Jeremy Rassen, Co-Founder, President, Chief Science Officer, Aetion
Stephanie Reisinger, Vice President and General Manager, Life Sciences, Veradigm Health
Debra A. Schaumberg, Vice President, Scientific Affairs, Evidera
Thomas Seck, Senior Vice President, Medicine & Regulatory Affairs, Boehringer Ingelheim
Lauren Silvis, Senior Vice President, Tempus
David Thompson, Senior Vice President, Syneos Health
Richard Wilke, Chief Science Officer, ISPOR
Marcus Wilson, Co-Founder and President, HealthCore
Observers
Jacqueline Corrigan-Curay, Director, Office of Medical Policy, CDER, U.S. Food and Drug Administration
Amanda Wagner-Gee, Program Officer, National Academies of Sciences, Engineering, and Medicine
Publications
Characterizing RWD Quality and Relevancy for Regulatory Purposes
Determining Real-World Data’s Fitness for Use and the Role of Reliability
A Roadmap for Developing Study Endpoints in Real-World Settings
Funding
This project is made possible through the generosity of the Margolis Family Foundation, which provides core resources for the Center, as well as a combination of financial and in-kind contributions from collaborative members including AbbVie; Amgen; Boehringer Ingelheim; Eli Lilly and Company; Genentech, a member of the Roche Group; GlaxoSmithKline; Merck; Novartis; Pfizer; and Teva.
Participation of private or non-profit organizations in Center activities does not imply an endorsement by Duke University or associated entities. As part of Duke University, Duke-Margolis honors the tradition of academic independence on the part of its faculty and scholars. Neither Duke nor the Margolis Center take partisan positions, but the individual members are free to speak their minds and express their opinions regarding important issues. Duke Margolis adheres to all institutional policies on research independence and conflict of interest.
