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 2020 priorities:

  • RWEndpoints Roadmap
  • The Science of Similarity—External Comparators/Controls
  • Identifying Shared Real-World Evidentiary Opportunities
  • Advancing International RWE Efforts
  • PGHD Pilot Implementation Case Studies

To learn more about the RWE Collaborative or to learn more about 2020 projects, please contact Nirosha Mahendraratnam Lederer (Nirosha.Mahendraratnam@duke.edu).

 

Advisory Group

Aylin Altan, Senior Vice President of Research, OptumLabs

Dorothee B. Bartels, Head of Global RWE, UCB

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

Adrian Cassidy, Global Head RWE, Novartis Pharmaceuticals Corporation

Cathy Critchlow, Vice President, Center for Observational Research, Amgen

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

Lisa LaVange, Professor and Associate Chair of the Department of Biostatistics, UNC Gillings School of Global Public Health

Jacqueline Law, Vice President, Global Head Real World Data, Genentech

Christina Mack, RWE Task Force, ISPE

Eileen Mack Thorley, Senior Research Scientist, PatientsLikeMe

Nicole Mahoney, Senior Director, Regulatory Policy, Flatiron Health

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

Stephanie Reisinger, Vice President and General Manager, Life Sciences, Veradigm Health

Debra A. Schaumberg, Vice President, Scientific Affairs, Evidera

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

Understanding the Need for Non-Interventional Studies Using Secondary Data to Generate Real-World Evidence for Regulatory Decision Making, and Demonstrating Their Credibility

Adding Real-World Evidence to a Totality of Evidence Approach for Evaluating Marketed Product Effectiveness

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; Eli Lilly and Company; Pfizer; Genentech, a member of the Roche Group; GlaxoSmithKline; Merck; Novartis; Teva and UCB.

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.