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 2019 priorities:

  • Real-World Data - Fit for Use Reporting
  • Assessing Individual Study Credibility for Observational Trials
  • Understanding the Role of Observational Studies in a Totality of Evidence (ToE) Approach
  • Establishing Guideposts for Developing Real-World Endpoints

To learn more about the RWE Collaborative or to learn more about 2019 projects, please contact Nirosha Mahendraratnam Lederer (Nirosha.Mahendraratnam@duke.edu).

Duke-Margolis Project Team

Research Associate
 

Joy Eckert
Senior Research Assistant 

Adrian Hernandez, MD, MHS
Vice Dean for Clinical Research, School of Medicine
Professor of Medicine, Cardiology
Associate Director, Duke Clinical Research Institute

Managing Associate
 

Mark B. McClellan, MD, PhD 
Director and Robert J. Margolis, M.D., Professor of Business, Medicine and Policy

Senior Research Assistant
 

Morgan Romine, MPA
Research Director

Christina Silcox, PhD
Managing Associate
 
 

Advisory Group

AbbVie
Ryan Kilpatrick, Head, Global Epidemiology
 
Eli Lilly and Company
Carlos Garner, Vice President, Global Regulatory Affairs
 
Evidation
Bray Patrick-Lake, Director, Strategic Partnerships
 
Evidera
Debra A. Schaumberg, Vice President, Scientific Affairs
 
Flatiron Health
Nicole Mahoney, Senior Director of Regulatory Policy
 
Genentech
Jacqueline Law, Vice President, Global Head Real World Data
 
GlaxoSmithKline
John Graham, Senior Vice President, Medical Engagement and Value Evidence & Outcomes
 
Harvard Medical School
Richard Platt, Executive Director, Harvard Pilgrim Health Care Institute and Chair, Department of Population Medicine
 
Healthcore
Marcus Wilson, Co-Founder and President
 
International Society for Pharmacoeconomics and Outcomes Research
Marc Berger, Consultant
 
International Society for Pharmacoeconomics and Outcomes Research
Richard Wilke, Chief Science Officer
 
IQVIA Real-World Insights
Nancy Dreyer, Global Chief of Scientific Affairs
 
Johnson & Johnson
Joanne Waldstreicher, Chief Medical Officer
 
Merck & Co.
Solomon Iyasu, Vice President, Head of Pharmacoepidemiology, Center for Observational and Real World Evidence
 
Multi-Regional Clinical Trials Center of Brigham and Women’s Hospital and Harvard
Barbara Bierer, Faculty Director
 
National Health Council
Eleanor Perfetto, Senior Vice President, Strategic Initiatives
 
Novartis Pharmaceuticals Corporation
Amy Rudolph, Vice President and Head of HE&OR and Digital Medicine
 
OptumLabs
Paul Bleicher, Chief Executive Officer
 
PatientsLikeMe
Sally Okun, Vice President Policy & Ethics
 
Syneos Health
David Thompson, Senior Vice President
 
Teva Pharmaceuticals
Riad Dirani, Vice President, Global Health Economics and Outcomes Research
 
UCB
David Miller, Senior Director, RWE Methods Analysts & Safety
 
Veradigm Health
Stephanie Reisinger, Vice President and General Manager, Life Sciences
 
 

Observers

National Academies of Sciences, Engineering, and Medicine
Amanda Wagner-Gee, Program Officer
 
People-Centered Research Foundation
Kathy Hudson, Executive Director
 
Patient-Centered Outcomes Research Institute 
Joe Selby, Executive Director
 
U.S. Food and Drug Administration
Jacqueline Corrigan-Curay, Director, Office of Medical Policy, CDER
 

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 consortium members including AbbVie; Eli Lilly and Company; Genentech, a member of the Roche Group; GlaxoSmithKline; Johnson & Johnson; 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.