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
Gregory W. Daniel, PhD, MPH, RPh
Deputy Director, Policy and Clinical Professor, Fuqua School of Business
Adrian Hernandez, MD, MHS
Vice Dean for Clinical Research, School of Medicine
Professor of Medicine, Cardiology
Associate Director, Duke Clinical Research Institute
Mark B. McClellan, MD, PhD
Director and Robert J. Margolis, M.D., Professor of Business, Medicine and Policy
Morgan Romine, MPA
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.