New Collaborative to Advance Regulatory Use of Real-World Evidence

December 7, 2017

The Robert J. Margolis, MD, Center for Health Policy at Duke University announced today that it is forming a new collaborative to advance policy development related to the regulatory acceptability of real-world evidence (RWE). The Collaborative will engage major stakeholders to help advise and guide high priority efforts aimed at improving the development and use of RWE, with the express aim of informing and supporting the FDA as it works to meet RWE milestones as established in the 21st Century Cures Act and the sixth Prescription Drug User Fee Act (PDUFA VI).

The RWE Collaborative will consist of an advisory group that will include senior representation from medical product developers, payers, research groups, providers, patient networks, and regulators and will steer working groups and contribute to the Collaborative’s recommendations.

“Our Collaborative members are national leaders in the development and use of real-world data and evidence to improve patient care. They share a common goal of using RWE to support not only biomedical innovation and regulatory review, but also better outcomes for patients at a more sustainable cost,” said Duke-Margolis director Mark McClellan, MD, PhD

Gregory Daniel, PhD, MPH, the Center’s deputy director, said, “We have the technical and methodological tools to incorporate real-world evidence into regulatory decision making, but more progress is needed on when and how to apply RWE within the regulatory framework. Ultimately, we hope that better clarity in how such high-quality evidence can inform FDA’s decisions will further bolster support for a more efficient evidence development infrastructure that makes it easier for RWE to guide a range of decisions to improve the value and quality of care.”

In its initial projects, the Collaborative will seek to overcome current barriers to the use of RWE in regulatory decision making by exploring primary considerations for assessing the quality of real-world data (RWD) sources, promising opportunities to improve the credibility of observational study designs, and lessons learned from actual industry experience with RWD- and RWE-enabled regulatory submissions.

“This group gathers the leadership needed to bring the vision of PDUFA IV and 21st Century Cures for RWE to life, and I can’t overstate the importance of the vision and integrating RWE into clinical research and practice,” said Adrian Hernandez, MD, MHS, vice dean for clinical research at the Duke University School of Medicine and associate director of the Duke Clinical Research Institute.

About Duke-Margolis

The Robert J. Margolis, MD, Center for Health Policy at Duke University brings together expertise from the Washington, DC policy community, Duke University and Duke Health to address the most pressing issues in health policy.

The Center’s mission is to improve health and the value of health care by developing and implementing evidence-based policy solutions locally, nationally, and globally. For more information, visit

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 Eli Lilly and Company; Genentech, a member of the Roche Group; GlaxoSmithKline; Johnson & Johnson; Novartis 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. For more details on relevant institutional policies on research independence and conflict of interest, please visit our web site.