Workshop on Regulatory Use of Real-World Evidence Draws Hundreds

Informal comment period to extend to October 11th 

The Robert J. Margolis, MD, Center for Health Policy at Duke University convened a workshop on September 13th to discuss the use of real-world data (RWD) and evidence (RWE) in drug development and regulatory decision-making. In advance of this workshop, the Center published a white paper that outlines considerations stakeholders should make when exploring the potential for regulatory use of RWE.

RWD, data relating to patient health status and/or the delivery of health care that is routinely collected from a variety of sources, and RWE, evidence derived from that data through the application of research methods, have the potential to be applied within a regulatory decision-making framework. However, progress has been limited by both an imprecise characterization of the kinds of RWD and RWE that could be useful and a clear assessment of how they can be applied in specific regulatory contexts.

The workshop, supported by a cooperative agreement with the U.S. Food and Drug Administration, explored the considerations that should guide the development of RWE that is fit for regulatory purposes and high priority opportunities to improve its development and use. Topics included an update on FDA’s implementation of the 21st Century Cures Act’s provisions related to RWE and panel discussions focused on opportunities to improve the data development activities, study designs, and analytical methods used to create robust RWE that supports specific regulatory decisions.

View the webcast and review presentation slides on the event page.

Duke Margolis would like to continue to solicit feedback on the white paper and its proposed framework through an informal comment period that will end Wednesday, October 11, 2017.

We will accept written comments from any and all experts and organizations that would like to provide feedback on the issues contained in the paper, as well as potential ways to further enhance a framework for regulatory use of RWE. Please submit written comments to Margolis.RWE@duke.edu.

Comments will be reviewed and synthesized for a follow-on summary document.

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Change will be needed to harness the knowledge of real-world evidence