FDA Draft Guidance: Submitting Documents Utilizing Real-World Data (RWD) and Real-World Evidence (RWE) to the FDA for Drugs and Biologics

Today the U.S. Food and Drug Administration (FDA) published draft guidance entitled “Submitting Documents Utilizing Real-World Data (RWD) and Real-World Evidence (RWE) to the FDA for Drugs and Biologics.”

This draft guidance encourages sponsors and applicants who are using real-world data (RWD) to generate real-world evidence (RWE) as part of a submission to provide information on their use of RWE to the FDA in a simple, uniform format. The FDA will use this information for internal tracking purposes only.

To inform the agency’s RWE program under the Cures Act and to help the FDA understand the scope and use of RWE submitted to support efficacy and/or safety, the Center for Drug Evaluation and Research (CDER), and the Center for Biologics Evaluation and Research (CBER) intend to identify and track certain types of submissions using RWE under an IND, NDA, or BLA. To aid in the tracking of these submissions, CDER and CBER encourage sponsors and applicants to identify in their cover letters submissions that include RWE to support regulatory decisions regarding efficacy and/or safety.  This guidance helps sponsors and applicants determine how to best identify and provide the requested information on submissions that include RWE. Under the guidance, FDA encourages sponsors to note in the cover letter the purpose of using RWE, the study design in which the RWE is being used, and the source of the RWD. 

While the FDA will post a companion webinar to this draft guidance in mid-to-late May, please check CDER’s webinar page for more information, two RWE/RWD-related webinars are currently available:  An Introduction to FDA MyStudies: An Open-Source, Digital Platform to Gather Real World Data for Clinical Trials and Research Studies and Framework for FDA’s Real-World Evidence Program.

Comments on this draft guidance will be accepted via the associated docket (FDA-2019-D-1263) and a notice has been issued in the Federal Register (Document Number 2019-09529).