On May 8, 2019, FDA issued draft guidance for the submission of documents using real-world data (RWD) and real-world evidence (RWE) to FDA for drugs and biologics. This draft guidance explains how RWD and RWE can be used as a part of regulatory submissions to FDA, and describes the information that should be included in those submissions.

RWD are data relating to the health status of a patient or to the delivery of health care to a patient. RWD are routinely collected from a number of sources, such as electronic health records, medical billing records, and mobile device applications. RWE is derived from the analysis of RWD, and can be used to demonstrate the usage and potential benefits or risks of a particular medical product.

The increasingly wide-spread availability of RWD, and the continued development of improved analytic techniques to generate RWE, has inspired the research and medical communities to use RWD and RWE to enhance clinical research and to support regulatory decision making. In response, FDA is currently evaluating the scope and use of RWE in IND, NDA, and BLA submissions relating to safety and effectiveness. To aid FDA’s evaluation, FDA encourages sponsors and applicants to identify submissions that include RWE tied to a specific product, and to provide information about the RWE used.

In each regulatory submission featuring RWE in support of safety or effectiveness, the sponsor or applicant should identify (1) the purpose for using RWE as part of the regulatory submission; (2) the design of any clinical study that includes RWE; and (3) all sources of RWD used to generate the RWE. This information should be highlighted in the submission’s cover letter or included in an accompanying table.

Readers are encouraged to read the draft guidance, also available on FDA’s website.