This draft FDA guidance is intended to encourage sponsors and applicants who are using real-world data (RWD) to generate real-world evidence (RWE) as part of a regulatory submission to FDA to provide information on their use of RWE in a simple, uniform format. FDA will use this information for internal tracking purposes only. This guidance applies to submissions for investigational new drug applications (INDs), new drug applications (NDAs), and biologics license application (BLAs) that contain RWE used to support regulatory decisions regarding safety and/or effectiveness.

>>> Draft FDA Guidance – Submitting Documents Using Real-World Data and Real-World Evidence to FDA for Drugs and Biologics (May 2019)

The availability of RWD and evolving analytic techniques to generate RWE has created interest within the research and medical communities to use RWD/RWE to enhance clinical research and support regulatory decision making. Exploring the potential for RWE to inform regulatory decisions is mandated by the 21 Cures Act (Cures Act). Section 3022 of the Cures Act requires FDA to establish a program to evaluate the potential use of RWE to help to support the approval of a new indication for a drug  approved under section 505(c) of the Federal Food, Drug, and Cosmetic Act (FD&C Act) and to help to support or satisfy post-approval study requirements.

To inform FDA’s RWE program under the Cures Act and to help FDA understand the scope and use of RWE submitted to support regulatory decisions regarding safety and/or effectiveness, the  Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER) intend to track certain types of submissions using RWE under an IND, NDA, or BLA. To aid in the tracking, CDER and CBER encourage sponsors and applicants to identify submissions that include RWE being used to support a regulatory decision(s) regarding safety and/or effectiveness.