In early December the FDA published their Framework for Real World Evidence (RWE) Program. The intent of the program is to allow the agency to work with sponsors to evaluate the use of RWE to support approval of new indications or satisfy post-approval study requirements for approved drugs and biological products. The RWE program is part of the requirements of the 21st Century Cures Act and is regulated under section 505F of the FD&C Act. The framework document discusses how the agency currently uses Real World Data (data relating to patient health or delivery of health care, abbreviated RWD) to generate the clinical evidence (RWE) on the use and potential benefits and risks of a drug or biologic. The framework does not cover medical devices; these are covered by the guidance “Use of Real-World Evidence to Support Regulatory Decision-Making for Medical Devices”.  The point of the document is to describe in broad terms the individual approaches the agency intends to take to expand the use of RWD/RWE to make regulatory decisions on the safety and efficacy to support a new indication or meet post approval study requirements for an already approved drug or biologic. The agency’s focus to date has been to use such data for safety evaluation, and the intent is to increase the use of RWD/RWE for efficacy evaluation. As always, the framework gives the intent and offers clues to how industry may structure pre-submission conversations, but we’ll need at least a draft guidance to understand how this approach will actually change trial operations.

Text Copyright © 2019 Katrina Rogers