The framework will evaluate the potential use of RWE to support changes to labeling about drug product effectiveness. This includes adding or modifying an indication, such as a change in dose, dose regimen, or route of administration; adding a new population; or adding comparative effectiveness or safety information. The RWE Program will establish demonstration projects, engage stakeholders, get input from FDA senior leadership when evaluating RWE, and promote shared learning and consistency in applying the framework. FDA will also develop guidance documents to assist sponsors interested in using RWE to support drug development.
Fine sentiments but divisional actions speak louder than policy statements.
In the framework, FDA identifies a three-part approach for assessing whether the use of real world data (RWD) to generate RWE is appropriate to answer a regulatory question:
(1) Are the RWD fit for use?
(2) Can the trial or study design used to generate RWE provide adequate scientific evidence to answer or help answer the regulatory question?
(3) Does the study conducted meet FDA regulatory requirements (e.g., for study monitoring and data collection)?
The agency is accepting comments on the framework and encourages interested parties to submit comments to the established docket (FDA-2018-N-4000).
It’s time to get real.