USA – FDA Will Allow Observational Studies to Support Effectiveness Determinations, New Framework Says

The US Food and Drug Administration (FDA) on Thursday unveiled a new framework discussing how the agency will use real world evidence (RWE) and real-world data (RWD) to help companies win new indications for approved drugs and biologics, expand labels or satisfy post-approval study requirements.

As far as what the framework will guide FDA on specifically, the agency said its RWE program will evaluate the potential use of RWE to support changes to labeling about drug product effectiveness, including adding or modifying an indication, such as a change in dose, dose regimen or route of administration; new populations; or the addition of comparative effectiveness or safety information.

“The RWE framework will also consider the evaluation of observational clinical studies using RWD to support product effectiveness determinations,” FDA said.

For determinations on how to use RWE, the agency said it will consider the following on a case-by-case basis:

  • Whether the RWD are fit for use
  • Whether the trial or study design used to generate RWE can provide adequate scientific evidence to answer or help answer the regulatory question
  • Whether the study conduct meets FDA regulatory requirements (e.g., for study monitoring and data collection)

“FDA intends to use this three-part approach to evaluate individual supplemental applications, as appropriate, and more generally to guide FDA’s RWE Program,” the framework says…