BRIEF—FDA's final guidance on use of real-world data and evidence

The US Food and Drug Administration (FDA) has issued a final guidance for industry titled Considerations for the Use of Real-World Data and Real-World Evidence to Support Regulatory Decision-Making for Drugs and Biological Products.

The guidance discusses the applicability of the FDA's investigational new drug (IND) application regulations under 21 CFR part 312 to clinical study designs that use real-world data (RWD) and clarifies the agency's expectations for sponsors submitting new drug applications or biologics license applications using RWD to support the safety or effectiveness of a drug in clinical studies that are not subject to 21 CFR part 312.

For non-interventional (observational) studies, which are not subject to the FDA’s IND regulations, the guidance outlines the agency’s general expectations for study conduct.

The FDA is issuing this guidance to assist stakeholders in meeting its expectations and to ensure that the agency can appropriately evaluate observational studies that are submitted in marketing applications to support the safety and effectiveness of a drug.

This guidance is part of a series of guidances that the FDA has already published, or plans to publish, as part of the agency’s  RWE Program and in support of the 21st Century Cures Act and the Prescription Drug User Fee Act.