FDA Issues Draft Guidance on Real-World Evidence

Rare Daily Staff

The U.S. Food and Drug Administration has issued a draft guidance for industry on the use of Real-Word Data derived from electronic health records and medical claims data to support regulatory decision making for drugs and biologics.

The agency announced the guidance in a listing in the Federal Register. The draft guidance is part of a series of guidance documents under the FDA’s Real-World Evidence Program. It is intended to provide sponsors, researchers, and other interested stakeholders with considerations when proposing to use electronic health records or medical claims data in clinical studies to support a regulatory decision for effectiveness or safety, the agency said.

The 21st Century Cures Act required the FDA to establish a program to evaluate the potential use of real-world evidence to support the approval of a new indication for an approved drug and to help support or satisfy post-approval study requirements. Under the law, the agency is required to inform guidance for industry on the circumstances under which sponsors of drugs may rely on real world evidence and the appropriate standards and methodologies for the collection and analysis of real world evidence.

The agency under the Prescription Drug User Fee Amendments of 2017 (PDUFA VI), also made a commitment to publish draft guidance on how real-world evidence can contribute to the assessment of safety and effectiveness in regulatory submissions.

Real-world evidence includes data derived from electronic health records, medical claims data, data from product and disease registries, patient-generated data including from in-home use, and data gathered from other sources that can inform on health status, such as digital health technologies. The draft guidance, though, is focused on just data from electronic health records and medical claims data.

The guidance complements the 2013 guidance by expanding on certain aspects of that guidance relating to the selection of data sources and also provides additional guidance for evaluating the relevance and reliability of both electronic health records and medical claims data for use in a clinical study.

The FDA is accepting electronic or written comments on the draft guidance. Comment should be filed by November 29, 2021.