The US Food and Drug Administration last week issued draft guidance for the use of “externally controlled clinical trials” to provide evidence of the safety and effectiveness of drugs and biologics. An externally controlled trial uses patient-level data from a source outside the clinical trial—such as a registry, electronic health records, or administrative claims data—to provide a historical or concurrent control group for the study.

Reviewers are asked to submit written comments on the draft guidance by May 2, 2023. Read the draft guidance.

Externally controlled trials can be useful when it would not be feasible or ethical to use an internal control in the study, such as in studies of populations with rare diseases. The FDA issued the draft guidance as part of a series of guidance documents under its Real-World Evidence Program to satisfy a mandate under the 21st Century Cures Act.