Considerations for the Design and Conduct of Externally Controlled Trials for Drug and Biological Products

Docket Number:FDA-2022-D-2983

Issued by:

Center for Drug Evaluation and Research

Center for Biologics Evaluation and Research

Oncology Center of Excellence

This guidance provides recommendations to sponsors and investigators considering the use of externally controlled clinical trials to provide evidence of the safety and effectiveness of a drug product. In an externally controlled trial, outcomes in participants receiving the test treatment according to a protocol are compared to outcomes in a group of people external to the trial who had not received the same treatment. The external control arm can be a group of people, treated or untreated, from an earlier time (historical control), or it can be a group of people, treated or untreated, during the same time period (concurrent control) but in another setting.

Source: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/considerations-design-and-conduct-externally-controlled-trials-drug-and-biological-products