Innovative trial designs that make use of the totality of available evidence can reduce the burden on patients and meaningfully improve the drug development process. In February 2023, the FDA released for public comment: “Considerations for the Design and Conduct of Externally Controlled Trials for Drug and Biological Products” (Docket FDA-2022-D-2983).

The draft guidance received over 200 responses from industry sponsors, patient advocacy organizations, academic research institutions, non-profit consortia, and patients. In July, CRDSA convened a roundtable panel of experts to discuss issues raised by commenting organizations and explore ways to ensure the expanded use of alternative control trial designs in regulatory submissions. The August 2023 FDA Draft External Controls Guidance: Situation Analysis and Recommendations report summarizes the roundtable discussion, key regulatory challenges, and recommendations.

Date: August 22, 2023

Work Group: Framework & Tools