The Food and Drug Administration (FDA, Agency, or we) is announcing the availability of a revised draft guidance for industry titled "Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products." FDA has revised and is reissuing this draft guidance in response to public comments and to changes in drug development by focusing on generating rigorous scientific evidence in the most efficient manner. Advances in our understanding of biological processes and the increasing availability of high-quality data have transformed the evidentiary landscape for drug development. Given these advances, the draft guidance discusses the many factors that can impact the strength of evidence of effectiveness for a drug and clarifies how sponsors can rely on one adequate and well-controlled clinical investigation with confirmatory evidence to satisfy the substantial evidence of effectiveness standard. When final, this guidance will replace the 1998 guidance titled "Providing Clinical Evidence of Effectiveness for Human Drug and Biological Products."
US Federal Register - FDA Guidance Notices · June 24, 2026
Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products; Revised Draft Guidance for Industry
Original title: Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products; Revised Draft Guidance for Industry; Availability
FDA revises draft guidance on clinical evidence standards for drug effectiveness, clarifying reliance on one adequate and well-controlled investigation with confirmatory evidence.
Published by US Federal Register - FDA Guidance Notices. HealthSignal links to the original document; the summary above is extracted from the source text.