Demonstrating Substantial Evidence of Effectiveness With One Adequate and Well-Controlled Clinical Investigation and Confirmatory Evidence

Issue Date: September 19, 2023
Issued by: Center for Drug Evaluation and Research
Topic(s): Clinical – Medical
Status: Draft

Demonstrating Substantial Evidence of Effectiveness With One Adequate and Well-Controlled Clinical Investigation and Confirmatory Evidence

The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled “Demonstrating Substantial Evidence of Effectiveness With One Adequate and Well-Controlled Clinical Investigation and Confirmatory Evidence.” This guidance complements the 2019 draft guidance for industry entitled “Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products” (the 2019 Effectiveness draft guidance) and the 1998 guidance for industry entitled “Providing Clinical Evidence of Effectiveness for Human Drug and Biological Products” (the 1998 Effectiveness guidance). Although FDA’s evidentiary standard has not changed since 1998, there is a need for more Agency guidance to describe how one adequate and well-controlled clinical investigation and confirmatory evidence can be used to meet the substantial evidence requirement.

Download the Guidance Document