Demonstrating Substantial Evidence of Effectiveness With One Adequate and Well-Controlled Clinical Investigation and Confirmatory Evidence September 2023

GUIDANCE DOCUMENT

• Docket Number: FDA-2023-D-2318
• Issued by: Center for Drug Evaluation and Research; Center for Biologics Evaluation and Research; Oncology Center of Excellence; Office of the Commissioner

The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled “Demonstrating Substantial Evidence of Effectiveness With One Adequate and Well-Controlled Clinical Investigation and Confirmatory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 This guidance complements the 2019 draft guidance for industry entitled “Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products” (the 2019 Effectiveness draft guidance) and the 1998 guidance for industry entitled “Providing Clinical Evidence of Effectiveness for Human Drug and Biological Products” (the 1998 Effectiveness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.

---

Submit Comments

Submit Comments Online

You can submit online or written comments on any guidance at any time (see 21 CFR 10.115(g)(5))

If unable to submit comments online, please mail written comments to:

Dockets Management
Food and Drug Administration
5630 Fishers Lane, Rm 1061
Rockville, MD 20852

All written comments should be identified with this document's docket number: FDA-2023-D-2318.