Multiple Endpoints in Clinical Trials Guidance for Industry

GUIDANCE DOCUMENT

Multiple Endpoints in Clinical Trials Guidance for Industry

OCTOBER 2022

Docket Number: FDA-2016-D-4460

Issued by: Center for Biologics Evaluation and Research

Center for Drug Evaluation and Research

This guidance provides sponsors and review staff with the Agency’s thinking about the problems posed by multiple endpoints in the analysis and interpretation of study results and how these problems can be managed in clinical trials for human drugs, including drugs subject to licensing as biological products. Most clinical trials performed in drug development contain multiple endpoints to assess the effects of the drug and to document the ability of the drug to favorably affect one or more disease characteristics. As the number of endpoints analyzed in a single trial increases, the likelihood of making false conclusions about a drug’s effects with respect to one or more of those endpoints becomes a concern if there is not appropriate adjustment for multiplicity. The purpose of this guidance is to describe various strategies for grouping and ordering endpoints for analysis and applying some well-recognized statistical methods for managing multiplicity within a study in order to control the chance of making erroneous conclusions about a drug’s effects. Basing a conclusion on an analysis where the risk of false conclusions has not been appropriately controlled can lead to false or misleading representations regarding a drug’s effects.

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:

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Food and Drug Administration

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Rockville, MD 20852

All written comments should be identified with this document’s docket number: FDA-2016-D-4460.