Multiple endpoints in clinical trials: guidance for industry
- Collection:
- Health Policy and Services Research
- Contributor(s):
- United States. Department of Health and Human Services, issuing body. United States. Food and Drug Administration, issuing body. Center for Drug Evaluation and Research (U.S.), issuing body. Center for Biologics Evaluation and Research (U.S.), issuing body.
- Publication:
- Silver Spring, MD : Center for Drug Evaluation and Research, October 2022
- Language(s):
- English
- Format:
- Text
- Subject(s):
- Biostatistics Clinical Trials as Topic Endpoint Determination Government Regulation United States United States. Food and Drug Administration
- Genre(s):
- Guideline Technical Report
- Abstract:
- 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. When more than one endpoint is analyzed in a single trial, the likelihood of making false conclusions about a drug’s effects with respect to one or more of those endpoints could increase if there is no appropriate adjustment for multiplicity. The purpose of this guidance is to describe various strategies for grouping and ordering endpoints for analysis of a drug’s effects and applying some well-recognized statistical methods for managing multiplicity within a study 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. The ICH guidance for industry E9 Statistical Principles for Clinical Trials (September 1998) is a broad ranging guidance that includes discussion of multiple endpoints. This guidance on multiple endpoints in clinical trials for human drugs provides greater detail on the topic. The issuance of this guidance represents partial fulfillment of an FDA commitment under the Food and Drug Administration Amendments Act (FDAAA) of 2007. In general, FDA’s guidance documents do not establish legally enforceable responsibilities. Instead, guidances describe the Agency’s current thinking on a topic and should be viewed only as recommendations, unless specific regulatory or statutory requirements are cited. The use of the word should in Agency guidances means that something is suggested or recommended, but not required.
- Copyright:
- The National Library of Medicine believes this item to be in the public domain. (More information)
- Extent:
- 1 online resource (1 PDF file (26 pages))
- Illustrations:
- Illustrations
- NLM Unique ID:
- 9918504488206676 (See catalog record)
- Permanent Link:
- http://resource.nlm.nih.gov/9918504488206676