Composite Endpoints in Clinical Trials

Peter C. O'Brien, Peter J. Dyck, Barbara C. Tilley

Research output: Chapter in Book/Report/Conference proceedingChapter

1 Scopus citations


Composite endpoints have become increasingly useful as a measure of a patient's disease status. They have been found useful in both clinical practice and medical research. This chapter examines the usefulness of composite endpoints as a primary endpoint in clinical trials. The basic rationale for their use is discussed and illustrated with examples, followed by a variety of procedures for forming composites including how they should be interpreted. The chapter considers alternate approaches to conducting a primary analysis with multiple endpoints, and compares these methods with the use of composite endpoints. It focuses on the outcome measures used in the typical phase III clinical trial for efficacy, for simplicity assuming that an experimental therapy is being compared with a placebo. Some useful methods for defining a composite endpoint for use as a primary endpoint in a clinical trial are considered.

Original languageEnglish (US)
Title of host publicationConcepts, Principles, Trials, and Designs
Number of pages6
ISBN (Electronic)9781118596005
ISBN (Print)9781118595923
StatePublished - Sep 26 2014


  • Cardiovascular disease (CVD)
  • Clinical trials
  • Composite endpoints
  • Phase III clinical trial

ASJC Scopus subject areas

  • General Medicine


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