Understanding the Use of Composite Endpoints in Clinical Trials

• Published in: The western journal of emergency medicine 2018-07, Vol.19 (4), p.631-634
• Main Author: McCoy, C Eric
• Format: Article
• Language: English
• Subjects: Decision Making; Drug Therapy; Endpoint Determination; Humans; Population Health Research Design; Randomized Controlled Trials as Topic; Risk Factors
• ISSN: 1936-9018; 1936-900X; 1936-9018
• DOI: 10.5811/westjem.2018.4.38383

Clinicians, institutions, healthcare networks, and policymakers use outcomes reported in clinical trials as the basis for medical decision-making when managing individual patients or populations. Therefore, the choice of a valid primary endpoint is crucial for randomized controlled trials (RCT) to demonstrate efficacy of new therapies. Recent improvements in treatment, however, have led to a decline in the morbidity and mortality of several common diseases, resulting in a reduction in relevant outcomes that can be used as clinical trial endpoints. Composite endpoints have been used as a solution to maintain the feasibility of RCTs, particularly when facing low event rates, high cost, and long follow-up. However, the benefits of using composite endpoints must be weighed against the risks of misinterpretation by clinicians and policymakers, as incorrect interpretation may have a detrimental effect on patients and populations. This paper defines a composite endpoint, discusses the rationale for its use, and provides a practical approach to interpreting results to aid in medical decision-making.