Making Sense of Composite Endpoints in Clinical Research

Daniela Baracaldo-Santamaría, John Edwin Feliciano-Alfonso, Raul Ramirez-Grueso, Luis Carlos Rojas-Rodriguez, Camilo Alberto Dominguez-Dominguez, Carlos-Alberto Calderon-Ospina

Research output: Contribution to journalResearch Articlepeer-review

5 Scopus citations


Multiple drugs currently used in clinical practice have been approved by regulatory agencies based on studies that utilize composite endpoints. Composite endpoints are appealing because they reduce sample size requirements, follow-up periods, and costs. However, interpreting composite endpoints can be challenging, and their misuse is not uncommon. Incorrect interpretation of composite outcomes can lead to misleading conclusions that impact patient care. To correctly interpret composite outcomes, several important questions should be considered. Are the individual components of the composite outcome equally important to patients? Did the more and less important endpoints occur with similar frequency? Do the component endpoints exhibit similar relative risk reductions? If these questions receive affirmative answers, the use and interpretation of the composite endpoint would be appropriate. However, if any component of the composite endpoint fails to satisfy the aforementioned criteria, interpretation can become difficult, necessitating additional steps. Regulatory agencies acknowledge these challenges and have specific considerations when approving drugs based on studies employing composite endpoints. In conclusion, composite endpoints are valuable tools for evaluating the efficacy and net clinical benefit of interventions; however, cautious interpretation is advised.

Keywords: randomized controlled trials; data interpretation; treatment outcome; outcome assessment; endpoint determination
Original languageEnglish (US)
Article number4371
JournalJournal of Clinical Medicine
Issue number13
StatePublished - Jun 29 2023

All Science Journal Classification (ASJC) codes

  • General Medicine


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