Making Sense of Composite Endpoints in Clinical Research
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...
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| Format: | Article |
| Language: | English |
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MDPI AG
2023-06-01
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| Series: | Journal of Clinical Medicine |
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| Online Access: | https://www.mdpi.com/2077-0383/12/13/4371 |
| _version_ | 1797591417226264576 |
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| author | Daniela Baracaldo-Santamaría John Edwin Feliciano-Alfonso Raul Ramirez-Grueso Luis Carlos Rojas-Rodríguez Camilo Alberto Dominguez-Dominguez Carlos Alberto Calderon-Ospina |
| author_facet | Daniela Baracaldo-Santamaría John Edwin Feliciano-Alfonso Raul Ramirez-Grueso Luis Carlos Rojas-Rodríguez Camilo Alberto Dominguez-Dominguez Carlos Alberto Calderon-Ospina |
| author_sort | Daniela Baracaldo-Santamaría |
| collection | DOAJ |
| description | 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. |
| first_indexed | 2024-03-11T01:37:07Z |
| format | Article |
| id | doaj.art-71a18fa5b66b495d84663e0e97f31ce5 |
| institution | Directory Open Access Journal |
| issn | 2077-0383 |
| language | English |
| last_indexed | 2024-03-11T01:37:07Z |
| publishDate | 2023-06-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Journal of Clinical Medicine |
| spelling | doaj.art-71a18fa5b66b495d84663e0e97f31ce52023-11-18T16:52:56ZengMDPI AGJournal of Clinical Medicine2077-03832023-06-011213437110.3390/jcm12134371Making Sense of Composite Endpoints in Clinical ResearchDaniela Baracaldo-Santamaría0John Edwin Feliciano-Alfonso1Raul Ramirez-Grueso2Luis Carlos Rojas-Rodríguez3Camilo Alberto Dominguez-Dominguez4Carlos Alberto Calderon-Ospina5Pharmacology Unit, Department of Biomedical Sciences, School of Medicine and Health Sciences, Universidad del Rosario, Bogotá 111221, ColombiaSchool of Medicine, Universidad El Bosque, Bogotá 110121, ColombiaPharmacology Unit, Department of Biomedical Sciences, School of Medicine and Health Sciences, Universidad del Rosario, Bogotá 111221, ColombiaPharmacology Unit, Department of Biomedical Sciences, School of Medicine and Health Sciences, Universidad del Rosario, Bogotá 111221, ColombiaSchool of Medicine and Health Sciences, Universidad del Rosario, Bogotá 111221, ColombiaPharmacology Unit, Department of Biomedical Sciences, School of Medicine and Health Sciences, Universidad del Rosario, Bogotá 111221, ColombiaMultiple 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.https://www.mdpi.com/2077-0383/12/13/4371randomized controlled trialsdata interpretationtreatment outcomeoutcome assessmentendpoint determination |
| spellingShingle | Daniela Baracaldo-Santamaría John Edwin Feliciano-Alfonso Raul Ramirez-Grueso Luis Carlos Rojas-Rodríguez Camilo Alberto Dominguez-Dominguez Carlos Alberto Calderon-Ospina Making Sense of Composite Endpoints in Clinical Research Journal of Clinical Medicine randomized controlled trials data interpretation treatment outcome outcome assessment endpoint determination |
| title | Making Sense of Composite Endpoints in Clinical Research |
| title_full | Making Sense of Composite Endpoints in Clinical Research |
| title_fullStr | Making Sense of Composite Endpoints in Clinical Research |
| title_full_unstemmed | Making Sense of Composite Endpoints in Clinical Research |
| title_short | Making Sense of Composite Endpoints in Clinical Research |
| title_sort | making sense of composite endpoints in clinical research |
| topic | randomized controlled trials data interpretation treatment outcome outcome assessment endpoint determination |
| url | https://www.mdpi.com/2077-0383/12/13/4371 |
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