American Journal of Clinical and Experimental Medicine

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Composite Endpoints: Sometimes More than a Solely Economic Consideration

Received: 18 June 2013    Accepted:     Published: 20 July 2013
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Abstract

Endpoints are response variables, or outcomes, that are measured during the course of a clinical trial. I consider endpoints that are either events (e.g., death) or the time to an occurrence of an event (e.g., time to disease progression). A composite endpoint (CEP) is an endpoint that consists of a number of component endpoints, and is considered to have occurred as soon as any one of its components occurs. For example if CEP = death + disease progression, the CEP is said to have occurred as soon as either the disease progresses or the patient dies. It is seen that one of the results of using a CEP is to increase the event rate; and this in turn can reduce the sample size or the time required to observe a specified number of events, thereby resulting in a speedier, less costly clinical trial. Many believe that the only reason CEPs are ever employed is to this end, viz., saving money. I argue that there may be other circumstances that suggest the use of CEPs – that the choice of the primary response variable should be driven by the question the trial is being designed to answer.

DOI 10.11648/j.ajcem.20130101.15
Published in American Journal of Clinical and Experimental Medicine (Volume 1, Issue 1, July 2013)
Page(s) 24-34
Creative Commons

This is an Open Access article, distributed under the terms of the Creative Commons Attribution 4.0 International License (http://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, distribution and reproduction in any medium or format, provided the original work is properly cited.

Copyright

Copyright © The Author(s), 2024. Published by Science Publishing Group

Keywords

Clinical Trials, Outcome Variables, Event Rates, Win Ratio, Quality of Life

References
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  • University of Michigan, Ann Arbor MI 48109 USA

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    Charles J Kowalski. (2013). Composite Endpoints: Sometimes More than a Solely Economic Consideration. American Journal of Clinical and Experimental Medicine, 1(1), 24-34. https://doi.org/10.11648/j.ajcem.20130101.15

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    Charles J Kowalski. Composite Endpoints: Sometimes More than a Solely Economic Consideration. Am. J. Clin. Exp. Med. 2013, 1(1), 24-34. doi: 10.11648/j.ajcem.20130101.15

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    AMA Style

    Charles J Kowalski. Composite Endpoints: Sometimes More than a Solely Economic Consideration. Am J Clin Exp Med. 2013;1(1):24-34. doi: 10.11648/j.ajcem.20130101.15

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  • @article{10.11648/j.ajcem.20130101.15,
      author = {Charles J Kowalski},
      title = {Composite Endpoints: Sometimes More than a Solely Economic Consideration},
      journal = {American Journal of Clinical and Experimental Medicine},
      volume = {1},
      number = {1},
      pages = {24-34},
      doi = {10.11648/j.ajcem.20130101.15},
      url = {https://doi.org/10.11648/j.ajcem.20130101.15},
      eprint = {https://download.sciencepg.com/pdf/10.11648.j.ajcem.20130101.15},
      abstract = {Endpoints are response variables, or outcomes, that are measured during the course of a clinical trial. I consider endpoints that are either events (e.g., death) or the time to an occurrence of an event (e.g., time to disease progression). A composite endpoint (CEP) is an endpoint that consists of a number of component endpoints, and is considered to have occurred as soon as any one of its components occurs. For example if CEP = death + disease progression, the CEP is said to have occurred as soon as either the disease progresses or the patient dies. It is seen that one of the results of using a CEP is to increase the event rate; and this in turn can reduce the sample size or the time required to observe a specified number of events, thereby resulting in a speedier, less costly clinical trial. Many believe that the only reason CEPs are ever employed is to this end, viz., saving money. I argue that there may be other circumstances that suggest the use of CEPs – that the choice of the primary response variable should be driven by the question the trial is being designed to answer.},
     year = {2013}
    }
    

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    AB  - Endpoints are response variables, or outcomes, that are measured during the course of a clinical trial. I consider endpoints that are either events (e.g., death) or the time to an occurrence of an event (e.g., time to disease progression). A composite endpoint (CEP) is an endpoint that consists of a number of component endpoints, and is considered to have occurred as soon as any one of its components occurs. For example if CEP = death + disease progression, the CEP is said to have occurred as soon as either the disease progresses or the patient dies. It is seen that one of the results of using a CEP is to increase the event rate; and this in turn can reduce the sample size or the time required to observe a specified number of events, thereby resulting in a speedier, less costly clinical trial. Many believe that the only reason CEPs are ever employed is to this end, viz., saving money. I argue that there may be other circumstances that suggest the use of CEPs – that the choice of the primary response variable should be driven by the question the trial is being designed to answer.
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