Composite Endpoints: Sometimes More than a Solely Economic Consideration

Charles J Kowalski

doi:doi:10.11648/j.ajcem.20130101.15

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Composite Endpoints: Sometimes More than a Solely Economic Consideration

Charles J Kowalski

Published in American Journal of Clinical and Experimental Medicine (Volume 1, Issue 1)

Received: 18 June 2013 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.

Published in	American Journal of Clinical and Experimental Medicine (Volume 1, Issue 1)
DOI	10.11648/j.ajcem.20130101.15
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), 2013. Published by Science Publishing Group

Keywords

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

References

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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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ACS 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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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://article.sciencepublishinggroup.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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TY  - JOUR
T1  - Composite Endpoints: Sometimes More than a Solely Economic Consideration
AU  - Charles J Kowalski
Y1  - 2013/07/20
PY  - 2013
N1  - https://doi.org/10.11648/j.ajcem.20130101.15
DO  - 10.11648/j.ajcem.20130101.15
T2  - American Journal of Clinical and Experimental Medicine
JF  - American Journal of Clinical and Experimental Medicine
JO  - American Journal of Clinical and Experimental Medicine
SP  - 24
EP  - 34
PB  - Science Publishing Group
SN  - 2330-8133
UR  - https://doi.org/10.11648/j.ajcem.20130101.15
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.
VL  - 1
IS  - 1
ER  -

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Author Information

Charles J Kowalski

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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ACS 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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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

Copy | Download

@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://article.sciencepublishinggroup.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}
}

Copy | Download

TY  - JOUR
T1  - Composite Endpoints: Sometimes More than a Solely Economic Consideration
AU  - Charles J Kowalski
Y1  - 2013/07/20
PY  - 2013
N1  - https://doi.org/10.11648/j.ajcem.20130101.15
DO  - 10.11648/j.ajcem.20130101.15
T2  - American Journal of Clinical and Experimental Medicine
JF  - American Journal of Clinical and Experimental Medicine
JO  - American Journal of Clinical and Experimental Medicine
SP  - 24
EP  - 34
PB  - Science Publishing Group
SN  - 2330-8133
UR  - https://doi.org/10.11648/j.ajcem.20130101.15
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.
VL  - 1
IS  - 1
ER  -

Copy | Download