Selecting the primary endpoint in a randomized clinical trial: the ARE method

The decision on the primary endpoint in a randomized clinical trial is of paramount importance and the combination of several endpoints might be a reasonable choice. Gómez and Lagakos (2013) have developed a method that quantifies how much more efficient it could be to use a composite instead of an...

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Autores: Plana-Ripoll, Oleguer, Gómez Melis, Guadalupe|||0000-0003-4252-4884
Tipo de recurso: artículo
Fecha de publicación:2016
País:España
Institución:Universitat Politècnica de Catalunya (UPC)
Repositorio:UPCommons. Portal del coneixement obert de la UPC
Idioma:inglés
OAI Identifier:oai:upcommons.upc.edu:2117/102360
Acceso en línea:https://hdl.handle.net/2117/102360
https://dx.doi.org/10.1080/10543406.2015.1094808
Access Level:acceso abierto
Palabra clave:Asymptotic relative efficiency
composite endpoint
copulas
logrank
randomized clinical trial
Classificació AMS::90 Operations research, mathematical programming
Àrees temàtiques de la UPC::Matemàtiques i estadística::Investigació operativa
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spelling Selecting the primary endpoint in a randomized clinical trial: the ARE methodPlana-Ripoll, OleguerGómez Melis, Guadalupe|||0000-0003-4252-4884Asymptotic relative efficiencycomposite endpointcopulaslogrankrandomized clinical trialClassificació AMS::90 Operations research, mathematical programmingÀrees temàtiques de la UPC::Matemàtiques i estadística::Investigació operativaThe decision on the primary endpoint in a randomized clinical trial is of paramount importance and the combination of several endpoints might be a reasonable choice. Gómez and Lagakos (2013) have developed a method that quantifies how much more efficient it could be to use a composite instead of an individual relevant endpoint. From the information provided by the frequencies of observing the component endpoints in the control group and by the relative treatment effects on each individual endpoint, the asymptotic relative efficiency (ARE) can be computed. This article presents the applicability of the ARE method as a practical and objective tool to evaluate which components, among the plausible ones, are more efficient in the construction of the primary endpoint. The method is illustrated with two real cardiovascular clinical trials and is extended to allow for different dependence structures between the times to the individual endpoints. The influence of this choice on the recommendation on whether or not to use the composite endpoint as the primary endpoint for the investigation is studied. We conclude that the recommendation between using the composite or the relevant endpoint only depends on the frequencies of the endpoints and the relative effects of the treatment.Peer Reviewed20162016-01-0120172017-03-13journal articlehttp://purl.org/coar/resource_type/c_6501AMhttp://purl.org/coar/version/c_ab4af688f83e57aainfo:eu-repo/semantics/articleapplication/pdfhttps://hdl.handle.net/2117/102360https://dx.doi.org/10.1080/10543406.2015.1094808reponame:UPCommons. Portal del coneixement obert de la UPCinstname:Universitat Politècnica de Catalunya (UPC)Inglésengopen accesshttp://purl.org/coar/access_right/c_abf2http://creativecommons.org/licenses/by-nc-nd/3.0/es/info:eu-repo/semantics/openAccessoai:upcommons.upc.edu:2117/1023602026-05-27T15:37:01Z
dc.title.none.fl_str_mv Selecting the primary endpoint in a randomized clinical trial: the ARE method
title Selecting the primary endpoint in a randomized clinical trial: the ARE method
spellingShingle Selecting the primary endpoint in a randomized clinical trial: the ARE method
Plana-Ripoll, Oleguer
Asymptotic relative efficiency
composite endpoint
copulas
logrank
randomized clinical trial
Classificació AMS::90 Operations research, mathematical programming
Àrees temàtiques de la UPC::Matemàtiques i estadística::Investigació operativa
title_short Selecting the primary endpoint in a randomized clinical trial: the ARE method
title_full Selecting the primary endpoint in a randomized clinical trial: the ARE method
title_fullStr Selecting the primary endpoint in a randomized clinical trial: the ARE method
title_full_unstemmed Selecting the primary endpoint in a randomized clinical trial: the ARE method
title_sort Selecting the primary endpoint in a randomized clinical trial: the ARE method
dc.creator.none.fl_str_mv Plana-Ripoll, Oleguer
Gómez Melis, Guadalupe|||0000-0003-4252-4884
author Plana-Ripoll, Oleguer
author_facet Plana-Ripoll, Oleguer
Gómez Melis, Guadalupe|||0000-0003-4252-4884
author_role author
author2 Gómez Melis, Guadalupe|||0000-0003-4252-4884
author2_role author
dc.subject.none.fl_str_mv Asymptotic relative efficiency
composite endpoint
copulas
logrank
randomized clinical trial
Classificació AMS::90 Operations research, mathematical programming
Àrees temàtiques de la UPC::Matemàtiques i estadística::Investigació operativa
topic Asymptotic relative efficiency
composite endpoint
copulas
logrank
randomized clinical trial
Classificació AMS::90 Operations research, mathematical programming
Àrees temàtiques de la UPC::Matemàtiques i estadística::Investigació operativa
description The decision on the primary endpoint in a randomized clinical trial is of paramount importance and the combination of several endpoints might be a reasonable choice. Gómez and Lagakos (2013) have developed a method that quantifies how much more efficient it could be to use a composite instead of an individual relevant endpoint. From the information provided by the frequencies of observing the component endpoints in the control group and by the relative treatment effects on each individual endpoint, the asymptotic relative efficiency (ARE) can be computed. This article presents the applicability of the ARE method as a practical and objective tool to evaluate which components, among the plausible ones, are more efficient in the construction of the primary endpoint. The method is illustrated with two real cardiovascular clinical trials and is extended to allow for different dependence structures between the times to the individual endpoints. The influence of this choice on the recommendation on whether or not to use the composite endpoint as the primary endpoint for the investigation is studied. We conclude that the recommendation between using the composite or the relevant endpoint only depends on the frequencies of the endpoints and the relative effects of the treatment.
publishDate 2016
dc.date.none.fl_str_mv 2016
2016-01-01
2017
2017-03-13
dc.type.none.fl_str_mv journal article
http://purl.org/coar/resource_type/c_6501
AM
http://purl.org/coar/version/c_ab4af688f83e57aa
dc.type.openaire.fl_str_mv info:eu-repo/semantics/article
format article
dc.identifier.none.fl_str_mv https://hdl.handle.net/2117/102360
https://dx.doi.org/10.1080/10543406.2015.1094808
url https://hdl.handle.net/2117/102360
https://dx.doi.org/10.1080/10543406.2015.1094808
dc.language.none.fl_str_mv Inglés
eng
language_invalid_str_mv Inglés
language eng
dc.rights.none.fl_str_mv open access
http://purl.org/coar/access_right/c_abf2

http://creativecommons.org/licenses/by-nc-nd/3.0/es/
dc.rights.openaire.fl_str_mv info:eu-repo/semantics/openAccess
rights_invalid_str_mv open access
http://purl.org/coar/access_right/c_abf2

http://creativecommons.org/licenses/by-nc-nd/3.0/es/
eu_rights_str_mv openAccess
dc.format.none.fl_str_mv application/pdf
dc.source.none.fl_str_mv reponame:UPCommons. Portal del coneixement obert de la UPC
instname:Universitat Politècnica de Catalunya (UPC)
instname_str Universitat Politècnica de Catalunya (UPC)
reponame_str UPCommons. Portal del coneixement obert de la UPC
collection UPCommons. Portal del coneixement obert de la UPC
repository.name.fl_str_mv
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