Design of trials with composite endpoints with the R package compAREdesign

Composite endpoints are widely used as primary endpoints in clinical trials. Designing trials with time-to-event endpoints can be particularly challenging because the proportional hazard assumption usually does not hold when using a composite endpoint, even when the premise remains true for their co...

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Autores: Cortés Martínez, Jordi|||0000-0002-3764-0795, Bofill Roig, Marta|||0000-0002-4400-7541, Gómez Melis, Guadalupe|||0000-0003-4252-4884
Tipo de recurso: artículo
Fecha de publicación:2022
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:2117/377308
Acceso en línea:https://hdl.handle.net/2117/377308
https://dx.doi.org/10.48550/arXiv.2211.02535
Access Level:acceso abierto
Palabra clave:Biometry
Statistics
Pharmaceutical technology
Composite endpoints
Clinical trial
Study design
ARE
Biometria
Estadística
Tecnologia farmacèutica
Àrees temàtiques de la UPC::Matemàtiques i estadística::Estadística matemàtica
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spelling Design of trials with composite endpoints with the R package compAREdesignCortés Martínez, Jordi|||0000-0002-3764-0795Bofill Roig, Marta|||0000-0002-4400-7541Gómez Melis, Guadalupe|||0000-0003-4252-4884BiometryStatisticsPharmaceutical technologyComposite endpointsClinical trialStudy designAREBiometriaEstadísticaTecnologia farmacèuticaÀrees temàtiques de la UPC::Matemàtiques i estadística::Estadística matemàticaComposite endpoints are widely used as primary endpoints in clinical trials. Designing trials with time-to-event endpoints can be particularly challenging because the proportional hazard assumption usually does not hold when using a composite endpoint, even when the premise remains true for their components. Consequently, the conventional formulae for sample size calculation do not longer apply. We present the R package CompAREdesign by means of which the key elements of trial designs, such as the sample size and effect sizes, can be computed based on the information on the composite endpoint components. CompAREdesign provides the functions to assess the sensitivity and robustness of design calculations to variations in initial values and assumptions. Furthermore, we describe other features of the package, such as functions for the design of trials with binary composite endpoints, and functions to simulate trials with composite endpoints under a wide range of scenarios.This work was supported by the Ministerio de Economía y Competitividad (Spain) under Grant PID2019- 104830RB-I00 and the Departament d’Empresa i Coneixement de la Generalitat de Catalunya (Spain) under Grant 2017 SGR 622 (GRBIO). Marta Bofill Roig is a member of the EU Patient Centric Clinical Trial Platforms (EU-PEARL). This Joint Undertaking receives support from the European Union’s Horizon 2020 research and innovation program and EFPIA and Children’s Tumor Foundation, Global Alliance for TB Drug Development nonprofit organization, Springworks Therapeutics Inc. This publication reflects the authors’ views. Neither IMI nor the European Union, EFPIA, nor any Associated Partners are responsible for any use that may be made of the information contained herein.Peer Reviewed20222022-11-0420222022-11-29journal articlehttp://purl.org/coar/resource_type/c_6501AMhttp://purl.org/coar/version/c_ab4af688f83e57aainfo:eu-repo/semantics/articleapplication/pdfhttps://hdl.handle.net/2117/377308https://dx.doi.org/10.48550/arXiv.2211.02535reponame:UPCommons. Portal del coneixement obert de la UPCinstname:Universitat Politècnica de Catalunya (UPC)InglésengAgencia Estatal de Investigación http://doi.org/10.13039/501100011033 Plan Estatal de Investigación Científica y Técnica y de Innovación 2017-2020 PID2019-104830RB-I00 METODOLOGIAS ESTADISTICAS PARA DATOS CLINICOS Y OMICOS Y SUS APLICACIONES EN CIENCIAS DE LA SALUDopen accesshttp://purl.org/coar/access_right/c_abf2Attribution-NonCommercial-NoDerivatives 4.0 Internationalhttp://creativecommons.org/licenses/by-nc-nd/4.0/info:eu-repo/semantics/openAccessoai:upcommons.upc.edu:2117/3773082026-05-27T15:37:01Z
dc.title.none.fl_str_mv Design of trials with composite endpoints with the R package compAREdesign
title Design of trials with composite endpoints with the R package compAREdesign
spellingShingle Design of trials with composite endpoints with the R package compAREdesign
Cortés Martínez, Jordi|||0000-0002-3764-0795
Biometry
Statistics
Pharmaceutical technology
Composite endpoints
Clinical trial
Study design
ARE
Biometria
Estadística
Tecnologia farmacèutica
Àrees temàtiques de la UPC::Matemàtiques i estadística::Estadística matemàtica
title_short Design of trials with composite endpoints with the R package compAREdesign
title_full Design of trials with composite endpoints with the R package compAREdesign
title_fullStr Design of trials with composite endpoints with the R package compAREdesign
title_full_unstemmed Design of trials with composite endpoints with the R package compAREdesign
title_sort Design of trials with composite endpoints with the R package compAREdesign
dc.creator.none.fl_str_mv Cortés Martínez, Jordi|||0000-0002-3764-0795
Bofill Roig, Marta|||0000-0002-4400-7541
Gómez Melis, Guadalupe|||0000-0003-4252-4884
author Cortés Martínez, Jordi|||0000-0002-3764-0795
author_facet Cortés Martínez, Jordi|||0000-0002-3764-0795
Bofill Roig, Marta|||0000-0002-4400-7541
Gómez Melis, Guadalupe|||0000-0003-4252-4884
author_role author
author2 Bofill Roig, Marta|||0000-0002-4400-7541
Gómez Melis, Guadalupe|||0000-0003-4252-4884
author2_role author
author
dc.subject.none.fl_str_mv Biometry
Statistics
Pharmaceutical technology
Composite endpoints
Clinical trial
Study design
ARE
Biometria
Estadística
Tecnologia farmacèutica
Àrees temàtiques de la UPC::Matemàtiques i estadística::Estadística matemàtica
topic Biometry
Statistics
Pharmaceutical technology
Composite endpoints
Clinical trial
Study design
ARE
Biometria
Estadística
Tecnologia farmacèutica
Àrees temàtiques de la UPC::Matemàtiques i estadística::Estadística matemàtica
description Composite endpoints are widely used as primary endpoints in clinical trials. Designing trials with time-to-event endpoints can be particularly challenging because the proportional hazard assumption usually does not hold when using a composite endpoint, even when the premise remains true for their components. Consequently, the conventional formulae for sample size calculation do not longer apply. We present the R package CompAREdesign by means of which the key elements of trial designs, such as the sample size and effect sizes, can be computed based on the information on the composite endpoint components. CompAREdesign provides the functions to assess the sensitivity and robustness of design calculations to variations in initial values and assumptions. Furthermore, we describe other features of the package, such as functions for the design of trials with binary composite endpoints, and functions to simulate trials with composite endpoints under a wide range of scenarios.
publishDate 2022
dc.date.none.fl_str_mv 2022
2022-11-04
2022
2022-11-29
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/2117/377308
https://dx.doi.org/10.48550/arXiv.2211.02535
url https://hdl.handle.net/2117/377308
https://dx.doi.org/10.48550/arXiv.2211.02535
dc.language.none.fl_str_mv Inglés
eng
language_invalid_str_mv Inglés
language eng
dc.relation.none.fl_str_mv Agencia Estatal de Investigación http://doi.org/10.13039/501100011033 Plan Estatal de Investigación Científica y Técnica y de Innovación 2017-2020 PID2019-104830RB-I00 METODOLOGIAS ESTADISTICAS PARA DATOS CLINICOS Y OMICOS Y SUS APLICACIONES EN CIENCIAS DE LA SALUD
dc.rights.none.fl_str_mv open access
http://purl.org/coar/access_right/c_abf2
Attribution-NonCommercial-NoDerivatives 4.0 International
http://creativecommons.org/licenses/by-nc-nd/4.0/
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
Attribution-NonCommercial-NoDerivatives 4.0 International
http://creativecommons.org/licenses/by-nc-nd/4.0/
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
repository.mail.fl_str_mv
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