Current state-of-the-art and gaps in platform trials: 10 things you should know, insights from EU-PEARL

Adaptive designs; Clinical research; Patient-centred

Detalles Bibliográficos
Autores: König, Franz, Spiertz, Cécile, Millar, Daniel, Machín, Núria, Van Dessel, Ann, Rodríguez Navarro, Sarai, Genesca, Joan, Pericas, Juan M
Tipo de recurso: artículo
Estado:Versión publicada
Fecha de publicación:2024
País:España
Institución:Departament de Salut de la Generalitat de Catalunya (DS)
Repositorio:Scientia. Dipòsit d'Informació Digital del Departament de Salut
OAI Identifier:oai:scientiasalut.gencat.cat:11351/11022
Acceso en línea:https://hdl.handle.net/11351/11022
Access Level:acceso abierto
Palabra clave:Medicaments - Desenvolupament
Medicina - Investigació
ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Therapeutics::Clinical Protocols
ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Investigative Techniques::Drug Development
TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::terapéutica::protocolos clínicos
TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::técnicas de investigación::desarrollo de medicamentos
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oai_identifier_str oai:scientiasalut.gencat.cat:11351/11022
network_acronym_str ES
network_name_str España
repository_id_str
dc.title.none.fl_str_mv Current state-of-the-art and gaps in platform trials: 10 things you should know, insights from EU-PEARL
title Current state-of-the-art and gaps in platform trials: 10 things you should know, insights from EU-PEARL
spellingShingle Current state-of-the-art and gaps in platform trials: 10 things you should know, insights from EU-PEARL
König, Franz
Medicaments - Desenvolupament
Medicina - Investigació
ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Therapeutics::Clinical Protocols
ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Investigative Techniques::Drug Development
TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::terapéutica::protocolos clínicos
TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::técnicas de investigación::desarrollo de medicamentos
title_short Current state-of-the-art and gaps in platform trials: 10 things you should know, insights from EU-PEARL
title_full Current state-of-the-art and gaps in platform trials: 10 things you should know, insights from EU-PEARL
title_fullStr Current state-of-the-art and gaps in platform trials: 10 things you should know, insights from EU-PEARL
title_full_unstemmed Current state-of-the-art and gaps in platform trials: 10 things you should know, insights from EU-PEARL
title_sort Current state-of-the-art and gaps in platform trials: 10 things you should know, insights from EU-PEARL
dc.creator.none.fl_str_mv König, Franz
Spiertz, Cécile
Millar, Daniel
Machín, Núria
Van Dessel, Ann
Rodríguez Navarro, Sarai
Genesca, Joan
Pericas, Juan M
author König, Franz
author_facet König, Franz
Spiertz, Cécile
Millar, Daniel
Machín, Núria
Van Dessel, Ann
Rodríguez Navarro, Sarai
Genesca, Joan
Pericas, Juan M
author_role author
author2 Spiertz, Cécile
Millar, Daniel
Machín, Núria
Van Dessel, Ann
Rodríguez Navarro, Sarai
Genesca, Joan
Pericas, Juan M
author2_role author
author
author
author
author
author
author
dc.contributor.none.fl_str_mv Institut Català de la Salut
[Koenig F] Medical University of Vienna, Center for Medical Data Science, Vienna, Austria. [Spiertz C] Janssen Biologics BV, Leiden, the Netherlands. [Millar D] Former Employee, Janssen Research & Development, LLC, Raritan, NJ, USA. [Rodríguez-Navarro S] Vall d’Hebron Institut de Recerca (VHIR), Barcelona, Spain. [Machín N] TEAM-IT Research S.L., Barcelona, Spain. [Van Dessel A] Janssen Pharmaceutica NV, Beerse, Belgium. [Genescà J, Pericàs JM] Vall d’Hebron Institut de Recerca (VHIR), Barcelona, Spain. Servei d’Hepatologia, Vall d’Hebron Hospital Universitari, Barcelona, Spain. Spanish Network of Biomedical Research Centers, Digestive and Liver Diseases (CIBERehd), Madrid, Spain. Universitat Autònoma de Barcelona, Bellaterra, Spain
Vall d'Hebron Barcelona Hospital Campus
dc.subject.none.fl_str_mv Medicaments - Desenvolupament
Medicina - Investigació
ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Therapeutics::Clinical Protocols
ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Investigative Techniques::Drug Development
TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::terapéutica::protocolos clínicos
TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::técnicas de investigación::desarrollo de medicamentos
topic Medicaments - Desenvolupament
Medicina - Investigació
ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Therapeutics::Clinical Protocols
ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Investigative Techniques::Drug Development
TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::terapéutica::protocolos clínicos
TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::técnicas de investigación::desarrollo de medicamentos
description Adaptive designs; Clinical research; Patient-centred
publishDate 2024
dc.date.none.fl_str_mv 2024
2024
2024
dc.type.none.fl_str_mv info:eu-repo/semantics/article
info:eu-repo/semantics/publishedVersion
format article
status_str publishedVersion
dc.identifier.none.fl_str_mv https://hdl.handle.net/11351/11022
url https://hdl.handle.net/11351/11022
dc.language.none.fl_str_mv Inglés
language_invalid_str_mv Inglés
dc.relation.none.fl_str_mv eClinicalMedicine;67
https://doi.org/10.1016/j.eclinm.2023.102384
info:eu-repo/grantAgreement/EC/H2020/847989
dc.rights.none.fl_str_mv Attribution-NonCommercial-NoDerivatives 4.0 International
http://creativecommons.org/licenses/by-nc-nd/4.0/
info:eu-repo/semantics/openAccess
rights_invalid_str_mv 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.publisher.none.fl_str_mv Elsevier
publisher.none.fl_str_mv Elsevier
dc.source.none.fl_str_mv Scientia
reponame:Scientia. Dipòsit d'Informació Digital del Departament de Salut
instname:Departament de Salut de la Generalitat de Catalunya (DS)
instname_str Departament de Salut de la Generalitat de Catalunya (DS)
reponame_str Scientia. Dipòsit d'Informació Digital del Departament de Salut
collection Scientia. Dipòsit d'Informació Digital del Departament de Salut
repository.name.fl_str_mv
repository.mail.fl_str_mv
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spelling Current state-of-the-art and gaps in platform trials: 10 things you should know, insights from EU-PEARLKönig, FranzSpiertz, CécileMillar, DanielMachín, NúriaVan Dessel, AnnRodríguez Navarro, SaraiGenesca, JoanPericas, Juan MMedicaments - DesenvolupamentMedicina - InvestigacióANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Therapeutics::Clinical ProtocolsANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Investigative Techniques::Drug DevelopmentTÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::terapéutica::protocolos clínicosTÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::técnicas de investigación::desarrollo de medicamentosAdaptive designs; Clinical research; Patient-centredDiseños adaptativos; Investigación clínica; Centrada en el pacienteDissenys adaptatius; Recerca clínica; Centrada en el pacientPlatform trials bring the promise of making clinical research more efficient and more patient centric. While their use has become more widespread, including their prominent role during the COVID-19 pandemic response, broader adoption of platform trials has been limited by the lack of experience and tools to navigate the critical upfront planning required to launch such collaborative studies. The European Union-Patient-cEntric clinicAl tRial pLatform (EU-PEARL) initiative has produced new methodologies to expand the use of platform trials with an overarching infrastructure and services embedded into Integrated Research Platforms (IRPs), in collaboration with patient representatives and through consultation with U.S. Food and Drug Administration and European Medicines Agency stakeholders. In this narrative review, we discuss the outlook for platform trials in Europe, including challenges related to infrastructure, design, adaptations, data sharing and regulation. Documents derived from the EU-PEARL project, alongside a literature search including PubMed and relevant grey literature (e.g., guidance from regulatory agencies and health technology agencies) were used as sources for a multi-stage collaborative process through which the 10 more important points based on lessons drawn from the EU-PEARL project were developed and summarised as guidance for the setup of platform trials. We conclude that early involvement of critical stakeholder such as regulatory agencies or patients are critical steps in the implementation and later acceptance of platform trials. Addressing these gaps will be critical for attaining the full potential of platform trials for patients.EU-PEARL has received funding from the Innovative Medicines Initiative 2 Joint Undertaking under grant agreement No 853966. This Joint Undertaking receives support from the European Union’s Horizon 2020 research and innovation programme and EFPIA.ElsevierInstitut Català de la Salut[Koenig F] Medical University of Vienna, Center for Medical Data Science, Vienna, Austria. [Spiertz C] Janssen Biologics BV, Leiden, the Netherlands. [Millar D] Former Employee, Janssen Research & Development, LLC, Raritan, NJ, USA. [Rodríguez-Navarro S] Vall d’Hebron Institut de Recerca (VHIR), Barcelona, Spain. [Machín N] TEAM-IT Research S.L., Barcelona, Spain. [Van Dessel A] Janssen Pharmaceutica NV, Beerse, Belgium. [Genescà J, Pericàs JM] Vall d’Hebron Institut de Recerca (VHIR), Barcelona, Spain. Servei d’Hepatologia, Vall d’Hebron Hospital Universitari, Barcelona, Spain. Spanish Network of Biomedical Research Centers, Digestive and Liver Diseases (CIBERehd), Madrid, Spain. Universitat Autònoma de Barcelona, Bellaterra, SpainVall d'Hebron Barcelona Hospital Campus202420242024info:eu-repo/semantics/articleinfo:eu-repo/semantics/publishedVersionapplication/pdfhttps://hdl.handle.net/11351/11022Scientiareponame:Scientia. Dipòsit d'Informació Digital del Departament de Salutinstname:Departament de Salut de la Generalitat de Catalunya (DS)IngléseClinicalMedicine;67https://doi.org/10.1016/j.eclinm.2023.102384info:eu-repo/grantAgreement/EC/H2020/847989Attribution-NonCommercial-NoDerivatives 4.0 Internationalhttp://creativecommons.org/licenses/by-nc-nd/4.0/info:eu-repo/semantics/openAccessoai:scientiasalut.gencat.cat:11351/110222026-06-12T09:38:37Z
score 15.81155

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