Regulatory issues of platform trials: learnings from EU-PEARL

Although platform trials have many benefits, the complexity of these designs may result not only in increased methodological but also regulatory and ethical challenges. These aspects were addressed as part of the IMI project EU Patient-Centric Clinical Trial Platforms (EU-PEARL). We reviewed the ava...

ver descrição completa

Detalhes bibliográficos
Autores: Lan Nguyen, Quynh, Hees, Katharina, Hernandez Penna, Sabina, König, Franz, Posch, Martin, Bofill Roig, Marta|||0000-0002-4400-7541, Meyer, Elias Laurin, Freitag, Michaela Maria, Parke, Tom, Otte, Maximilian, Dauben, Hans-Peter, Mielke, Tobias, Spiertz, Cecile, Mesenbrink, Peter, Gidh-Jain, Madhavi, Pierre, Suzanne, Morello, Salvatore, Hofner, Benjamin
Formato: artículo
Fecha de publicación:2024
País:España
Recursos:Universitat Politècnica de Catalunya (UPC)
Repositorio:UPCommons. Portal del coneixement obert de la UPC
Idioma:inglés
OAI Identifier:oai:upcommons.upc.edu:2117/418072
Acesso em linha:https://hdl.handle.net/2117/418072
https://dx.doi.org/10.1002/cpt.3244
Access Level:acceso abierto
Palavra-chave:Àrees temàtiques de la UPC::Economia i organització d'empreses::Gestió i direcció
id ES_e7eef950e9d20c021ae35a1a6a33cceb
oai_identifier_str oai:upcommons.upc.edu:2117/418072
network_acronym_str ES
network_name_str España
repository_id_str
spelling Regulatory issues of platform trials: learnings from EU-PEARLLan Nguyen, QuynhHees, KatharinaHernandez Penna, SabinaKönig, FranzPosch, MartinBofill Roig, Marta|||0000-0002-4400-7541Meyer, Elias LaurinFreitag, Michaela MariaParke, TomOtte, MaximilianDauben, Hans-PeterMielke, TobiasSpiertz, CecileMesenbrink, PeterGidh-Jain, MadhaviPierre, SuzanneMorello, SalvatoreHofner, BenjaminÀrees temàtiques de la UPC::Economia i organització d'empreses::Gestió i direccióAlthough platform trials have many benefits, the complexity of these designs may result not only in increased methodological but also regulatory and ethical challenges. These aspects were addressed as part of the IMI project EU Patient-Centric Clinical Trial Platforms (EU-PEARL). We reviewed the available guidelines on platform trials in the European Union and the United States. This is supported and complemented by feedback received from regulatory interactions with the European Medicines Agency and the US Food and Drug Administration. Throughout the project we collected the needs of all relevant stakeholders including ethics committees, regulators, and health technology assessment bodies through active dialog and dedicated stakeholder workshops. Furthermore, we focused on methodological aspects and where applicable identified the corresponding guidance. Learnings from the guideline review, regulatory interactions, and workshops are provided. Based on these, a master protocol template was developed. Issues that still need harmonization or clarification in guidelines or where further methodological research is needed are also presented. These include questions around clinical trial submissions in Europe, the need for multiplicity control across the whole master protocol, the use of non-concurrent controls, and the impact of different randomization schemes. Master protocols are an efficient and patient-centered clinical trial design that can expedite drug development. However, they can also introduce additional operational and regulatory complexities. It is important to understand the different requirements of stakeholders upfront and address them in the trial. While relevant guidance is increasing, early dialog with relevant stakeholders can help to further support such designs.EU-PEARL has received funding from the Innovative Medicines Initiative (IMI) 2 Joint Undertaking under grant agreement No. 853966. This Joint Undertaking receives support from the European Union's Horizon 2020 research and innovation program and EFPIA. See http://www.imi.europa.eu for further details. This publication reflects the authors' views. Neither the IMI nor the European Union, EFPIA, or any Associated Partners are responsible for any use that may be made of the information contained herein.Peer Reviewed20242024-07-0120242024-11-15journal articlehttp://purl.org/coar/resource_type/c_6501VoRhttp://purl.org/coar/version/c_970fb48d4fbd8a85info:eu-repo/semantics/articleapplication/pdfhttps://hdl.handle.net/2117/418072https://dx.doi.org/10.1002/cpt.3244reponame:UPCommons. Portal del coneixement obert de la UPCinstname:Universitat Politècnica de Catalunya (UPC)Inglésengopen accesshttp://purl.org/coar/access_right/c_abf2Attribution-NonCommercial 4.0 Internationalhttp://creativecommons.org/licenses/by-nc/4.0/info:eu-repo/semantics/openAccessoai:upcommons.upc.edu:2117/4180722026-05-27T15:37:01Z
dc.title.none.fl_str_mv Regulatory issues of platform trials: learnings from EU-PEARL
title Regulatory issues of platform trials: learnings from EU-PEARL
spellingShingle Regulatory issues of platform trials: learnings from EU-PEARL
Lan Nguyen, Quynh
Àrees temàtiques de la UPC::Economia i organització d'empreses::Gestió i direcció
title_short Regulatory issues of platform trials: learnings from EU-PEARL
title_full Regulatory issues of platform trials: learnings from EU-PEARL
title_fullStr Regulatory issues of platform trials: learnings from EU-PEARL
title_full_unstemmed Regulatory issues of platform trials: learnings from EU-PEARL
title_sort Regulatory issues of platform trials: learnings from EU-PEARL
dc.creator.none.fl_str_mv Lan Nguyen, Quynh
Hees, Katharina
Hernandez Penna, Sabina
König, Franz
Posch, Martin
Bofill Roig, Marta|||0000-0002-4400-7541
Meyer, Elias Laurin
Freitag, Michaela Maria
Parke, Tom
Otte, Maximilian
Dauben, Hans-Peter
Mielke, Tobias
Spiertz, Cecile
Mesenbrink, Peter
Gidh-Jain, Madhavi
Pierre, Suzanne
Morello, Salvatore
Hofner, Benjamin
author Lan Nguyen, Quynh
author_facet Lan Nguyen, Quynh
Hees, Katharina
Hernandez Penna, Sabina
König, Franz
Posch, Martin
Bofill Roig, Marta|||0000-0002-4400-7541
Meyer, Elias Laurin
Freitag, Michaela Maria
Parke, Tom
Otte, Maximilian
Dauben, Hans-Peter
Mielke, Tobias
Spiertz, Cecile
Mesenbrink, Peter
Gidh-Jain, Madhavi
Pierre, Suzanne
Morello, Salvatore
Hofner, Benjamin
author_role author
author2 Hees, Katharina
Hernandez Penna, Sabina
König, Franz
Posch, Martin
Bofill Roig, Marta|||0000-0002-4400-7541
Meyer, Elias Laurin
Freitag, Michaela Maria
Parke, Tom
Otte, Maximilian
Dauben, Hans-Peter
Mielke, Tobias
Spiertz, Cecile
Mesenbrink, Peter
Gidh-Jain, Madhavi
Pierre, Suzanne
Morello, Salvatore
Hofner, Benjamin
author2_role author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
dc.subject.none.fl_str_mv Àrees temàtiques de la UPC::Economia i organització d'empreses::Gestió i direcció
topic Àrees temàtiques de la UPC::Economia i organització d'empreses::Gestió i direcció
description Although platform trials have many benefits, the complexity of these designs may result not only in increased methodological but also regulatory and ethical challenges. These aspects were addressed as part of the IMI project EU Patient-Centric Clinical Trial Platforms (EU-PEARL). We reviewed the available guidelines on platform trials in the European Union and the United States. This is supported and complemented by feedback received from regulatory interactions with the European Medicines Agency and the US Food and Drug Administration. Throughout the project we collected the needs of all relevant stakeholders including ethics committees, regulators, and health technology assessment bodies through active dialog and dedicated stakeholder workshops. Furthermore, we focused on methodological aspects and where applicable identified the corresponding guidance. Learnings from the guideline review, regulatory interactions, and workshops are provided. Based on these, a master protocol template was developed. Issues that still need harmonization or clarification in guidelines or where further methodological research is needed are also presented. These include questions around clinical trial submissions in Europe, the need for multiplicity control across the whole master protocol, the use of non-concurrent controls, and the impact of different randomization schemes. Master protocols are an efficient and patient-centered clinical trial design that can expedite drug development. However, they can also introduce additional operational and regulatory complexities. It is important to understand the different requirements of stakeholders upfront and address them in the trial. While relevant guidance is increasing, early dialog with relevant stakeholders can help to further support such designs.
publishDate 2024
dc.date.none.fl_str_mv 2024
2024-07-01
2024
2024-11-15
dc.type.none.fl_str_mv journal article
http://purl.org/coar/resource_type/c_6501
VoR
http://purl.org/coar/version/c_970fb48d4fbd8a85
dc.type.openaire.fl_str_mv info:eu-repo/semantics/article
format article
dc.identifier.none.fl_str_mv https://hdl.handle.net/2117/418072
https://dx.doi.org/10.1002/cpt.3244
url https://hdl.handle.net/2117/418072
https://dx.doi.org/10.1002/cpt.3244
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
Attribution-NonCommercial 4.0 International
http://creativecommons.org/licenses/by-nc/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 4.0 International
http://creativecommons.org/licenses/by-nc/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
_version_ 1869422897918902272
score 15.81155

Registos relacionados