Review article: The need for more efficient and patient-oriented drug development pathways in NASH—setting the scene for platform trials

Drug development; Non-alcoholic steatohepatitis; Study designs

Detalles Bibliográficos
Autores: Di Prospero, Nicholas A., Anstee, Quentin M., Mesenbrinck, Peter, Kjær, Mette S, Rivera-Esteban, Jesús, Sena, Elena, Genesca, Joan, Pericas, Juan M, Manzano-Nunez, Ramiro
Tipo de recurso: artículo
Estado:Versión publicada
Fecha de publicación:2023
País:España
Institución:Varias* (Consorci de Biblioteques Universitáries de Catalunya, Centre de Serveis Científics i Acadèmics de Catalunya)
Repositorio:Recercat. Dipósit de la Recerca de Catalunya
OAI Identifier:oai:recercat.cat:11351/9931
Acceso en línea:https://hdl.handle.net/11351/9931
http://hdl.handle.net/11351/9931
Access Level:acceso abierto
Palabra clave:Medicaments - Desenvolupament
Esteatosi hepàtica - Tractament
ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Investigative Techniques::Drug Development
DISEASES::Digestive System Diseases::Liver Diseases::Fatty Liver::Non-alcoholic Fatty Liver Disease
Other subheadings::Other subheadings::Other subheadings::/drug therapy
TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::técnicas de investigación::desarrollo de medicamentos
ENFERMEDADES::enfermedades del sistema digestivo::enfermedades hepáticas::hígado graso::esteatosis hepática no alcohólica
Otros calificadores::Otros calificadores::Otros calificadores::/farmacoterapia
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oai_identifier_str oai:recercat.cat:11351/9931
network_acronym_str ES
network_name_str España
repository_id_str
dc.title.none.fl_str_mv Review article: The need for more efficient and patient-oriented drug development pathways in NASH—setting the scene for platform trials
title Review article: The need for more efficient and patient-oriented drug development pathways in NASH—setting the scene for platform trials
spellingShingle Review article: The need for more efficient and patient-oriented drug development pathways in NASH—setting the scene for platform trials
Di Prospero, Nicholas A.
Medicaments - Desenvolupament
Esteatosi hepàtica - Tractament
ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Investigative Techniques::Drug Development
DISEASES::Digestive System Diseases::Liver Diseases::Fatty Liver::Non-alcoholic Fatty Liver Disease
Other subheadings::Other subheadings::Other subheadings::/drug therapy
TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::técnicas de investigación::desarrollo de medicamentos
ENFERMEDADES::enfermedades del sistema digestivo::enfermedades hepáticas::hígado graso::esteatosis hepática no alcohólica
Otros calificadores::Otros calificadores::Otros calificadores::/farmacoterapia
title_short Review article: The need for more efficient and patient-oriented drug development pathways in NASH—setting the scene for platform trials
title_full Review article: The need for more efficient and patient-oriented drug development pathways in NASH—setting the scene for platform trials
title_fullStr Review article: The need for more efficient and patient-oriented drug development pathways in NASH—setting the scene for platform trials
title_full_unstemmed Review article: The need for more efficient and patient-oriented drug development pathways in NASH—setting the scene for platform trials
title_sort Review article: The need for more efficient and patient-oriented drug development pathways in NASH—setting the scene for platform trials
dc.creator.none.fl_str_mv Di Prospero, Nicholas A.
Anstee, Quentin M.
Mesenbrinck, Peter
Kjær, Mette S
Rivera-Esteban, Jesús
Sena, Elena
Genesca, Joan
Pericas, Juan M
Manzano-Nunez, Ramiro
author Di Prospero, Nicholas A.
author_facet Di Prospero, Nicholas A.
Anstee, Quentin M.
Mesenbrinck, Peter
Kjær, Mette S
Rivera-Esteban, Jesús
Sena, Elena
Genesca, Joan
Pericas, Juan M
Manzano-Nunez, Ramiro
author_role author
author2 Anstee, Quentin M.
Mesenbrinck, Peter
Kjær, Mette S
Rivera-Esteban, Jesús
Sena, Elena
Genesca, Joan
Pericas, Juan M
Manzano-Nunez, Ramiro
author2_role author
author
author
author
author
author
author
author
dc.contributor.none.fl_str_mv Institut Català de la Salut
[Pericàs JM, Rivera-Esteban J, Sena E, Manzano R, Genescà J] Unitat Hepàtica, Servei de Medicina Interna, Vall d'Hebron Hospital Universitari, Barcelona, Spain. Vall d'Hebron Institut de Recerca (VHIR), Barcelona, Spain. [Di Prospero NA] Janssen Research and Development, Raritan, New Jersey, USA. [Anstee QM] Liver Unit, The Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK. Translational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne, UK. [Mesenbrinck P] Analytics Department, Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA. [Kjaer MS] Novo Nordisk A/S, Bagsværd, Denmark
Vall d'Hebron Barcelona Hospital Campus
dc.subject.none.fl_str_mv Medicaments - Desenvolupament
Esteatosi hepàtica - Tractament
ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Investigative Techniques::Drug Development
DISEASES::Digestive System Diseases::Liver Diseases::Fatty Liver::Non-alcoholic Fatty Liver Disease
Other subheadings::Other subheadings::Other subheadings::/drug therapy
TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::técnicas de investigación::desarrollo de medicamentos
ENFERMEDADES::enfermedades del sistema digestivo::enfermedades hepáticas::hígado graso::esteatosis hepática no alcohólica
Otros calificadores::Otros calificadores::Otros calificadores::/farmacoterapia
topic Medicaments - Desenvolupament
Esteatosi hepàtica - Tractament
ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Investigative Techniques::Drug Development
DISEASES::Digestive System Diseases::Liver Diseases::Fatty Liver::Non-alcoholic Fatty Liver Disease
Other subheadings::Other subheadings::Other subheadings::/drug therapy
TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::técnicas de investigación::desarrollo de medicamentos
ENFERMEDADES::enfermedades del sistema digestivo::enfermedades hepáticas::hígado graso::esteatosis hepática no alcohólica
Otros calificadores::Otros calificadores::Otros calificadores::/farmacoterapia
description Drug development; Non-alcoholic steatohepatitis; Study designs
publishDate 2023
dc.date.none.fl_str_mv 2023
2023
2023
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/11351/9931
http://hdl.handle.net/11351/9931
url https://hdl.handle.net/11351/9931
http://hdl.handle.net/11351/9931
dc.language.none.fl_str_mv Inglés
language_invalid_str_mv Inglés
dc.relation.none.fl_str_mv Alimentary Pharmacology & Therapeutics;57(9)
https://doi.org/10.1111/apt.17456
info:eu-repo/grantAgreement/EC/H2020/847989
info:eu-repo/grantAgreement/ES/PE2017-2020/PI18%2F00947
info:eu-repo/grantAgreement/ES/PEICTI2021-2023/PI21%2F00691
dc.rights.none.fl_str_mv Attribution-NonCommercial 4.0 International
http://creativecommons.org/licenses/by-nc/4.0/
info:eu-repo/semantics/openAccess
rights_invalid_str_mv 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.publisher.none.fl_str_mv Wiley
publisher.none.fl_str_mv Wiley
dc.source.none.fl_str_mv Scientia
reponame:Recercat. Dipósit de la Recerca de Catalunya
instname:Varias* (Consorci de Biblioteques Universitáries de Catalunya, Centre de Serveis Científics i Acadèmics de Catalunya)
instname_str Varias* (Consorci de Biblioteques Universitáries de Catalunya, Centre de Serveis Científics i Acadèmics de Catalunya)
reponame_str Recercat. Dipósit de la Recerca de Catalunya
collection Recercat. Dipósit de la Recerca de Catalunya
repository.name.fl_str_mv
repository.mail.fl_str_mv
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spelling Review article: The need for more efficient and patient-oriented drug development pathways in NASH—setting the scene for platform trialsDi Prospero, Nicholas A.Anstee, Quentin M.Mesenbrinck, PeterKjær, Mette SRivera-Esteban, JesúsSena, ElenaGenesca, JoanPericas, Juan MManzano-Nunez, RamiroMedicaments - DesenvolupamentEsteatosi hepàtica - TractamentANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Investigative Techniques::Drug DevelopmentDISEASES::Digestive System Diseases::Liver Diseases::Fatty Liver::Non-alcoholic Fatty Liver DiseaseOther subheadings::Other subheadings::Other subheadings::/drug therapyTÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::técnicas de investigación::desarrollo de medicamentosENFERMEDADES::enfermedades del sistema digestivo::enfermedades hepáticas::hígado graso::esteatosis hepática no alcohólicaOtros calificadores::Otros calificadores::Otros calificadores::/farmacoterapiaDrug development; Non-alcoholic steatohepatitis; Study designsDesenvolupament de fàrmacs; Esteatohepatitis no alcohòlica; Dissenys d'estudiDesarrollo de fármacos; Esteatohepatitis no alcohólica; Diseños de estudioBackground and Aims Non-alcoholic steatohepatitis (NASH) constitutes a significant unmet medical need with a burgeoning field of clinical research and drug development. Platform trials (PT) might help accelerate drug development while lowering overall costs and creating a more patient-centric environment. This review provides a comprehensive and nuanced assessment of the NASH clinical development landscape. Methods Narrative review and expert opinion with insight gained during the EU Patient-cEntric clinicAl tRial pLatforms (EU-PEARL) project. Results Although NASH represents an opportunity to use adaptive trial designs, including master protocols for PT, there are barriers that might be encountered owing to distinct and sometimes opposing priorities held by these stakeholders and potential ways to overcome them. The following aspects are critical for the feasibility of a future PT in NASH: readiness of the drug pipeline, mainly from large drug companies, while there is not yet an FDA/EMA-approved treatment; the most suitable design (trial Phase and type of population, e.g., Phase 2b for non-cirrhotic NASH patients); the operational requirements such as the scope of the clinical network, the use of concurrent versus non-concurrent control arms, or the re-allocation of participants upon trial adaptations; the methodological appraisal (i.e. Bayesian vs. frequentist approach); patients' needs and patient-centred outcomes; main regulatory considerations and the funding and sustainability scenarios. Conclusions PT represent a promising avenue in NASH but there are a number of conundrums that need addressing. It is likely that before a global NASH PT becomes a reality, ‘proof-of-platform’ at a smaller scale needs to be provided.JMP reports having received consulting fees from Boehringer-Ingelheim, MSD and Novo Nordisk. He has received speaking fees from Gilead, Intercept, and Novo Nordisk, and travel expenses from Gilead, Rubió, Pfizer, Astellas, MSD, CUBICIN, and Novo Nordisk. He has received educational and research support from Madrigal, Gilead, Pfizer, Astellas, Accelerate, Novartis, Abbvie, ViiV, and MSD. Funds from European Commission/EFPIA IMI2 853966-2, IMI2 777377, H2020 847989, and ISCIII PI19/01898. NAdP works for Janssen. QMA is Coordinator of the EU IMI-2 LITMUS consortium, which is funded by the EU Horizon 2020 programme and EFPIA. This multistakeholder consortium includes industry partners. He reports research Grant Funding: Allergan/Tobira, AstraZeneca, Boehringer-Ingelheim, Glaxo SmithKline, Glympse Bio, Intercept, Novartis Pharma AG, Pfizer Ltd. Consultancy: 89Bio, Abbvie/Allergan, Akero, Altimentiv, Altimmune, AstraZeneca, Axcella, Blade, BMS, BNN Cardio, Boehringer-Ingelheim, Cirius, CymaBay, EcoR1, E3Bio, Eli Lilly & Company Ltd., Galmed, Genentech, Genfit SA, Gilead, Grunthal, HistoIndex, Indalo, Intercept Pharma Europe Ltd., Inventiva, IQVIA, Janssen, Johnson & Johnson, Madrigal, MedImmune, Medpace, Merck, Metacrine, NGMBio, North Sea Therapeutics, Novartis, Novo Nordisk A/S, PathAI, Pfizer Ltd., Poxel, ProSciento, Raptor Pharma, Roche, Servier, Shionogi, Terns, The Medicines Company, Viking Therapeutics. Speaker: Abbott Laboratories, Allergan/Tobira, BMS, Clinical Care Options, Falk, Fishawack, Genfit SA, Gilead, Integritas Communications, Kenes, Medscape. Royalties: Elsevier Ltd. PM works for Novartis. MSK is an employee of and a shareholder in Novo Nordisk A/S. JG has received consulting fees from Boehringer-Ingelheim, speaking fees from Echosens and travel expenses from Gilead and Abbie. Funds from ISCIII PI18/00947 and PI21/00691. JRE has received speaking fees from Gilead. FT lab’ work has been supported by the German Research Foundation (DFG, CRC/TR 362) and research grants from Gilead, Allergan, Bristol-Myers Squibb and Inventiva. VR consults for and Intercept, Novo Nordisk, Galmed, Poxel, NGM, Madrigal, Enyo, Sagimet, 89 Bio, Prosciento, Terns, and Theratechnologies, and received grants from Intercept and Gilead.WileyInstitut Català de la Salut[Pericàs JM, Rivera-Esteban J, Sena E, Manzano R, Genescà J] Unitat Hepàtica, Servei de Medicina Interna, Vall d'Hebron Hospital Universitari, Barcelona, Spain. Vall d'Hebron Institut de Recerca (VHIR), Barcelona, Spain. [Di Prospero NA] Janssen Research and Development, Raritan, New Jersey, USA. [Anstee QM] Liver Unit, The Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK. Translational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne, UK. [Mesenbrinck P] Analytics Department, Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA. [Kjaer MS] Novo Nordisk A/S, Bagsværd, DenmarkVall d'Hebron Barcelona Hospital Campus202320232023info:eu-repo/semantics/articleinfo:eu-repo/semantics/publishedVersionapplication/pdfhttps://hdl.handle.net/11351/9931http://hdl.handle.net/11351/9931Scientiareponame:Recercat. Dipósit de la Recerca de Catalunyainstname:Varias* (Consorci de Biblioteques Universitáries de Catalunya, Centre de Serveis Científics i Acadèmics de Catalunya)InglésAlimentary Pharmacology & Therapeutics;57(9)https://doi.org/10.1111/apt.17456info:eu-repo/grantAgreement/EC/H2020/847989info:eu-repo/grantAgreement/ES/PE2017-2020/PI18%2F00947info:eu-repo/grantAgreement/ES/PEICTI2021-2023/PI21%2F00691Attribution-NonCommercial 4.0 Internationalhttp://creativecommons.org/licenses/by-nc/4.0/info:eu-repo/semantics/openAccessoai:recercat.cat:11351/99312026-05-29T05:05:01Z
score 15.811543

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