Platform trials to overcome major shortcomings of traditional clinical trials in non-alcoholic steatohepatitis? Pros and cons

Non-alcoholic steatohepatitis; Drug development; Non-invasive biomarkers

Detalles Bibliográficos
Autores: Pericàs Pulido, Juan Manuel, Tacke, Frank, Di Prospero, Nicholas A., Kjær, Mette Skalshøj, Mesenbrink, Peter, Koenig, Franz, Genescà Ferrer, Joan, Ratziu, Vlad, Anstee, Quentin M.
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:11351/9089
Acceso en línea:https://hdl.handle.net/11351/9089
http://hdl.handle.net/11351/9089
Access Level:acceso abierto
Palabra clave:Esteatosi hepàtica - Complicacions
Medicaments - Desenvolupament
DISEASES::Digestive System Diseases::Liver Diseases::Fatty Liver::Non-alcoholic Fatty Liver Disease
Other subheadings::Other subheadings::Other subheadings::/complications
ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Investigative Techniques::Drug Development
ENFERMEDADES::enfermedades del sistema digestivo::enfermedades hepáticas::hígado graso::esteatosis hepática no alcohólica
Otros calificadores::Otros calificadores::Otros calificadores::/complicaciones
TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::técnicas de investigación::desarrollo de medicamentos
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oai_identifier_str oai:recercat.cat:11351/9089
network_acronym_str ES
network_name_str España
repository_id_str
dc.title.none.fl_str_mv Platform trials to overcome major shortcomings of traditional clinical trials in non-alcoholic steatohepatitis? Pros and cons
title Platform trials to overcome major shortcomings of traditional clinical trials in non-alcoholic steatohepatitis? Pros and cons
spellingShingle Platform trials to overcome major shortcomings of traditional clinical trials in non-alcoholic steatohepatitis? Pros and cons
Pericàs Pulido, Juan Manuel
Esteatosi hepàtica - Complicacions
Medicaments - Desenvolupament
DISEASES::Digestive System Diseases::Liver Diseases::Fatty Liver::Non-alcoholic Fatty Liver Disease
Other subheadings::Other subheadings::Other subheadings::/complications
ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Investigative Techniques::Drug Development
ENFERMEDADES::enfermedades del sistema digestivo::enfermedades hepáticas::hígado graso::esteatosis hepática no alcohólica
Otros calificadores::Otros calificadores::Otros calificadores::/complicaciones
TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::técnicas de investigación::desarrollo de medicamentos
title_short Platform trials to overcome major shortcomings of traditional clinical trials in non-alcoholic steatohepatitis? Pros and cons
title_full Platform trials to overcome major shortcomings of traditional clinical trials in non-alcoholic steatohepatitis? Pros and cons
title_fullStr Platform trials to overcome major shortcomings of traditional clinical trials in non-alcoholic steatohepatitis? Pros and cons
title_full_unstemmed Platform trials to overcome major shortcomings of traditional clinical trials in non-alcoholic steatohepatitis? Pros and cons
title_sort Platform trials to overcome major shortcomings of traditional clinical trials in non-alcoholic steatohepatitis? Pros and cons
dc.creator.none.fl_str_mv Pericàs Pulido, Juan Manuel
Tacke, Frank
Di Prospero, Nicholas A.
Kjær, Mette Skalshøj
Mesenbrink, Peter
Koenig, Franz
Genescà Ferrer, Joan
Ratziu, Vlad
Anstee, Quentin M.
author Pericàs Pulido, Juan Manuel
author_facet Pericàs Pulido, Juan Manuel
Tacke, Frank
Di Prospero, Nicholas A.
Kjær, Mette Skalshøj
Mesenbrink, Peter
Koenig, Franz
Genescà Ferrer, Joan
Ratziu, Vlad
Anstee, Quentin M.
author_role author
author2 Tacke, Frank
Di Prospero, Nicholas A.
Kjær, Mette Skalshøj
Mesenbrink, Peter
Koenig, Franz
Genescà Ferrer, Joan
Ratziu, Vlad
Anstee, Quentin M.
author2_role author
author
author
author
author
author
author
author
dc.contributor.none.fl_str_mv Institut Català de la Salut
[Pericàs JM, Genescà J] Unitat Hepàtica, Servei de Medicina Interna, Vall d'Hebron Hospital Universitari, Barcelona, Spain. Vall d'Hebron Institut de Recerca (VHIR), Barcelona, Spain. Universitat Autònoma de Barcelona, Bellaterra, Spain. Centros de Investigación Biomédica en Red en Enfermedades Hepáticas y Digestivas (CIBERehd), Barcelona, Spain. [Tacke F] Department of Hepatology and Gastroenterology, Charité - Universitätsmedizin Berlin, Berlin, Germany. [Anstee QM] Translational and Clinical Research Institute, Faculty of Medical Sciences, Newcastle University, Newcastle NIHR Biomedical Research Centre, Newcastle Upon Tyne Hospitals NHS Foundation Trust, Newcastle Upon Tyne, UK. [Di Prospero NA] Janssen Research and Development, Raritan, New Jersey, USA. [Kjær MS] Novo Nordisk, Bagsværd, Denmark. [Mesenbrink P] Analytics Department, Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA. [Koenig F] Center for Medical Statistics, Informatics and Intelligent Systems, Medical University of Vienna, Vienna, Austria. [Ratziu V] Department of Hepatology, Pitié-Salpêtrière Hospital, France
Vall d'Hebron Barcelona Hospital Campus
dc.subject.none.fl_str_mv Esteatosi hepàtica - Complicacions
Medicaments - Desenvolupament
DISEASES::Digestive System Diseases::Liver Diseases::Fatty Liver::Non-alcoholic Fatty Liver Disease
Other subheadings::Other subheadings::Other subheadings::/complications
ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Investigative Techniques::Drug Development
ENFERMEDADES::enfermedades del sistema digestivo::enfermedades hepáticas::hígado graso::esteatosis hepática no alcohólica
Otros calificadores::Otros calificadores::Otros calificadores::/complicaciones
TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::técnicas de investigación::desarrollo de medicamentos
topic Esteatosi hepàtica - Complicacions
Medicaments - Desenvolupament
DISEASES::Digestive System Diseases::Liver Diseases::Fatty Liver::Non-alcoholic Fatty Liver Disease
Other subheadings::Other subheadings::Other subheadings::/complications
ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Investigative Techniques::Drug Development
ENFERMEDADES::enfermedades del sistema digestivo::enfermedades hepáticas::hígado graso::esteatosis hepática no alcohólica
Otros calificadores::Otros calificadores::Otros calificadores::/complicaciones
TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::técnicas de investigación::desarrollo de medicamentos
description Non-alcoholic steatohepatitis; Drug development; Non-invasive biomarkers
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/11351/9089
http://hdl.handle.net/11351/9089
url https://hdl.handle.net/11351/9089
http://hdl.handle.net/11351/9089
dc.language.none.fl_str_mv Inglés
language_invalid_str_mv Inglés
dc.relation.none.fl_str_mv Journal of Hepatology;78(2)
https://doi.org/10.1016/j.jhep.2022.09.021
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: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
_version_ 1869406570223239168
spelling Platform trials to overcome major shortcomings of traditional clinical trials in non-alcoholic steatohepatitis? Pros and consPericàs Pulido, Juan ManuelTacke, FrankDi Prospero, Nicholas A.Kjær, Mette SkalshøjMesenbrink, PeterKoenig, FranzGenescà Ferrer, JoanRatziu, VladAnstee, Quentin M.Esteatosi hepàtica - ComplicacionsMedicaments - DesenvolupamentDISEASES::Digestive System Diseases::Liver Diseases::Fatty Liver::Non-alcoholic Fatty Liver DiseaseOther subheadings::Other subheadings::Other subheadings::/complicationsANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Investigative Techniques::Drug DevelopmentENFERMEDADES::enfermedades del sistema digestivo::enfermedades hepáticas::hígado graso::esteatosis hepática no alcohólicaOtros calificadores::Otros calificadores::Otros calificadores::/complicacionesTÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::técnicas de investigación::desarrollo de medicamentosNon-alcoholic steatohepatitis; Drug development; Non-invasive biomarkersEsteatohepatitis no alcohólica; Desarrollo de fármacos; Biomarcadores no invasivosEsteatohepatitis no alcohòlica; Desenvolupament de medicaments; Biomarcadors no invasiusNon-alcoholic fatty liver disease is a condition that affects 25% of the population. Non-alcoholic steatohepatitis (NASH) is a progressive form of the disease that can lead to severe complications such as cirrhosis and hepatocellular carcinoma. Despite its high prevalence, no drugs are currently approved for the treatment of NASH. The drug development pipeline in NASH is very active, yet most assets do not progress to phase III trials and those that do reach phase III often fail to achieve the endpoints necessary for approval by regulatory agencies. Amongst other reasons, the methodological and operational features of traditional clinical trials in NASH might impede optimal drug development. In this regard, platform trials might be an attractive complement or alternative to conventional clinical trials. Platform trials use a master protocol which enables evaluation of multiple investigational medicinal products concurrently or sequentially with a single, shared control arm. Through Bayesian interim analyses, these trials allow for early exit of drugs from the trial based on success or futility, while providing participants better chances of receiving active compounds through adaptive randomisation. Overall, platform trials represent an alternative for patients, pharmaceutical companies, and clinicians in the quest to accelerate the approval of pharmacologic treatments for NASH.EU-PEARL has received funding from the Innovative Medicines Initiative 2 Joint Undertaking under grant agreement No 853966-2. This Joint Undertaking receives support from the European Union’s Horizon 2020 research and innovation programme and EFPIA and Children’s Tumor Foundation, Global Alliance for TB Drug Development Non-profit Organisation, Springworks Therapeutics Inc.ElsevierInstitut Català de la Salut[Pericàs JM, Genescà J] Unitat Hepàtica, Servei de Medicina Interna, Vall d'Hebron Hospital Universitari, Barcelona, Spain. Vall d'Hebron Institut de Recerca (VHIR), Barcelona, Spain. Universitat Autònoma de Barcelona, Bellaterra, Spain. Centros de Investigación Biomédica en Red en Enfermedades Hepáticas y Digestivas (CIBERehd), Barcelona, Spain. [Tacke F] Department of Hepatology and Gastroenterology, Charité - Universitätsmedizin Berlin, Berlin, Germany. [Anstee QM] Translational and Clinical Research Institute, Faculty of Medical Sciences, Newcastle University, Newcastle NIHR Biomedical Research Centre, Newcastle Upon Tyne Hospitals NHS Foundation Trust, Newcastle Upon Tyne, UK. [Di Prospero NA] Janssen Research and Development, Raritan, New Jersey, USA. [Kjær MS] Novo Nordisk, Bagsværd, Denmark. [Mesenbrink P] Analytics Department, Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA. [Koenig F] Center for Medical Statistics, Informatics and Intelligent Systems, Medical University of Vienna, Vienna, Austria. [Ratziu V] Department of Hepatology, Pitié-Salpêtrière Hospital, FranceVall d'Hebron Barcelona Hospital Campus202320232023info:eu-repo/semantics/articleinfo:eu-repo/semantics/publishedVersionapplication/pdfhttps://hdl.handle.net/11351/9089http://hdl.handle.net/11351/9089Scientiareponame:Recercat. Dipósit de la Recerca de Catalunyainstname:Varias* (Consorci de Biblioteques Universitáries de Catalunya, Centre de Serveis Científics i Acadèmics de Catalunya)InglésJournal of Hepatology;78(2)https://doi.org/10.1016/j.jhep.2022.09.021Attribution-NonCommercial-NoDerivatives 4.0 Internationalhttp://creativecommons.org/licenses/by-nc-nd/4.0/info:eu-repo/semantics/openAccessoai:recercat.cat:11351/90892026-05-29T05:05:01Z
score 15,811543

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