Current landscape of clinical development and approval of advanced therapies
Clinical trials advanced therapies; Genetic therapy; Research design
| Autores: | , , , |
|---|---|
| Tipo de recurso: | artículo |
| Estado: | Versión publicada |
| Fecha de publicación: | 2021 |
| 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:11351/7533 |
| Acceso en línea: | https://hdl.handle.net/11351/7533 |
| Access Level: | acceso abierto |
| Palabra clave: | Teràpia cel·lular Medicaments - Desenvolupament Terapèutica ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Therapeutics::Therapies, Investigational ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Therapeutics::Biological Therapy::Cell- and Tissue-Based Therapy ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Investigative Techniques::Drug Development::Drug Approval TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::terapéutica::tratamientos en investigación TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::terapéutica::terapia biológica::tratamientos basados en células y tejidos TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::técnicas de investigación::desarrollo de medicamentos::autorización de medicamentos |
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Current landscape of clinical development and approval of advanced therapiesVallano, AntoniObach, MercèIglesias-Lopez, CarolinaAgustí, AntòniaTeràpia cel·lularMedicaments - DesenvolupamentTerapèuticaANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Therapeutics::Therapies, InvestigationalANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Therapeutics::Biological Therapy::Cell- and Tissue-Based TherapyANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Investigative Techniques::Drug Development::Drug ApprovalTÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::terapéutica::tratamientos en investigaciónTÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::terapéutica::terapia biológica::tratamientos basados en células y tejidosTÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::técnicas de investigación::desarrollo de medicamentos::autorización de medicamentosClinical trials advanced therapies; Genetic therapy; Research designEnsayos clínicos de terapias avanzadas; Terapia genética; Diseño de la investigaciónAssajos clínics de teràpies avançades; Teràpia genètica; Disseny de recercaAdvanced therapy medicinal products (ATMPs) are innovative therapies that mainly target orphan diseases and high unmet medical needs. The uncertainty about the product's benefit-risk balance at the time of approval, the limitations of nonclinical development, and the complex quality aspects of those highly individualized advanced therapies are playing a key role in the clinical development, approval, and post-marketing setting for these therapies. This article reviews the current landscape of clinical development of advanced therapies, its challenges, and some of the efforts several stakeholders are conducting to move forward within this field. Progressive iteration of the science, methodologically sound clinical developments, establishing new standards for ATMPs development with the aim to ensure consistency in clinical development, and the reproducibility of knowledge is required, not only to increase the evidence generation for approval but to set principles to achieve translational success in this field.This research did not receive any specific grant from funding agencies in the public, commercial, or not-for-profit sectors.Cell PressInstitut Català de la Salut[Iglesias-Lopez C] Departament de Farmacologia, Terapèutica i Toxicologia, Universitat Autònoma de Barcelona, Bellaterra, Spain. [Agustí A] Departament de Farmacologia, Terapèutica i Toxicologia, Universitat Autònoma de Barcelona, Bellaterra, Spain. Servei de Farmacologia Clínica, Vall d’Hebron Hospital Universitari, Barcelona, Spain. [Vallano A] Departament de Farmacologia, Terapèutica i Toxicologia, Universitat Autònoma de Barcelona, Bellaterra, Spain. Medicines Department, Catalan Healthcare Service, Barcelona, Spain. [Obach M] Medicines Department, Catalan Healthcare Service, Barcelona, SpainVall d'Hebron Barcelona Hospital Campus202220222021info:eu-repo/semantics/articleinfo:eu-repo/semantics/publishedVersionapplication/pdfhttps://hdl.handle.net/11351/7533Scientiareponame:Scientia. Dipòsit d'Informació Digital del Departament de Salutinstname:Departament de Salut de la Generalitat de Catalunya (DS)InglésMolecular Therapy: Methods & Clinical Development;23https://doi.org/10.1016/j.omtm.2021.11.003Attribution-NonCommercial-NoDerivatives 4.0 Internationalhttp://creativecommons.org/licenses/by-nc-nd/4.0/info:eu-repo/semantics/openAccessoai:scientiasalut.gencat.cat:11351/75332026-06-12T09:38:37Z |
| dc.title.none.fl_str_mv |
Current landscape of clinical development and approval of advanced therapies |
| title |
Current landscape of clinical development and approval of advanced therapies |
| spellingShingle |
Current landscape of clinical development and approval of advanced therapies Vallano, Antoni Teràpia cel·lular Medicaments - Desenvolupament Terapèutica ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Therapeutics::Therapies, Investigational ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Therapeutics::Biological Therapy::Cell- and Tissue-Based Therapy ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Investigative Techniques::Drug Development::Drug Approval TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::terapéutica::tratamientos en investigación TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::terapéutica::terapia biológica::tratamientos basados en células y tejidos TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::técnicas de investigación::desarrollo de medicamentos::autorización de medicamentos |
| title_short |
Current landscape of clinical development and approval of advanced therapies |
| title_full |
Current landscape of clinical development and approval of advanced therapies |
| title_fullStr |
Current landscape of clinical development and approval of advanced therapies |
| title_full_unstemmed |
Current landscape of clinical development and approval of advanced therapies |
| title_sort |
Current landscape of clinical development and approval of advanced therapies |
| dc.creator.none.fl_str_mv |
Vallano, Antoni Obach, Mercè Iglesias-Lopez, Carolina Agustí, Antònia |
| author |
Vallano, Antoni |
| author_facet |
Vallano, Antoni Obach, Mercè Iglesias-Lopez, Carolina Agustí, Antònia |
| author_role |
author |
| author2 |
Obach, Mercè Iglesias-Lopez, Carolina Agustí, Antònia |
| author2_role |
author author author |
| dc.contributor.none.fl_str_mv |
Institut Català de la Salut [Iglesias-Lopez C] Departament de Farmacologia, Terapèutica i Toxicologia, Universitat Autònoma de Barcelona, Bellaterra, Spain. [Agustí A] Departament de Farmacologia, Terapèutica i Toxicologia, Universitat Autònoma de Barcelona, Bellaterra, Spain. Servei de Farmacologia Clínica, Vall d’Hebron Hospital Universitari, Barcelona, Spain. [Vallano A] Departament de Farmacologia, Terapèutica i Toxicologia, Universitat Autònoma de Barcelona, Bellaterra, Spain. Medicines Department, Catalan Healthcare Service, Barcelona, Spain. [Obach M] Medicines Department, Catalan Healthcare Service, Barcelona, Spain Vall d'Hebron Barcelona Hospital Campus |
| dc.subject.none.fl_str_mv |
Teràpia cel·lular Medicaments - Desenvolupament Terapèutica ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Therapeutics::Therapies, Investigational ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Therapeutics::Biological Therapy::Cell- and Tissue-Based Therapy ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Investigative Techniques::Drug Development::Drug Approval TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::terapéutica::tratamientos en investigación TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::terapéutica::terapia biológica::tratamientos basados en células y tejidos TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::técnicas de investigación::desarrollo de medicamentos::autorización de medicamentos |
| topic |
Teràpia cel·lular Medicaments - Desenvolupament Terapèutica ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Therapeutics::Therapies, Investigational ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Therapeutics::Biological Therapy::Cell- and Tissue-Based Therapy ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Investigative Techniques::Drug Development::Drug Approval TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::terapéutica::tratamientos en investigación TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::terapéutica::terapia biológica::tratamientos basados en células y tejidos TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::técnicas de investigación::desarrollo de medicamentos::autorización de medicamentos |
| description |
Clinical trials advanced therapies; Genetic therapy; Research design |
| publishDate |
2021 |
| dc.date.none.fl_str_mv |
2021 2022 2022 |
| dc.type.none.fl_str_mv |
info:eu-repo/semantics/article info:eu-repo/semantics/publishedVersion |
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article |
| status_str |
publishedVersion |
| dc.identifier.none.fl_str_mv |
https://hdl.handle.net/11351/7533 |
| url |
https://hdl.handle.net/11351/7533 |
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Inglés |
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Inglés |
| dc.relation.none.fl_str_mv |
Molecular Therapy: Methods & Clinical Development;23 https://doi.org/10.1016/j.omtm.2021.11.003 |
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Attribution-NonCommercial-NoDerivatives 4.0 International http://creativecommons.org/licenses/by-nc-nd/4.0/ info:eu-repo/semantics/openAccess |
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Attribution-NonCommercial-NoDerivatives 4.0 International http://creativecommons.org/licenses/by-nc-nd/4.0/ |
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openAccess |
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application/pdf |
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Cell Press |
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Cell Press |
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Scientia reponame:Scientia. Dipòsit d'Informació Digital del Departament de Salut instname:Departament de Salut de la Generalitat de Catalunya (DS) |
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Departament de Salut de la Generalitat de Catalunya (DS) |
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Scientia. Dipòsit d'Informació Digital del Departament de Salut |
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15,81155 |