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dc.contributorVall d'Hebron Barcelona Hospital Campus
dc.contributor.authorIglesias-Lopez, Carolina
dc.contributor.authorAgustí Escasany, Maria Antònia
dc.contributor.authorVallano, Antoni
dc.contributor.authorObach, Mercè
dc.date.accessioned2022-05-13T12:55:10Z
dc.date.available2022-05-13T12:55:10Z
dc.date.issued2021-12
dc.identifier.citationIglesias-Lopez C, Agustí A, Vallano A, Obach M. Current landscape of clinical development and approval of advanced therapies. Mol Ther Methods Clin Dev. 2021 Dec;23:606–18.
dc.identifier.issn2329-0501
dc.identifier.urihttp://hdl.handle.net/11351/7533
dc.descriptionClinical trials advanced therapies; Genetic therapy; Research design
dc.description.abstractAdvanced 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.
dc.language.isoeng
dc.publisherCell Press
dc.relation.ispartofseriesMolecular Therapy: Methods & Clinical Development;23
dc.rightsAttribution-NonCommercial-NoDerivatives 4.0 International
dc.rights.urihttp://creativecommons.org/licenses/by-nc-nd/4.0/
dc.sourceScientia
dc.subjectTeràpia cel·lular
dc.subjectMedicaments - Desenvolupament
dc.subjectTerapèutica
dc.subject.meshTherapies, Investigational
dc.subject.meshCell- and Tissue-Based Therapy
dc.subject.meshDrug Approval
dc.titleCurrent landscape of clinical development and approval of advanced therapies
dc.typeinfo:eu-repo/semantics/article
dc.identifier.doi10.1016/j.omtm.2021.11.003
dc.subject.decstratamientos en investigación
dc.subject.decstratamientos basados en células y tejidos
dc.subject.decsautorización de medicamentos
dc.relation.publishversionhttps://doi.org/10.1016/j.omtm.2021.11.003
dc.type.versioninfo:eu-repo/semantics/publishedVersion
dc.audienceProfessionals
dc.contributor.organismesInstitut Català de la Salut
dc.contributor.authoraffiliation[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
dc.identifier.pmid34901306
dc.identifier.wos000757164400032
dc.rights.accessrightsinfo:eu-repo/semantics/openAccess


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