dc.contributor | Vall d'Hebron Barcelona Hospital Campus |
dc.contributor.author | Agustí Escasany, Maria Antònia |
dc.contributor.author | Vallano, Antoni |
dc.contributor.author | Obach, Mercè |
dc.contributor.author | Iglesias-Lopez, Carolina |
dc.date.accessioned | 2022-05-13T12:55:10Z |
dc.date.available | 2022-05-13T12:55:10Z |
dc.date.issued | 2021-12 |
dc.identifier.citation | Iglesias-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.issn | 2329-0501 |
dc.identifier.uri | https://hdl.handle.net/11351/7533 |
dc.description | Clinical trials advanced therapies; Genetic therapy; Research design |
dc.description.abstract | Advanced 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.iso | eng |
dc.publisher | Cell Press |
dc.relation.ispartofseries | Molecular Therapy: Methods & Clinical Development;23 |
dc.rights | Attribution-NonCommercial-NoDerivatives 4.0 International |
dc.rights.uri | http://creativecommons.org/licenses/by-nc-nd/4.0/ |
dc.source | Scientia |
dc.subject | Teràpia cel·lular |
dc.subject | Medicaments - Desenvolupament |
dc.subject | Terapèutica |
dc.subject.mesh | Therapies, Investigational |
dc.subject.mesh | Cell- and Tissue-Based Therapy |
dc.subject.mesh | Drug Approval |
dc.title | Current landscape of clinical development and approval of advanced therapies |
dc.type | info:eu-repo/semantics/article |
dc.identifier.doi | 10.1016/j.omtm.2021.11.003 |
dc.subject.decs | tratamientos en investigación |
dc.subject.decs | tratamientos basados en células y tejidos |
dc.subject.decs | autorización de medicamentos |
dc.relation.publishversion | https://doi.org/10.1016/j.omtm.2021.11.003 |
dc.type.version | info:eu-repo/semantics/publishedVersion |
dc.audience | Professionals |
dc.contributor.organismes | Institut 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.pmid | 34901306 |
dc.identifier.wos | 000757164400032 |
dc.rights.accessrights | info:eu-repo/semantics/openAccess |