Current landscape of clinical development and approval of advanced therapies

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...

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Autores principales:	Carolina Iglesias-Lopez, Antonia Agustí, Antoni Vallano, Merce Obach
Formato:	article
Lenguaje:	EN
Publicado:	Elsevier 2021
Materias:	drug development drug approval research design methods clinical trials advanced therapies cell- and tissue-based therapy Genetics QH426-470 Cytology QH573-671
Acceso en línea:	https://doaj.org/article/43c715acca8f4480a4c1e57dcd181861
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spelling	oai:doaj.org-article:43c715acca8f4480a4c1e57dcd1818612021-11-24T04:31:25ZCurrent landscape of clinical development and approval of advanced therapies2329-050110.1016/j.omtm.2021.11.003https://doaj.org/article/43c715acca8f4480a4c1e57dcd1818612021-12-01T00:00:00Zhttp://www.sciencedirect.com/science/article/pii/S2329050121001753https://doaj.org/toc/2329-0501Advanced 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.Carolina Iglesias-LopezAntonia AgustíAntoni VallanoMerce ObachElsevierarticledrug developmentdrug approvalresearch designmethodsclinical trials advanced therapiescell- and tissue-based therapyGeneticsQH426-470CytologyQH573-671ENMolecular Therapy: Methods & Clinical Development, Vol 23, Iss , Pp 606-618 (2021)
institution	DOAJ
collection	DOAJ
language	EN
topic	drug development drug approval research design methods clinical trials advanced therapies cell- and tissue-based therapy Genetics QH426-470 Cytology QH573-671
spellingShingle	drug development drug approval research design methods clinical trials advanced therapies cell- and tissue-based therapy Genetics QH426-470 Cytology QH573-671 Carolina Iglesias-Lopez Antonia Agustí Antoni Vallano Merce Obach Current landscape of clinical development and approval of advanced therapies
description	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.
format	article
author	Carolina Iglesias-Lopez Antonia Agustí Antoni Vallano Merce Obach
author_facet	Carolina Iglesias-Lopez Antonia Agustí Antoni Vallano Merce Obach
author_sort	Carolina Iglesias-Lopez
title	Current landscape of clinical development and approval of advanced therapies
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
publisher	Elsevier
publishDate	2021
url	https://doaj.org/article/43c715acca8f4480a4c1e57dcd181861
work_keys_str_mv	AT carolinaiglesiaslopez currentlandscapeofclinicaldevelopmentandapprovalofadvancedtherapies AT antoniaagusti currentlandscapeofclinicaldevelopmentandapprovalofadvancedtherapies AT antonivallano currentlandscapeofclinicaldevelopmentandapprovalofadvancedtherapies AT merceobach currentlandscapeofclinicaldevelopmentandapprovalofadvancedtherapies
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Current landscape of clinical development and approval of advanced therapies

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