Methodological Characteristics of Clinical Trials Supporting the Marketing Authorisation of Advanced Therapies in the European Union

Several advanced therapy medicinal products (ATMPs) have been approved in the European Union (EU). The aim of this study is to analyse the methodological features of the clinical trials (CT) that supported the marketing authorization (MA) of the approved ATMPs in the EU. A systematic review of the c...

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Autores principales:	Carolina Iglesias-Lopez, Antònia Agustí, Antonio Vallano, Merce Obach
Formato:	article
Lenguaje:	EN
Publicado:	Frontiers Media S.A. 2021
Materias:	drug development drug approval research design methods clinical trials advanced therapies Therapeutics. Pharmacology RM1-950
Acceso en línea:	https://doaj.org/article/77ef42e3a7644035a068caa9e761def1
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spelling	oai:doaj.org-article:77ef42e3a7644035a068caa9e761def12021-12-01T14:05:47ZMethodological Characteristics of Clinical Trials Supporting the Marketing Authorisation of Advanced Therapies in the European Union1663-981210.3389/fphar.2021.773712https://doaj.org/article/77ef42e3a7644035a068caa9e761def12021-11-01T00:00:00Zhttps://www.frontiersin.org/articles/10.3389/fphar.2021.773712/fullhttps://doaj.org/toc/1663-9812Several advanced therapy medicinal products (ATMPs) have been approved in the European Union (EU). The aim of this study is to analyse the methodological features of the clinical trials (CT) that supported the marketing authorization (MA) of the approved ATMPs in the EU. A systematic review of the characteristics of pivotal CT of ATMPs approved in the EU until January 31st, 2021 was carried out. A total of 17 ATMPs were approved and 23 CT were conducted to support the MA (median, 1, range, 1–3). Of those studies, 8 (34.78%) were non-controlled and 7 (30.43%) used historical controls. Only 7 (30.4%) were placebo or active-controlled studies. Among all CT, 21 (91.3%) were open-label and 13 (56.52%) had a single-arm design. To evaluate the primary endpoint, 18 (78.26%) studies used an intermediate and single variable. The median (IQR) number of patients enrolled in the studies was 75 (22–118). To date, ATMPs’ approval in the EU is mainly supported by uncontrolled, single-arm pivotal CT. Although there is a trend toward an adaptive or a life cycle approach, a switch to more robust clinical trial designs is expected to better define the benefit and the therapeutic added value of ATMPs.Carolina Iglesias-LopezAntònia AgustíAntònia AgustíAntonio VallanoAntonio VallanoMerce ObachFrontiers Media S.A.articledrug developmentdrug approvalresearch designmethodsclinical trialsadvanced therapiesTherapeutics. PharmacologyRM1-950ENFrontiers in Pharmacology, Vol 12 (2021)
institution	DOAJ
collection	DOAJ
language	EN
topic	drug development drug approval research design methods clinical trials advanced therapies Therapeutics. Pharmacology RM1-950
spellingShingle	drug development drug approval research design methods clinical trials advanced therapies Therapeutics. Pharmacology RM1-950 Carolina Iglesias-Lopez Antònia Agustí Antònia Agustí Antonio Vallano Antonio Vallano Merce Obach Methodological Characteristics of Clinical Trials Supporting the Marketing Authorisation of Advanced Therapies in the European Union
description	Several advanced therapy medicinal products (ATMPs) have been approved in the European Union (EU). The aim of this study is to analyse the methodological features of the clinical trials (CT) that supported the marketing authorization (MA) of the approved ATMPs in the EU. A systematic review of the characteristics of pivotal CT of ATMPs approved in the EU until January 31st, 2021 was carried out. A total of 17 ATMPs were approved and 23 CT were conducted to support the MA (median, 1, range, 1–3). Of those studies, 8 (34.78%) were non-controlled and 7 (30.43%) used historical controls. Only 7 (30.4%) were placebo or active-controlled studies. Among all CT, 21 (91.3%) were open-label and 13 (56.52%) had a single-arm design. To evaluate the primary endpoint, 18 (78.26%) studies used an intermediate and single variable. The median (IQR) number of patients enrolled in the studies was 75 (22–118). To date, ATMPs’ approval in the EU is mainly supported by uncontrolled, single-arm pivotal CT. Although there is a trend toward an adaptive or a life cycle approach, a switch to more robust clinical trial designs is expected to better define the benefit and the therapeutic added value of ATMPs.
format	article
author	Carolina Iglesias-Lopez Antònia Agustí Antònia Agustí Antonio Vallano Antonio Vallano Merce Obach
author_facet	Carolina Iglesias-Lopez Antònia Agustí Antònia Agustí Antonio Vallano Antonio Vallano Merce Obach
author_sort	Carolina Iglesias-Lopez
title	Methodological Characteristics of Clinical Trials Supporting the Marketing Authorisation of Advanced Therapies in the European Union
title_short	Methodological Characteristics of Clinical Trials Supporting the Marketing Authorisation of Advanced Therapies in the European Union
title_full	Methodological Characteristics of Clinical Trials Supporting the Marketing Authorisation of Advanced Therapies in the European Union
title_fullStr	Methodological Characteristics of Clinical Trials Supporting the Marketing Authorisation of Advanced Therapies in the European Union
title_full_unstemmed	Methodological Characteristics of Clinical Trials Supporting the Marketing Authorisation of Advanced Therapies in the European Union
title_sort	methodological characteristics of clinical trials supporting the marketing authorisation of advanced therapies in the european union
publisher	Frontiers Media S.A.
publishDate	2021
url	https://doaj.org/article/77ef42e3a7644035a068caa9e761def1
work_keys_str_mv	AT carolinaiglesiaslopez methodologicalcharacteristicsofclinicaltrialssupportingthemarketingauthorisationofadvancedtherapiesintheeuropeanunion AT antoniaagusti methodologicalcharacteristicsofclinicaltrialssupportingthemarketingauthorisationofadvancedtherapiesintheeuropeanunion AT antoniaagusti methodologicalcharacteristicsofclinicaltrialssupportingthemarketingauthorisationofadvancedtherapiesintheeuropeanunion AT antoniovallano methodologicalcharacteristicsofclinicaltrialssupportingthemarketingauthorisationofadvancedtherapiesintheeuropeanunion AT antoniovallano methodologicalcharacteristicsofclinicaltrialssupportingthemarketingauthorisationofadvancedtherapiesintheeuropeanunion AT merceobach methodologicalcharacteristicsofclinicaltrialssupportingthemarketingauthorisationofadvancedtherapiesintheeuropeanunion
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Methodological Characteristics of Clinical Trials Supporting the Marketing Authorisation of Advanced Therapies in the European Union

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