Associação Brasileira de Hematologia, Hemoterapia e Terapia Celular Consensus on genetically modified cells. Special Article: Advanced therapy medicinal products in Brazil: regulatory panorama

Advanced therapy medicinal products, considered special medications, requires Anvisa approval for use and commercialization in Brazil. They include the advanced cellular therapy products, tissue engineering products and gene therapy products, which due to their complexity involve innovation and risk...

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Autores principales:	João Batista Silva Junior, Antonio Alfredo Rodrigues e Silva, Francielli Cristine Cunha Melo, Melina Cossote Kumoto, Renata Miranda Parca
Formato:	article
Lenguaje:	EN
Publicado:	Elsevier 2021
Materias:	Gene therapy Advanced therapy medicinal product Anvisa Clinical trials Regulatory approval Diseases of the blood and blood-forming organs RC633-647.5
Acceso en línea:	https://doaj.org/article/2f80208415ca4fd091bc2f52a997b650
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spelling	oai:doaj.org-article:2f80208415ca4fd091bc2f52a997b6502021-11-18T04:51:06ZAssociação Brasileira de Hematologia, Hemoterapia e Terapia Celular Consensus on genetically modified cells. Special Article: Advanced therapy medicinal products in Brazil: regulatory panorama2531-137910.1016/j.htct.2021.09.010https://doaj.org/article/2f80208415ca4fd091bc2f52a997b6502021-11-01T00:00:00Zhttp://www.sciencedirect.com/science/article/pii/S2531137921001462https://doaj.org/toc/2531-1379Advanced therapy medicinal products, considered special medications, requires Anvisa approval for use and commercialization in Brazil. They include the advanced cellular therapy products, tissue engineering products and gene therapy products, which due to their complexity involve innovation and risks, optimized regulatory channels for their development and life cycle monitoring. The scientific elements and the compliance with applicable regulatory aspects are fundamental pillars for the advancement of clinical trials, the positive evidence of the benefit-risk profile and the definition of the critical quality attributes, from the perspective of making safe, effective and high-quality products available to the population. The approval models of these products in Brazil adapt to the specificities and characteristics of the technology and the patient target population, with accelerated regulatory analyses, use in emergency situations by risk controls and specific monitoring mechanisms, principally those related to rare diseases without other therapeutic alternatives. The opportune access to the advance therapy product with safety, efficacy and quality involves innovative normative elements that include the long-term follow-up of the safety and efficacy and of the adaptive pharmacovigilance requisites, as well as the traceability mechanisms for the start-off materials, products and patients.João Batista Silva JuniorAntonio Alfredo Rodrigues e SilvaFrancielli Cristine Cunha MeloMelina Cossote KumotoRenata Miranda ParcaElsevierarticleGene therapyAdvanced therapy medicinal productAnvisaClinical trialsRegulatory approvalDiseases of the blood and blood-forming organsRC633-647.5ENHematology, Transfusion and Cell Therapy, Vol 43, Iss , Pp S68-S77 (2021)
institution	DOAJ
collection	DOAJ
language	EN
topic	Gene therapy Advanced therapy medicinal product Anvisa Clinical trials Regulatory approval Diseases of the blood and blood-forming organs RC633-647.5
spellingShingle	Gene therapy Advanced therapy medicinal product Anvisa Clinical trials Regulatory approval Diseases of the blood and blood-forming organs RC633-647.5 João Batista Silva Junior Antonio Alfredo Rodrigues e Silva Francielli Cristine Cunha Melo Melina Cossote Kumoto Renata Miranda Parca Associação Brasileira de Hematologia, Hemoterapia e Terapia Celular Consensus on genetically modified cells. Special Article: Advanced therapy medicinal products in Brazil: regulatory panorama
description	Advanced therapy medicinal products, considered special medications, requires Anvisa approval for use and commercialization in Brazil. They include the advanced cellular therapy products, tissue engineering products and gene therapy products, which due to their complexity involve innovation and risks, optimized regulatory channels for their development and life cycle monitoring. The scientific elements and the compliance with applicable regulatory aspects are fundamental pillars for the advancement of clinical trials, the positive evidence of the benefit-risk profile and the definition of the critical quality attributes, from the perspective of making safe, effective and high-quality products available to the population. The approval models of these products in Brazil adapt to the specificities and characteristics of the technology and the patient target population, with accelerated regulatory analyses, use in emergency situations by risk controls and specific monitoring mechanisms, principally those related to rare diseases without other therapeutic alternatives. The opportune access to the advance therapy product with safety, efficacy and quality involves innovative normative elements that include the long-term follow-up of the safety and efficacy and of the adaptive pharmacovigilance requisites, as well as the traceability mechanisms for the start-off materials, products and patients.
format	article
author	João Batista Silva Junior Antonio Alfredo Rodrigues e Silva Francielli Cristine Cunha Melo Melina Cossote Kumoto Renata Miranda Parca
author_facet	João Batista Silva Junior Antonio Alfredo Rodrigues e Silva Francielli Cristine Cunha Melo Melina Cossote Kumoto Renata Miranda Parca
author_sort	João Batista Silva Junior
title	Associação Brasileira de Hematologia, Hemoterapia e Terapia Celular Consensus on genetically modified cells. Special Article: Advanced therapy medicinal products in Brazil: regulatory panorama
title_short	Associação Brasileira de Hematologia, Hemoterapia e Terapia Celular Consensus on genetically modified cells. Special Article: Advanced therapy medicinal products in Brazil: regulatory panorama
title_full	Associação Brasileira de Hematologia, Hemoterapia e Terapia Celular Consensus on genetically modified cells. Special Article: Advanced therapy medicinal products in Brazil: regulatory panorama
title_fullStr	Associação Brasileira de Hematologia, Hemoterapia e Terapia Celular Consensus on genetically modified cells. Special Article: Advanced therapy medicinal products in Brazil: regulatory panorama
title_full_unstemmed	Associação Brasileira de Hematologia, Hemoterapia e Terapia Celular Consensus on genetically modified cells. Special Article: Advanced therapy medicinal products in Brazil: regulatory panorama
title_sort	associação brasileira de hematologia, hemoterapia e terapia celular consensus on genetically modified cells. special article: advanced therapy medicinal products in brazil: regulatory panorama
publisher	Elsevier
publishDate	2021
url	https://doaj.org/article/2f80208415ca4fd091bc2f52a997b650
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Associação Brasileira de Hematologia, Hemoterapia e Terapia Celular Consensus on genetically modified cells. Special Article: Advanced therapy medicinal products in Brazil: regulatory panorama

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