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...
| Main Authors: | , , , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
Elsevier
2021-11-01
|
| Series: | Hematology, Transfusion and Cell Therapy |
| Subjects: | |
| Online Access: | http://www.sciencedirect.com/science/article/pii/S2531137921001462 |
| _version_ | 1818569400320524288 |
|---|---|
| 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 |
| collection | DOAJ |
| 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. |
| first_indexed | 2024-12-14T06:47:38Z |
| format | Article |
| id | doaj.art-2f80208415ca4fd091bc2f52a997b650 |
| institution | Directory Open Access Journal |
| issn | 2531-1379 |
| language | English |
| last_indexed | 2024-12-14T06:47:38Z |
| publishDate | 2021-11-01 |
| publisher | Elsevier |
| record_format | Article |
| series | Hematology, Transfusion and Cell Therapy |
| spelling | doaj.art-2f80208415ca4fd091bc2f52a997b6502022-12-21T23:12:59ZengElsevierHematology, Transfusion and Cell Therapy2531-13792021-11-0143S68S77Associação Brasileira de Hematologia, Hemoterapia e Terapia Celular Consensus on genetically modified cells. Special Article: Advanced therapy medicinal products in Brazil: regulatory panoramaJoão Batista Silva Junior0Antonio Alfredo Rodrigues e Silva1Francielli Cristine Cunha Melo2Melina Cossote Kumoto3Renata Miranda Parca4Faculdade de Ciências da Saúde da Universidade de Brasília (UnB), Brasília, DF, Brazil; Agência Nacional de Vigilância Sanitária (Anvisa), Brasília, DF, Brazil; Corresponding author at: Gerência de Sangue, Tecidos, Células e Órgãos – GSTCO. Agência Nacional de Vigilância Sanitária (Anvisa), Brasília, DF, Brazil.Agência Nacional de Vigilância Sanitária (Anvisa), Brasília, DF, BrazilAgência Nacional de Vigilância Sanitária (Anvisa), Brasília, DF, BrazilAgência Nacional de Vigilância Sanitária (Anvisa), Brasília, DF, BrazilAgência Nacional de Vigilância Sanitária (Anvisa), Brasília, DF, BrazilAdvanced 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.http://www.sciencedirect.com/science/article/pii/S2531137921001462Gene therapyAdvanced therapy medicinal productAnvisaClinical trialsRegulatory approval |
| spellingShingle | 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 Hematology, Transfusion and Cell Therapy Gene therapy Advanced therapy medicinal product Anvisa Clinical trials Regulatory approval |
| 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_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_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_sort | associacao brasileira de hematologia hemoterapia e terapia celular consensus on genetically modified cells special article advanced therapy medicinal products in brazil regulatory panorama |
| topic | Gene therapy Advanced therapy medicinal product Anvisa Clinical trials Regulatory approval |
| url | http://www.sciencedirect.com/science/article/pii/S2531137921001462 |
| work_keys_str_mv | AT joaobatistasilvajunior associacaobrasileiradehematologiahemoterapiaeterapiacelularconsensusongeneticallymodifiedcellsspecialarticleadvancedtherapymedicinalproductsinbrazilregulatorypanorama AT antonioalfredorodriguesesilva associacaobrasileiradehematologiahemoterapiaeterapiacelularconsensusongeneticallymodifiedcellsspecialarticleadvancedtherapymedicinalproductsinbrazilregulatorypanorama AT franciellicristinecunhamelo associacaobrasileiradehematologiahemoterapiaeterapiacelularconsensusongeneticallymodifiedcellsspecialarticleadvancedtherapymedicinalproductsinbrazilregulatorypanorama AT melinacossotekumoto associacaobrasileiradehematologiahemoterapiaeterapiacelularconsensusongeneticallymodifiedcellsspecialarticleadvancedtherapymedicinalproductsinbrazilregulatorypanorama AT renatamirandaparca associacaobrasileiradehematologiahemoterapiaeterapiacelularconsensusongeneticallymodifiedcellsspecialarticleadvancedtherapymedicinalproductsinbrazilregulatorypanorama |