دورية أكاديمية
Regulatory Framework, Challenges, and Initial Strategic Planning for Advanced Therapy Products (PTAs) Development in Brazil.
| العنوان: | Regulatory Framework, Challenges, and Initial Strategic Planning for Advanced Therapy Products (PTAs) Development in Brazil. |
|---|---|
| المؤلفون: | Fachel FNS; Células, Tecidos e Genes (CTG), Centro de Pesquisa Experimental, Hospital de Clínicas de Porto Alegre, R. Ramiro Barcelos 2350, Porto Alegre, RS, 90035-903, Brazil. flavia.fachel@ufrgs.br.; Programa de Pós-Graduação em Ciências Farmacêuticas da Universidade Federal do Rio Grande Do Sul (UFRGS), Faculdade de Farmácia, Av. Ipiranga 2752, Porto Alegre, RS, 90610-000, Brazil. flavia.fachel@ufrgs.br., Schuh RS; Células, Tecidos e Genes (CTG), Centro de Pesquisa Experimental, Hospital de Clínicas de Porto Alegre, R. Ramiro Barcelos 2350, Porto Alegre, RS, 90035-903, Brazil.; Programa de Pós-Graduação em Ciências Farmacêuticas da Universidade Federal do Rio Grande Do Sul (UFRGS), Faculdade de Farmácia, Av. Ipiranga 2752, Porto Alegre, RS, 90610-000, Brazil., Grudzinski PB; Células, Tecidos e Genes (CTG), Centro de Pesquisa Experimental, Hospital de Clínicas de Porto Alegre, R. Ramiro Barcelos 2350, Porto Alegre, RS, 90035-903, Brazil., Teixeira HF; Células, Tecidos e Genes (CTG), Centro de Pesquisa Experimental, Hospital de Clínicas de Porto Alegre, R. Ramiro Barcelos 2350, Porto Alegre, RS, 90035-903, Brazil.; Programa de Pós-Graduação em Ciências Farmacêuticas da Universidade Federal do Rio Grande Do Sul (UFRGS), Faculdade de Farmácia, Av. Ipiranga 2752, Porto Alegre, RS, 90610-000, Brazil., Baldo G; Células, Tecidos e Genes (CTG), Centro de Pesquisa Experimental, Hospital de Clínicas de Porto Alegre, R. Ramiro Barcelos 2350, Porto Alegre, RS, 90035-903, Brazil.; Programa de Pós-Graduação em Fisiologia da Universidade Federal do Rio Grande do Sul (UFRGS), Instituto de Ciências Básicas da Saúde, R. Sarmento Leite, n° 500, Porto Alegre, RS, 90050-170, Brazil.; Programa de Pós-Graduação em Genética e Biologia Molecular da Universidade Federal do Rio Grande do Sul (UFRGS), Campus do Vale, Av. Bento Gonçalves 9500, Porto Alegre, RS, 91501-970, Brazil., Matte U; Células, Tecidos e Genes (CTG), Centro de Pesquisa Experimental, Hospital de Clínicas de Porto Alegre, R. Ramiro Barcelos 2350, Porto Alegre, RS, 90035-903, Brazil.; Programa de Pós-Graduação em Genética e Biologia Molecular da Universidade Federal do Rio Grande do Sul (UFRGS), Campus do Vale, Av. Bento Gonçalves 9500, Porto Alegre, RS, 91501-970, Brazil. |
| المصدر: | Therapeutic innovation & regulatory science [Ther Innov Regul Sci] 2024 Jan; Vol. 58 (1), pp. 21-33. Date of Electronic Publication: 2023 Oct 10. |
| نوع المنشور: | Journal Article; Review |
| اللغة: | English |
| بيانات الدورية: | Publisher: Springer International Publishing Country of Publication: Switzerland NLM ID: 101597411 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2168-4804 (Electronic) Linking ISSN: 21684790 NLM ISO Abbreviation: Ther Innov Regul Sci Subsets: MEDLINE |
| أسماء مطبوعة: | Publication: 2020- : Cham, Switzerland : Springer International Publishing Original Publication: Thousand Oaks, CA : Sage Publications, [2013]- |
| مواضيع طبية MeSH: | Strategic Planning* , Biological Products*/therapeutic use, Animals ; Brazil ; Cell- and Tissue-Based Therapy ; Tissue Engineering |
| مستخلص: | Advanced Therapies are a class of innovative complex biological products used for therapeutic purposes, encompassing cell therapy, tissue engineering, and gene therapy products. These are promising therapeutic strategies for several complex diseases with low or non-existent therapeutic alternatives. The proper transposition of basic research in this area into medicinal products must comply with regulatory requirements. Here we review the main regulatory recommendations, emphasizing on the Brazilian regulation. The critical points are the manufacturing process, challenges in characterizing the product, development of non-clinical trials, lack of adequate animal models representative of the clinical situation, and absence of valid and measurable therapeutic endpoints. Based on that, we propose a framework for strategic planning of pre-clinical studies in this field. The detailed example involves producing a nonviral vector-based gene editing product, but the regulations and methods may be extrapolated for developing different types of advanced therapies. (© 2023. The Author(s), under exclusive licence to The Drug Information Association, Inc.) |
| References: | Pimenta C, Bettiol V, Alencar-Silva T, Franco OL, Pogue R, Carvalho JL, Felipe MSS. Advanced therapies and regulatory framework in different areas of the globe: past, present, and future. Clin Ther. 2021;43:e103–38. (PMID: 10.1016/j.clinthera.2021.02.00633892966) Iglesias-López C, Agustí A, Obach M, Vallano A. Regulatory framework for advanced therapy medicinal products in Europe and United States. Front Pharmacol. 2019;10:1–14. (PMID: 10.3389/fphar.2019.00921) Iglesias-Lopez C, Obach M, Vallano A, Agustí A. Comparison of regulatory pathways for the approval of advanced therapies in the European Union and the United States. Cytotherapy. 2021;23:261–74. (PMID: 10.1016/j.jcyt.2020.11.00833483292) Flory E, Reinhardt J. European regulatory tools for advanced therapy medicinal products. Transfusion Medicine and Hemotherapy. 2013;40:409–12. (PMID: 10.1159/000356364244748903901598) Bachtarzi H. Ex vivo and in vivo genome editing: A regulatory scientific framework from early development to clinical implementation. Regenerative Med. 2017;12:1015–30. (PMID: 10.2217/rme-2017-0095) Schacker M, Seimetz D. From fiction to science: clinical potentials and regulatory considerations of gene editing. Clin Transl Med. 2019. https://doi.org/10.1186/s40169-019-0244-7 . (PMID: 10.1186/s40169-019-0244-7316375416803602) European Parliament (2007) Regulation (EC) No. 1394/2007 of the European Parliament and of the Council of 13 November 2007 on Advanced Therapy Medicinal Products and Amending Directive 2001/83/EC and Regulation /EC) No 726/2004. https://eur-lex.europa.eu/LexUriServ/LexUriServ.do?uri=OJ:L:2007:324:0121:0137:en:PDF . Accessed 31 Aug 2021. Carvalho M, Sepodes B, Martins AP. Regulatory and scientific advancements in gene therapy: State-of-the-art of clinical applications and of the supporting european regulatory framework. Front Med (Lausanne). 2017. https://doi.org/10.3389/fmed.2017.00182 . (PMID: 10.3389/fmed.2017.0018229124055) López-Paniagua M, de la Mata A, Galindo S, Blázquez F, Calonge M, Nieto-Miguel T. Advanced therapy medicinal products for the eye: Definitions and regulatory framework. Pharmaceutics. 2021;13:1–18. (PMID: 10.3390/pharmaceutics13030347) European Medicines Agency (2009) ChondroCelect—characterised viable autologous cartilage-forming cells expanded ex vivo expressing specific marker proteins. https://www.ema.europa.eu/en/medicines/human/EPAR/chondrocelect . Accessed 31 Aug 2021. Food and Drug Administration (2010) Provenge® (sipuleucel-T). 13 september 2021. ANVISA (2020) Carta de aprovação de Produto de Terapia Avançada—Luxturna®. https://www.gov.br/anvisa/pt-br/assuntos/sangue/terapias-avancadas/cartas-de-aprovacao/carta-de-aprovacao-luxturna.pdf/view . Accessed 13 Sep 2021. RENETA (2020) Cenário de crescimento de produtos de terapias avançadas no Brasil e no mundo. https://bef226c9-51c9-4f4d-b45d-485faa781332.filesusr.com/ugd/92716b_1460bdc1e1da4d56813cb91f27c853d9.pdf . de Freitas DRC. The regulatory pathway for advanced cell therapy and gene therapy products in brazil: a road to be built. Adv Exp Med Biol. 2015. https://doi.org/10.1007/978-3-319-18618-4_4 . (PMID: 10.1007/978-3-319-18618-4_426374221) Parca RM, Takao MRM, da Silva JB. Proposal of regulatory framework for advanced therapy medicinal products in Brazil. Vigilância Sanitária em Debate. 2018;6:15–22. (PMID: 10.22239/2317-269x.01078) Batista J, Junior S, Rodrigues E Silva AA, Cunha Melo C, Kumoto MC, Parca RM (2021) Produtos de terapias avançadas no Brasil: panorama regulatório. Consenso da Associação Brasileira de Hematologia, Hemoterapia e Terapia Celular sobre Células Geneticamente Modificadas Agosto:. Anvisa (2016) Portaria N o 1.731, de 9 de Setembro de 2016. 2016. Anvisa (2021) Resolução da Diretoria Colegiada—RDC N o 505, de 27 de Maio de 2021. https://www.in.gov.br/en/web/dou/-/resolucao-rdc-n-505-de-27-de-maio-de-2021-323002775 . Anvisa (2021) Resolução da diretoria Colegiada—RDC N o 506, de 27 de Maio de 2021. https://www.in.gov.br/en/web/dou/-/resolucao-rdc-n-506-de-27-de-maio-de-2021-323008725 . Anvisa (2021) Resolução da diretoria Colegiada—RDC N o 508, de 27 de Maio de 2021. https://www.in.gov.br/en/web/dou/-/resolucao-rdc-n-508-de-27-de-maio-de-2021-323013606#:~:text=Dispõe sobre as Boas Práticas,clínica%2C e dá outras providências.&text=1° Esta Resolução estabelece,Uso Terapêutico e pesquisa clínica. da Silva JB, Takao MRM, Parca RM. Advanced therapy medicinal products: an introduction to risk management. Vigilância Sanitária em Debate. 2018;6:23–31. Shim G, Kim D, Park GT, Jin H, Suh SK, Oh YK. Therapeutic gene editing: Delivery and regulatory perspectives. Acta Pharmacol Sin. 2017;38:738–53. (PMID: 10.1038/aps.2017.2283925685520188) Fachel FNS, Frâncio L, Poletto É, Schuh RS, Teixeira HF, Giugliani R, Baldo G, Matte U. Gene editing strategies to treat lysosomal disorders: The example of mucopolysaccharidoses. Adv Drug Deliv Rev. 2022. https://doi.org/10.1016/j.addr.2022.114616 . (PMID: 10.1016/j.addr.2022.11461636356930) Pramod K, Ma T, Charoo N, Ansari S, Ali J. Pharmaceutical product development: A quality by design approach. Int J Pharm Investig. 2016;6:129. (PMID: 10.4103/2230-973X.187350276062564991121) Barkholt L, Voltz-Girolt C, Raine J, Salmonson T, Schüssler-Lenz M. European regulatory experience with advanced therapy medicinal products. Nat Rev Drug Discov. 2018;18:8–9. (PMID: 10.1038/nrd.2018.20030498203) |
| معلومات مُعتمدة: | 312714/2018-1 Conselho Nacional de Desenvolvimento Científico e Tecnológico; 442425/2020-1 Conselho Nacional de Desenvolvimento Científico e Tecnológico; 403587/2021-2 Conselho Nacional de Desenvolvimento Científico e Tecnológico |
| فهرسة مساهمة: | Keywords: Advanced therapy products; Brazil; Regulatory framework; Strategic planning |
| المشرفين على المادة: | 0 (Biological Products) |
| تواريخ الأحداث: | Date Created: 20231010 Date Completed: 20240105 Latest Revision: 20240209 |
| رمز التحديث: | 20240209 |
| DOI: | 10.1007/s43441-023-00578-2 |
| PMID: | 37815738 |
| قاعدة البيانات: | MEDLINE |
Find this article in full text from Springer Verlag. 
Full Text Finder | تدمد: | 2168-4804 |
|---|---|
| DOI: | 10.1007/s43441-023-00578-2 |