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. |
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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.) |
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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 |
تدمد: | 2168-4804 |
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DOI: | 10.1007/s43441-023-00578-2 |