Challenges and opportunities for access to Advanced Therapy Medicinal Products in Brazil.

Autor: Sachetti CG; Graduate Program for Collective Health, Faculty of Health Sciences, University of Brasilia (UnB), Brasilia, Brazil; Oswaldo Cruz Foundation (Fiocruz), Rio de Janeiro, Brazil. Electronic address: camile.sachetti@fiocruz.br., Barbosa A Jr; Departament of Hemotherapy and Cell Therapy, Israelita Albert Einstein Hospital, São Paulo, Brazil., de Carvalho ACC; Carlos Chagas Filho Biophysics Institute, Federal University of Rio de Janeiro, Rio de Janeiro, Brazil., Araujo DV; Medical School, State University of Rio de Janeiro, Rio de Janeiro, Brazil., da Silva EN; Graduate Program for Collective Health, Faculty of Health Sciences, University of Brasilia (UnB), Brasilia, Brazil; Collective Health School, Faculty of Ceilandia, University of Brasilia, Brasília, Brazil.
Jazyk: angličtina
Zdroj: Cytotherapy [Cytotherapy] 2024 Aug; Vol. 26 (8), pp. 939-947. Date of Electronic Publication: 2024 Mar 29.
DOI: 10.1016/j.jcyt.2024.03.492
Abstrakt: Background Aims: The marketing authorization of Advanced Therapy Medicinal Products (ATMPs) in Brazil is recent. The features of these therapies impose specialized regulatory action and are consequently challenging for developers. The goal of this study was to identify the industry's experience in clinical development, marketing authorization and access to ATMPs through the Unified Health System (SUS, acronym in Portuguese), from a regulatory perspective.
Methods: A survey containing structured questions was conducted among research participants who work at companies that commercialize ATMPs. A descriptive analysis was performed.
Results: We invited 15 foreign pharmaceutical companies, of which 10 agreed to participate. Overall, participants assessed that Brazil has a well-established regulatory system, especially the sanitary registration by the National Health Surveillance Agency (Anvisa), which ensures the quality, safety, and efficacy of the products. The Agency's good interaction with the regulated sector, the harmonization of sanitary and ethical assessment systems with other countries, and the analysis time in the biosafety assessment of Genetically Modified Organisms (GMOs) stand out as positive in industry's evaluation. On the other hand, it is important to advance the pricing regulation for these products since Brazilian regulations do not establish specific criteria for ATMP. One of the biggest challenges is the difficulty for the SUS in reimbursing these very high-cost therapies, especially using current Health Technology Assessment (HTA) methods.
Conclusions: Considering the increasing number of approvals of cell and gene therapies in Brazil in the coming years, a close dialogue between the industry and the public sector is recommended to advance regulatory improvements (pricing and HTA). Additionally, the construction of policies to promote the national Health Economic-Industrial Complex, based on a mission-oriented vision that encourages innovative models of financing, especially those that consider risk-sharing and co-financing technologies, will help provide the population with universal, equitable and sustainable access to ATMP in the SUS.
Competing Interests: Declaration of Competing Interest The authors have no commercial, proprietary, or financial interest in the products or companies described in this article.
(Copyright © 2024 International Society for Cell & Gene Therapy. Published by Elsevier Inc. All rights reserved.)
Databáze: MEDLINE
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