Graduate Program for Collective Health, Faculty of Health Sciences, University of Brasilia, Brasilia, Brazil; Oswaldo Cruz Foundation, Rio de Janeiro, Brazil.
Departament of Hemotherapy and Cell Therapy, Israelita Albert Einstein Hospital, São Paulo, Brazil.
Cytotherapy. 2022 Nov;24(11):1158-1165. doi: 10.1016/j.jcyt.2022.06.004. Epub 2022 Aug 6.
Advanced therapy medicinal products (ATMPs) have reached the forefront of biotechnological innovation, partly due to public funders' efforts in the early stages of research and development (R&D). Data on investment in R&D of ATMPs are recognized as scarce, particularly in developing countries. Because of the numerous peculiarities of the Brazilian health system and the science and technology (S&T) system, the country is a good example for the evaluation of public investments in R&D of ATMPs. The aim of this study is to analyze the evolution of investments made by the Ministry of Health (MoH) of Brazil and partners in the ATMP field between 2004 and 2020.
A descriptive analysis was performed based on secondary data. The analysis was based on S&T and innovation research and support for research infrastructure in the field. The database was stratified by year of funding, ATMP type, type of study or research infrastructure project, amount invested in the project, targeted disease for which clinical trials in ATMPs were developed and financing sector (health, education, S&T and economic).
The investments coordinated by MoH (61.5%) in partnership with the S&T, education and economic sectors (38.5%) consisted of Int$137.35 million in 282 ATMP projects. Funding included S&T and innovation research (67% of the total amount) and projects to implement or maintain infrastructure in selected research centers (32.98%). With regard to global convergence, cell therapy was the type of ATMP that most benefited from public investment, totaling 82.23% of the total funding in the analyzed period. Cardiology (29%) and neurology (21%) were the main focus of clinical trials. Following the global trend of public sector R&D funding, the number of basic and pre-clinical research projects represented 78.06% of the total number of projects.
Despite the need to implement improvements in ATMP R&D financing policy in Brazil, the country has made important steps in the field and can serve as a benchmark for other countries with socioeconomic similarities. Among the main lessons are the prioritization of research aligned with the health needs of the population, cross-sector articulation by the health policymaker to coordinate R&D efforts of the sector and formulation of a specific sector policy (Programa Genomas Brasil, the Brazilian National Program of Genomic and Precision Medicine) to promote knowledge translation.
先进治疗药物产品（ATMPs）已成为生物技术创新的前沿领域，部分原因是公共资助者在研发（R&D）的早期阶段所做的努力。关于 ATMPs 的 R&D 投资的数据被认为是稀缺的，尤其是在发展中国家。由于巴西卫生系统和科学技术（S&T）系统的诸多特殊性，巴西是评估 ATMPs 的 R&D 公共投资的一个很好的例子。本研究的目的是分析 2004 年至 2020 年期间巴西卫生部（MoH）及其合作伙伴在 ATMP 领域投资的演变。
采用二次数据分析进行描述性分析。分析基于 S&T 和创新研究以及该领域研究基础设施的支持。数据库按资助年份、ATMP 类型、研究或研究基础设施项目类型、项目投资金额、开发 ATMP 临床试验的目标疾病以及融资部门（卫生、教育、S&T 和经济）进行分层。
MoH （61.5%）与 S&T、教育和经济部门（38.5%）合作协调的投资包括 282 个 ATMP 项目中的 1.3735 亿美元。资金包括 S&T 和创新研究（占总金额的 67%）和在选定研究中心实施或维持基础设施的项目（占 32.98%）。在全球趋同方面，细胞疗法是最受益于公共投资的 ATMP 类型，占分析期间总资金的 82.23%。心脏病学（29%）和神经病学（21%）是临床试验的主要重点。遵循公共部门 R&D 资金的全球趋势，基础和临床前研究项目的数量占项目总数的 78.06%。
尽管巴西需要改进 ATMP R&D 融资政策，但该国在该领域已经迈出了重要的步伐，可以为其他具有相似社会经济条件的国家提供借鉴。主要经验教训包括优先考虑与人口健康需求相一致的研究、卫生政策制定者通过跨部门协调部门的研发工作以及制定特定部门政策（巴西基因组和精准医学国家计划）来促进知识转化。
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