Post-marketing authorisation safety and efficacy surveillance of advanced therapy medicinal products in Brazil, the European Union, the United States and Japan.

BACKGROUND AIMS: Advanced therapy medicinal products (ATMPs) are a class of biological products for human use that are based on gene, tissues or cells. ATMPs have peculiar characteristics when compared with traditional medicines. In this regard, long-term safety and efficacy follow-up systems of individuals treated with ATMPs have become necessary and may present unique challenges, because unlike conventional drugs and biologics, these products can exert their effects for years after administration. This work seeks to assess the requirements foreseen in the regulatory frameworks for the post-marketing authorization safety and efficacy surveillance for ATMPs in Brazil, European Union (EU), Japan and United States, which are some of the members of the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use. METHODS: We reviewed the scientific literature and official documents of regulatory agencies (RAs) in Brazil, the EU, Japan and the United States. RESULTS AND CONCLUSIONS: RAs in the EU, US and Japan have developed regulatory guidelines for the post-marketing surveillance of ATMPs. These guidelines aim at implementing surveillance plans for monitoring adverse events, including late ones, after marketing authorization. All the ATMPs authorized by the RAs studied, submitted some type of post-marketing requirement to supplement safety and efficacy data, according to the regulations and terminology used by those jurisdictions.

Pesquisa Translacional: o desempenho dos Institutos Nacionais de Ciência e Tecnologia na área da saúde / Translational Research: the performance of the National Institutes of Science and Technology in the field of health

RESUMO O Programa Institutos Nacionais de Ciência e Tecnologia foi criado para promover a excelência nas atividades de ciência e tecnologia e sua internacionalização, fomentar a interação destas com o sistema empresarial, melhorar a educação científica e a participação homogênea das regiões do País na produção do conhecimento. Medicina translacional compreende a aceleração de transmissão de conhecimentos da pesquisa básica à aplicação clínica, o aprofundamento de observações clínicas e a aplicação desses conhecimentos à população geral. Este artigo buscou identificar e analisar as contribuições dos Institutos Nacionais de Ciência e Tecnologia em Saúde (INCT-Saúde) no atendimento às demandas da saúde brasileira, baseado no conceito de Pesquisa Translacional. Foi realizada uma pesquisa qualiquantitativa, exploratório-descritiva das atividades desenvolvidas pelos INCT-Saúde. Os 39 INCT-Saúde têm contribuído para melhoria da saúde brasileira por meio da transferência de conhecimentos diretos e para a formulação/implantação de Políticas Públicas de Saúde. Os INCT-Saúde têm potencial para o atendimento da demanda da população brasileira, na área da saúde, e têm-se destacado na interação com empresas e na criação de startups. Contudo, é necessária a divulgação dos resultados alcançados pelos INCT-Saúde, bem como a elaboração e a aplicação de uma metodologia de monitoramento e avaliação da Ciência, Tecnologia e Inovação no Brasil.

ABSTRACT The National Institutes of Science and Technology Program was created to promote excellence in science and technology activities and their internationalization, foster their interaction with the business system, improve scientific education and the homogeneous participation of the regions of the Country in the production of knowledge. Translational medicine comprises accelerating the transmission of knowledge from basic research to clinical application, deepening clinical observations and applying this knowledge to the general population. This article aimed to identify and analyze the contributions of the National Science and Technology Institute for Translational Medicine (INCT-Health) in meeting the demands of Brazilian health, based on the concept of Translational Research. A qualitative, exploratory and descriptive research of the activities developed by INCT-Health was carried out. The 39 INCT-Health have contributed to the improvement of Brazilian health through the transfer of direct knowledge and the formulation/implementation of Public Health Policies. The INCT-Health has potential to meet the demand of the Brazilian population, in the health area, and have stood out in the interaction with companies and in the creation of startups. However, it is necessary to disseminate the results achieved by INCT-Health, as well as the elaboration and application of a methodology for monitoring and evaluation of Science, Technology and Innovation in Brazil.