Programas assistenciais para medicamentos no Brasil nos últimos três anos (2019–2021): uso compassivo, acesso expandido e fornecimento de medicamento pós-estudo / Drug assistance programs in Brazil over the past three years (2019–2021): compassionate use, expanded access, and post-study drug supply

Abstract

Introduction. Medication assistance programs have emerged as an option for patients to have access to innovative medications that are still unregistered in the country. There are currently three types of drug assistance programs in Brazil: Compassionate Use, Expanded Access, and Post-study Drug Supply. Objective. Carry out a survey on the assistance programs for medicines in Brazil in the last three years ((2019-2021), as well as a bibliographic review on the ethical and regulatory aspects involving the Compassionate Use Programs, Expanded Access, and Post-study Medicine Supply. Method. An observational, cross-sectional study was carried out, using a public database, through the Activities Report issued by the Coordination of Clinical Research in Medicines and Biological Products (COPEC) of the General Management of Medicines and Biological Products (GGMED) of the National Health Agency. Sanitary Surveillance (ANVISA), and data provided by ANVISA, through the “fale conosco” system on September 9, 2022, referring to the periods of 2019, 2020 and 2021. Results. During the period from 2019 to 2021, it was observed that Neoplasms and Rare Diseases represented most cases involved in drug assistance programs in Brazil. According to the results found, the Post-study Drug Supply and Expanded Access Programs in Oncology are directly related to the clinical studies approved by ANVISA during the analyzed period. Rare Diseases represented most cases involved in Compassionate Use Programs. Although the volunteer participating in clinical research has the right to continue treatment as provided for in ANVISA and CONEP/CNS resolutions, the judicialization of health is still one of the great challenges for the Unified Health System (SUS) regarding programs medication assistance. Conclusion. There are currently three types of drug assistance programs in Brazil: Compassionate Use, Expanded Access, and Post-study Drug Supply. According to the results found, such programs focus on Oncology, Rare Diseases or Ultra-Rare Diseases. Brazil is not the only country to have some kind of regulation to guarantee that the volunteer participating in clinical research receives continuity of treatment after the end of the clinical study. Despite the unquestionable humanitarian importance of drug supply assistance programs, there is concern about the impracticability of research and development (R&D) of high-cost innovative drugs due to the fact that in Brazil this supply should be for an indefinite period after completion of the research clinic and not only until the registration of the product in ANVISA or the availability of the medicine in the SUS. Therefore, similarly to other countries, it is important to have a specific law that regulates clinical research in Brazil, in order to find a balance between the legitimate welfare, humanitarian and commercial interests that make both the investments necessary for the R&D of new drugs such as drug assistance programs.