0276/2025 - Acesso judicial a medicamentos para doenças raras: características das demandas contra a União Federal Judicial Access to Medicines for Rare Diseases: Characteristics of Lawsuits Against the Federal Government
As doenças raras são importantes causas de mortalidade infantil, mas existem inúmeros desafios para seu cuidado. As demandas judiciais têm permitido o acesso a medicamentos, entretanto há preocupações sobre a interferência na política de saúde e na segurança dos usuários. A pesquisa analisou 912 ações judiciais, de 23 medicamentos para 18 doenças raras, entre 2008 e 2022. Predominaram mulheres, crianças de 0 a 5 anos, residentes em São Paulo e Minas Gerais. A maior parte com gratuidade de justiça e registrados 36 óbitos. As demandas originaram-se, principalmente, do Distrito Federal, com baixo uso de métodos alternativos de resolução de conflitos. Predominaram advogados privados e prescrição por consultórios privados. As decisões foram predominantemente favoráveis aos autores, mas apenas 4,2% das ações indicavam a inclusão dos demandantes no SUS. Embora a judicialização da saúde seja relevante no Brasil, alguns destes medicamentos são registrados sem evidências robustas de eficácia e segurança. Há necessidade de monitoramento da utilização de medicamentos em contextos judiciais para fundamentar decisões futuras. É essencial o desenvolvimento de políticas inovadoras e a coordenação entre diferentes setores para garantir um atendimento eficaz e integral às pessoas com doenças raras.
Palavras-chave:
Doenças Raras, Judicialização da Saúde, Medicamentos, Direito à Saúde
Abstract:
Rare diseases are an important cause of infant mortality, yet their care presents many challenges. Judicial demands have enabled access to medicines, but there are concerns about interference in health policy and safety. The research analyzed 912 lawsuits involving 23 medicines for 18 rare diseases between 2008 and 2022. Most plaintiffs were women and children aged 0 to 5 years, residing in São Paulo and Minas Gerais. Legal aid was commonly granted, and 36 deaths were reported. The demands originated from the Federal District, with limited use of alternative resolution methods. Private attorneys and prescriptions from private practices predominated. The decisions were predominantly favorable to the plaintiffs, but only 4.2% of the actions indicated the inclusion of claimants in the SUS (Unified Health System). Despite Brazil’s high rate of health litigation, some medicines involved lacked strong evidence of efficacy and safety. Monitoring drug use in this context is crucial to inform future decisions. It is essential to develop innovative policies and coordination among different sectors to ensure effective and comprehensive care for individuals with rare diseases.
Keywords:
Rare diseases, Health's Judicialization, Medicines, Unified Health System, Right to Health
Judicial Access to Medicines for Rare Diseases: Characteristics of Lawsuits Against the Federal Government
Resumo (abstract):
Rare diseases are an important cause of infant mortality, yet their care presents many challenges. Judicial demands have enabled access to medicines, but there are concerns about interference in health policy and safety. The research analyzed 912 lawsuits involving 23 medicines for 18 rare diseases between 2008 and 2022. Most plaintiffs were women and children aged 0 to 5 years, residing in São Paulo and Minas Gerais. Legal aid was commonly granted, and 36 deaths were reported. The demands originated from the Federal District, with limited use of alternative resolution methods. Private attorneys and prescriptions from private practices predominated. The decisions were predominantly favorable to the plaintiffs, but only 4.2% of the actions indicated the inclusion of claimants in the SUS (Unified Health System). Despite Brazil’s high rate of health litigation, some medicines involved lacked strong evidence of efficacy and safety. Monitoring drug use in this context is crucial to inform future decisions. It is essential to develop innovative policies and coordination among different sectors to ensure effective and comprehensive care for individuals with rare diseases.
Palavras-chave (keywords):
Rare diseases, Health's Judicialization, Medicines, Unified Health System, Right to Health
Pepe, VLE, Baptista, MR, Ventura, M, Hora, MFVP, Alvarez, NR, Pinto, GS, Silva, RS, Vidal, TJ. Acesso judicial a medicamentos para doenças raras: características das demandas contra a União Federal. Cien Saude Colet [periódico na internet] (2025/ago). [Citado em 27/01/2026].
Está disponível em: http://cienciaesaudecoletiva.com.br/artigos/acesso-judicial-a-medicamentos-para-doencas-raras-caracteristicas-das-demandas-contra-a-uniao-federal/19752?id=19752
