Judicial access to medicines for rare diseases: characteristics of lawsuits against the Federal Government

Abstract Rare diseases are an important cause of infant mortality, yet their care presents many challenges. Legal claims have enabled access to medicines, but there are concerns about interference in health policy and patient safety. The present study 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 lawsuits originated from the Federal District, with limited use of alternative resolution methods. Private attorneys and prescriptions from private health services predominated. Court decisions were predominantly favorable to the plaintiffs, but only 4.2% of the actions indicated the inclusion of claimants in the Unified Health System (SUS). Despite Brazil’s high rate of health litigation, some medicines involved in these lawsuits lacked strong evidence of efficacy and safety. Monitoring medicine use in this context is crucial to support 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.