Access to Medicines for Rare Diseases in Brazil

Brazil is a continental country with socioeconomic inequities, hampering access to essential goods and services, including health care. Brazil's Unified Health System (SUS) is a universal, publicly funded health system, warranting free access to health care, including medicines. Significant advances followed the creation of SUS, the implementation of the National Medicines Policy (PNM) and the National Pharmaceutical Services Policy (PNAF) between 1990 and 2000, such as country-wise organization and management of pharmaceutical services and provision of high-cost medicines, a segment including most medicines for rare diseases. The National Policy for Comprehensive Care for Individuals with Rare Diseases has fostered discussion and action on comprehensive care for individuals with rare conditions. However, insufficient funding, setbacks in provision, shortages and the pressures of innovation, patents and prices of medicines have burdened access initiatives. The paper presents the Brazilian scenario and discusses the main pathways for access to medicines for rare diseases in the country, beginning by a comprehensive description of administrative routes, as marketing authorization and incorporation into the health system, which constitute the more straightforward and norm-adherent paths. However, the alternative pathway, litigation for access, guaranteed by the Brazilian Constitution that upholds the right to health, has consolidated itself in the last 20 years, taking an enormous toll on expenditures and appropriate use of medicines. To overcome these challenges and expand access to drugs for rare diseases Brazil must ensure robust evidence-based use for adequate indications, monitoring and managed entry of new medicines and ethical allocation of resources.