PUBLIC POLICIES AND ACCESS TO TREATMENT FOR RARE DISEASES: CHALLENGES OF CYSTIC FIBROSIS IN THE BRAZILIAN UNIFIED HEALTH SYSTEM (SUS)

Abstract

This article aimed to describe public policies and access to treatment for rare diseases, highlighting the main challenges related to the care of people with cystic fibrosis within the Brazilian Unified Health System (SUS). To achieve this objective, an integrative literature review was conducted in the LILACS, MEDLINE/BVS, and SciELO databases, using the descriptors "Cystic Fibrosis," "Rare Diseases," "Public Health Policies," "Unified Health System," and "Access to Health Services," combined with the Boolean operators AND and OR. Articles published between 2021 and 2026, available in full text and in Portuguese, English, and Spanish, were included, excluding duplicate studies and publications that did not directly address the topic. The results showed advances in the organization of care for rare diseases, especially in the expansion of neonatal screening, the development of clinical protocols, and the strengthening of multidisciplinary follow-up. However, challenges related to regional inequality, limited availability of specialized services, and access to high-cost medications persist. It is concluded that, although there have been significant advances in public policies aimed at cystic fibrosis, investments and strategies are still needed to ensure greater equity, comprehensiveness, and effectiveness in access to treatment within the Brazilian Unified Health System (SUS).