Equity in access to high-cost drugs for rare diseases in Brazil: dornase alfa case

Abstract Objective To analyse equity in access to dornase alfa for cystic fibrosis (CF) treatment within the SUS, using indirect indicators of availability, consumption, and regional coverage. Methods A cross-sectional study was conducted using secondary data from the SUS Outpatient Information System (SIA/SUS), focusing on Outpatient Procedure Authorizations (APAC) for dornase alfa. Medicine prices were retrieved from Health Price Bank (BPS), which registers public health procurement prices in Brazil. Three indicators were constructed by macro-region: average per capita expenditure, average per capita consumption, and the proportion of treated patients relative to the estimated CF population adjusted to SUS coverage. Dornase alfa was adopted as a tracer medicine to assess access to high-cost therapies for rare diseases. Key findings Only 41% of CF patients relying on SUS received dornase alfa treatment. Marked regional disparities were observed: while the Central-West treated 50.9% of patients, the Northeast treated only 34.3%. Per capita expenditure ranged from US$123.50 to US$140.70, and annual consumption per patient varied between 97.2 and 136.1 vials. Conclusions Unequal access to dornase alfa across Brazil highlights structural inequities in the SUS. The proposed methodology supports systematic monitoring of access to high-cost technologies and may inform targeted public policies to enhance justice in rare disease care.