Abstract
Introduction (with objective): Spinal Muscular Atrophy (SMA) is a genetic disease characterized by the progressive degeneration of motor neurons in the spinal cord, leading to varying degrees of muscle weakness and atrophy. In the most severe presentations, there is a risk of impairment of vital functions, such as breathing and swallowing, as well as delays or loss in the acquisition of motor development milestones (e.g., walking or sitting). The condition is divided into five types (0 to IV) according to the age of onset of symptoms and the highest motor milestone achieved, highlighting great clinical heterogeneity and complex care demands. Despite therapeutic advances, equitable access to disease-modifying therapies remains a significant challenge due to the high cost and limited robustness of evidence for some subtypes. In Brazil, the Clinical Protocol and Therapeutic Guidelines (PCDT) for SMA were established in 2022, restricted to patients with SMA types I and II, maintaining judicialization as an important access strategy. The objective is to evaluate the number of lawsuits and public spending related to the treatment of SMA in Brazil, comparing the periods before and after the implementation of the PCDT (Clinical Protocol and Therapeutic Guidelines). Material and Method: Cross-sectional and retrospective study based on national data on lawsuits related to SMA treatment, obtained through requests via the Access to Information Law. Lawsuits from the period of regulatory approvals of medications until March 2026 were included. The number of lawsuits and public spending associated with the provision of therapies were analyzed. Results: In the pre-PCDT period (2019–2022), 256 judicialization processes involving nusinersen and risdiplam were registered, with a median (min-max) of 44.5 (3-164) processes per year, and R$ 185,649,305.55 was spent, with a median (min-max) of R$ 26,069,841.12 (R$ 1,595,051.12 - R$ 131,914,572.20). In the post-PCDT period (2023–2026), 272 processes related to the same medications were identified, with a median (min-max) of 59.5 (11-142) and the total expenditure was R$ 389,728,669.64, with a median (min-max) of R$ 99,488,170.19 (R$ 29,556,938.94 - R$ 161,195,390.32). Additionally, 50 lawsuits were registered between 2024 and 2026 for onasemnogene abeparvovec-xioi, a gene therapy approved for commercialization in Brazil in 2020 and incorporated into the SUS (Brazilian Unified Health System) in 2022. This was only incorporated into the PCDT (Clinical Protocol and Therapeutic Guidelines) for SMA (Spinal Muscular Atrophy) in 2025. The expenses associated with these lawsuits totaled R$ 345,903,235.45 in the analyzed period. Conclusions: The establishment of the PCDT for SMA in Brazil was not accompanied by a significant reduction in the volume of litigation or public spending related to the treatment of the disease. Despite the partial incorporation of therapies into the SUS, litigation remained a central access strategy, indicating limitations in the protocol design. The introduction of onasemnogene abeparvovec xioi generated a significant short-term financial impact, even after its incorporation into the SUS. The persistence of lawsuits, especially for patients with SMA types III and IV, reveals allocative inefficiency and indicates the need to revise the protocol criteria for greater system sustainability.
