Spinal muscular atrophy (SMA-5q) care-associated costs in Brazil

Abstract

Objective: This study estimated the individual cost over a year of monitoring a Brazilian cohort of patients diagnosed with spinal muscular atrophy 5q (5q SMA) using a microcosting technique, including direct and indirect costs. Methods: The hospital, home care, and transportation costs of a cohort of patients monitored at the Hospital de Clínicas de Porto Alegre were evaluated along 2019. The costing method used for the evaluation of hospital costs was the Time-driven Activity-based Costing (TDABC). For the costs of home care, interviews were conducted with family members. For the estimation of transportation costs, reference values of cost per kilometer traveled were considered. Annual hospital, home, and transportation costs per patient were calculated and stratified according to the use or not of the drug nusinersen, the type of 5q SMA and the patients’ age group. Results: Data from 43 patients were analyzed, of which 5 were treated with nusinersen. The average cost of the 38 patients who did not use the medication was R$ 4.870 (min R$ 190 - max R$ 67.138), while the average cost of the 5 patients who were treated with nusinersen was R$ 600.347 (min R$ 342.679 - max R$ 813.513). In the group of patients who used nusinersen, 91% of the total cost was due to medication. Concerning transportation, an average annual cost of R$ 4.895 (min R$ 18 - max R$ 73.382) was observed and the average annual family expenditure per patient reported was R$ 32.262 (min R $ 240 -max R$ 110,952). Patients with 5q SMA type 1 or 2 on average had a higher cost than those with 5q SMA type 3 or 4, as well as patients under 6 years of age in comparison to the others. Conclusions: The cost of medical care for patients with 5q SMA is expressive from the health system’s perspective and patients’ families. These estimates are directly related to the type of disease and the therapy used.