RESUMO
OBJECTIVES: The aim of the budget impact analysis (BIA) was to determine the economic impact of introducing risdiplam in the treatment of type 3 spinal muscular atrophy (SMA) patients in a rare diseases reference centre on a 3-year time horizon, as compared with nusinersen. METHODS: Public databases were used to estimate the target population. Two market scenarios were assessed over a 3-year time horizon: with nusinersen and with the introduction of risdiplam. Drug acquisition and administration costs were considered. BIA is calculated as the difference between scenarios with nusinersen - scenario with risdiplam. RESULTS: The introduction of risdiplam would generate a 3-year saving of 3411.50. There could be a saving in the administration of risdiplam in the treatment of patients under the age of 2 with a weight of 5 kg (26 382.08). CONCLUSION: The BIA shows the overlapping costs of these therapies, although the oral administration of risdiplam could be a decisive factor for the therapeutical switch from nusinersen.
