Cost-effectiveness analysis of Risdiplam and Nusinersen for the treatment of Chinese patients with spinal muscular atrophy

As a rare disease with the largest number of cases in China, spinal muscular atrophy (SMA) causes severe motor deficits as well as multi-system organ damage. Some potent drugs for SMA, such as Nusinersen (NU) and Risdiplam (RI) represented by targeted survival motor neuron gene therapy, have become a new trend in the treatment of SMA. RI has gradually attracted attention due to its ease of administration, safety, and efficacy. The study was to analyze the cost-effectiveness of RI and NU for the treatment of SMA in China from the perspective of the healthcare system. We constructed a five-state Markov model based on the different stages of SMA. The willingness-to-pay threshold was chosen to be 1–3 times the gross domestic product (GDP) per capita. The base-case analysis was used to calculate the incremental cost-effectiveness ratio (ICER), and one-way and probabilistic sensitivity analyses were performed. The ICER value was ¥132,402.10/QALY, which was in the range of 1–3 times GDP per capita. Sensitivity analysis results showed the cost of RI and NU had a significant effect on ICER as well as proving the stability of results. RI is a cost-effective option compared to NU in SMA treatment.