Abstract

Objectives

This study aimed to evaluate the potential budget impact of nusinersen in the treatment of spinal muscular atrophy (SMA) from the perspective of the basic medical insurance payer in China.

Methods

A budget impact analysis model was developed according to ISPOR budget impact analysis guidelines; we integrated health resource consumption, epidemiology, and market data for the treatment of patients with SMA in China, to estimate the impact of nusinersen on basic medical insurance. The microcosting method was conducted to evaluate the treatment cost. One-way sensitivity analysis was performed to explore the stability of the results.

Results

If nusinersen could be reimbursed, the impact on the medical insurance funds in the next 1 to 3 years will be 253 million, 333 million, and 426 million, respectively. In addition, it can reduce 0.73 million, 2.17 million, and 2.22 million in complication costs, respectively.

Conclusions

SMA is a rare disease with a very low prevalence; reimbursement of nusinersen will lead to a limited impact on the basic medical insurance and improve the accessibility of treatment.