Economic evaluation of risdiplam in the treatment of pediatric spinal muscular atrophy under health insurance scenarios
10.12206/j.issn.2097-2024.202601017
- VernacularTitle:医保情景下利司扑兰治疗儿童脊髓性肌萎缩症的经济学评价
- Author:
Dan HAN
1
;
Fei YU
2
;
Xintong ZHU
2
;
Yue WU
3
;
Wei SHAO
2
;
Ruimin LI
1
Author Information
1. School of Pharmacy, Naval Medical University, Shanghai 200433, China.
2. Liaoning Institute of Basic Medical Sciences, Shenyang 110101, China;School of Pharmacy, Liaoning Vocational College of Medicine, Shenyang 110101, China.
3. Department of Ophthalmology, The Fourth People’s Hospital of Shenyang, Shenyang 110031, China.
- Publication Type:Pharmacyadministration
- Keywords:
health insurance;
multi-tiered healthcare security;
risdiplam;
spinal muscular atrophy;
cost-utility analysis;
rare disease;
pharmacoeconomics
- From:
Journal of Pharmaceutical Practice and Service
2026;44(8):431-436
- CountryChina
- Language:Chinese
-
Abstract:
Objective To evaluate the cost-utility of risdiplam in the treatment of pediatric spinal muscular atrophy （SMA） and analyze changes in patients’ out-of-pocket economic burden under the medical insurance scenarios. Methods A Markov model was developed from the healthcare system perspective to compare the long-term costs and quality-adjusted life years （QALYs） of risdiplam treatment versus conventional therapy. The incremental cost-utility ratio （ICUR） was calculated. One-way sensitivity analysis and probabilistic sensitivity analysis were conducted to assess the robustness of the model. On this basis, health insurance scenarios were simulated to evaluate patients’ individual economic burden. Results Base-case analysis showed that risdiplam treatment generated additional health benefits but substantially increased medical costs. The resulting ICUR did not demonstrate a clear cost-utility advantage under the current willingness-to-pay threshold. Sensitivity analyses indicated that the model results were relatively robust, with drug costs and key utility parameters being the main drivers of economic outcomes. Health insurance scenario analysis demonstrated that increasing reimbursement levels and reducing patient copayment rates could significantly alleviate patients’ financial burden. Conclusion Risdiplam for pediatric SMA faces economic challenges under the current health insurance payment framework in China. However, optimizing health insurance reimbursement policies and developing multi-tiered healthcare security mechanisms may improve its affordability and accessibility.
