依库珠单抗治疗阵发性睡眠性血红蛋白尿症的成本-效用分析

Cost-utility analysis of eculizumab in the treatment of paroxysmal nocturnal hemoglobinuria

目的 评估依库珠单抗对比支持治疗对阵发性睡眠性血红蛋白尿症(paroxysmal nocturnal hemoglobinuria,PNH)的经济性。方法 基于中国全社会角度,构建分区生存模型模拟患者终生(40年)的总成本、质量调整生命年(quality-adjusted life years,QALYs)和增量成本-效果比(incremental cost-effectiveness ratio,ICER),贴现率为5%。采用单因素和概率敏感性分析检验模型的稳健性。结果 基础分析结果显示,患者使用依库珠单抗相较支持治疗可多获得8.50个QALYs,费用增加639 784元,ICER值为75 297元/QALY,低于1倍2023年人均国内生产总值(GDP)。单因素敏感性分析显示,年自发缓解率对模型影响最大。概率敏感性分析显示,当意愿支付阈值为3倍人均GDP时,依库珠单抗具有成本-效果的概率为99.98%。结论 在中国全社会角度下,与支持治疗相比,依库珠单抗治疗PNH具有经济性。

Objective To evaluate the cost-utility of eculizumab versus supportive therapy in the treatment of paroxysmal nocturnal hemoglobinuria(PNH).Methods From the perspective of whole Chinese society,a partitioned survival model was constructed to simulate the total cost,quality-adjusted life years(QALYs)and incremental costeffectiveness ratio(ICER)of patients over a lifetime(40 years)with a discount rate of 5%.One-way sensitivity analysis and probabilistic sensitivity analysis were used to verify the robustness of the model.Results The base-case results showed that compared with supportive therapy,patients who used eculizumab could obtaine 8.50 more QALYs and an incremental cost of 639784 CNY,the ICER was 75297 CNY/QALY,which was lower than the GDP per capita in 2023.The oneway sensitivity analysis showed that the annual spontaneous remission rate had the most significant impact on the final result.The probabilistic sensitivity analysis showed that the probability of cost-effective of eculizumab was 99.98%when the willingness-to-pay threshold was 3 times of GDP per capital.Conclusion From the perspective of the Chinese society,eculizumab was a cost-effective treatment regimen compared to supportive therapy in the treatment of PNH patients.