富马酸二甲酯治疗复发缓解型多发性硬化的成本效果分析

Cost-utility analysis of dimethyl fumarate compared with terifunomide for the treatment of relapsing multiple sclerosis in China

目的 评估富马酸二甲酯(dimethyl fumarate,DMF)对比特立氟胺(terifunomide,TFM)治疗复发缓解型多发性硬化的成本效果。方法 研究从卫生体系角度出发,构建Markov模型模拟患者终生(37年)。成本包括药品成本、监测成本、疾病管理成本、复发治疗成本和不良事件处理成本。结局指标使用质量调整生命年,效用值的选取包括模型状态和不良事件的效用值,参数来自发表文献、公开文件及专家访谈,并对结果进行单因素敏感性分析和概率敏感性分析。结果 在模拟时限内,DMF组以及TFM组的总成本分别为513 105和642 028元,前者较后者低128 923元,节约主要体现在DMF组有更低的药品、治疗监测、不良事件处理、疾病管理和复发治疗成本。DMF组与TFM组的总质量调整生命年分别为4.10和3.94年,前者较后者增加0.16年。单因素敏感性分析结果显示TFM和DMF的残疾进展风险值是影响结果的主要因素。概率敏感性分析结果显示,在1倍人均GDP的阈值下,DMF相较于TFM具有经济性的概率为100%。结论 基于卫生体系角度,DMF相较TFM在治疗成人复发缓解型多发性硬化的经济性上具有绝对优势。

Objective This study aims to evaluate the cost-utility of dimethyl fumarate(DMF) compared with terifunomide(TFM) for relapsing multiple sclerosis(RMS) in China. Method A Markov model was constructed based on a from healthcare system perspective in China over a 37-year horizon. Drug acquisition costs, monitoring costs, disease management costs, disease relapse costs and adverse events management costs were included. QALYs was used as the outcome. Utility of different model statuses and disutility caused by adverse events were also considered in the inputs.Costs and utility inputs were derived from published studies, public documents, and expert interviews. One-way sensitivity analysis(DSA) and probability sensitivity analysis(PSA) were conducted to evaluate the robustness of the results. Results Over a 37-year time horizon, DMF led to an incremental QALY gain of 0.16(4.10 of DMF and vs 3.94 of TFM) and reduced the total cost of ￥128,923 yuan(￥513,105 of DMF and vs ￥642,028 of TFM) compared with TFM. Cost savings are mainly drug acquisition costs, monitoring costs, disease management costs, disease relapse costs and adverse events management costs. DSA results showed that the disability progression hazard ratio values of TFM and DMF were the main drivers.PSA results showed a 100% probability of DMF being cost-effective compared to TFM at a threshold of 1 GDP per capita.Conclusion DMF dominates the TFM for the treatment of RMS from healthcare system perspective in China.