恩沙替尼一线治疗间变性淋巴瘤激酶阳性晚期非小细胞肺癌的药物经济学评价

Pharmacoeconomic evaluation of ensatinib in the first-line treatment of anaplastic lymphoma kinasepositive advanced non-small cell lung cancer

目的评价恩沙替尼一线治疗间变性淋巴瘤激酶(ALK)阳性晚期非小细胞肺癌(NSCLC)的经济性,为我国医保决策及临床合理用药提供参考。方法从我国卫生体系角度出发,基于国际多中心Ⅲ期临床试验(eXalt3研究)数据构建三状态分区生存模型,模拟时限为10年,循环周期为30 d。比较恩沙替尼相对于克唑替尼(标准治疗方案)一线治疗ALK阳性晚期NSCLC的经济性,以质量调整生命年(QALY)作为效用指标并计算增量成本-效果比(ICER),再通过不确定性分析来验证基础分析结果的稳健性。结果基础分析结果显示,相对于克唑替尼组,恩沙替尼组患者的人均增量成本为-343370.36元,人均增量效用为0.76 QALYs,ICER为-454292.25元/QALY,远低于1倍2021年我国人均国内生产总值(GDP,即80976元)这一意愿支付(WTP)阈值。单因素敏感性分析结果显示,无进展生存状态效用值、疾病进展状态效用值、克唑替尼后续治疗成本等参数对ICER的影响较大,但这些参数均不能导致基础分析结果翻转。概率敏感性分析结果显示,在以1倍2021年我国人均GDP作为WTP阈值的前提下,恩沙替尼组方案具有经济性的概率为100%。情境分析结果显示,改变恩沙替尼组后续治疗方案所得的ICER为-217671.43元/QALY,仍远低于WTP阈值。结论针对我国ALK阳性晚期NSCLC患者,相对于目前应用较多的标准一线治疗方案(克唑替尼),恩沙替尼更具经济性且为绝对优势方案。

OBJECTIVE To evaluate the economical efficiency of ensatinib in the first-line treatment of anaplastic lymphoma kinase(ALK)positive-advanced non-small cell lung cancer(NSCLC),so as to provide reference for China’s medical insurance decision-making and rational drug use in clinic. METHODS A three-state partitioned survival model was constructed from the perspective of China’s health system,based on the data of the international multi-center phase Ⅲ clinical trial(eXalt3 trail),with simulation time limit of 10 years,cycle period of 30 days. The economical efficiency of ensatinib was compared with that of crizotinib(standard treatment)in the first-line treatment of ALK positive-advanced NSCLC. The incremental cost-effectiveness ratio(ICER) was calculated with quality-adjusted life years(QALYs) as utility index. The stability of basic analysis results was validated through uncertainty analysis. RESULTS The basic analysis results showed that compared with crizotinib group,incremental cost per capita of ensatinib group was-343 370.36 yuan,incremental utility per capita was 0.76 QALYs,ICER was-454 292.25 yuan/QALY,which was far lower than the willingness-to-pay(WTP)threshold of 1 time of China’s per capita gross domestic product(GDP,80 976 yuan)in 2021. The results of univariate sensitivity analysis showed that progression-free survival(PFS)status utility,progression of disease(PD)status utility and subsequent treatment cost of crizotinib had a greater impact on ICER,but these parameters could not cause the reversal of basic analysis results. Probabilistic sensitivity analysis showed that with1 time of China’s per capita GDP in 2021 as the WTP threshold,the probability of ensatinib group’s treatment possessed economical efficiency was 100%. In the situational analysis,ICER obtained by changing the ensatinib group’s follow-up treatment regimen was-217 671.43 yuan/QALY,which was far below WTP threshold. CONCLUSIONS For Chinese patients with ALK positive-advanced NSCLC,compared with commonly used the first-line treatment(crizotinib),ensartinib is economical and absolutely dominant.