基于分区生存模型的帕博利珠单抗单药与化疗一线治疗PD-L1肿瘤比例分数不同的非小细胞肺癌的成本-效果分析

目的：从卫生体系角度出发，评价帕博利珠单抗单药与化疗一线治疗PD-L1肿瘤比例分数≥1%的局部晚期或转移性非小细胞肺癌的经济性，为相关卫生决策提供参考。方法：根据疾病发展进程建立无进展、进展和死亡三状态分区生存模型，以质量调整生命年（QALY）作为产出指标计算增量成本-效果比。生存分析数据来自于一项多中心随机对照非盲的Ⅲ期临床试验KEYNOTE-042，成本数据来自于米内网和8个省市的医疗卫生服务项目价格中位数，效用数据源于已发表文献，并对关键参数进行敏感性分析和情景分析。结果：基础分析结果表明，相较于标准化疗组，帕博利珠单抗单药治疗相对于化疗的增量成本效果比在PD-L1肿瘤比例分数不同的人群中分别为228 254.12元/QALY（TPS≥50%）、351 267.03元/QALY（TPS≥20%）和256 990.96元/QALY（TPS≥1%）。单因素敏感性分析显示帕博利珠单抗价格和PFS状态效用等对ICER的变化有较大影响。概率敏感度分析结果表明模型结构稳定，稳健性较好。结论：在中国3倍人均GDP（193 932.00元）的意愿支付阈值下，帕博利珠单抗单药治疗与化疗相比不具有成本-效果，其经济性未来可通过降低价格来实现。

Cost-effectiveness analysis of pembrolizumab versus chemotherapy as first-line treatment in non-small cell lung cancer with different PD-L1 expression levels based on partitioned survival model

OBJECTIVE To evaluate the economics of pembrolizumab monotherapy and chemotherapy in the first-line treatment of PD-L1 tumors with PD-L1 tumor proportion scores ≥ 1% in locally advanced or metastatic non-small cell lung cancer from the perspective of the health care system.METHODS According to the disease development process, a three-state partitioned survival model:progression-free, progressive disease and death was established, and the incremental cost-effectiveness ratio was calculated using quality-adjusted life years (QALYs) as the output index.Survival analysis data were obtained from a multicenter randomized controlled unblinded phase Ⅲ clinical trial KEYNOTE-042, cost data were obtained from the median price of medical and health service items in MineNet and 8 provinces and cities, utility data were obtained from the published literature, and sensitivity analysis and scenario analysis were performed for key parameters.RESULTS Based on the results of the basic analysis, compared to the standard chemotherapy group, the incremental cost-effectiveness ratio of pembrolizumab versus chemotherapy was ￥228 254.12/QALY (TPS ≥ 50%), ￥351 267.03/QALY (TPS ≥ 20%) and ￥256 990.96/QALY. The deterministic sensitivity analysis showed that the price of pembrolizumab and the utility of PFS status had a greater impact on the changes in ICER. The results of probability sensitivity analysis showed that the model structure was stable and robust.CONCLUSION Under the willingness-to-pay threshold of 3 times GDP per capita (193 932.00 yuan) in China, pembrolizumab treatment has no cost-effectiveness compared with chemotherapy, and its economy can be realized by reducing the price in the future.