帕博利珠单抗一线治疗不可切除或转移性MSI-H/dMMR结直肠癌的成本效果分析

Cost-effectiveness analysis of pembrolizumab in first-line treatment of patients with unresectable or metastatic MSI-H/dMMR colorectal cancer

目的评价帕博利珠单抗用于不可切除或转移性微卫星高度不稳定/错配修复缺陷(MSI-H/dMMR)结直肠癌患者一线治疗的经济性。方法基于中国卫生体系角度,利用KEYNOTE-177临床试验数据,构建分区生存模型评价帕博利珠单抗对比标准治疗的成本与效用,模型周期与研究时限分别设置为21 d与10年。以增量成本效果比(ICER)为评价指标,并对关键参数进行情景分析和敏感性分析。结果与标准治疗相比,帕博利珠单抗的ICER为221546.85元·QALY^(-1),低于2021年3倍中国人均国内生产总值(GDP)。若有患者援助计划,帕博利珠单抗为绝对优势。单因素敏感性分析表明,对ICER影响最大的3个参数分别为标准治疗后续治疗中帕博利珠单抗比例、帕博利珠单抗价格和贴现率。概率敏感性分析显示,在2021年3倍中国人均GDP下,帕博利珠单抗具有经济性的概率为63.36%。结论相比标准治疗,帕博利珠单抗在不可切除或转移性MSI-H/dMMR结直肠癌的一线治疗中具有经济学优势。

AIM To evaluate the economics of pembrolizumab in the first-line treatment of patients with unresectable or metastatic microsatellite instability-high/mismatch repair-deficient(MSI-H/dMMR)colorectal cancer.METHODS Based on the Chinese healthcare system,a partitioned survival model was constructed to compare the cost and utility between pembrolizumab and standard of care using KEYNOTE-177 clinical trial data.The cycle length and time horizon of the model was set as 21 days and 10 years,respectively.The incremental cost-effectiveness ratio(ICER)was used as the evaluation indicator.Scenario analysis and sensitivity analysis were also performed.RESULTS Compared with standard of care,the ICER of pembrolizumab was 221546.85 yuan·QALY^(-1),which was lower than 3 times China's per capita gross domestic product(GDP)in 2021.The pembrolizumab was dominant when patient assistance program was considered.The results of one-way sensitivity analysis showed that the proportion of pembrolizumab on subsequent treatment in the standard of care,pembrolizumab price and discounting exhibited the significant impact on the ICER.The results of probability sensitivity analysis showed that under the 3 times China's per capita GDP in 2021,the probability of pembrolizumab being cost-effectiveness was 63.36%.CONCLUSION Pembrolizumab has a cost-effective advantage over standard of care as first-line treatment for unresectable or metastatic MSI-H/dMMR colorectal cancer in China.