Cost-Effectiveness Analysis of Enfortumab Vedotin plus Pembrolizumab as First-Line Treatment for Advanced Urothelial Carcinoma

目的：评估维恩妥尤单抗(EV)联合帕博利珠单抗(Pembro)作为晚期尿路上皮癌(UC)一线治疗的成本效果，为临床决策和医保政策提供参考。方法：基于EV-302试验数据，从中国卫生体系视角构建分区生存模型(Partitioned Survival Model, PSM)，评估EV + Pembro相较于吉西他滨联合顺铂/卡铂(Chemo)的成本效益。研究纳入直接医疗成本，包括药品费用、管理费用、检查费用、随访费用、不良事件管理费用等，并计算增量成本效果比(ICER)。同时，进行单因素敏感性分析和概率敏感性分析，并开展情景分析评估EV价格下降对ICER的影响。结果：EV + Pembro较Chemo方案可额外获得0.78质量调整生命年(QALY)，但总治疗成本增加1910981.51元，ICER为2440191.76元/QALY，远超我国支付意愿阈值(275,100元/QALY)。敏感性分析表明，EV和Pembro的药品成本、贴现率及无进展生存期(PFS)状态效用值对ICER影响最大。情景分析显示，即使EV价格下降90%，ICER仍高于WTP阈值，该方案在当前定价下不具备成本效益。结论：尽管EV + Pembro在晚期UC患者中展现出显著的临床获益，但在我国当前支付意愿水平下，该方案经济性不足。价格调整、医保覆盖、精准筛选适宜人群及真实世界数据分析或有助于提高其经济可行性。Objective: To evaluate the cost-effectiveness of Enfortumab Vedotin (EV) combined with Pembrolizumab (Pembro) as a first-line treatment for advanced urothelial carcinoma (UC) and provide insights for clinical decision-making and health insurance policy. Methods: A partitioned survival model (PSM) was developed from the perspective of the Chinese healthcare system using data from the EV-302 trial to assess the cost-effectiveness of EV + Pembro compared to gemcitabine plus cisplatin/carboplatin (Chemo). The study incorporated direct medical costs, including drug costs, administration fees, examination expenses, follow-up costs, and adverse event management costs, to calculate the incremental cost-effectiveness ratio (ICER). One-way sensitivity analysis and probabilistic sensitivity analysis were conducted, along with scenario analyses to evaluate the impact of EV price reductions on the ICER. Results: Compared to Chemo, EV + Pembro provided an additional 0.78 quality-adjusted life years (QALYs) but increased total treatment costs by ¥1910981.51, resulting in an ICER of ¥2440191.76/QALY, which far exceeds China’s willingness-to-pay (WTP) threshold of ¥275,100/QALY. Sensitivity analysis indicated that the drug costs of EV and Pembro, the discount rate, and the utility value of the progression-free survival (PFS) state had the greatest impact on ICER. Scenario analysis demonstrated that even with a 90% price reduction in EV, the ICER remained above the WTP threshold, indicating that the regimen is not cost-effective at current pricing. Conclusion: Although EV + Pembro demonstrates significant clinical benefits for patients with advanced UC, its cost-effectiveness remains unfavorable under the current WTP threshold in China. Adjustments in pricing, health insurance coverage, targeted patient selection, and real-world data analysis may improve its economic feasibility.