Chen Qiuping, Sun Quan, Li Baixue
School of Basic Medical Sciences, Chengdu University of Traditional Chinese Medicine, Chengdu, Sichuan, China.
Health Care Security and Pharmacoeconomic Evaluation Branch, China Medical Education Association, Beijing, China.
Front Oncol. 2025 Jun 5;15:1459695. doi: 10.3389/fonc.2025.1459695. eCollection 2025.
The GEMSTONE-304 trial established the clinical benefits of sugemalimab plus chemotherapy in advanced esophageal squamous-cell carcinoma (ESCC). This study evaluates the cost-effectiveness of this regimen versus chemotherapy alone as the first-line treatment for advanced ESCC patients from the perspective of China's health system.
We established a partitioned survival model based on GEMSTONE-304 trial data, we simulated lifetime outcomes through three health states: progression-free survival, progressive disease, and death. Key parameters included quality-adjusted life years (QALYs) and incremental cost-effectiveness ratio (ICER), analyzed with 5% discounting. Sensitivity analyses encompassed probabilistic, one-way, and scenario evaluations.
The sugemalimab combination yielded 0.336 incremental QALYs at $ 44,182.03 additional cost (ICER = $ 131,544.70/QALY). PD-L1 subgroup ICERs exhibited dose-dependent efficacy: $ 187,421.63/QALY (Combined Positive Score (CPS) < 1), $ 175,689.56 (1 ≤ CPS < 10), and $ 130,349.21 (CPS ≥ 10). Scenario analysis demonstrated ICER reduction to $ 51,454.12/QALY under consideration of patient assistance program. None of the results demonstrated cost-effectiveness for this therapeutic regimen. Sensitivity analyses identified sugemalimab pricing as the dominant driver of ICER, while simultaneously validating the model's internal and external validity. Price cap simulations determined that a minimum 91.20% price reduction is required to achieve cost-effectiveness.
Current pricing renders sugemalimab combination therapy economically unfavorable as first-line ESCC treatment in China. Strategic price adjustments could enhance cost-effectiveness potential.
GEMSTONE-304试验确立了舒格利单抗联合化疗在晚期食管鳞状细胞癌（ESCC）中的临床获益。本研究从中国卫生系统的角度评估了该方案与单纯化疗作为晚期ESCC患者一线治疗方案的成本效益。
我们基于GEMSTONE-304试验数据建立了一个分区生存模型，通过无进展生存期、疾病进展和死亡三种健康状态模拟终生结局。关键参数包括质量调整生命年（QALY）和增量成本效益比（ICER），采用5%的贴现率进行分析。敏感性分析包括概率分析、单因素分析和情景评估。
舒格利单抗联合方案产生了0.336个增量QALY，但额外成本为44,182.03美元（ICER = 131,544.70美元/QALY）。PD-L1亚组的ICER显示出剂量依赖性疗效：187,421.63美元/QALY（联合阳性评分（CPS）<1）、175,689.56美元（1≤CPS<10）和130,349.21美元（CPS≥10）。情景分析表明，在考虑患者援助计划的情况下，ICER降至51,454.12美元/QALY。所有结果均未表明该治疗方案具有成本效益。敏感性分析确定舒格利单抗定价是ICER的主要驱动因素，同时验证了模型的内部和外部有效性。价格上限模拟确定，至少需要降低91.20%的价格才能实现成本效益。
目前的定价使舒格利单抗联合疗法在中国作为ESCC一线治疗在经济上不利。战略性价格调整可能会增强成本效益潜力。
