Department of Pharmaceutical Regulatory Science and Pharmacoeconomics, School of Pharmacy, Nanjing Medical University, 101, Longmian Avenue, Jiangning District, Nanjing, People's Republic of China.
Department of Pharmacy, Lianyungang No. 2 People's Hospital, 41, Hailian East Road, Haizhou District, Lianyungang, People's Republic of China.
Int J Clin Pharm. 2023 Jun;45(3):641-649. doi: 10.1007/s11096-023-01540-w. Epub 2023 Feb 17.
Toripalimab is an immune checkpoint inhibitor (ICI) against programmed death ligand 1 (PD-L1). It has been approved for advanced esophageal squamous cell carcinoma (ESCC) as the first-line treatment due to significantly improved progression-free survival (PFS) and overall survival (OS) in the JUPITER-06 trial.
This study aimed to compare the cost-effectiveness between toripalimab plus chemotherapy and placebo plus chemotherapy from the perspective of the Chinese health system.
The study developed a 3-year partitioned survival model to assess costs and outcomes in two treatment groups with or without toripalimab. The critical indicator was the incremental cost-effectiveness ratio (ICER). Scenario and sensitivity analyses were performed to evaluate the robustness of the findings and identify the parameters with the greatest impact on cost-effectiveness.
In the base case analysis, the incremental effectiveness and cost of toripalimab plus chemotherapy versus placebo plus chemotherapy were 0.26 quality-adjusted life year (QALYs) and $11,254.84, respectively, resulting in an ICER of $43,405.09/QALY, higher than the 2021 willingness-to-pay threshold in China ($37,658.70/QALY). The results were sensitive to the utility of PFS, the incidence of neutropenia in the toripalimab group, and the cost of toripalimab. The toripalimab plus chemotherapy group was cost-effective only if the price of toripalimab decreased by more than 40%.
Adding toripalimab to chemotherapy was not cost-effective in patients with advanced ESCC in China.
特瑞普利单抗是一种针对程序性死亡配体 1(PD-L1)的免疫检查点抑制剂(ICI)。由于在 JUPITER-06 试验中显著改善了无进展生存期(PFS)和总生存期(OS),特瑞普利单抗已被批准用于晚期食管鳞状细胞癌(ESCC)的一线治疗。
本研究旨在从中国卫生系统的角度比较特瑞普利单抗联合化疗与安慰剂联合化疗的成本效益。
本研究建立了一个 3 年的分区生存模型,以评估两个治疗组(有无特瑞普利单抗)的成本和结果。关键指标是增量成本效益比(ICER)。进行了情景和敏感性分析,以评估研究结果的稳健性,并确定对成本效益影响最大的参数。
在基础情况分析中,特瑞普利单抗联合化疗与安慰剂联合化疗的增量效果和成本分别为 0.26 个质量调整生命年(QALY)和 11254.84 美元,导致 ICER 为 43405.09 美元/QALY,高于中国 2021 年的意愿支付阈值(37658.70 美元/QALY)。结果对 PFS 的效用、特瑞普利单抗组中性粒细胞减少症的发生率和特瑞普利单抗的成本敏感。只有当特瑞普利单抗的价格下降超过 40%时,特瑞普利单抗联合化疗组才具有成本效益。
在中国,晚期 ESCC 患者中添加特瑞普利单抗联合化疗并不具有成本效益。