Zhan Mei, Xu Ting, Zheng Hanrui, He Zhiyao
Department of Pharmacy, State Key Laboratory of Biotherapy and Cancer Center, National Clinical Research Center for Geriatrics, West China Hospital, Sichuan University, Chengdu, China.
West China School of Pharmacy, Sichuan University, Chengdu, China.
Front Public Health. 2022 Mar 2;10:790225. doi: 10.3389/fpubh.2022.790225. eCollection 2022.
The KEYNOTE-181 study demonstrated that pembrolizumab for advanced or metastatic esophageal cancer in patients with programmed death ligand-1 (PD-L1) combined positive score (CPS) ≥ 10 had a survival advantage and better tolerability than chemotherapy. However, at the same time, pembrolizumab places an economic burden on patients. This study assessed the cost-effectiveness of pembrolizumab based on the KEYNOTE181 study.
A three-state Markov model [progression-free survival (PFS), progressive disease (PD), and death] based on data from the KEYNOTE-181 study was used to estimate the incremental cost-effectiveness ratio (ICER) of pembrolizumab versus chemotherapy for advanced or metastatic esophageal cancer. The model evaluates the outcomes from the Chinese society's perspective. Costs, quality-adjusted life-years (QALYs), and the ICER in terms of 2021 US$ per QALY gained, were calculated. one-way and probabilistic sensitivity analyses were performed to evaluate the model robustness.
Compared with chemotherapy, pembrolizumab increased costs by $37,201.68, while gaining 0.23 QALYs, resulting in an ICER of $163,165.26 per QALY in patients with PD-L1 CPS ≥ 10. The ICER is $202,708.62 per QALY and $163,643.19 per QALY in the total population and patients with esophageal squamous cell carcinoma, respectively. The ICER was much higher than the commonly accepted willingness-to-pay threshold ($11,105.8 per QALY). One-way and sensitivity analyses showed that the costs of pembrolizumab and the utility of PD were the crucial factors in determining the ICER, and probabilistic sensitivity analyses demonstrated pembrolizumab is unlikely to be cost-effective at a willingness-to-pay threshold of $11,105.8 per QALY. The result was robust across sensitivity analyses.
Pembrolizumab is not a cost-effective treatment option for the second-line treatment of esophageal cancer from the perspective of Chinese society.
KEYNOTE-181研究表明,帕博利珠单抗用于程序性死亡配体1(PD-L1)联合阳性评分(CPS)≥10的晚期或转移性食管癌患者,与化疗相比具有生存优势且耐受性更好。然而,与此同时,帕博利珠单抗给患者带来了经济负担。本研究基于KEYNOTE-181研究评估了帕博利珠单抗的成本效益。
基于KEYNOTE-181研究的数据,使用三状态马尔可夫模型[无进展生存期(PFS)、疾病进展(PD)和死亡]来估计帕博利珠单抗与化疗用于晚期或转移性食管癌的增量成本效益比(ICER)。该模型从中国社会的角度评估结果。计算了成本、质量调整生命年(QALY)以及每获得一个QALY以2021年美元计的ICER。进行了单向和概率敏感性分析以评估模型的稳健性。
与化疗相比,帕博利珠单抗使成本增加了37,201.68美元,同时获得了0.23个QALY,导致PD-L1 CPS≥10的患者每QALY的ICER为163,165.26美元。在总体人群和食管鳞状细胞癌患者中,每QALY的ICER分别为202,708.62美元和163,643.19美元。ICER远高于普遍接受的支付意愿阈值(每QALY 11,105.8美元)。单向和敏感性分析表明,帕博利珠单抗的成本和PD的效用是决定ICER的关键因素,概率敏感性分析表明,在每QALY支付意愿阈值为11,105.8美元时,帕博利珠单抗不太可能具有成本效益。该结果在敏感性分析中具有稳健性。
从中国社会的角度来看,帕博利珠单抗并非食管癌二线治疗的具有成本效益的治疗选择。