Data_Sheet_1_Cost-effectiveness of nivolumab plus ipilimumab as first-line treatment for American patients with unresectable malignant pleural mesothelioma.pdf

BackgroundThe treatment paradigm of unresectable malignant pleural mesothelioma (MPM) has changed in recent years. Checkmate 743 demonstrate that nivolumab plus ipilimumab showed good clinical benefits compared with chemotherapy in the treatment of MPM. The study is aim to evaluate the cost-effectiveness of Nivolumab plus ipilimumab vs. platinum plus chemotherapy for the first-line treatment of unresectable MPM.
MethodsA Markov model was developed to compare the cost and quality-adjusted life-year (QALY) of nivolumab plus ipilimumab and chemotherapy over a 10-year time horizon. Clinical efficacy and safety data were extracted from the CheckMate 743 trials. Health state utilities were obtained from published literature. Costs were collected from an US payer perspective. One-way and probabilistic sensitivity analyses were conducted to explore the impact of uncertainties on the cost-effectiveness's results.
ResultsIn the base case analysis, the incremental healthcare costs and QALYs for Nivolumab plus Ipilimumab vs. chemotherapy are $196,604.22 and 0.53, respectively, resulting an incremental cost-effectiveness ratio (ICER) of $372,414.28/QALYs for the model cohort of patients with locally advanced or metastatic MPM. However, Probabilistic sensitivity analysis showed that there was no probability that Nivolumab plus ipilimumab was cost-effective within the fluctuation range of other model parameters in first-line in unresectable MPM. The results of one-way sensitivity analysis showed that the cost of Nivolumab was the most sensitive parameter.
ConclusionsThe ICER of Nivolumab plus ipilimumab is above the theoretical willingness-to-pay threshold in the U.S, which suggests that first-line nivolumab plus ipilimumab for unresectable MPM may be not a cost-effective choice.

研究背景
近年来，不可切除性恶性胸膜间皮瘤（unresectable malignant pleural mesothelioma, MPM）的治疗范式已发生改变。CheckMate 743研究证实，相较于化疗，纳武利尤单抗（nivolumab）联合伊匹木单抗（ipilimumab）在治疗MPM中展现出良好的临床获益。本研究旨在评估纳武利尤单抗联合伊匹木单抗对比含铂化疗方案用于不可切除性MPM一线治疗的成本效益。

研究方法
本研究构建马尔可夫模型（Markov model），在10年时间跨度下对比纳武利尤单抗联合伊匹木单抗与化疗方案的成本及质量调整生命年（quality-adjusted life-year, QALY）。临床疗效与安全性数据提取自CheckMate 743研究，健康状态效用值来自已发表文献，成本数据基于美国支付方视角获取。本研究开展单因素敏感性分析与概率敏感性分析，以探究不确定性因素对成本效益分析结果的影响。

研究结果
基础案例分析显示，相较于化疗方案，纳武利尤单抗联合伊匹木单抗的增量医疗成本与增量质量调整生命年分别为196604.22美元与0.53，针对局部晚期或转移性MPM患者的模型队列计算得到的增量成本效果比（incremental cost-effectiveness ratio, ICER）为372414.28美元/QALY。然而，概率敏感性分析结果表明，在不可切除性MPM一线治疗的其他模型参数波动范围内，纳武利尤单抗联合伊匹木单抗不存在具备成本效益的可能性。单因素敏感性分析结果显示，纳武利尤单抗的成本是最具影响力的敏感参数。

研究结论
在美国医疗体系下，纳武利尤单抗联合伊匹木单抗的增量成本效果比高于理论支付意愿阈值，提示该方案用于不可切除性MPM的一线治疗或许并非具备成本效益的优选方案。