McCrea Charles, Johal Sukhvinder, Yang Shuo, Doan Justin
Health Economic Modelling Unit, PAREXEL Access Consulting, Evergreen Building North, 160 Euston Road, London, NW1 2DX UK.
2Bristol-Myers Squibb, Princeton, NJ USA.
Exp Hematol Oncol. 2018 Feb 9;7:4. doi: 10.1186/s40164-018-0095-8. eCollection 2018.
We evaluated the cost-effectiveness of nivolumab versus everolimus in patients with advanced renal cell carcinoma (RCC) from a US payer perspective.
A partitioned survival model consisting of three health states, progression-free survival (PFS), progressive disease, and death, was developed to evaluate the cost-effectiveness of intravenous nivolumab versus oral everolimus over a lifetime. The proportion of patients in each state was calculated based on parametric distributions fitted to PFS and overall survival (OS) data from CheckMate 025 (N = 821), a large randomized phase 3 trial of nivolumab versus everolimus for advanced RCC. Health state utility data were derived from CheckMate 025 EQ-5D data. Scenario analyses and deterministic and probabilistic sensitivity analyses assessed the impact of uncertainty in model inputs on outcomes.
Over a 25-year lifetime horizon, treatment with nivolumab resulted in a gain of 0.64 quality-adjusted life-years (QALYs) versus everolimus. Nivolumab had greater total costs versus everolimus ($US197,089 vs. $US163,902), mainly due to higher acquisition costs. The incremental cost-utility ratio (ICUR), a measure of incremental costs divided by incremental QALYs, was $US51,714 per QALY gained for nivolumab versus everolimus, and an incremental cost-effectiveness ratio was $US44,576 per life-year gained for nivolumab versus everolimus. In sensitivity analyses, average body weight had the greatest impact on the ICUR for nivolumab versus everolimus (base case $US51,714; range $US8863-$US94,566). At a $US150,000 willingness-to-pay (WTP) threshold, nivolumab had a 91.7% probability of being cost-effective versus everolimus.
In the United States, at a WTP threshold of $US150,000 per QALY, nivolumab was found to be cost-effective. Key drivers of cost-effectiveness were survival inputs for OS and the average weight of patients; the latter directly affects nivolumab drug acquisition cost.
我们从美国医保支付方的角度评估了纳武利尤单抗与依维莫司治疗晚期肾细胞癌（RCC）患者的成本效益。
建立了一个由无进展生存期（PFS）、疾病进展和死亡三种健康状态组成的分割生存模型，以评估静脉注射纳武利尤单抗与口服依维莫司终身治疗的成本效益。根据对CheckMate 025（N = 821）试验中PFS和总生存期（OS）数据拟合的参数分布来计算每个状态下的患者比例，CheckMate 025是一项纳武利尤单抗与依维莫司治疗晚期RCC的大型随机3期试验。健康状态效用数据来自CheckMate 025的EQ - 5D数据。情景分析以及确定性和概率性敏感性分析评估了模型输入的不确定性对结果的影响。
在25年的生存期内，与依维莫司相比，纳武利尤单抗治疗可使质量调整生命年（QALY）增加0.64。纳武利尤单抗的总成本高于依维莫司（197,089美元对163,902美元），主要是由于获取成本较高。增量成本效用比（ICUR，即增量成本除以增量QALY），纳武利尤单抗相对于依维莫司为每获得一个QALY 51,714美元，增量成本效益比为纳武利尤单抗相对于依维莫司每获得一个生命年44,576美元。在敏感性分析中，平均体重对纳武利尤单抗与依维莫司的ICUR影响最大（基础情况为51,714美元；范围为8863美元至94,566美元）。在支付意愿（WTP）阈值为150,000美元时，纳武利尤单抗相对于依维莫司具有成本效益的概率为91.7%。
在美国，每QALY的WTP阈值为150,000美元时，纳武利尤单抗被认为具有成本效益。成本效益的关键驱动因素是OS的生存输入和患者的平均体重；后者直接影响纳武利尤单抗的药物获取成本。
