Lai Yizhen, Bensimon Arielle G, Gao Emily, Bhattacharya Rituparna, Xu Ruifeng, Chevure Jestinah, Imai Kentaro, Haas Naomi B
Merck & Co., Inc., Rahway, NJ.
Health Economics and Outcomes Research, Analysis Group, Inc., Boston, MA.
Clin Genitourin Cancer. 2023 Oct;21(5):612.e1-612.e11. doi: 10.1016/j.clgc.2023.03.016. Epub 2023 Apr 6.
Pembrolizumab was recently approved as an adjuvant treatment of renal cell carcinoma (RCC), based on prolonged disease-free survival compared to placebo in the phase III KEYNOTE-564 trial. The objective of this study was to evaluate the cost-effectiveness of pembrolizumab as monotherapy in the adjuvant treatment of RCC post-nephrectomy, from a US health sector perspective.
A Markov model with 4 health states (disease-free, locoregional recurrence, distant metastases, and death) was developed to compare the cost and effectiveness of pembrolizumab versus routine surveillance or sunitinib. Transition probabilities were estimated using patient-level KEYNOTE-564 data (cutoff: June 14, 2021), a retrospective study, and published literature. Costs of adjuvant and subsequent treatments, adverse events, disease management, and terminal care were estimated in 2022 US$. Utilities were based on EQ-5D-5L data collected in KEYNOTE-564. Outcomes included costs, life-years (LYs), and quality-adjusted LYs (QALYs). Robustness was assessed through one-way and probabilistic sensitivity analyses.
Total cost per patient was $549,353 for pembrolizumab, $505,094 for routine surveillance, and $602,065 for sunitinib. Over a lifetime, pembrolizumab provided gains of 0.96 QALYs (1.00 LYs) compared to routine surveillance, yielding an incremental cost-effectiveness ratio of $46,327/QALY. Pembrolizumab dominated sunitinib with 0.89 QALYs (0.91 LYs) gained while saving costs. At a $150,000/QALY threshold, pembrolizumab was cost-effective versus both routine surveillance and sunitinib in 84.2% of probabilistic simulations.
Pembrolizumab is projected to be cost-effective as an adjuvant RCC treatment versus routine surveillance or sunitinib based on a typical willingness-to-pay threshold.
帕博利珠单抗最近被批准用于肾细胞癌（RCC）的辅助治疗，这是基于III期KEYNOTE-564试验中与安慰剂相比延长的无病生存期。本研究的目的是从美国卫生部门的角度评估帕博利珠单抗作为单一疗法在肾切除术后RCC辅助治疗中的成本效益。
建立了一个具有4种健康状态（无病、局部区域复发、远处转移和死亡）的马尔可夫模型，以比较帕博利珠单抗与常规监测或舒尼替尼的成本和效果。使用患者层面的KEYNOTE-564数据（截止日期：2021年6月14日）、一项回顾性研究和已发表的文献来估计转移概率。辅助治疗和后续治疗、不良事件、疾病管理和终末期护理的成本以2022年美元估算。效用基于KEYNOTE-564中收集的EQ-5D-5L数据。结果包括成本、生命年（LYs）和质量调整生命年（QALYs）。通过单因素和概率敏感性分析评估稳健性。
帕博利珠单抗每位患者的总成本为549,353美元，常规监测为505,094美元，舒尼替尼为602,065美元。与常规监测相比，帕博利珠单抗在一生中可提供0.96个QALY（1.00个LYs）的收益，增量成本效益比为46,327美元/QALY。帕博利珠单抗优于舒尼替尼，获得了0.89个QALY（0.91个LYs），同时节省了成本。在150,000美元/QALY的阈值下，在84.2%的概率模拟中，帕博利珠单抗与常规监测和舒尼替尼相比具有成本效益。
基于典型的支付意愿阈值，预计帕博利珠单抗作为RCC辅助治疗与常规监测或舒尼替尼相比具有成本效益。
