背景：派姆单抗是一种潜在有价值的治疗方法。然而，患者、医生和医疗保健决策者对其成本效益和这种新疗法的适当定价并不确定。本研究旨在评估派姆单抗作为中国和美国晚期胆道癌 (BTC) 患者一线治疗的成本效益。方法：从中美两国医疗卫生体系角度构建马尔可夫模型进行药物经济学评价。患者基线特征和关键临床数据来自 KEYNOTE-966 试验（ClinicalTrials.gov，NCT04003636）。成本和公用事业是从药品成本网站和出版的文献中收集的。测量并比较了累积成本（美元）、生命年（LY）、质量调整生命年（QALY）和增量成本效益比（ICER）。在给定的支付意愿（WTP）阈值下进行价格模拟，为定价方案提供参考。通过单向敏感性分析和概率敏感性分析来分析模型的稳健性。结果：基础数据分析表明，在中国，在 WTP 阈值为 38,201.19 美元时，派姆单抗（2662.41 美元/100 mg）联合化疗方案相对于化疗方案并不具有成本效益，相对于化疗方案的额外成本为 77,114.94 美元（ICER 556,689.47 美元） /QALY），同时增加 0.14 QALY。 Pembrolizumab（54.71 美元/1 mg）在美国的疗效也提高了 0.14 QALY，但在美国 WTP 阈值 229,044 美元下仍然不具有成本效益，总成本增加了 160,425.24 美元（ICER 1,109,462.92 美元/QALY）。结论：与化疗相比，派姆单抗联合化疗减轻了疾病负担。然而，以目前的价格来看，对于中国和美国的先进比特币来说，这可能不是一种具有成本效益的治疗方法。这项研究可以帮助决策者做出最佳选择。版权所有 © 2024 罗、蔡、吴、李、王和马。

Background: Pembrolizumab is a potentially valuable treatment. However, patients, doctors, and healthcare decision-makers are uncertain about its cost-effectiveness and an appropriate pricing for this new therapy. This study aims to appraise the cost-effectiveness of pembrolizumab as a first-line treatment for advanced biliary tract cancer (BTC) patients in China and the United States (US). Methods: A Markov model was constructed from the perspectives of healthcare systems in both China and the US for pharmacoeconomic evaluation. Patient baseline characteristics and key clinical data were sourced from the KEYNOTE-966 trial (ClinicalTrials.gov, NCT04003636). Costs and utilities were collected from drug cost websites and published literature. Cumulative costs (in USD), life years (LYs), quality-adjusted life years (QALYs), and incremental cost-effectiveness ratios (ICERs) were measured and compared. Price simulations were conducted under given willingness-to-pay (WTP) thresholds to provide pricing scheme references. The model's robustness was analyzed through one-way sensitivity analysis and probabilistic sensitivity analysis. Results: Basic data analysis illustrates that pembrolizumab ($2662.41/100 mg) in combination with chemotherapy regimen was not cost-effective relative to chemotherapy regimens at the WTP threshold of $38,201.19 in China, and the additional cost relative to chemotherapy regimens was $77,114.94 (ICER $556,689.47/QALY) while increasing 0.14 QALYs. Pembrolizumab ($54.71/1 mg) also increased efficacy by 0.14 QALYs in the US, but remained also not cost-effective at the US WTP threshold of $229,044, and the total cost increased by $160,425.24 (ICER $1,109,462.92/QALY). Conclusion: Compared with chemotherapy, pembrolizumab plus chemotherapy reduces the disease of burden. However, at its current price, it may not be a cost-effective treatment for advanced BTC in both China and the US. This study can aid decision-makers in making optimal choices.Copyright © 2024 Luo, Cai, Wu, Li, Wang and Ma.