Luo Xianmei, Cai Tingting, Wu Jinyan, Li Xingyu, Wang Xiaofan, Ma Haiying
Department of Pharmacy, The Fourth Affiliated Hospital of China Medical University, Shenyang, China.
School of Pharmacy, China Medical University, Shenyang, China.
Front Pharmacol. 2024 Aug 14;15:1393559. doi: 10.3389/fphar.2024.1393559. eCollection 2024.
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). 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. 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). 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.
帕博利珠单抗是一种具有潜在价值的治疗方法。然而，患者、医生和医疗保健决策者对其成本效益以及这种新疗法的合理定价并不确定。本研究旨在评估帕博利珠单抗作为中国和美国晚期胆管癌（BTC）患者一线治疗的成本效益。从中国和美国医疗保健系统的角度构建了一个马尔可夫模型用于药物经济学评估。患者基线特征和关键临床数据来自KEYNOTE - 966试验（ClinicalTrials.gov，NCT04003636）。成本和效用值从药品成本网站和已发表的文献中收集。测量并比较了累积成本（以美元计）、生命年（LYs）、质量调整生命年（QALYs）和增量成本效益比（ICERs）。在给定的支付意愿（WTP）阈值下进行价格模拟，以提供定价方案参考。通过单向敏感性分析和概率敏感性分析对模型的稳健性进行了分析。基础数据分析表明，在中国，支付意愿阈值为38,201.19美元时，帕博利珠单抗（2662.41美元/100毫克）联合化疗方案相对于化疗方案不具有成本效益，相对于化疗方案的额外成本为77,114.94美元（ICER为556,689.47美元/QALY），同时增加了0.14个QALYs。在美国，帕博利珠单抗（54.71美元/1毫克）也使疗效提高了0.14个QALYs，但在美国支付意愿阈值为229,044美元时同样不具有成本效益，总成本增加了160,425.24美元（ICER为1,109,462.92美元/QALY）。与化疗相比，帕博利珠单抗联合化疗减轻了疾病负担。然而，以其当前价格，在中国和美国它可能都不是晚期BTC的成本效益高的治疗方法。本研究有助于决策者做出最优选择。
