Wan Junjie, Xu Yizhou, Wan Bin, Ding Haixia
Department of Pharmaceutical Regulatory Science and Pharmacoeconomics, School of Pharmacy, Nanjing Medical University, Nanjing, China.
Department of Pharmacy, The First Affiliated Hospital with Nanjing Medical University, Nanjing, China.
Int J Clin Pharm. 2025 Jul 24. doi: 10.1007/s11096-025-01968-2.
Benmelstobart and anlotinib plus etoposide-carboplatin (EC) group has demonstrated substantial clinical efficacy in improving survival outcomes for patients with extensive-stage small-cell lung cancer (ES-SCLC). However, the high treatment cost raises concerns regarding its affordability and cost-effectiveness across healthcare systems with heterogeneous pricing and reimbursement mechanisms.
This study aimed to evaluate the cost-effectiveness of benmelstobart and anlotinib plus EC group compared to EC alone group and anlotinib plus EC group from both US and Chinese payer perspectives. The findings are intended to inform value-based pricing strategies and evidence-based reimbursement decision-making.
A partitioned survival model (PSM) with a lifetime horizon and 21-day cycles was constructed using clinical data from the ETER701 trial. Direct medical costs and health utility inputs were obtained from national databases, local hospitals, and published literature. The primary outcome was the incremental cost-effectiveness ratio (ICER), calculated by comparing costs and quality-adjusted life years (QALYs) between treatment strategies. Scenario analyses, including drug price simulations and deterministic and probabilistic sensitivity analyses, were conducted to evaluate model robustness. Willingness-to-pay (WTP) thresholds were set at $100,000/QALY and $150,000/QALY (US) and $40,011/QALY (China).
In the US, the benmelstobart and anlotinib plus EC group yielded ICER of $121,560.40/QALY versus EC alone group and $127,579.09/QALY versus anlotinib plus EC group, both below the $150,000/QALY threshold. However, at the $100,000/QALY threshold, cost-effectiveness would require reducing benmelstobart's price to $1316.12/600 mg. In China, the ICER of $117,667.17/QALY exceeded the local threshold. Price simulations suggested that cost-effectiveness could be achieved if prices were reduced below $2230.60/600 mg (US) and $328.47/600 mg (China). Sensitivity analyses identified progression-free survival (PFS) utility and benmelstobart pricing as major cost drivers. Probabilistic analysis indicated a 75.1% probability of cost-effectiveness at $150,000/QALY in the US. However, the probability of cost-effectiveness is 0% at WTP thresholds of $100,000/QALY in the US and $40,011/QALY in China.
Benmelstobart plus anlotinib and EC group is likely to be cost-effective in the US at a WTP threshold of $150,000/QALY, but not in China at current prices. An 80% price reduction in China would be necessary to align with its WTP threshold, emphasizing the need for policy interventions in drug pricing and reimbursement to improve patient access.
苯美司托巴特联合安罗替尼加依托泊苷-卡铂（EC）方案已在广泛期小细胞肺癌（ES-SCLC）患者的生存结局改善方面展现出显著的临床疗效。然而，高昂的治疗成本引发了人们对其在定价和报销机制各异的医疗体系中的可承受性及成本效益的担忧。
本研究旨在从美国和中国支付方的角度，评估苯美司托巴特联合安罗替尼加EC方案与单纯EC方案及安罗替尼加EC方案相比的成本效益。研究结果旨在为基于价值的定价策略和循证报销决策提供参考。
使用来自ETER701试验的临床数据构建了一个具有终身时间范围和21天周期的分区生存模型（PSM）。直接医疗成本和健康效用数据来自国家数据库、当地医院及已发表的文献。主要结局为增量成本效益比（ICER），通过比较各治疗策略之间的成本和质量调整生命年（QALY）来计算。进行了情景分析，包括药品价格模拟以及确定性和概率性敏感性分析，以评估模型的稳健性。支付意愿（WTP）阈值设定为100,000美元/QALY和150,000美元/QALY（美国）以及40,011美元/QALY（中国）。
在美国，苯美司托巴特联合安罗替尼加EC方案与单纯EC方案相比的ICER为121,560.40美元/QALY，与安罗替尼加EC方案相比为127,579.09美元/QALY，均低于150,000美元/QALY的阈值。然而，在100,000美元/QALY的阈值下，要实现成本效益则需将苯美司托巴特的价格降至1316.12美元/600毫克。在中国，ICER为117,667.17美元/QALY，超过了当地阈值。价格模拟表明，如果价格降至2230.60美元/600毫克（美国）和328.47美元/600毫克（中国）以下，则可实现成本效益。敏感性分析确定无进展生存期（PFS）效用和苯美司托巴特定价为主要成本驱动因素。概率分析表明，在美国150,000美元/QALY的WTP阈值下，成本效益的概率为75.1%。然而，在美国100,000美元/QALY和中国40,011美元/QALY的WTP阈值下，成本效益的概率为0%。
在美国，苯美司托巴特联合安罗替尼和EC方案在150,000美元/QALY的WTP阈值下可能具有成本效益，但按当前价格在中国则不然。在中国，要达到其WTP阈值需要降价80%，这凸显了在药品定价和报销方面进行政策干预以改善患者可及性的必要性。
