研究概要
相对于标准治疗,cilta-cel 预计在治疗来那度胺难治性多发性骨髓瘤中展现出经济上的绝对成本效益优势。
研究思路结论见上方概要
目的
基于III期CARTITUDE-4试验中显示的生存优势,本研究从美国混合支付方角度量化了ciltacabtagene autoleucel(cilta-cel)与标准方案(PVd或DPd)用于来那度胺难治性多发性骨髓瘤的成本效益。
方法
开发了一个周期为3或4周的Markov模型,用以模拟长期临床轨迹。通过整合成本和质量调整生命年(QALYs)计算增量成本效果比(ICERs),并以150,000美元/QALY的支付意愿(WTP)阈值作为基准,判定其经济可行性。通过敏感性和情景分析验证了稳健性。
结果
在10年时间范围内,cilta-cel队列累计获得3.99个预期QALYs,而标准治疗组为2.96个QALYs。同时,cilta-cel组的预期总医疗费用为$574,425.45,低于标准治疗组的$645,495.60。与传统治疗相比,cilta-cel方案显示出经济学绝对优势,在获得更优健康结局的同时降低了总体成本。情景分析和敏感性分析证实了模型的稳健性。cilta-cel的采购成本、标准组的后续治疗成本以及贴现率被确定为三个影响最大的因素。
展开英文摘要原文
OBJECTIVE
Building upon the survival advantages demonstrated in the Phase III CARTITUDE-4 trial, this study quantifies the cost-effectiveness of ciltacabtagene autoleucel (cilta-cel) versus standard regimens (PVd or DPd) for lenalidomide-refractory multiple myeloma from a US mixed payer perspective.
METHODS
A Markov model with 3 or 4-week cycles was developed to simulate long-term clinical trajectories. The economic viability was determined by calculating incremental cost-effectiveness ratios (ICERs) through integrated costs and quality-adjusted life years (QALYs), benchmarked against a willingness-to-pay (WTP) threshold of $150,000/QALY. Robustness was verified via sensitivity and scenario analyses.
RESULTS
Under the 10-year time horizon, patients in the cilta-cel cohort accumulated an expected 3.99 QALYs, compared with 2.96 QALYs in the standard-of-care group. Concurrently, the expected total medical costs for the cilta-cel group were $574,425.45, which were lower than the $645,495.60 incurred by the standard therapy group. Compared with conventional treatment, the cilta-cel regimen demonstrated economic absolute dominance, achieving superior health outcomes alongside reduced overall costs. Scenario and sensitivity analyses confirmed the robustness of the model. The acquisition cost of cilta-cel, the subsequent treatment costs in the standard group and the discount rate were identified as the three most influential factors.
CONCLUSIONS
Relative to standard therapy, cilta-cel is projected to exhibit an economic absolute cost-effectiveness advantage in the treatment of lenalidomide-refractory multiple myeloma.
论文信息
- 作者
- Zhang Y、Li Y、Chen J、Sun G、Zhang Y
- 第一作者单位
- College of Pharmacy, Zhejiang University of Technology, Hangzhou, Zhejiang Province, China.China
- 通讯作者单位
- College of Pharmacy, Zhejiang University of Technology, Hangzhou, Zhejiang Province, 313100 China. Electronic address: oksgj@163.com.China
- 期刊
- Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research2026 Sep 11