卡非佐米不同剂量方案治疗复发/难治多发性骨髓瘤的经济学评价

Economic Evaluation of Different Dosing Regimens of Carfilzomib for the Treatment of Relapsed/Refractory Multiple Myeloma

关键词:  卡非佐米  复发/难治多发性骨髓瘤  Markov 模型  成本-效用分析

Key words:  carfilzomib  relapsed/refractory multiple myeloma  markov model  Cost-Utility Analysis

摘要:

摘要：目的 从中国卫生体系角度评价卡非佐米不同剂量方案治疗复发/难治多发性骨髓瘤的经济性。方法 利用临床试验数据和相关文献数据构建包含肿瘤“无进展”、“进展”、“死亡”三状态的Markov模型。循环周期为1月，模拟RRMM患者10年内的疾病进展状况，成本和效用数据均采用5%的贴现率进行贴现处理。以增量成本-效用比（Incremental cost-utility ratio，ICUR）为评价指标，意愿支付阈值（willingness-to-pay，WTP）设为 2024 年中国1~3 倍人均国内生产总值(gross domestic product，GDP)(95 749 元~ 287 247元)，并采用单因素敏感性分析评价模型参数变化时模型结果的稳定性。结果 基础分析结果显示，Kd27方案和Kd70方案属于高成本高健康获益方案，与Kd56方案相比的ICUR值分别为Kd27：38 341.25元/QALYs，Kd70：46 889.94元/QALYs。单因素敏感性分析结果表明疾病无进展生存期（PFS）效用值是影响整体治疗经济性结果的关键因素，但不足以翻转结果。结论 研究表明，在中国医保体系下，卡非佐米不同剂量方案治疗复发/难治多发性骨髓瘤，与Kd56方案相比，Kd27方案、Kd70方案有效性和经济性具有一定优势，且Kd27方案成本效用最佳。患者可根据自身需要和医生建议选择合适治疗方案。

Abstract:

ABSTRACT: OBJECTIVE To evaluate the economics of different dosage regimens of carfilzomib in the treatment of relapsed/refractory multiple myeloma (RRMM) from the perspective of the Chinese healthcare system. METHODS A Markov model encompassing three health states—"progression-free," "progressed," and "dead"—was constructed using clinical trial data and relevant literature. The cycle length was set at one month, simulating the disease progression of RRMM patients over a 10-year period. Cost and utility data were discounted at a rate of 5%. The incremental cost-utility ratio (ICUR) was used as the evaluation metric, with the willingness-to-pay (WTP) threshold set at 1-3 times the per capita gross domestic product (GDP) of China in 2024 (ranging from 95,749 yuan to 287,247 yuan). One-way sensitivity analysis was conducted to assess the stability of the model results when parameters were varied. RESULTS The base-case analysis revealed that both the Kd27 and Kd70 regimens were associated with higher costs and greater health benefits compared to the Kd56 regimen. The ICUR values for Kd27 and Kd70 compared to Kd56 were 38,341.25 yuan/QALYs and 46,889.94 yuan/QALYs, respectively. One-way sensitivity analysis indicated that the utility value during progression-free survival (PFS) was a key factor influencing the overall economic evaluation results, but it was not sufficient to reverse the findings. CONCLUSION The study demonstrated that, within the Chinese healthcare insurance system, the Kd27 and Kd70 regimens of carfilzomib for treating RRMM offer certain advantages in terms of effectiveness and economics compared to the Kd56 regimen. Specifically, the Kd27 regimen exhibits the best cost-utility. Patients may choose an appropriate treatment regimen based on their individual needs and physician recommendations.