酶替代疗法在中国法布雷病患者肾脏并发症中的成本效用分析  

Cost-effectiveness analysis of enzyme replacement therapy for renal complications in Chinese patients with Fabry disease

作　　者：陈登云 郑斌[1] 刘茂柏[1] CHEN Dengyun;ZHENG Bin;LIU Maobai(Department of Pharmacy,Fujian Medical University Union Hos-pital,Fujian Fuzhou 350001,China;Department of Pharmacy,Pingtan Comprehensive Experimental Area Hospital,Fujian Pingtan 350400,China)

机构地区：[1]福建医科大学附属协和医院药学部,福建福州350001 [2]平潭综合实验区医院药剂科,福建平潭350400

出　　处：《中国医院药学杂志》2025年第5期503-509,共7页Chinese Journal of Hospital Pharmacy

摘　　要：目的:比较中国人群中酶替代疗法(enzyme replacement therapy,ERT)与常规对症治疗在法布雷病患者肾脏并发症中的经济性。方法:从中国医疗卫生体系角度,基于中国人群回顾性队列研究的个体患者数据,采用匹配调整间接比较法调整患者基线特征,间接比较两种方案的临床疗效。通过构建Markov模型,模拟10年内法布雷病患者的肾病健康状况变化,转移概率数据基于中国人群的个体随访数据,效用值数据来源于文献,成本数据参考相关文献和中国某省省属公立医疗机构收费标准,主要的结果指标是终末期肾损伤,以2023年3倍国内生产总值作为意愿支付(willingness to pay,WTP)阈值进行成本效用分析,同时进行单因素敏感性分析、概率敏感性分析和情境分析来验证结果的稳健性。结果:相比常规对症治疗,ERT组患者获得的质量调整生命年(quality-adjusted life-years,QALYs)为0.7,增量成本为2311405.4元,增量成本效用比为3314875.70元/QALY,显著高于WTP阈值(268074元/QALY)。当阿加糖酶α的药品价格下降至37891.266元时,得到ICER≤WTP,则认为ERT具有经济性。单因素敏感性分析、概率敏感性分析和情境分析验证了结果的稳健性。结论:该研究强调了优化ERT治疗策略和降低治疗成本的重要性,并为医保决策提供参考依据,以制定更合理的医疗保障政策。OBJECTIVE To compare the cost-effectiveness of enzyme replacement therapy(ERT)versus standard medical care for Chinese patients with Fabry disease.METHODS From the perspective of the Chinese healthcare system,the clinical efficacy of the two treatment regimens was indirectly compared based on individual patient data of a retrospective cohort study in the Chinese population.The matching adjusted indirect comparison method was used to adjust the baseline characteristics of patients to indirectly compare the clinical efficacy of the two regimens.A Markov model was constructed to simulate the 10-year progression of kidney disease in patients with Fabry disease.Transition probabilities were derived from individual follow-up data in the Chinese population.Meanwhile,utility values were sourced from the literatures,and cost data were based on relevant stud⁃ies and fee schedules from provincial public healthcare institutions in China.The primary outcome was the end-stage renal injury.Cost-utility analysis was conducted using three times the 2023 gross domestic product(GDP)per capita as the willingness to pay(WTP)threshold.One-way sensitivity analysis,probabilistic sensitivity analysis,and scenario analysis were performed to vali⁃date the robustness of the results.RESULTS Compared to standard medical care,ERT provided an additional 0.7 quality-adjusted life years(QALYs),with an incremental cost of 2311405.40 RMB.This resulted in an incremental cost-effectiveness ratio(ICER)of 3314875.70 RMB/QALY that significantly exceeded the WTP threshold of 268074 RMB/QALY.When the price of agalsidase alfa was reduced to 37891.266 RMB,the ICER fell below or equaled the WTP threshold,making ERT cost-effective.The robustness of the results was confirmed through one-way sensitivity analysis,probabilistic sensitivity analysis,and scenario analysis.CONCLUSION This study emphasizes the importance of optimizing ERT strategies and reducing treatment costs,and provides a reference for medical insurance decision-making to formulate more ratio

关 键 词：法布雷病 酶替代疗法 匹配调整间接比较 成本效用分析 

分 类 号：R956[医药卫生—药学]