司美替尼治疗中国3~18岁Ⅰ型神经纤维瘤中有症状、不能手术的丛状神经纤维瘤儿童患者的成本效用分析  

Cost-effectiveness analysis of selumetinib in the treatment of symptomatic and inoperable plexiform neurofibromas associated with neurofibromatosis type 1 in Chinese children aged 3–18 years

作　　者：周琬忻 杨盟雨 常峰[1] 路云[1] 朱语眉 ZHOU Wanxin;YANG Mengyu;CHANG Feng;LU Yun;ZHU Yumei(School of International Pharmaceutical Business,China Pharmaceutical University,Jiangsu Nanjing 211198,China)

机构地区：[1]中国药科大学国际医药商学院,江苏南京211198

出　　处：《中国医院药学杂志》2025年第2期181-187,共7页Chinese Journal of Hospital Pharmacy

摘　　要：目的:评价司美替尼治疗中国3~18岁Ⅰ型神经纤维瘤(neurofibromatosis type 1,NF1)中有症状、不能手术的丛状神经纤维瘤(plexiform neurofibroma,PN)儿童患者的经济性。方法:分别从全社会和中国卫生体系2个视角出发,基于SPRINTⅡ期临床试验(NCT01362803)构建分区生存(partitioned survival model,PSM)模型,进行成本效用分析,比较司美替尼和疾病自然进展(最佳支持治疗)治疗中国3~18岁NF1中有症状、不能手术的PN儿童患者的健康结果和疾病经济负担,并对关键参数进行情境分析和敏感性分析。结果:基础分析结果显示,与疾病自然进展相比,全社会视角下,司美替尼方案的增量效果为4.91质量调整生命年(QALYs),增量费用为256567元,增量成本效果比(ICER)为52223元/QALY;中国卫生体系视角下的增量效果为3.73 QALYs,增量费用为290891元,ICER为77929元/QALY。以1倍2023年中国人均国内生产总值(GDP)为意愿支付(WTP)时,两视角下司美替尼均具有经济性;情境分析结果与基础分析一致。两视角下单因素敏感性分析结果显示,对ICER影响最大的因素均是年贴现率;在WTP阈值为1倍2023年中国人均GDP的情况下,司美替尼具有经济性的概率均大于疾病自然进展。结论:对于中国3~18岁NF1中有症状、不能手术的PN儿童患者来说,和疾病自然进展相比,司美替尼更具成本效用,是一种十分经济的药物治疗方案。OBJECTIVE To evaluate the cost-effectiveness of Selumetinib in the treatment of symptomatic and inoperable plexiform neurofibromas(PN) associated with neurofibromatosis type 1(NF1) in Chinese children aged 3 – 18 years.METHODS From the two perspectives of the whole society and Chinese health system,a partitioned survival model(PSM) model was constructed based on the SPRINT phase Ⅱ clinical trial(NCT01362803) to perform a cost-effectiveness analysis of health outcomes and economic burden of disease in Chinese children aged 3–18 years with NF1-PN who were treated with Selumetinib versus natural progression(optimal supportive care).Scenario analysis and sensitivity analysis were performed on key parameters.RESULTS The baseline analysis revealed that,compared with natural progression of NF1-PN,the incremental effects of the Selumetinib were 4.91 quality-adjusted life years(QALYs),incremental cost of ￥256 567,and incremental costeffectiveness ratio of 52 223 yuan/QALY from the perspectives of whole society.From the perspectives of Chinese health system,the incremental effects of the Selumetinib were 3.73 QALYs,incremental cost of ￥290 891,and incremental costeffectiveness ratio of 77 929 yuan/QALY.When taking one time of China's per capita gross domestic product(GDP) in 2023 as the willingness to pay(WTP),Selumetinib was cost-effective from the both perspectives.The results of the situational analysis were consistent with the basic analysis.Univariate sensitive analysis from the both perspectives showed that the annual discount rate made the greatest influence on ICER.When the WTP threshold is one time of China's per capita GDP in 2023,the economic probability of Selumetinib was greater than the natural progression of disease.CONCLUSION For symptomatic and inoperable NF1-PN children aged 3–18 years in China,Selumetinib is a very economical drug treatment option than the natural progression.

关 键 词：司美替尼 丛状神经纤维瘤 Ⅰ型神经纤维瘤 罕见病 成本效果分析 

分 类 号：R979.1[医药卫生—药品]