瑞戈非尼治疗经索拉非尼治疗失败后的肝细胞癌的经济性

Cost-effectiveness of regorafenib for hepatocellular carcinoma after failure of sorafenib

目的 从我国卫生体系角度出发,评估瑞戈非尼治疗经索拉非尼治疗失败后的肝细胞癌的经济性。方法 基于一项Ⅲ期临床试验(RESORCE研究),分别构建分区生存模型(PSM)和Markov模型,循环周期设为4周,研究时限为患者终身,年贴现率为5%,药品成本数据来自药智网,其他成本数据来源于安徽省医保局数据和相关文献,效用数据来源于文献。以增量成本-效果比(ICER)为评价指标,意愿支付值为3倍的我国2022年人均国内生产总值(GDP),采用单因素敏感性分析和概率敏感性分析验证基础分析结果的稳健性。结果 瑞戈非尼组对比安慰剂组在PSM和Markov模型中的增量成本分别为112 116.95元、96 617.19元;增量效果分别为0.31、0.32 QALYs;ICER分别为360 751.01、301 114.45元/QALY,ICER均大于意愿支付值,瑞戈非尼不具有经济性。单因素敏感性分析结果显示,无进展生存期状态和疾病进展状态的效用值以及瑞戈非尼单价对结果的影响较大,但在各参数浮动范围内,ICER始终大于意愿支付值;在3倍的我国2022年人均GDP的意愿支付值下,瑞戈非尼具有经济性的概率为0.8%(PSM)、11.4%(Markov模型)。结论 在3倍的我国人均GDP下,瑞戈非尼对比安慰剂治疗经索拉非尼治疗失败后的肝细胞癌不具有经济性。

OBJECTIVE To evaluate the cost-effectiveness of regorafenib in the treatment of hepatocellular carcinoma after failure of sorafenib from the perspective of Chinese health system.METHODS Based on a phaseⅢtrial(RESORCE),the partition survival model(PSM)and Markov model were constructed.The cycle was set as four weeks,the duration of the study lasted for lifetime,the annual discount rate was 5%.Drug cost data was obtained from yaozhi.com,other cost data were obtained from Anhui Provincial Medical Insurance Bureau and related literature,and utility values were obtained from literature.The incremental cost-effectiveness ratio(ICER)was used as the evaluation index,and the value of willingness to pay(WTP)was three times of China’s gross domestic product(GDP)per capita in 2022;one-way sensitivity analysis and probabilistic sensitivity analysis were used to verify the robustness of the basic analysis results.RESULTS The incremental cost of regorafenib group versus placebo group in PSM and Markov model was 112116.95 yuan and 96617.19 yuan,respectively.The incremental effectiveness was 0.31 QALYs and 0.32 QALYs,respectively.The ICERs were 360751.01 yuan/QALY and 301114.45 yuan/QALY,which were both greater than the value of WTP;regorafenib was not cost-effective.Results of one-way sensitivity analysis showed that the utility of progression-free survival and progressive disease,the unit cost of regorafenib had the greatest influence on the results,but ICER was always greater than the WTP within the floating range of each parameter.Under the WTP of 3 times China’s per capita GDP in 2022,the probabilities of regorafenib with cost-effectiveness were 0.8%(PSM)and 11.4%(Markov).CONCLUSIONS Under the WTP of 3 times the per capita GDP of China,regorafenib is not cost-effective in the treatment of hepatocellular carcinoma after failure of sorafenib treatment,compared with placebo.