国际指南建议使用 ivosidenib 后联合改良 FOLFOX (mFOLFOX) 治疗具有异柠檬酸脱氢酶 1 (IDH1) 突变的晚期肝内胆管癌 (ICC)。台湾国民健康保险仅承保该 ICC 组的氟尿嘧啶/甲酰四氢叶酸 (5-FU/LV) 化疗，并且之前没有对 ivosidenib 进行过经济评估。因此，我们的目的是评估 ivosidenib 与 mFOLFOX 或 5-FU/LV 相比，在既往治疗的晚期 ICC 呈现 IDH1 突变中的成本效益。采用三态分区生存模型来评估 ivosidenib 10 年的成本效益3%的贴现率，将支付意愿门槛设定为2022年人均GDP的3倍。 Ivosidenib、mFOLFOX 和 5-FU/LV 的疗效数据分别来自 ClarIDHy、ABC06 和 NIFTY 试验。 Ivosidenib 的成本假设为新台币 10,402 元/500 毫克。主要成果包括增量成本效益比（ICER）和净货币效益。采用确定性敏感性分析（DSA）和概率敏感性分析（PSA）来评估不确定性并探索降价方案。与 mFOLFOX 和 5-FU/LV 相比，Ivosidenib 的 ICER 分别为新台币 6,268,528 元和新台币 5,670,555 元，均超过既定阈值。 PSA 显示，ivosidenib 不太可能具有成本效益，除非与 mFOLFOX 和 5-FU/LV 相比，其分别降低至 NT$4,161 和 NT$5,201/500 mg。 DSA 强调了 ivosidenib 的成本和效用值对估计不确定性的显着影响。与 mFOLFOX 或 5-FU/LV 相比，ivosidenib 对于 IDH1 突变 ICC 患者的成本效益为 NT$10,402/500 mg，表明 50-60 % 为了使 ivosidenib 在该患者群体中具有成本效益，有必要降价。© 2024。作者。

International guidelines recommend ivosidenib followed by modified FOLFOX (mFOLFOX) for advanced intrahepatic cholangiocarcinoma (ICC) with isocitrate dehydrogenase 1 (IDH1) mutations. Taiwan National Health Insurance covers only fluorouracil/leucovorin (5-FU/LV) chemotherapy for this ICC group, and there has been no prior economic evaluation of ivosidenib. Therefore, we aimed to assess ivosidenib's cost-effectiveness in previously treated, advanced ICC-presenting IDH1 mutations compared with mFOLFOX or 5-FU/LV.A 3-state partitioned survival model was employed to assess ivosidenib's cost-effectiveness over a 10-year horizon with a 3% discount rate, setting the willingness-to-pay threshold at 3 times the 2022 GDP per capita. Efficacy data for Ivosidenib, mFOLFOX, and 5-FU/LV were sourced from the ClarIDHy, ABC06, and NIFTY trials, respectively. Ivosidenib's cost was assumed to be NT$10,402/500 mg. Primary outcomes included incremental cost-effectiveness ratios (ICERs) and net monetary benefit. Deterministic sensitivity analyses (DSA) and probabilistic sensitivity analyses (PSA) were employed to evaluate uncertainty and explore price reduction scenarios.Ivosidenib exhibited ICERs of NT$6,268,528 and NT$5,670,555 compared with mFOLFOX and 5-FU/LV, respectively, both exceeding the established threshold. PSA revealed that ivosidenib was unlikely to be cost-effective, except when it was reduced to NT$4,161 and NT$5,201/500 mg when compared with mFOLFOX and 5-FU/LV, respectively. DSA underscored the significant influence of ivosidenib's cost and utility values on estimate uncertainty.At NT$10,402/500 mg, ivosidenib was not cost-effective for IDH1-mutant ICC patients compared with mFOLFOX or 5-FU/LV, indicating that a 50-60% price reduction is necessary for ivosidenib to be cost-effective in this patient group.© 2024. The Author(s).