Institute of Health and Welfare Policy, National Yang Ming Chiao Tung University, Taipei, Taiwan.
Clinical Informatics and Medical Statistics Research Center, Chang Gung University, Taoyuan, Taiwan.
BMC Cancer. 2024 May 22;24(1):622. doi: 10.1186/s12885-024-12362-y.
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.
国际指南推荐对于存在异柠檬酸脱氢酶 1（IDH1）突变的晚期肝内胆管癌（ICC）患者，采用ivosidenib 联合改良 FOLFOX（mFOLFOX）方案进行治疗。然而，台湾地区的全民健康保险仅覆盖氟尿嘧啶/亚叶酸（5-FU/LV）化疗用于 ICC 这一适应证，此前尚未对ivosidenib 进行过经济性评价。因此，我们旨在评估与 mFOLFOX 或 5-FU/LV 相比，ivosidenib 用于既往治疗的 IDH1 突变的晚期 ICC 患者的成本效果。
采用三状态分区生存模型，以 3%的贴现率评估 10 年的成本效果，设定意愿支付阈值为 2022 年人均国内生产总值的 3 倍。Ivosidenib、mFOLFOX 和 5-FU/LV 的疗效数据分别来源于 ClarIDHy、ABC06 和 NIFTY 试验。假设 ivosidenib 的价格为新台币 10,402 元/500mg。主要结局包括增量成本效果比（ICER）和净货币收益。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，除非将其价格降低至新台币 4,161 元和新台币 5,201 元/500mg，分别与 mFOLFOX 和 5-FU/LV 相比，否则 ivosidenib 不太可能具有成本效果。DSA 强调了 ivosidenib 的成本和效用值对估计不确定性的显著影响。
在新台币 10,402 元/500mg 的价格下，与 mFOLFOX 或 5-FU/LV 相比，ivosidenib 对 IDH1 突变型 ICC 患者不具有成本效果，这表明需要将 ivosidenib 的价格降低 50%-60%，才能使其在该患者人群中具有成本效果。
