2022年12月，台湾国家健康保险管理局（NHIA）宣布，为治疗具有纤维母细胞生长因子受体2（FGFR2）融合/重排的晚期肝内胆管癌（ICC），将对三种剂量的帕米吉替尼（4.5毫克、9毫克和13.5毫克）进行报销，并将帕米吉替尼4.5毫克的报销价格设定为6600新台币。 本研究旨在从台湾国家健保局的角度，分析13.5毫克帕米吉替尼作为二线治疗与mFOLFOX和5-FU化疗相比，对具有FGFR2融合/重排的晚期ICC患者的成本效益。本研究采用3状态分区生存模型，分析对于首线含紫嘌呤的化疗失败的ICC患者而言，帕米吉替尼作为二线治疗的5年成本效益，并将结果与mFOLFOX和5-FU化疗方案进行比较。整体生存和无进展生存来自FIGHT-202试验（帕米吉替尼）、ABC-06试验（mFOLFOX）和NIFTY试验（5-FU）。帕米吉替尼13.5mg的价格设定为潜在的最高列示价格（17,820新台币）。与ICC相关的效用、不效用和成本参数来自已发表的文献。愿意支付阈值为2022年预测人均国内生产总值的3倍（2,928,570新台币）。质量调整的生命年（QALY）和成本应用了3%的折现率。进行了几种情景分析，包括帕米吉替尼价格逐渐降低的情况。进行了确定性敏感性分析、概率敏感性分析（PSA）和信息价值评估以评估不确定性。在基准情景分析中，与mFOLFOX或5-FU相比，帕米吉替尼在成本效益方面并不划算。当帕米吉替尼的价格降低了50％或更多时，帕米吉替尼获得了正面的净经济效益（mFOLFOX：55,374新台币；5-FU：92,437新台币）以及72％（mFOLFOX）和77.1％（5-FU）的成本效益可能性。大部分的不确定性来自帕米吉替尼的药物费用、健康状态效用以及与帕米吉替尼相关的整体生存期。根据NCCN指南，在假定的NHIA价格（17,820新台币/13.5mg）下，每天使用13.5mg帕米吉替尼与mFOLFOX或5-FU相比并不具备成本效益。价格降低的情景建议，针对具有FGFR2融合/重排的晚期ICC患者，13.5mg帕米吉替尼的价格应降低50%，即8910新台币。© 2023. BioMed Central Ltd., part of Springer Nature.

In December 2022, the Taiwan National Health Insurance Administration (NHIA) announced the reimbursement of three dosages of pemigatinib 4.5 mg, 9 mg, and 13.5 mg for treating advanced intrahepatic cholangiocarcinoma (ICC) with fibroblast growth factor receptor 2 (FGFR2) fusions/rearrangements and set the reimbursement price for pemigatinib 4.5 mg at NT$6600. This study aims to analyze the cost-effectiveness of pemigatinib 13.5 mg as a second-line treatment compared to mFOLFOX and 5-FU chemotherapy for advanced ICC patients with FGFR2 fusions/rearrangements from the perspective of Taiwan's NHIA.This study used a 3-state partitioned survival model to analyze the 5 year cost-effectiveness of pemigatinib as a second-line treatment for advanced ICC patients in whom first-line gemcitabine-based chemotherapy failed and to compare the results with those for the mFOLFOX and 5-FU chemotherapy regimens. Overall survival and progression-free survival were estimated from the FIGHT-202 trial (pemigatinib), ABC-06 trial (mFOLFOX), and NIFTY trial (5-FU). The price of pemigatinib 13.5 mg was set at the potentially highest listing price (NT$17,820). Other parameters of utility, disutility, and costs related to advanced ICC were obtained from the published literature. The willingness-to-pay threshold was three times the forecasted gross domestic product per capita in 2022 (NT$2,928,570). A 3% discount rate was applied to quality-adjusted life-years (QALYs) and costs. Several scenario analyses were performed, including a gradual price reduction for pemigatinib. Deterministic sensitivity analysis, probabilistic sensitivity analysis (PSA), and value of information were performed to assess uncertainty.Pemigatinib was not cost-effective compared to mFOLFOX or 5-FU in the base-case analysis. When the price of pemigatinib was reduced by 50% or more, pemigatinib gained a positive net monetary benefit (mFOLFOX: NT$55,374; 5-FU: NT$92,437) and a 72% (mFOLFOX) and 77.1% (5-FU) probability of being cost-effective. Most of the uncertainty came from the medication cost of pemigatinib, health state utility, and the overall survival associated with pemigatinib.According to the NCCN guidelines, the daily use of pemigatinib 13.5 mg at the hypothesized NHIA price of NT$17,820/13.5 mg was not cost-effective compared to mFOLFOX or 5-FU. The price reduction scenario suggested a 50% price reduction, NT$8910 per 13.5 mg, for advanced ICC patients with FGFR2 fusions/rearrangements.© 2023. BioMed Central Ltd., part of Springer Nature.