OBJECTIVES: The National Comprehensive Cancer Network recommends a second-line treatment of pemigatinib for intrahepatic cholangiocarcinoma (ICC) with fibroblast growth factor receptor 2 (FGFR2) fusion/rearrangement, and modified FOLFOX (mFOLFOX) for those without FGFR2 alternations. However, these regimens are not yet under the coverage of Taiwan's National Health Insurance (NHI). This study evaluated the cost-effectiveness of pemigatinib/mFOLFOX and provided pemigatinib pricing reference as the second-line treatment for advanced ICC based on FGFR2 status in comparison with 5-FU.

METHODS: A 3-state partitioned survival model with a 5-year time horizon was constructed for 1,000 hypothetical advanced ICC patients who failed the first-line therapy. Overall and progression-free survival of pemigatinib, mFOLFOX, and 5-FU were estimated from the FIGHT-202, ABC-06, and NIFTY trials, respectively. Utility of health states and disutility of adverse events were obtained from the literature. Medical costs related to advanced ICC were calculated using NHI claim data. The willingness-to-pay threshold was three times the GDP in 2021 (NT$2,889,684). A 3% discount rate was applied to quality-adjusted life-years (QALYs) and costs. Scenario analyes included gradual price reduction of pemigatinib, alternative survival models, and parameter distributions. One-way sensitivity and probabilistic sensitivity analysis (PSA) were performed.

RESULTS: The new regimen provided an incremental 0.13 QALY, with incremental costs of NT$459,697, resulting in an incremental cost-effectiveness ratio (ICER) of 3,411,098 per QALY, which was found to be 55.8% cost-effective in PSA. In scenario analyses, the expected incremental net monetary benefit (INMB) was positive when the price of pemigatinib was reduced by 30% or more. The parameter uncertainty was mainly due to the utility of the progression-free state and the costs of the new regimen.

CONCLUSIONS: The new regimen with pemigatinib is likely to be cost-effective and will plausibly gain a positive INMB when the cost of pemigatinib is reduced by more than 30%.