Battle against time for innovative cancer treatment: an updated cost-effectiveness analysis of pemigatinib in intrahepatic cholangiocarcinoma

Advanced intrahepatic cholangiocarcinoma (ICC) is a rare cancer. In 2023, Taiwan’s National Health Insurance Administration granted provisional payment for pemigatinib for patients with advanced ICC and FGFR2 fusions/rearrangements. Previous analyses of the 2020 FIGHT-202 trial found pemigatinib was not cost-effective; however, updated 2024 data showed promising long-term benefits. This study aimed to reassess the cost-effectiveness of pemigatinib for advanced ICC with FGFR2 fusions/rearrangements from the perspective of the Taiwanese healthcare payer. We used a three-state partitioned survival model to evaluate the lifetime cost-effectiveness of pemigatinib compared to mFOLFOX or 5-FU/LV, based on data from the updated FIGHT-202, ABC-06, and updated NIFTY pivotal trials. Utility, disutility, and cost parameters were derived from published sources. The primary measure was the incremental cost-effectiveness ratio (ICER). Scenario and sensitivity analyses were performed to determine the break-even year and main parameters influencing cost-effectiveness analysis results. Pemigatinib was found to be cost-effective with high certainty (mFOLFOX: ICER = US$83,475, probability = 81.4%; 5-FU/LV: ICER = US$84,386, probability = 79.9%). The updates to the FIGHT-202 and NIFTY trials significantly increased the expected value of information (mFOLFOX: US$30,405; 5-FU/LV: US$28,851). Although pemigatinib incurred higher cumulative costs initially, the break-even point was reached between 7.6 and 7.7 years. The key factors influencing the cost-effectiveness results were the use of updated FIGHT-202 trial data and a lifetime simulation horizon. Pemigatinib is cost-effective with updated long-term benefits from the pivotal trial in the lifetime simulation for advanced patients with advanced ICC and FGFR2 fusions/rearrangements. Given the rarity of advanced ICC, applying updated pivotal trial data with local real-world data for cost-effectiveness reassessment can be an efficient way under Taiwan’s Provisional Payment scheme.