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Background <p>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 <i>FGFR2</i> 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 <i>FGFR2</i> fusions/rearrangements from the perspective of the Taiwanese healthcare payer.</p> Methods <p>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.</p> Results <p>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.</p> Conclusion <p>Pemigatinib is cost-effective with updated long-term benefits from the pivotal trial in the lifetime simulation for advanced patients with advanced ICC and <i>FGFR2</i> 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.</p>

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

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2026年2月10日

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  • I-Ting Wang,
  • Hsiao-Lin Chen,
  • Wei-Ming Huang,
  • Nai-Jung Chiang,
  • Li-Jiuan Shen,
  • Chen-Han Chueh,
  • Yi-Wen Tsai

摘要

Background

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.

Methods

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.

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.

Conclusion

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.