Wei-Ming Huang

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.

Aims: Predetermined treatment duration limits (PTDLs) are often used by Taiwan's National Health Insurance Administration to contain healthcare costs, but they may compromise patient outcomes. Therefore, we studied Taiwan's 2017 extension of the bevacizumab PTDL from 24 to 36 weeks in metastatic colorectal cancer (mCRC) to evaluate whether prolonged treatment duration improves clinical outcomes, specifically treatment continuity, therapy duration and survival. Methods: We identified two cohorts based on initial bevacizumab prescription dates from the Taiwan Health and Welfare Database: PTDL‐24 weeks (1 April 2013–31 March 2016; n = 2051) and PTDL‐36 weeks (1 April 2017–31 March 2020; n = 2484). We assessed overall survival (OS), progression‐free survival (PFS) and time on treatment (ToT) for first‐line therapy over 2 years. A doubly robust approach was applied, including inverse probability of treatment weighting (IPTW) to balance covariates and Cox proportional hazards models adjusted for calendar year. A two‐stage residual inclusion (2‐SRI) model estimated the impact of the policy on bevacizumab duration and subsequent survival outcomes. Results: After IPTW adjustment, baseline characteristics were well‐balanced. The 36‐week PTDL policy was associated with improved PFS (adjusted hazard ratio: 0.814 [95% confidence interval: 0.691–0.959]) and ToT (0.821 [0.703–0.959]), but not OS (0.926 [0.751–1.142]). The 2‐SRI analysis showed the policy extended bevacizumab treatment by approximately 8 weeks and was associated with improvements in OS (0.912 [0.89–0.934]), PFS (0.941 [0.923–0.959]) and ToT (0.966 [0.95–0.984]). Conclusions: Relaxing the PTDL can extend bevacizumab treatment exposure, improving PFS and ToT.