Yi-Wen Tsai

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.

Background: The combination of lenvatinib and pembrolizumab (LP) for advanced or recurrent endometrial carcinoma has demonstrated improved survival in a clinical study and has been endorsed by the European Society for Medical Oncology. However, the LP regimen is currently not covered by Taiwan’s National Health Insurance (NHI). This study aimed to evaluate the cost-effectiveness of LP as a second-line treatment for advanced endometrial carcinoma (aEC) under Taiwan’s NHI. Methods: A 3-state partitioned survival model was used to analyze a hypothetical population of patients with aEC over 20 years. The overall and progression-free survival curves were obtained from the updated results of the Phase III trial 309/KEYNOTE-775 and extrapolated using a hybrid method. The pricing of the LP was based on the NHI fee schedule for other cancer treatments. Health state utilities and disutilities related to adverse events were obtained from the published literature. The willingness-to-pay (WTP) threshold was three times the GDP per capita in 2022 (NT$2,917,650). Quality-adjusted life-years (QALYs) and costs were discounted at 3%. Scenario analyses included LP price reductions and time horizon adjustments. Deterministic sensitivity, probabilistic sensitivity, and value of information analyses were performed to assess the uncertainty. Results: LP provided an incremental gain of 0.92 QALYs at an incremental cost of NT$2,929,046, resulting in an incremental cost-effectiveness ratio of NT$3,197,177, which exceeded the WTP threshold. The major factors causing uncertainty were the time horizon and cost of the LP. Conclusions: LP is not a cost-effective second-line therapy for aEC unless its price is reduced by more than 20%.

Brain metastases (BM) significantly impact prognosis and healthcare utilization but remain underrepresented in economic evaluations. The IMpower133 trial demonstrated the efficacy of atezolizumab plus chemotherapy (carboplatin and etoposide), particularly among patients without baseline BM, leading to its approval for extensive-stage small-cell lung cancer (EX-SCLC) in several countries. As previous studies did not incorporate BM status, we addressed this gap by including BM in the cost-effectiveness analysis of atezolizumab for EX-SCLC. A four-state Markov model was constructed with the following health states: progression-free, progressive disease (PD) without brain metastases, PD with brain metastases, and death, over a 15-year time horizon. Time-varying efficacy and utility were derived from IMpower133; direct medical costs were estimated using population-based databases. Both deterministic and probabilistic sensitivity analyses were conducted to assess uncertainty in input parameters. Adding atezolizumab to chemotherapy increased QALYs by 0.721 at an incremental cost of NT$2,111,359, yielding an incremental net monetary benefit of NT$68,264, and a 53.22% probability of cost-effectiveness. Key sources of uncertainty include efficacy, cost of atezolizumab, time horizon, and utility. In conclusion, adding atezolizumab to chemotherapy is cost-effective for patients with EX-SCLC without baseline BM. Incorporating BM status into CEA could support more nuanced policy recommendations.

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.

Background/Objectives: Sugemalimab demonstrated clinical efficacy in the GEMSTONE-303 trial, but its cost-effectiveness remains unclear. This study aims to evaluate the cost-effectiveness of sugemalimab in combination with chemotherapy (CAPOX) as a first-line treatment for patients with advanced or metastatic gastric or gastroesophageal junction (G/GEJ) adenocarcinoma, compared to chemotherapy alone, from the perspective of Taiwan’s healthcare payer. Methods: A partitioned survival model was developed to simulate outcomes over a 40-year time horizon, and model parameters were derived from GEMSTONE-303 and the wider literature. Health benefits were measured in quality-adjusted life-years (QALYs), and only direct medical costs were included, with both discounted at an annual rate of 3%. The willingness-to-pay threshold was set at three times the 2024 GDP per capita. Deterministic and probabilistic sensitivity analyses were conducted alongside scenario analyses. Results: Compared to capecitabine and oxaliplatin (CAPOX) alone, adding sugemalimab yielded an incremental gain of 0.39 QALYs at an additional cost of USD 47,020, resulting in an incremental net monetary benefit of −USD 7478. Conclusions: Sugemalimab plus CAPOX is not cost-effective for advanced or metastatic G/GEJ adenocarcinoma from the Taiwan payer’s perspective. Achieving cost-effectiveness would require a 20–30% price reduction for sugemalimab (to USD 1204–USD 1376 per 600 mg), assuming first-line therapy is administered for the median treatment duration observed in the GEMSTONE-303 trial. If reimbursement continued until disease progression, a reduction of approximately 68% would be required (USD 550 per 600 mg).

Aim: In 2017, Taiwan’s National Health Insurance Administration (NHIA) extended the Predetermined Treatment Duration Limit (PTDL) for bevacizumab in metastatic colorectal cancer (mCRC) from 24 to 36 weeks. However, its impact on treatment continuity and survival outcomes remains unclear. In this population-based cohort study, we evaluated the effects of PTDL relaxation on survival outcomes, first-line treatment continuity, and bevacizumab therapy duration in patients with mCRC. Methods: Using the Taiwan Health and Welfare Database, we identified two cohorts based on initial bevacizumab prescription dates: PTDL-24 weeks (April 1, 2013–March 31, 2016; N = 2,051) and PTDL-36 weeks (April 1, 2017–March 31, 2020; N = 2,484). 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 time trends. 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, improving OS (0.912 [0.89–0.934]), PFS (0.941 [0.923–0.959]), and ToT (0.966 [0.95–0.984]). Conclusion: Relaxing the PTDL improved PFS and ToT by extending bevacizumab treatment exposure.

Background: Hormone therapy (HT) use among menopausal women declined after negative information from the 2002 Women’s Health Initiative (WHI) HT study. The 2017 post-intervention follow-up WHI study revealed that HT did not increase long-term mortality. However, studies on the effects of the updated WHI findings are lacking. Thus, we assessed the impact of the 2017 WHI findings on HT use in Taiwan. Methods: We identified 1,869,050 women aged 50–60 years, between June and December 2017, from health insurance claims data to compare HT use in the 3 months preceding and following September 2017. To address the limitations associated with interval-censored data, we employed an emulated repeated cross-sectional design. Using logistic regression analysis, we evaluated the impact of the 2017 WHI study on menopausal symptom-related outpatient visits and HT use. In a scenario analysis, we examined the impact of the 2002 trial on HT use to validate our study design. Results: Study participants’ baseline characteristics before and after the 2017 WHI study were not significantly different. Logistic regressions demonstrated that the 2017 study had no significant effect on outpatient visits for menopause-related symptoms or HT use among women with outpatient visits. The scenario analysis confirmed the negative impact of the 2002 WHI trial on HT use. Conclusions: The 2017 WHI study did not demonstrate any impact on either menopause-related outpatient visits or HT use among middle-aged women in Taiwan. Our emulated cross-sectional study design may be employed in similar population-based policy intervention studies using interval-censored data.