Stage-Specific Cost-Effectiveness of Early vs. Delayed Tafamidis for Transthyretin Amyloid Cardiomyopathy in Japan
Abstract
BACKGROUND: Tafamidis improves survival in patients with transthyretin amyloid cardiomyopathy (ATTR-CM); however, its cost raises concerns regarding healthcare sustainability. We evaluated the stage-specific cost-effectiveness of tafamidis in Japan. METHODS AND RESULTS: Individual patient-level data were reconstructed from published Kaplan-Meier curves from the ATTR-ACT long-term extension study. Time-dependent survival benefit between early (continuous tafamidis) and delayed (placebo-to-tafamidis) tafamidis initiation was quantified using restricted mean survival time (RMST). Stage-specific incremental cost-effectiveness ratios (ICERs) were estimated according to National Amyloidosis Centre (NAC) stage. Drug costs reflected the official Japanese reimbursement price, and quality-adjusted life years (QALYs) were derived from RMST gains. The primary endpoint was cardiovascular mortality. At maximum follow-up, RMST gains were 15.7, 13.1, and 1.2 months in NAC stage I, II, and III, respectively ICERs declined over time but remained above commonly accepted Japanese willingness-to-pay thresholds (5,000,000-15,000,000 yen/QALY). The lowest ICER was 47,000,000 yen/QALY in NAC stage I at 90 months. Sensitivity analyses showed that results were robust across utility assumptions, whereas drug cost was the principal determinant of cost-effectiveness. CONCLUSIONS: The survival benefit and economic value of tafamidis varied by disease stage. Earlier-stage patients derived the greatest benefit and economic value; however, ICERs remained above Japanese willingness-to-pay thresholds across all NAC stages. Earlier diagnosis may maximize clinical benefit, but substantial price reduction is likely required for sustainable healthcare in ATTR-CM.
Related
- Impact of Tafamidis on Transthyretin Concentration and Its Relation to All-Cause Mortality in Patients with Transthyretin Amyloid Cardiomyopathy: An Analysis from ATTR-ACT
- When Earlier Is Better But Still Not Cost-Effective ― Reframing the Value of Tafamidis in Transthyretin Amyloid Cardiomyopathy ―
- Survival in a Contemporary, Real-World, Matched Cohort of Patients with Transthyretin Amyloid Cardiomyopathy Treated vs Not Treated with Tafamidis
- Cardiovascular-Related Mortality and Hospitalizations in Patients with Transthyretin Amyloid Cardiomyopathy Treated with Tafamidis: A Post Hoc Analysis of the Phase 3 ATTR-ACT and Long-Term Extension
- Sex Differences in a Contemporary, Real-World Cohort of Patients with Transthyretin Amyloid Cardiomyopathy Treated with Tafamidis
BibTeX
@article{nagata2026stagespecific,
title = {{Stage-Specific Cost-Effectiveness of Early vs. Delayed Tafamidis for Transthyretin Amyloid Cardiomyopathy in Japan}},
author = {Yasufumi Nagata and Yosuke Nabeshima and Tetsuji Kitano and Masaaki Takeuchi and Masaharu Kataoka},
journal = {Circulation Journal},
year = {2026},
month = oct,
doi = {10.1253/circj.cj-26-0576},
url = {https://www.jstage.jst.go.jp/article/circj/90/11/90_CJ-26-0576/_article},
note = {CC BY-NC-ND},
}
RIS
TY - JOUR
TI - Stage-Specific Cost-Effectiveness of Early vs. Delayed Tafamidis for Transthyretin Amyloid Cardiomyopathy in Japan
AU - Yasufumi Nagata
AU - Yosuke Nabeshima
AU - Tetsuji Kitano
AU - Masaaki Takeuchi
AU - Masaharu Kataoka
PY - 2026
DA - 2026/10/23
JO - Circulation Journal
DO - 10.1253/circj.cj-26-0576
UR - https://www.jstage.jst.go.jp/article/circj/90/11/90_CJ-26-0576/_article
AB - BACKGROUND: Tafamidis improves survival in patients with transthyretin amyloid cardiomyopathy (ATTR-CM); however, its cost raises concerns regarding healthcare sustainability. We evaluated the stage-specific cost-effectiveness of tafamidis in Japan. METHODS AND RESULTS: Individual patient-level data were reconstructed from published Kaplan-Meier curves from the ATTR-ACT long-term extension study. Time-dependent survival benefit between early (continuous tafamidis) and delayed (placebo-to-tafamidis) tafamidis initiation was quantified using restricted mean survival time (RMST). Stage-specific incremental cost-effectiveness ratios (ICERs) were estimated according to National Amyloidosis Centre (NAC) stage. Drug costs reflected the official Japanese reimbursement price, and quality-adjusted life years (QALYs) were derived from RMST gains. The primary endpoint was cardiovascular mortality. At maximum follow-up, RMST gains were 15.7, 13.1, and 1.2 months in NAC stage I, II, and III, respectively ICERs declined over time but remained above commonly accepted Japanese willingness-to-pay thresholds (5,000,000-15,000,000 yen/QALY). The lowest ICER was 47,000,000 yen/QALY in NAC stage I at 90 months. Sensitivity analyses showed that results were robust across utility assumptions, whereas drug cost was the principal determinant of cost-effectiveness. CONCLUSIONS: The survival benefit and economic value of tafamidis varied by disease stage. Earlier-stage patients derived the greatest benefit and economic value; however, ICERs remained above Japanese willingness-to-pay thresholds across all NAC stages. Earlier diagnosis may maximize clinical benefit, but substantial price reduction is likely required for sustainable healthcare in ATTR-CM.
SN - 1347-4820
SN - 1346-9843
ER -