When Earlier Is Better But Still Not Cost-Effective ― Reframing the Value of Tafamidis in Transthyretin Amyloid Cardiomyopathy ―
Abstract
required substantially longer follow-up.The supplementary analysis summarized "time to benefit" as approximately 18 months in stage I and 56 months in stage II, with no demonstrable time to benefit in stage III within the available follow-up. 6,9The economic message is equally important.Even where clinical benefit was greatest, economic value at the current Japanese reimbursement price remained unfavorable: the lowest estimated incremental cost-effectiveness ratios (ICERs) were approximately ¥47 million/QALY in stage I and ¥52 million/QALY in stage II, both above commonly cited Japanese willingness-to-pay ranges.6,7 Stage III was far less favorable.Utility assumptions changed estimates only modestly, whereas drug price dominated the model; reductions of roughly 68% and 71% were estimated to bring stages I and II, respectively, to ¥15 million/QALY.6 Thus, "earlier is better" and "earlier is cost-effective" are not synonymous.This distinction is clinically useful.Earlier detection is supported by Japanese evidence, including hs-cTnT-based screening and opportunistic imaging approaches, 2,3 while long-term observational data suggest biomarkers may help identify poorer prognosis during tafamidis therapy.4 Nagata et al. add the missing value dimension: diagnostic acceleration is most meaningful when it moves treatment into a stage in which patients can accumulate substantial survival benefit, but diagnosis alone cannot solve affordability.6 However, several uncertainties should prevent these ICERs from being interpreted as definitive reimbursement thresholds.First, this is not a conventional lifetime costutility model.The comparator is delayed tafamidis rather than persistent no treatment; non-drug healthcare costs are omitted; outcomes are not discounted; and QALYs are derived from RMST using assumed utility weights rather than longitudinal patient-level EQ-5D measurements.6 These choices are understandable, but the analysis is best viewed as a stage-specific value signal rather than a complete health technology assessment.Second, reconstructing
Related
- Stage-Specific Cost-Effectiveness of Early vs. Delayed Tafamidis for Transthyretin Amyloid Cardiomyopathy in Japan
- Impact of Tafamidis on Transthyretin Concentration and Its Relation to All-Cause Mortality in Patients with Transthyretin Amyloid Cardiomyopathy: An Analysis from ATTR-ACT
- 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{kishi2026when,
title = {{When Earlier Is Better But Still Not Cost-Effective ― Reframing the Value of Tafamidis in Transthyretin Amyloid Cardiomyopathy ―}},
author = {Takuya Kishi},
journal = {Circulation Journal},
year = {2026},
month = oct,
doi = {10.1253/circj.cj-26-0898},
url = {https://www.jstage.jst.go.jp/article/circj/90/11/90_CJ-26-0898/_article},
note = {CC BY-NC-ND},
}
RIS
TY - JOUR
TI - When Earlier Is Better But Still Not Cost-Effective ― Reframing the Value of Tafamidis in Transthyretin Amyloid Cardiomyopathy ―
AU - Takuya Kishi
PY - 2026
DA - 2026/10/23
JO - Circulation Journal
DO - 10.1253/circj.cj-26-0898
UR - https://www.jstage.jst.go.jp/article/circj/90/11/90_CJ-26-0898/_article
AB - required substantially longer follow-up.The supplementary analysis summarized "time to benefit" as approximately 18 months in stage I and 56 months in stage II, with no demonstrable time to benefit in stage III within the available follow-up. 6,9The economic message is equally important.Even where clinical benefit was greatest, economic value at the current Japanese reimbursement price remained unfavorable: the lowest estimated incremental cost-effectiveness ratios (ICERs) were approximately ¥47 million/QALY in stage I and ¥52 million/QALY in stage II, both above commonly cited Japanese willingness-to-pay ranges.6,7 Stage III was far less favorable.Utility assumptions changed estimates only modestly, whereas drug price dominated the model; reductions of roughly 68% and 71% were estimated to bring stages I and II, respectively, to ¥15 million/QALY.6 Thus, "earlier is better" and "earlier is cost-effective" are not synonymous.This distinction is clinically useful.Earlier detection is supported by Japanese evidence, including hs-cTnT-based screening and opportunistic imaging approaches, 2,3 while long-term observational data suggest biomarkers may help identify poorer prognosis during tafamidis therapy.4 Nagata et al. add the missing value dimension: diagnostic acceleration is most meaningful when it moves treatment into a stage in which patients can accumulate substantial survival benefit, but diagnosis alone cannot solve affordability.6 However, several uncertainties should prevent these ICERs from being interpreted as definitive reimbursement thresholds.First, this is not a conventional lifetime costutility model.The comparator is delayed tafamidis rather than persistent no treatment; non-drug healthcare costs are omitted; outcomes are not discounted; and QALYs are derived from RMST using assumed utility weights rather than longitudinal patient-level EQ-5D measurements.6 These choices are understandable, but the analysis is best viewed as a stage-specific value signal rather than a complete health technology assessment.Second, reconstructing
SN - 1347-4820
SN - 1346-9843
ER -