European heart journal. Quality of care & clinical outcomes · 2026
Affordability of innovation: cost-effectiveness and budget impact analysis of acoramidis in transthyretin amyloid cardiomyopathy in the German healthcare system.
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Estler Bent, Fröhlich Hanna, Täger Tobias, Frankenstein Lutz
Аннотация
AIMS: Transthyretin amyloid cardiomyopathy (ATTR-CM) causes heart failure with substantial morbidity, mortality, and healthcare utilization. ATTRibute-CM [Efficacy and Safety of Acoramidis in Transthyretin Amyloid Cardiomyopathy (Phase 3 trial)] showed that acoramidis improves clinical outcomes, but its cost-effectiveness and budget impact in the German statutory health insurance (SHI) system remain uncertain.
METHODS AND RESULTS: We developed a cohort Markov model (lifetime horizon) discounting costs and outcomes at 3% annually. Overall survival was modelled with recalibrated Weibull functions fitted to randomized-phase ATTRibute-CM data and anchored to match Month 30 survival. Health-related quality of life used Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS) trajectories mapped to EQ-5D utilities, with a plateau after 30 months in the base case. Direct medical costs (2025€) included drug, background management, and hospitalization costs. Uncertainty was assessed with sensitivity analyses, scenario analyses, and a budget impact analysis. Discounted mean lifetime costs were €13 881.50 (placebo) and €828 127.07 (acoramidis), for an incremental €814 245.57. Quality-adjusted life-years (QALYs) increased from 2.743 to 4.029 (incremental 1.286) and life-years from 4.023 to 6.045 (incremental 2.022), yielding an incremental cost-effectiveness ratio (ICER) of €633 081 per QALY. Survival scenario analyses produced lifetime ICERs from €458 953/QALY in an optimistic scenario to €2 139 712/QALY in a pessimistic waning-benefit scenario. At a willingness-to-pay (WTP) of €100 000/QALY, the probability of cost-effectiveness was very low and the results were driven primarily by acoramidis acquisition cost. In the budget impact analysis, Year 1 SHI expenditure was €80 813 995, €134 689 992, and €269 379 983 for 30, 50, and 100% uptake, respectively.
CONCLUSION: Despite benefits, acoramidis is unlikely to be cost-effective at current prices in Germany and could increase SHI spending. Price reductions could better align value with affordability.
METHODS AND RESULTS: We developed a cohort Markov model (lifetime horizon) discounting costs and outcomes at 3% annually. Overall survival was modelled with recalibrated Weibull functions fitted to randomized-phase ATTRibute-CM data and anchored to match Month 30 survival. Health-related quality of life used Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS) trajectories mapped to EQ-5D utilities, with a plateau after 30 months in the base case. Direct medical costs (2025€) included drug, background management, and hospitalization costs. Uncertainty was assessed with sensitivity analyses, scenario analyses, and a budget impact analysis. Discounted mean lifetime costs were €13 881.50 (placebo) and €828 127.07 (acoramidis), for an incremental €814 245.57. Quality-adjusted life-years (QALYs) increased from 2.743 to 4.029 (incremental 1.286) and life-years from 4.023 to 6.045 (incremental 2.022), yielding an incremental cost-effectiveness ratio (ICER) of €633 081 per QALY. Survival scenario analyses produced lifetime ICERs from €458 953/QALY in an optimistic scenario to €2 139 712/QALY in a pessimistic waning-benefit scenario. At a willingness-to-pay (WTP) of €100 000/QALY, the probability of cost-effectiveness was very low and the results were driven primarily by acoramidis acquisition cost. In the budget impact analysis, Year 1 SHI expenditure was €80 813 995, €134 689 992, and €269 379 983 for 30, 50, and 100% uptake, respectively.
CONCLUSION: Despite benefits, acoramidis is unlikely to be cost-effective at current prices in Germany and could increase SHI spending. Price reductions could better align value with affordability.