ArticlePharmacoEconomics - open2026
A Cost-Effectiveness Analysis for Treatments of Patients with Early Stage Huntington's Disease in the USA.
Article in PharmacoEconomics - open, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
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Authors and funding
4 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
BACKGROUND/
objectiveHuntington's disease (HD) is a rare neurodegenerative condition caused by mutations in the huntingtin gene. Emerging therapies such as Tominersen and AMT-130 have potential to treat HD, highlighting the importance of evaluating their cost-effectiveness. This study evaluates the cost-effectiveness of Tominersen and AMT-130 versus standard of care (SoC) for early stage HD in the US.
methodsA Markov model with four health states (early, middle, late, and death) was developed to estimate lifetime costs and benefits from a modified societal perspective, with death as an absorbing state. The model used 1-year cycle length with half-cycle correction, and an annual discount rate of 3% was used for all future costs and utilities. Health benefits were measured in quality-adjusted life years (QALYs). All costs were adjusted to 2024 US dollars. As per the incremental cost-effectiveness ratio (ICER) value assessment framework, a willingness-to-pay threshold (WTP) of $500,000/QALY gained was considered given that HD is a rare disease. Scenario analysis assessed the impact of adding value of hope to the base-case utility values. Deterministic and probabilistic sensitivity analyses were conducted to capture uncertainty across all model parameters.
resultsCompared with SoC, Tominersen increased costs by $2.11 million and QALYs by 0.93, resulting in an ICER of $2.28 million per QALY gained, whereas AMT-130 increased costs by $1.49 million and QALYs by 5.23, resulting in an ICER of $285,703 per QALY gained in the base case. Probabilistic analysis showed AMT-130 had 80% probability of being cost-effective at $350,000/QALY gained.
conclusionsFrom a modified US societal perspective, this analysis suggests that AMT-130 may be cost-effective relative to SoC, whereas Tominersen exceeds the WTP threshold of $500,000 per QALY gained. Further evidence on long-term effectiveness and durability is needed to inform policy and reimbursement decisions.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the Socratic graph.