What does this research mean for the field?

AMT-130 is a cost-effective treatment for early-stage Huntington's disease in the US at a $500,000/QALY threshold, whereas Tominersen is not cost-effective compared to standard of care. Novelty: ClaimNovelty.INCREMENTAL. Consensus alignment: ConsensusAlignment.NEUTRAL.

What question did this study set out to answer?

To evaluate the lifetime cost-effectiveness of emerging therapies Tominersen and AMT-130 compared with standard of care for early-stage Huntington's disease in the United States.

August 30, 2026PharmacoEconomics - OpenOpen Access

A Cost-Effectiveness Analysis for Treatments of Patients with Early Stage Huntington’s Disease in the USA

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Authors

DPDeepti Patil JSJulia F. Slejko JSJoseph M. Savitt

Discussion

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Overview

Economic evaluation reveals AMT-130, but not tominersen, is cost-effective for early-stage Huntington’s disease, highlighting substantial divergence in economic value among emerging therapies.

Key Points

To evaluate the lifetime cost-effectiveness of emerging therapies Tominersen and AMT-130 compared with standard of care for early-stage Huntington's disease in the United States.
Constructed a four-state Markov model (early, middle, late, and death) using 1-year cycle lengths, a 3% annual discount rate, and a modified societal perspective in 2024 US dollars.
Assessed health gains in quality-adjusted life years (QALYs) against a rare-disease willingness-to-pay threshold of $500,000 per QALY gained, with deterministic and probabilistic sensitivity analyses.
Tominersen increased lifetime costs by $2.11 million and gained 0.93 QALYs relative to standard of care, generating an incremental cost-effectiveness ratio of $2.28 million per QALY gained.
AMT-130 increased lifetime costs by $1.49 million and gained 5.23 QALYs relative to standard of care, generating an incremental cost-effectiveness ratio of $285,703 per QALY gained.
Probabilistic sensitivity analysis indicated that AMT-130 has an 80% probability of being cost-effective at a threshold of $350,000 per QALY gained.

Cite This Study

Patil et al. (2026) studied this question.

synapsesocial.com/papers/6a93f08e6c1a8fb52e79cbe8 https://doi.org/10.1007/s41669-026-00668-5

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August 30, 2026PharmacoEconomics - OpenOpen Access

A Cost-Effectiveness Analysis for Treatments of Patients with Early Stage Huntington’s Disease in the USA

View Full Paper

Ask AI

Bookmark

Authors

DPDeepti Patil JSJulia F. Slejko JSJoseph M. Savitt

Discussion

Member takes

Overview

Economic evaluation reveals AMT-130, but not tominersen, is cost-effective for early-stage Huntington’s disease, highlighting substantial divergence in economic value among emerging therapies.

Key Points

To evaluate the lifetime cost-effectiveness of emerging therapies Tominersen and AMT-130 compared with standard of care for early-stage Huntington's disease in the United States.
Constructed a four-state Markov model (early, middle, late, and death) using 1-year cycle lengths, a 3% annual discount rate, and a modified societal perspective in 2024 US dollars.
Assessed health gains in quality-adjusted life years (QALYs) against a rare-disease willingness-to-pay threshold of $500,000 per QALY gained, with deterministic and probabilistic sensitivity analyses.
Tominersen increased lifetime costs by $2.11 million and gained 0.93 QALYs relative to standard of care, generating an incremental cost-effectiveness ratio of $2.28 million per QALY gained.
AMT-130 increased lifetime costs by $1.49 million and gained 5.23 QALYs relative to standard of care, generating an incremental cost-effectiveness ratio of $285,703 per QALY gained.
Probabilistic sensitivity analysis indicated that AMT-130 has an 80% probability of being cost-effective at a threshold of $350,000 per QALY gained.

Cite This Study

Patil et al. (2026) studied this question.

synapsesocial.com/papers/6a93f08e6c1a8fb52e79cbe8 https://doi.org/10.1007/s41669-026-00668-5

View Full Paper

Ask AI

Bookmark