Year: 2024
Type: conference-abstract
Abstract: Background We recently developed an exploratory health economic decision-analytic model to estimate the natural history and potential health and cost impacts of future disease-modifying treatments (DMTs) for Huntington’s disease (HD) in the United States (US). Aims To adapt our US-based HD model to the United Kingdom (UK) and EU-4 countries Spain, France, Germany, and Italy. Methods/Techniques Advisory boards composed of HD patient advocates, clinicians, and health economists aided in development of a model to evaluate the US costs and health impacts of hypothetical DMTs on three groups of 40-year-old populations : (1) pre-functional decline (PFD, total functional score [TFC] 13), (2) active functional decline Shoulson & Fahn (SF) category 1 (SF1, TFC 13-11), and (3) SF2 (TFC 10-7) individuals. The Enroll-HD database was used to validate our approach to long-term modeling of HD progression. The model estimated lifetime health state costs, quality-adjusted life-years (QALYs), and overall survival. We utilized localized health-state-related costs and utilities for each of the five included countries. Results/Outcome: The model estimated that the PFD, SF1, and SF2 populations had an expected range of QALYs of 14.0-15.6, 6.7-8.9, and 4.2-6.3, respectively. The introduction of hypothetical DMTs led to cost-savings and gains in both QALYs and life-years in the PFD population. Conclusions Our HD decision-analytic model continually evolves to better assess the long-term effectiveness and affordability of disease-modifying therapies (DMTs) in countries other than the US, with ultimate goals of global applicability and providing a foundation for future cost-effectiveness evaluations once clinical trial evidence becomes available.
Language: English
Subfield: Economics and Econometrics
Domain: Social Sciences
Open Access status: gold
OpenAlex ID: W4402374852