A Cost-Effectiveness Analysis Model Framework For Treatments Of Early-Stage Huntington’s Disease Patients In The United States

Abstract

Huntington's disease (HD) is a rare neurodegenerative condition caused by a mutation in the huntingtin gene. The emergence of drugs such as Tominersen and AMT-130, which have the potential to treat HD highlights the importance of evaluating their cost-effectiveness. This study aims to fill this gap and evaluate the incremental cost-effectiveness of these treatments compared with the current standard of care for HD. A health state transition Markov model was developed to estimate the costs and benefits of each treatment over a lifetime time horizon from a societal perspective. Our findings showed that Tominersen and AMT-130 were associated with higher costs but also provided greater benefits than the standard of care. AMT-130 was found to be a cost-effective option compared to the standard of care and Tominersen, considering the willingness to pay threshold. This study provides valuable insights into the economic impact of HD which can inform healthcare policy and treatment decisions.