OBJECTIVES: HD is a rare neurodegenerative disorder currently without a cure or treatment to slow disease progression. Current treatment is symptomatic, focusing on alleviating motor symptoms as well as improving depression and other mood disorders. This study aimed to determine the current pharmacy costs in HD as well as identify potential DMTs in development

METHODS: An SLR was conducted March 28, 2023 in Embase to identify the economic burden associated with HD. Eligible studies were published from 2008 (2020 for conference proceedings) and presented data on healthcare resource utilization or direct/indirect costs. Citations were assessed by two reviewers, with discrepancies reconciled by a third. For this sub-analysis, pharmacy costs relating to HD treatment in the US were extracted. Clinicaltrials.gov was searched January 5, 2024 to identify potential future DMTs. Trials of interest were Phase 2 or 3 with a status of completed, active not recruiting, or recruiting, initiated within the last 10 years in North America.

RESULTS: 238 citations were screened, with 62 studies included; 9 provided pharmacy costs. Annual HD pharmacy costs ranged from $6,930 to $19,182 per patient, increasing as disease severity increases ($860-$14,831 for early stage, $3,094-$22,672 for late stage). This symptom management accounts for 14%-46% of direct costs in HD. Motor symptoms incur the greatest annual drug cost, with high median costs per patient (2019) for tetrabenazine ($24,996), deutetrabenazine ($69,972), and valbenazine ($76,908). Ten clinical trials are investigating potential future DMTs: two-antisense oligonucleotides (n=5; Tominersen [RO7234292, ISIS 443139], WVE-003), two-RNA-targeting small molecules (n=2; PTC518, Branaplam), monoclonal antibodies (n=2; ANX005, VX15/2503), and gene therapy (n=1; rAAV5-miHTT).

CONCLUSIONS: Pharmacy costs for symptomatic treatment are substantial in HD, with these costs increasing as HD progresses and symptom severity increases. This significant burden illustrates the importance of developing new DMTs that can prevent or slow disease progression for patients with HD.