Presentation (CTI)

OBJECTIVES: This analysis aimed to estimate the budget impact of introducing tividenofusp alfa for the treatment of pediatric patients weighing at least 5 kg with mucopolysaccharidosis type II (MPS II; also known as Hunter syndrome) with neurologic manifestations from a US payer perspective.
METHODS: A budget-impact model was developed for a hypothetical 10-million-member plan to compare a scenario in which tividenofusp alfa is available with a scenario in which it is not, over a 3-year time horizon. The model incorporated epidemiology-based population estimates, age- and weight-informed dosing assumptions, treatment-specific costs, market-share assumptions, and enrollee mix (commercial and Medicaid). In the scenario with tividenofusp alfa being available, uptake among addressable patients was assumed to increase over time based on data-informed assumptions. Drug acquisition costs for modeled therapies were based on WAC, and budget-impact results were reported on a per-member-per-month (PMPM) basis.
RESULTS: Across the modeled time horizon, PMPM costs increased with uptake of tividenofusp alfa but remained manageable. The PMPM budget impact for the base case was US$0.005 in Year 1, US$0.013 in Year 2, and US$0.016 in Year 3. In the sensitivity analyses, budget-impact estimates were most sensitive to variation in drug-acquisition costs and market-uptake assumptions. Across all sensitivity analyses conducted, PMPM budget impact remained below $0.025.
CONCLUSIONS: This analysis suggests that the adoption of tividenofusp alfa would be associated with a manageable short-term budget impact for US payers. The rarity of MPS II results in a small treated population within the health plan, which contributes to the limited magnitude of the modeled PMPM budget impact. In the sensitivity analyses, the projected budget impact remained small across assumptions, indicating that the results are robust and sustained even with variation of key model inputs.