OBJECTIVES: Facial angiofibroma (FA), one of the predominant dermatological manifestations of tuberous sclerosis complex (TSC) disorder, occurs in 75%-88% of patients. Managing FA with TSC adds a substantial clinical and economic burden on the healthcare system. We assessed the budget impact of including sirolimus 0.2% gel (HYFTOR™), the first and only US FDA approved topical medication for FA associated with TSC from a US third-party payer perspective (Commercial, Medicaid, and Medicare).

METHODS: A prevalence-based budget impact model with a 3-year time horizon was developed using Microsoft^® Excel comparing the total costs for treating TSC patients with FA (≥6 years) in two scenarios: with and without sirolimus gel 0.2%. Model inputs (epidemiological, clinical and costs inputs; drug acquisition, inpatient hospitalization, emergency department visit, office visit, other outpatient services and prescription refill costs) were obtained from published literature and a real-world study. Scenario analyses were considered as per ISPOR 2014 BIA Best Practice Guidelines.

RESULTS: In a hypothetical 1-million-member health plan, including topical sirolimus 0.2% gel at a wholesale acquisition cost (WAC) of $1,750 resulted in total budget impact of $287,450, $242,208, and $403,693 per million plan population, and a minimal per member per month (PMPM) cost of $0.02, $0.02, $0.03 in the Commercial, Medicaid and Medicare population, respectively. Scenario analyses by varying several parameters like longer time horizon (5 years), response rate assessed by Independent Review Committee, response rate assessed at 52 weeks, and proportion of patients associated with TSC from Kingswood et al. (2022), and pooled proportion suggested that the PMPM costs did not exceed $0.06 under alternative model assumptions across payers.

CONCLUSIONS: The introduction of topical sirolimus 0.2% gel for management of facial angiofibroma associated with TSC in the US is likely to have a very low impact on the US health plans budget.