Budget impact analysis of Skytrofa for the treatment of pediatric growth hormone deficiency in a US health plan

Daily administration of growth hormone (GH) can be challenging for patients with pediatric growth hormone deficiency (pGHD) and their caregivers. Lonapegsomatropin, a prodrug of somatropin, is administered once weekly and designed to provide sustained release of active, unmodified somatropin. International guidelines recognize the burden associated with daily injections and suggest long-acting GH to improve adherence and outcomes. The design of the auto-injector for lonapegsomatropin results in zero medication wastage leading to potential cost savings. To estimate the budget impact to a US health plan of introducing lonapegsomatropin to a market with existing daily and weekly GH treatments and to identify possible cost savings associated with its use. The budget impact model (BIM) was developed to compare the costs associated with two hypothetical scenarios for pediatric pGHD: one without lonapegsomatropin, where daily and weekly GHs are available, and one with lonapegsomatropin introduced to the market alongside daily and weekly GHs. The model combined per-patient costs with expected market shares for GHs to estimate the total financial expenditure for both scenarios over a five-year period, simulating costs for a US payer covering one million lives. This analysis included daily somatropin, and weekly somatrogon and somapacitan, as comparators. The introduction of lonapegsomatropin resulted in a cumulative total saving of $959,629 over a 5-year time horizon, with cost savings of $40,314 in Year 1, increasing to $373,258 in Year 5. The estimated average cost savings were $3,283 per patient per month (PPPM) and $0.08 per member per month (PMPM). Scenario and sensitivity analyses demonstrated that the cost-savings with lonapegsomatropin are robust across various assumptions, including alternative market share and dosing scenarios. The primary drivers of the cost reduction were decreased acquisition costs for daily somatropin and the elimination of wastage for weekly GHs. The inclusion of lonapegsomatropin in the ...