OBJECTIVES: Daily growth hormone (GH) treatment can be burdensome for patients with pediatric GH deficiency (pGHD) and their caregivers, and poor adherence has been associated with suboptimal growth outcomes. Skytrofa^® (lonapegsomatropin), a prodrug of somatropin administered once-weekly and designed to provide sustained release of active, unmodified somatropin, is FDA and EC approved for pGHD treatment. In the heiGHt trial, annualized height velocity with Skytrofa was non-inferior to daily GH and statistically significant in favor of Skytrofa. The objective of this budget impact analysis (BIA) is to estimate the financial significance of introducing Skytrofa to a market with daily GH and to identify potential costs averted with Skytrofa introduced.

METHODS: This BIA compares two hypothetical scenarios: (1) only current daily GHs available (Norditropin^®, Genotropin^®, and Humatrope^®) and (2) Skytrofa, a once-weekly alternative, is introduced to the market. The analysis, from a US payer perspective with 1 million members, has a time horizon of 5 years, and assumes no price discounts.

RESULTS: Based on pGHD incidence rate, an estimated 21 patients will start GH treatment in the first year of the model, increasing to 27 patients treated with GH in the fifth year. With the introduction of Skytrofa, estimated decrease in GH use is 3,173 milligrams (118 milligrams per patient over 5 years), primarily due to reduced Norditropin^® use, which is administered at one-third above recommended dosage based on ANSWER registry real-world results. The estimated plan budget increase would be $475,147 over 5 years, which equals $0.04 per member per month (PMPM). This includes an investment of $3,351,596 for adopting Skytrofa and a cost offset of $2,876,449 due to reduction in milligrams GH used.

CONCLUSIONS: Adding Skytrofa to the formulary for the treatment of pGHD has a minimal budget impact from US payers’ perspective while maintaining or improving growth outcomes.