OBJECTIVES: Pediatric growth hormone deficiency (pGHD) is defined as growth failure associated with inadequate growth hormone (GH) production. Daily injections of recombinant human GH (dGH) [somatropin] is the current standard of care, which has been shown to be safe and effective, however with suboptimal adherence, leading to reduced effectiveness.

Somatrogon, a once-weekly injectable long-acting human GH, has demonstrated clinical non-inferiority and significantly lower life interference vs. somatropin.

This work aimed to evaluate cost-effectiveness and cost-utility of somatrogon vs dGHs across 5 countries (United States, Canada, Spain, Sweden, Ireland)

METHODS: A Markov model was developed for patients starting somatrogon or dGH treatment at 3-12 years and continuing up to 18 years of age, with growth driven by trial-based height velocity (HV) and treatment-specific adherence. Patients could discontinue treatment at the end of Year 1 (ranges: 0%-20.4% across countries). DGH adherence (US: 88.7%-58.5% from Year 1 to Year 15 of treatment, Canada: 88.7%-82.2%, Spain: 95.9%-93.9%, Sweden: 88.7%-58.5%, Ireland: 95.3%-65%) and adherence-growth relationship were based on published evidence. Higher Year 1 adherence of 4%-5%, tapering over time, for somatrogon vs. dGHs was based on clinical consultation. Treatment costs, monitoring costs and costs due to different wastage types were sourced from local data. Health utilities based on height and injection frequency were derived from published literature. Scenario analysis, deterministic and probabilistic sensitivity analysis were performed.

RESULTS: Treating with somatrogon led to 1.71-4.11 cm near adult height (NAH) gain and 0.19-0.43 higher QALYs vs. dGHs, across the 5 countries, and the scenarios evaluated. Somatrogon was generally cost-effective vs dGHs, with dGH dosing, injection frequency disutility, dGHs unit costs and somatrogon adherence being the key cost-effectiveness drivers.

CONCLUSIONS: Somatrogon weekly injections were estimated to result in higher NAH, higher QALYs and favorable cost-effectiveness vs. dGHs, in pGHD.