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Estimated cost-effectiveness of growth hormone therapy for idiopathic short stature
Joyce M Lee1, Matthew M Davis, Sarah J Clark
1Division of Pediatric Endocrinology, University of Michigan, Ann Arbor 48109-0456, USA. joyclee@umich.edu
Insights
Growth hormone (GH) therapy for idiopathic short stature (ISS) costs $52,634 per inch of height gained. Targeting children with the best growth potential is key to improving cost-effectiveness.
Area of Science:
- Pediatric Endocrinology
- Health Economics
- Pharmacoeconomics
Background:
- Idiopathic short stature (ISS) affects children's growth.
- Growth hormone (GH) therapy is a treatment option for ISS.
- Estimating the economic value of GH therapy is crucial for clinical decision-making.
Purpose of the Study:
- To estimate the cost-effectiveness of GH therapy for children with ISS.
- To evaluate the economic impact of different GH treatment strategies.
Main Methods:
- A cost-effectiveness analysis using a decision model.
- Compared a cohort of 10-year-old boys with ISS treated with GH for 5 years versus no intervention.
- Measured incremental cost per child, incremental growth, and incremental cost per inch of final height gain.
Main Results:
- The incremental cost-effectiveness ratio was $52,634 per inch ($99,959 per child) for an average growth increase of 1.9 inches.
- Alternative strategies like extended duration or higher pubertal doses did not significantly improve cost-effectiveness.
- Growth response variability to GH therapy was the most impactful factor on cost-effectiveness.
Conclusions:
- Targeted GH therapy for children with ISS who have the highest growth potential is essential for maximizing cost-effectiveness.
- The true utility gains from height increases achieved with GH therapy for ISS require further investigation to fully assess cost per inch.
Objective:
To estimate the cost-effectiveness of growth hormone (GH) therapy for idiopathic short stature (ISS).
Design:
Cost-effectiveness analysis.
Setting:
Decision model.
Patients:
A cohort of 10-year-old prepubertal boys with ISS treated with GH.
Interventions:
Comparison of children treated for 5 years with GH therapy vs children receiving no intervention.
Main Outcome Measures:
Incremental cost per child, incremental growth per child, and incremental cost per inch of final height gain.
Results:
The estimated incremental cost-effectiveness ratio of GH therapy for ISS in the base case analysis compared with no therapy was 52,634 dollars per inch (per 2.54 cm), or 99,959 dollars per child, reflecting an incremental growth of 1.9 in (4.8 cm). Alternate treatment strategies such as increased duration of GH treatment and high pubertal dosing of GH did not substantially improve the cost-effectiveness ratio. Probabilistic sensitivity analyses showed that growth variability in response to GH had the greatest impact on the cost-effectiveness of GH therapy.
Conclusions:
Targeted treatment of children with ISS with the greatest potential for growth appears critical for maximizing cost-effectiveness of GH treatment. However, the significance of the cost per inch is difficult to judge until the utility gains associated with height gain after GH therapy for ISS can be ascertained.
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