Cost-effectiveness of somatropin for the treatment of short children born small for gestational age.
BACKGROUND
Short children born small for gestational age (SGA) may be at increased risk for long-term morbidity and reduced health-related quality of life (HRQoL) due to their short stature. Normalization of height in childhood and adolescence is possible in such children via the use of the recombinant human growth hormone somatropin.


OBJECTIVE
The aim of this study was to determine whether somatropin was a cost-effective treatment option in short children born SGA.


METHODS
A decision analytic model was constructed to calculate the cost-effectiveness of somatropin treatment versus no treatment over the lifetime of a short individual born SGA, from the perspective of the UK National Health Service (NHS). The model was based on patient-level data from a multicenter, double-blind, randomized controlled trial that reported the effects of somatropin on final (adult) height in short children born SGA. Health care resource and drug costs associated with each of the treatment arms were considered, and published utility scores were used to calculate improvement in HRQoL. The model calculated incremental costs and incremental quality-adjusted life-years (QALYs) associated with somatropin treatment compared with no treatment. Cost-effectiveness was expressed as incremental cost per QALY and cost per centimeter of height gained.


RESULTS
Over a patient's lifetime, somatropin (0.033 mg/kg/d) treatment was associated with a height gain of 16.12 cm and a cost per centimeter of height gained of pound4359 compared with no treatment. The incremental cost of somatropin treatment was pound70,263, with a QALY gain of 2.95, resulting in an incremental cost per QALY of pound23,807-below the widely accepted cost-effectiveness threshold in the United Kingdom of pound30,000.


CONCLUSIONS
In this model, somatropin was a cost-effective treatment option for short children born SGA from the perspective of the UK NHS.
Christensen
Torsten
T
Novo Nordisk A/S, Copenhagen, Denmark.
Buckland
Andrew
A
Bentley
Anthony
A
Djurhuus
Christian
C
Baker-Searle
Rachael
R
D001827:Body Height / Q000187:drug effects*; D001835:Body Weight; D002648:Child; D003362:Cost-Benefit Analysis; D006130:Growth Disorders / Q000188:drug therapy*; D019382:Human Growth Hormone / Q000191:economics* / Q000627:therapeutic use*; D006801:Humans; D007231:Infant, Newborn; D007236:Infant, Small for Gestational Age; D018803:Models, Economic; D011788:Quality of Life; D019057:Quality-Adjusted Life Years; D016032:Randomized Controlled Trials as Topic; D011994:Recombinant Proteins / Q000191:economics / Q000627:therapeutic use; D012959:Socioeconomic Factors
D011994:Recombinant Proteins; D019382:Human Growth Hormone