Cost-effectiveness of somatropin for the treatment of short children born small
for gestational age.
Author(s): Christensen T, Buckland A, Bentley A, Djurhuus C, Baker-Searle R.
Affiliation(s): Novo Nordisk A/S, Copenhagen, Denmark.
Publication date & source: 2010, Clin Ther. , 32(6):1068-82
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.
CONCLUSION: In this model, somatropin was a cost-effective treatment option for
short children born SGA from the perspective of the UK NHS.
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