Technology and Condition
Recombinant human growth hormone (rhGH) for treatment of Turner syndrome (TS).
Given the high cost of rhGH treatment and the evolving evidence base for its clinical effect in patients with TS, policy makers need evidence
to inform reimbursement decisions about rhGH.
Methods and Results
A systematic review was conducted to identify randomized controlled trials (RCTs) and comparative observational studies comparing rhGH with placebo or no treatment in patients with TS. The outcomes of interest were growth, adverse events (AEs), and quality of life (QoL). A meta-analysis was conducted where appropriate. Primary economic analyses were also undertaken, using the perspective of the public health care system and a lifetime horizon. Six RCTs and nine observational studies were included, ranging in duration from one to eight years. The included studies showed that rhGH treatment accelerates growth and results in improvement in height. No serious AEs were reported in the included studies. QoL data, derived from two RCTs, were variable, precluding any conclusion about rhGH’s
influence on QoL. Base case economic analysis showed that the incremental cost-effectiveness ratio (ICER) of rhGH treatment versus no treatment was C$23,630 per centimetre of final height improvement or C$243,078 per quality-adjusted life year (QALY) gained.
Implications for Decision Making
Treatment with rhGH has a demonstrated impact on final height but its effect on QoL is uncertain. The available evidence suggests that, compared with patients receiving placebo or no treatment, patients who are treated with rhGH experience accelerated growth and improvement in final height. Treatment appears to be safe with no serious AEs and few, if any, AEs reported. QoL data, reported in two studies, were variable and inconclusive.
- For the average patient, rhGH is cost effective if a payer is willing to pay more than C$200,000 for a QALY. However, from an ethics perspective, the provision and funding of rhGH could be supported until those with TS reach the lower end of the normal adult height range.
- Publicly funding rhGH treatment will require additional investment. If it were assumed that all TS patients aged 10 to 15 years were eligible for rhGH therapy, the corresponding annual budget impact for covering ~400 patients across Canada would be C$11.3 million. The more likely scenario would be that 40% to 50% of eligible patients would receive treatment, with a proportionate decrease in expenditure.
This summary is based on a comprehensive health technology assessment available from CADTH’s web site (www.cadth.ca): Li H, Banerjee S, Dunfield L, Kirby J, Jones M, Hamilton J, Deal C, Hadjiyannakis S, Normandin S, Tsakonas E. Recombinant Human Growth Hormone for Treatment of Turner Syndrome: Systematic Review and Economic Evaluation