This is a bibliographic record of a published health technology assessment from a member of INAHTA. No evaluation of the quality of this assessment has been made for the HTA database.

"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." (executive summary)

Implications for Decision Making

1) 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.

2) For the average patient, rhGH is cost effective if a payer is willing to pay more than CAD200,000 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.

3) 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 CAD11.3 million. The more likely scenario would be that 40% to 50% of eligible patients would receive treatment, with a proportionate decrease in expenditure.