Three‐year experience with access to nationally funded growth hormone (GH) replacement for GH‐deficient adults

Abstract

Carefully designed access to nationally funded GH replacement in GH-deficient adults was associated with a significant improvement in quality of life over a 3-year period with mean daily GH doses lower than in the majority of previously reported studies.

Limited adherence to growth hormone replacement in patients with traumatic brain injury

“…GHD due to other aetiologies (primarily hypothalamic and pituitary diseases), when hGH therapy was provided in a full reimbursement setting, adherence at 2 years exceeded 80% (4). Long-term adherence to hGH may therefore serve as a pragmatic indicator of patient-perceived efficacy, especially given that ongoing clinical follow-up was one of the only requisites for continuing treatment in this previous study (4).…”

“…GHD due to other aetiologies (primarily hypothalamic and pituitary diseases), when hGH therapy was provided in a full reimbursement setting, adherence at 2 years exceeded 80% (4). Long-term adherence to hGH may therefore serve as a pragmatic indicator of patient-perceived efficacy, especially given that ongoing clinical follow-up was one of the only requisites for continuing treatment in this previous study (4). This is similar to our practice locally; after patients are proven to have GHD by dynamic testing, they are prescribed a 1-year supply of hGH (which is reimbursed by all insurers operating in our province) and must self-initiate a follow-up appointment at 12 months if they wish to have ongoing prescriptions for this therapy.…”

A will and a way to fund medicines for rare diseases: the story of human growth hormone replacement for adults with growth hormone deficiency

“…8 The working group therefore began by commissioning a researcher (WL) (paid by the two professional societies) to conduct a reanalysis of the trials that had been presented to PBAC. The only important new data that had emerged since the 2011 PBAC submission was an observational study from New Zealand reporting the efficacy of somatropin since it had been approved by PHARMAC (New Zealand's equivalent to PBAC).…”

“…The only important new data that had emerged since the 2011 PBAC submission was an observational study from New Zealand reporting the efficacy of somatropin since it had been approved by PHARMAC (New Zealand's equivalent to PBAC). 8 The working group therefore began by commissioning a researcher (WL) (paid by the two professional societies) to conduct a reanalysis of the trials that had been presented to PBAC. This analysis included all relevant studies, including those that demonstrated a neutral or negative effect on the parameters of interest.…”

“…Indeed, a significant weight of the evidence presented to PBAC was provided from long-term open-label studies, most notably a 3-year observational study from New Zealand. 8 It is also noteworthy that, while consumers were not directly involved in the working group's activities, PBAC stated in its Public Summary Document that it had taken account of clinical and consumer input and had found this helpful in making their recommendation. They made note of input from individual patients, carers and clinicians, from the Australian Pituitary Foundation (which presented the results of a survey of its members) and from international consumer advocacy organisations.…”