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

Lipworth, Wendy; Ambler, Geoffrey; Burt, Morton G.; Fairchild, Jan; Inder, Warrick J.; Werther, George A.; K, Ho

doi:10.1111/imj.13943

Cited by 3 publications

(3 citation statements)

References 12 publications

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“…The result was that patients were either not treated, relied on their local district medicine committee to grant compassionate access or paid costs out‐of‐pocket. This prompted the Endocrine Society of Australia and the Australasian Paediatric Endocrine Group to submit ‘public interest’ application for subsidy, for which the application fee of AU$250 000 was waived 10,11 . Although this was ultimately successful in August 2017, considerable additional resources, technical understanding and support from a generic manufacturer were still required to achieve subsidy for this indication.…”

Section: Overcoming the Barriers To Medicine Repurposingmentioning

confidence: 99%

Can old drugs learn new tricks? Achieving registration and public subsidy listing for off‐patent medicines with novel therapeutic applications

Brett

Bahceci

Lipworth

et al. 2023

Internal Medicine Journal

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With the increasing costs of drug development, repurposing of low-cost medicines for new indications has never been more important. However, there are multiple barriers to repurposing, particularly for off-patent medicines, and limited incentives for the pharmaceutical industry to sponsor registration and public subsidy listing. Here, we explore these barriers and their consequences and provide examples of successful repurposing strategies.

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Section: Overcoming the Barriers To Medicine Repurposingmentioning

confidence: 99%

Can old drugs learn new tricks? Achieving registration and public subsidy listing for off‐patent medicines with novel therapeutic applications

Brett

Bahceci

Lipworth

et al. 2023

Internal Medicine Journal

Self Cite

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“…However, reimbursement was only obtained in 2017 after two unsuccessful applications from the industry. The successful application was unusual in that it was lodged by a professional organisation (the Endocrine Society of Australia and the Australian Paediatric Endocrine Group) as a public interest submission providing information meeting the rigorous threshold assessment for costeffectiveness (Lipworth et al 2018).…”

Section: Cost-effectivenessmentioning

confidence: 99%

The physiology of growth hormone (GH) in adults: translational journey to GH replacement therapy

O’Sullivan

Burt

2023

Journal of Endocrinology

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That GH plays an important role in health after the cessation of growth requiring replacement therapy in adult life, has only been recognised in the last three decades. This has only been made possible by recombinant technology providing GH supplies required to undertake investigations of the physiology of GH action and benefits of replacement therapy in patients identified by rigorously validated diagnostic tests for GHD. Human studies have revealed important regulatory roles in substrate metabolism, sodium homeostasis, body composition and physical function. GH-induced anabolism is achieved by stimulating amino acid incorporation into while reducing oxidative loss simultaneously enhancing lipid utilisation by stimulating fatty acid oxidation and reducing lipid storage. Sodium and fluid retention are enhanced by activating the renin-angiotensin system and distal renal tubular reabsorption. GH stimulates the aerobic and anaerobic energy systems that underpin muscle and cardiovascular function. These pleiotropic actions explain the clinical picture of increased adiposity, reduced lean mass, and impaired physical and psychological function in the GHD adult, all of which are reversed when GH is replaced. Women require a greater replacement dose of GH than men. This is because androgens enhance while oestrogens attenuate GH action. The oestrogen effect is route-dependent, occurring with oral delivery blunting the liver-mediated actions of GH by directly inhibiting GH receptor signalling. Global experience spanning over 30 years have attested to the safety, efficacy and benefits of replacement therapy for adults with GHD.

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“…Recombinant GH has been approved for the treatment of adult GHD since 1996 and subsequently has been reimbursed for use in several countries for many years 7‐11 . However, GH was only approved for subsidy in Australia in December 2018 following advocacy of a professional medical working party to the relevant government agencies 12 . Criteria for subsidised therapy in Australia require biochemical confirmation of GHD established by one of three dynamic tests: the insulin tolerance test (ITT), the glucagon stimulation test (GST) or the arginine infusion test.…”

Section: Introductionmentioning

confidence: 99%

Reducing adverse events associated with the glucagon stimulation test for the assessment of growth hormone deficiency in adults with a high prevalence of pituitary hormone deficiencies

Gogna

Jung

McLachlan

et al. 2021

Clinical Endocrinology

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Design A retrospective review of the adverse events (AEs) in 78 patients during the glucagon stimulation test (GST) for the assessment of growth hormone deficiency (GHD) before and after protocol amendments which aimed to reduce AEs in a group of patients with a high prevalence of pituitary hormone deficiencies. Patients Based on our observations of frequent AEs during the standard GST protocol in an initial 25 patients (cohort 1), a modified protocol was introduced to include the routine administration of 20 mg of hydrocortisone pre‐GST in a subsequent 53 patients (cohort 2). Post hoc analysis of the effect of glucocorticoid dosing pre‐GST on AEs was examined in those receiving <20 mg hydrocortisone (group A, n = 19) vs ≥20 mg hydrocortisone (group B, n = 59). Measurements AEs including hypotension, hypoglycaemia and nausea/vomiting. Results Of the 78 patients undergoing the GST, 79% had ≥2 hormone deficiencies. Rates of AEs were 41% vs 30% for hypotension, 60% vs 28% for hypoglycaemia (p < .05) and 20% vs 13% for nausea/vomiting in cohort 1 compared with cohort 2, respectively. Post hoc analysis revealed lower rates of AEs in those receiving ≥20 mg hydrocortisone (group B) compared to those receiving <20 mg due to a reduction in hypoglycaemic events (82% vs 26%, p < .001) and hypotension (50% vs 27%, p = .05). Similar numbers of patients in group A and group B met criteria for GHD. Conclusions In patients with a high prevalence of pituitary deficiencies, a modified GST protocol of additional stress dose glucocorticoid attenuated the frequency of AEs without appearing to compromise the performance of the GST.

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

Cited by 3 publications

References 12 publications

Can old drugs learn new tricks? Achieving registration and public subsidy listing for off‐patent medicines with novel therapeutic applications

Can old drugs learn new tricks? Achieving registration and public subsidy listing for off‐patent medicines with novel therapeutic applications

The physiology of growth hormone (GH) in adults: translational journey to GH replacement therapy

Reducing adverse events associated with the glucagon stimulation test for the assessment of growth hormone deficiency in adults with a high prevalence of pituitary hormone deficiencies

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