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Intern Med J. 2018 Aug;48(8):999-1002. doi: 10.1111/imj.13943.

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

Author information

1
Sydney Health Ethics, University of Sydney, Sydney, New South Wales, Australia.
2
Department of Endocrinology and Diabetes, Sydney Children's Hospital Network-Westmead, Sydney, New South Wales, Australia.
3
Discipline of Child and Adolescent Health, The University of Sydney, Sydney, New South Wales, Australia.
4
Southern Adelaide Diabetes and Endocrine Services, Flinders Medical Centre, Flinders University, Adelaide, South Australia, Australia.
5
School of Medicine, Flinders University, Adelaide, South Australia, Australia.
6
Department of Endocrinology and Diabetes, Women's and Children's Hospital, Adelaide, South Australia, Australia.
7
Department of Diabetes and Endocrinology, Princess Alexandra Hospital, Brisbane, Queensland, Australia.
8
Faculty of Medicine, University of Queensland, Brisbane, Queensland, Australia.
9
Department of Endocrinology and Diabetes, Royal Children's Hospital, Victoria, Australia.
10
Centre for Hormone Research, Murdoch Children's Research Institute, Victoria, Australia.
11
Department of Paediatrics, The University of Melbourne, Victoria, Australia.
12
Centres for Health Research, Princess Alexandra Hospital, Brisbane, Queensland, Australia.

Abstract

Growth hormone (GH) replacement therapy was recently recommended by the Pharmaceutical Benefits Advisory Committee (PBAC) for listing on the Pharmaceutical Benefits Scheme for adults with severe GH deficiency and impaired quality of life. This approval was significant for two reasons. First, the application was initiated and coordinated by a health professional working group, who prepared a 'public interest' submission to PBAC. Second, it resulted in a recommendation to subsidise therapy for a rare disease after two prior rejections on the basis of uncertainty about efficacy and cost effectiveness. There are important lessons to learn about the power of professional groups to drive health policy and attain funding for rare diseases.

KEYWORDS:

clinical advocacy; growth hormone; pharmaceutical benefits scheme; public interest submission; rare disease

PMID:
30133980
DOI:
10.1111/imj.13943
[Indexed for MEDLINE]

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