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

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.

journal_name

Intern Med J

authors

Lipworth W,Ambler G,Burt MG,Fairchild J,Inder WJ,Werther G,Ho K

doi

10.1111/imj.13943

subject

Has Abstract

pub_date

2018-08-01 00:00:00

pages

999-1002

issue

8

eissn

1444-0903

issn

1445-5994

journal_volume

48

pub_type

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