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

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

Research output: Contribution to journalArticle

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.

Original languageEnglish
Pages (from-to)999-1002
Number of pages4
JournalInternal Medicine Journal
Volume48
DOIs
Publication statusPublished - 2018
Externally publishedYes

Keywords

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

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