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 journalArticlepeer-review

    1 Citation (Scopus)

    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

    Keywords

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

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