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

作者：Lipworth Wendy^*; Ambler Geoffrey; Burt Morton G; Fairchild Jan; Inder Warrick J; Werther George; Ho Ken

来源：Internal Medicine Journal, 2018, 48(8): 999-1002.

DOI：10.1111/imj.13943

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

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.

• 出版日期2018-8