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First, the application was initiated and coordinated by a health professional working group, who prepared a \u2018public interest\u2019 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.<\/jats:p>","DOI":"10.1111\/imj.13943","type":"journal-article","created":{"date-parts":[[2018,8,8]],"date-time":"2018-08-08T01:09:06Z","timestamp":1533690546000},"page":"999-1002","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":3,"title":["A will and a way to fund medicines for rare diseases: the story of human growth hormone replacement for adults with growth hormone deficiency"],"prefix":"10.1111","volume":"48","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-0234-657X","authenticated-orcid":false,"given":"Wendy","family":"Lipworth","sequence":"first","affiliation":[{"name":"Sydney Health Ethics University of Sydney  Sydney New South Wales Australia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Geoffrey","family":"Ambler","sequence":"additional","affiliation":[{"name":"Department of Endocrinology and Diabetes Sydney Children's Hospital Network\u2010Westmead  Sydney New South Wales Australia"},{"name":"Discipline of Child and Adolescent Health The University of Sydney  Sydney New South Wales Australia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6468-7727","authenticated-orcid":false,"given":"Morton G.","family":"Burt","sequence":"additional","affiliation":[{"name":"Southern Adelaide Diabetes and Endocrine Services, Flinders Medical Centre Flinders University  Adelaide South Australia Australia"},{"name":"School of Medicine Flinders University  Adelaide South Australia Australia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jan","family":"Fairchild","sequence":"additional","affiliation":[{"name":"Department of Endocrinology and Diabetes Women's and Children's Hospital  Adelaide South Australia Australia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5078-4980","authenticated-orcid":false,"given":"Warrick J.","family":"Inder","sequence":"additional","affiliation":[{"name":"Department of Diabetes and Endocrinology Princess Alexandra Hospital  Brisbane Queensland Australia"},{"name":"Faculty of Medicine University of Queensland  Brisbane Queensland Australia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"George","family":"Werther","sequence":"additional","affiliation":[{"name":"Department of Endocrinology and Diabetes Royal Children's Hospital  Victoria Australia"},{"name":"Centre for Hormone Research Murdoch Children's Research Institute  Victoria Australia"},{"name":"Department of Paediatrics The University of Melbourne  Victoria Australia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ken","family":"Ho","sequence":"additional","affiliation":[{"name":"Department of Diabetes and Endocrinology Princess Alexandra Hospital  Brisbane Queensland Australia"},{"name":"Faculty of Medicine University of Queensland  Brisbane Queensland Australia"},{"name":"Centres for Health Research Princess Alexandra Hospital  Brisbane Queensland Australia"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2018,8,7]]},"reference":[{"key":"e_1_2_4_2_1","doi-asserted-by":"publisher","DOI":"10.1210\/jc.2011-0179"},{"key":"e_1_2_4_3_1","doi-asserted-by":"publisher","DOI":"10.1530\/EJE-07-0631"},{"key":"e_1_2_4_4_1","unstructured":"National Institute for Clinical Excellence. 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