Lipworth Wendy, Ambler Geoffrey, Burt Morton G, Fairchild Jan, Inder Warrick J, Werther George, Ho Ken
Sydney Health Ethics, University of Sydney, Sydney, New South Wales, Australia.
Department of Endocrinology and Diabetes, Sydney Children's Hospital Network-Westmead, Sydney, New South Wales, Australia.
Intern Med J. 2018 Aug;48(8):999-1002. doi: 10.1111/imj.13943.
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.
生长激素（GH）替代疗法最近被药品福利咨询委员会（PBAC）推荐列入药品福利计划，用于治疗严重生长激素缺乏且生活质量受损的成年人。这一批准具有重要意义，原因有两点。其一，该申请由一个卫生专业人员工作小组发起并协调，他们向PBAC提交了一份“公共利益”申请。其二，此前基于疗效和成本效益的不确定性曾两次遭到拒绝，而此次却促成了对一种罕见病治疗给予补贴的建议。关于专业团体推动卫生政策以及为罕见病获取资金的能力，有重要的经验教训可供汲取。
