Ong Natalie, McMeniman Erin, Pillans Peter, Soyer H Peter
Department of Dermatology, Princess Alexandra Hospital, Brisbane, Australia.
Dermatology Research Centre, The University of Queensland, School of Medicine, Translational Research Institute, Brisbane, Australia.
Australas J Dermatol. 2017 Feb;58(1):30-34. doi: 10.1111/ajd.12392. Epub 2015 Sep 7.
BACKGROUND/OBJECTIVES: The use of high-cost, off-label, unsubsidised drugs has become valuable in the management of dermatology patients with challenging conditions unresponsive to conventional therapy. Currently, there is no dedicated funding and a paucity of evidence for such drugs. The aim of this audit was to review outcomes and costs.
A retrospective clinical audit of high-cost off-label dermatology drug applications to the High Cost Drug's Subcommittee was undertaken at a tertiary public hospital in Brisbane, Queensland, between 2002 and 2013.
Of 28 applications, 25 were approved. The medications included thalidomide, mycophenolate mofetil, cyclosporin, infliximab, rituximab and adalimumab. Over 70% of patients responded to treatment. Individual annual costs for these medications ranged from $2068 to $36 984. Adverse effects resulted in drug withdrawal in five cases.
Despite the absence of dedicated funding for high-cost drugs and a rigorous committee approval process, most applications were approved, of which >70% benefited from treatment. This audit provides useful clinical experience with off-label use of high-cost drugs in difficult dermatological conditions.
背景/目的：在治疗对传统疗法无反应的具有挑战性皮肤病患者时，使用高成本、未注册、无补贴的药物已变得很有价值。目前，此类药物没有专项资金，且证据不足。本次审计的目的是审查疗效和成本。
2002年至2013年期间，在昆士兰州布里斯班的一家三级公立医院，对向高成本药物小组委员会提交的高成本未注册皮肤科药物申请进行了回顾性临床审计。
28份申请中，25份获得批准。这些药物包括沙利度胺、吗替麦考酚酯、环孢素、英夫利昔单抗、利妥昔单抗和阿达木单抗。超过70%的患者对治疗有反应。这些药物的个人年度费用从2068美元到36984美元不等。不良反应导致5例患者停药。
尽管高成本药物没有专项资金，且需经过严格的委员会审批程序，但大多数申请仍获得批准，其中超过70%的患者从治疗中获益。本次审计为在困难皮肤病情况下使用高成本未注册药物提供了有益的临床经验。
