罕见病仍是无人问津的孤儿还是已被欣然接纳？开发和使用孤儿药面临的挑战。
Are rare diseases still orphans or happily adopted? The challenges of developing and using orphan medicinal products.
作者信息
Dear James W, Lilitkarntakul Pajaree, Webb David J
机构信息
Clinical Pharmacology Unit, Centre for Cardiovascular Science, University of Edinburgh, Queen's Medical Research Institute, Edinburgh, UK.
出版信息
Br J Clin Pharmacol. 2006 Sep;62(3):264-71. doi: 10.1111/j.1365-2125.2006.02654.x.
Abstract
Orphan medicinal products (OMPs) are targeted at the diagnosis, prevention or treatment of rare diseases and have a special status in European law. This status brings incentives for pharmaceutical companies to invest in OMP development. The goal of the legislation is to encourage the development of more treatments for life-threatening rare disorders, but increased availability of OMPs raises important issues surrounding the public funding of very expensive treatments by national health services. In this article we review OMPs and the incentives for their development and discuss the challenges presented by funding these treatments.
摘要
孤儿药是针对罕见病的诊断、预防或治疗的药物，在欧洲法律中具有特殊地位。这种地位为制药公司投资孤儿药研发带来了激励。该立法的目标是鼓励开发更多针对危及生命的罕见疾病的治疗方法，但孤儿药可及性的提高引发了围绕国家医疗服务体系为非常昂贵的治疗提供公共资金的重要问题。在本文中，我们回顾了孤儿药及其研发激励措施，并讨论了为这些治疗提供资金所带来的挑战。
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