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.

译文

:孤儿药(OMP)专门用于罕见疾病的诊断,预防或治疗,在欧洲法律中具有特殊地位。这种状况为制药公司投资OMP开发带来了动力。立法的目的是鼓励开发更多威胁生命的罕见疾病的治疗方法,但是OMPs的可用性增加引起了重要问题,围绕着由国家卫生服务机构提供昂贵治疗的公共资金。在本文中,我们回顾了OMP及其发展的诱因,并讨论了为这些治疗提供资金所带来的挑战。

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