Are rare diseases still orphans or happily adopted? The challenges of developing and using orphan medicinal products

James W Dear, Pajaree Lilitkarntakul, David J Webb

Research output: Contribution to journalArticlepeer-review

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.
Original languageEnglish
Pages (from-to)264-71
Number of pages8
JournalBritish Journal of Clinical Pharmacology
Volume62
Issue number3
DOIs
Publication statusPublished - 28 Apr 2006

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