Orphan drugs in haematology

More than half (55%) of the drugs that received a positive opinion on orphan designation are for medical conditions affecting children (Fig. 1). Orphan drug designations for haematological conditions represent a substantial portion of all OD designations granted in the period 2000–2009 in EU.

According to the EU regulation 141/2000 [1], all cancers, with the exception of breast, lung, colorectal, prostate and bladder, are rare diseases, with the consequence that all haematological diseases are orphan diseases, with 70% of orphan designated conditions affecting less that 1 in 10,000 people. In the period 2000–2007 orphan designed medicines to treat oncological (17%) and haematological (33%) conditions have received more marketing authorisations from EC than those of any other therapeutic area.