Abstract: 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 , 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.