Orphan drugs in haematology

Article type: Research Article

Authors: Zanon, Paola

Affiliations: Celgene, Milan, Italy. E-mail: pzanon@celgene.com

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 [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.

Keywords: Haematology, cancer, rare disease, orphan

DOI: 10.3233/PPL-2010-0292

Journal: Pharmaceuticals, Policy and Law, vol. 12, no. 1-2, pp. 157-159, 2010