Orphan drug development

PubMed, the Internet portal of biomedical and life sciences literature, indexed an interesting article, entitled “Orphan drug development is progressing too slowly” (Br J Clin Pharmacol. 2006 Mar;61(3):355-360). Authors are Joppi R, Bertele V and Garattini S from the Mario Negri Institute for Pharmacological Research, Milan, Italy. The aims are to assess the methodological quality of OMP (Orphan Medical Products) dossiers and to discuss possible reasons for the small number of products licensed. Information about orphan drug designation and approval was obtained from the website of the European Commission-Enterprise and Industry DG and from the European Public Assessment Reports. Conclusions are that the paucity of European incentives for manufacturers and the poor documentation underpinning the applications may have limited the number of new OMP. The over 5000 rare diseases awaiting therapy are an important public health issue. To access the full abstract of the article, click here.