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Orphan drug development is progressing too slowly
Article first published online: 20 JAN 2006
DOI: 10.1111/j.1365-2125.2006.02579.x
Additional Information
How to Cite
Joppi, R., Bertele, V. and Garattini, S. (2006), Orphan drug development is progressing too slowly. British Journal of Clinical Pharmacology, 61: 355–360. doi: 10.1111/j.1365-2125.2006.02579.x
Publication History
- Issue published online: 20 JAN 2006
- Article first published online: 20 JAN 2006
- Received 27 May 2005 Accepted 3 October 2005
- Abstract
- Article
- References
- Cited By
Keywords:
- development;
- orphan drugs;
- rare diseases
Aims
To assess the methodological quality of OMP dossiers and to discuss possible reasons for the small number of products licensed.
Methods
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.
Results
Out of 255 OMP designations, only 18 were approved (7.1%). Their dossiers often showed methodological limitations such as inappropriate clinical design, lack of active comparator where available and use of surrogate end-points.
Conclusions
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.