Orphan drug development is progressing too slowly.
OBJECTIVE
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.
Joppi
Roberta
R
Mario Negri Institute for Pharmacological Research, Milan, Italy.
Bertele
Vittorio
V
Garattini
Silvio
S
D000818:Animals; D004282:Documentation; D004345:Drug Industry; D005062:European Union; D006307:Health Systems Agencies; D004364:Pharmaceutical Preparations; D012106:Research / Q000639:trends; D012189:Retrospective Studies
D004364:Pharmaceutical Preparations