The Pharmaceutical Journal
Vol 277 No 7408 p39
8 July 2006

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Orphan drug system works, Europe concludes

Europe’s orphan drug incentive programme has led to 22 new products being licensed for 20 rare conditions in the five years that the system has been in existence (PDF, 560K). Previously, there were no satisfactory treatments for eight of the 20 conditions. Development work is continuing on a further 246 orphan drugs out of a total of 450 applications for orphan designation.

European commissioner for enterprise and industry Günter Verheugen said:“The results show that we are on the right track. Already, more than one million patients suffering from orphan diseases in the EU may benefit from the new orphan medicines. In addition, the EU legislation has stimulated industrial activity leading to company creation with promising high-tech potential.”

Companies can seek orphan drug status for any treatment intended for conditions affecting less than five in 10,000 people in the European Community or which would be unlikely to be marketed because the return on investment would be too small. Only two orphan drug applications have been made on grounds of insufficient return.

Drugs granted orphan status benefit from a 10-year period of market exclusivity, plus reduced European Medicines Agency (EMEA) licensing fees, research support and advice from the EMEA on testing the drugs during development. Just over half (53 per cent) of applications for orphan drug status were for innovative products. The remainder were for new indications for existing drugs.