The Pharmaceutical Journal
Vol 276 No 7392 p313
18 March 2006

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Fewer than one in every 14 orphan drug applications is approved

Developers of orphan drugs need more support from governments, the authors of a paper analysing European Medicines Agency (EMEA) approval data have concluded (British Journal of Clinical Pharmacology 2006;61:355). Fewer than one in every 14 applications for orphan drug designation are approved, compared with 80 per cent of all marketing authorisation applications, the researchers found.

Between August 2000, when new EU legislation came into force, and December 2004, 255 orphan medicinal product designation applications were made, but only 18 of these (7.1 per cent) were approved. Over the same period a total of 193 marketing authorisation applications were submitted to the EMEA and 153 drugs were licensed (79.3 per cent).

“The fact that it took four years to develop 18 drugs and there are still several thousand rare diseases awaiting therapy is a public health issue that cannot be neglected,” the authors say. “There is an urgent need to establish programmes setting aside a special fund and providing tax relief for sponsors producing orphan medicinal products,” they add.

Of the 18 that were approved, 10 were authorised “under exception circumstances” because additional studies were required. In general, the application dossiers lacked dose-finding studies, controlled studies and studies with active comparators. Applications were also frequently based on insufficient numbers of patients and endpoints with little clinical relevance, the authors say.