Orphan drugs rarely approved 

A report recently published in the British Journal of Clinical Pharmacology assessed the methodological quality of OMP (orphan medical products) dossiers and discussed possible reasons for the small number of products licensed in Europe. 

According to the report, 255 possible drugs for rare diseases were reviewed by the EMEA 's (European Medicines Agency) committee for orphan medical products between August 2000 and December 2004. Only 18 products (7.1%) were approved, of which 10 under exceptional circumstances requiring additional studies to maintain market authorisation. During the same period, 79% of other drug applications submitted to EMEA were approved. 

The study states that the OMP dossiers were often poor in methodology, such as inappropriate clinical design, and suggests that the poor documentation supporting the applications may have limited the number of new orphan medicinal products. It also concludes with a suggestion that manufacturers need more EU incentives to develop OMP.

"It’s difficult to find a balance between the urgent need for drugs for patients with rare diseases and guaranteeing their quality, efficacy, safety and, where necessary, making comparisons with existing drugs," said co-author of the study Professor Silvio Garattini. 

Orphan medicines are those intended for rare and life threatening diseases or medicines that are unlikely to be developed under normal market conditions. Over 5000 rare diseases are waiting for a cure to be invented.