EMA approved a record number of medicines for rare diseases in 2014

According to an EMA press release, the European Medicines Agency in 2014 recommended the highest number of orphan drugs for marketing authorisation in a year. 17 out of 82 medicines that were approved in 2014, are inteded for the treatment of rare diseases. The new medicines for rare diseases include the first medicine for the treatment of Duchenne’s Muscular Dystrophy (DMD), and eight new medicines for rare cancer. DMD as well as rare cancers are among those diseases which the IDeAl group intends to study in detail. The increasing number of medicines receiving a recommendation for marketing authority in 2014 emphasizes the importance of the innovative statistical methods for small population groups that are investigated and developed by the IDeAl project.