Orphan Drug Designations reach 1000
The Committee for Orphan Medicinal Products has reached its 1000th positive opinion for orphan drug designations – an encouraging sign of an increasingly active environment for drug development for rare diseases in Europe.
This will be very welcome news for the rare disease community meeting this week in Brussels at the European Conference on Rare Diseases and Orphan Products (ECRD 2012 Brussels).
The 1000th positive opinion was provided by the Committee for Orphan Medicinal Products (COMP). Most designations are for rare cancers (39%); neuromuscular diseases (11%) and metabolic diseases (9%). More than half of designations are for very rare conditions. To date, 75 of these designated medicinal products have received marketing authorisation, and potentially can benefit 3 million patients in Europe.
Since 2007 a common EMA/FDA orphan drug designation application is used however 97 designations were granted in 2011 compared to 200 designations in the US for the same period. Therefore EURORDIS encourages stronger collaboration between the European Medicines Agency and the US Food and Drug Administration.
“EURORDIS celebrates these 12 years of success but still has concerns,” says Yann Le Cam, EURORDIS’ Chief Executive Officer and former COMP Vice Chair. “We call for a stronger commitment of the European Commission, Member States and industry to improve patients’ access to orphan drugs and to stimulate research for diseases for which there are currently no treatments. In addition the success rate between orphan drug designations and market authorisations must be improved.”
More on orphan drug designations on EURORDIS website’s new section About orphan drugs
View EMA video on medicines for rare diseases featuring Lesley Greene, EURORDIS patient representative at the COMP