European Journal of Molecular & Clinical Medicine

Abstract

The last two decades has seen a significant increase in the development of medicinal products to treat rare “orphan” diseases, largely due to the EU Orphan Medicinal Product Regulation (2000), but also because of the consistent advocacy by patient groups prior to this regulation. These groups from across Europe joined forces under the umbrella of EURORDIS, to secure the implementation of the regulation and ensure that there was patient representation on the committee at EMA (the European Medicines Agency) which would be responsible for assessing and giving opinions on applications, namely COMP (The Committee for Orphan Medicinal Products). As its Vice-Chair I have the privilege to ensure the patient perspective is always considered during these assessments.