The European Union Policy in the Field of Rare Diseases

Abstract

Rare diseases, are defined by the European Union as life-threatening or chronically debilitating diseases with low prevalence (less than 5 per 10,000). The specificities of rare diseases – limited number of patients and scarcity of relevant knowledge and expertise – single them out as a unique domain of very high European added-value.

The legal instruments at the disposal of the European Union, in terms of the Article 168 of the Treaties, are very limited. However a combination of instruments using the research and the pharmaceutical legal basis and an intensive and creative use of funding from the Health Programmes has permitted to create a solid basis that Member States have considered enough to put rare diseases in a privileged position in the health agenda.

The adoption of the Commission Communication, in November 2008, and of the Council Recommendation, in June 2009, and in 2011 the adoption of the Directive on Cross-border healthcare., have created an operational framework to act in the field of rare disease with European coordination in several areas (classification and codification, European Reference Networks, orphan medicinal products, the Commission expert group on rare diseases, etc.).

Rare diseases is an area with high and practical potential for the European cooperation.