Division Rare diseases

Technical action to support the development of the Orphanet database on rare diseases and orphan drugs which is run by a large consortium of European partners and which is the most important rare diseases database in the world. In order to implement the establishment of a dynamic EU inventory of rare diseases it will be necessary to further develop the database. The aim is to implement actions in the Commission Communication and in the Council Recommendation for a European Action in the field of rare diseases^f1 by providing a single reference point adding value at European level by bringing together all relevant information on rare diseases.

[Joint action]

Technical action to support the European Surveillance on Congenital Anomalies (Eurocat) network which is run by a large consortium of European partners in order to create a sustainable prevalence data system for 90 congenital anomaly subgroups which are to be updated annually. The aim is to implement actions identified in the Commission Communication and in the Council Recommendation for a European Action in the field of rare diseases.

[Joint action]

Creation of a mechanism for the exchange of knowledge between Member States and European authorities on the scientific assessment of the clinical added value for orphan medicines. The aim is to implement actions in the Commission Communication and in the Council Recommendation for a European Action in the field of rare diseases by sharing information in this area between Member States as efficiently as possible.

[Call for tenders]

Improving and refining definition of rare diseases taking into account incidence aspects as defined in the Commission Communication and in the Council Recommendation for a European Action in the field of rare diseases. The aim is to implement actions in the Commission Communication and in the Council Recommendation for a European Action in the field of rare diseases, reflecting in particular the concerns around the definition of rare diseases expressed during the discussion in the European Parliament.

[Call for proposals for projects]

Support to the creation of new registers on rare diseases. These registries should constitute key instruments to increase knowledge on rare diseases and develop clinical research. They are the only way to pool data in order to achieve a sufficient sample size for epidemiological research and/or clinical research. Collaborative efforts to establish data collection and maintain them will be considered, provided that these resources are open and accessible. The aim is to implement actions in the Commission Communication and in the Council Recommendation for a European Action in the field of rare diseases as well as in the High level Pharmaceutical Forum recommendations.

[Call for proposals for projects]

Support to the continuation of existing performing EU networks on information and registers on rare diseases in several areas (e.g. Ercusyn, Regiscar, EuroMyasthenia). The aim is to implement actions in the Commission Communication and in the Council Recommendation for a European Action in the field of rare diseases as well as in the High level Pharmaceutical Forum recommendations.

[Operating grants]