Rare diseases — improving care and quality of life

SUMMARY OF:

Recommendation (2009/C 151/02) – action in the field of rare diseases

WHAT IS THE AIM OF THE RECOMMENDATION?

The 2009 recommendation from European Union (EU) countries on diagnosing, treating and caring for sufferers followed on from an EU strategy on rare diseases adopted in 2008.

KEY POINTS

• National plans and strategies: the EUCERD joint action programme (March 2012 to August 2015) supports EU governments in developing national strategies. By early 2014, 16 had national plans in place (compared to only four in 2008) and seven others were well advanced.
• Definition and codification: for EU purposes, a rare disease affects no more than 5 per 10,000 persons. The EU has established a database, Orphanet, which links together over 6,000 diseases and operates in 7 languages. By the end of 2013, it had identified 2,512 specific rare diseases patient organisations.
• Research: the EU has funded almost 120 collaborative research projects with a total budget of over €620 million. In early 2011, it launched the International Rare Diseases Research Consortium (IRDiRC). This aims to deliver 200 new therapies by 2020. It supports the creation of patient registries and databases and is developing a European platform on rare diseases registration.
• Centres of expertise: the EU-co-funded 4-year rare diseases best practices project (January 2013-December 2016) is helping develop standards and procedures for clinical practice guidelines. In 2013 an expert group was created to advise the Commission.

The 2014 report presents an overview of the implementation of the rare diseases strategy to date. This focuses on improving the recognition and visibility of rare diseases, measures to help EU countries address the phenomenon and moves to develop wider EU-level cooperation, coordination and regulation.

BACKGROUND

• Some 5,000 to 8,000 rare diseases exist, affecting between 27 and 36 million people in the EU. The patients, because in national terms their numbers are relatively limited, often spend years waiting for a diagnosis. The EU and cooperation between EU countries can make a difference by pooling knowledge, expertise, research and Europe-wide authorisation for the best possible medicines.
• For more information, see:
  • ‘Rare diseases’ on the European Commission's website.

MAIN DOCUMENT

Council Recommendation of 8 June 2009 on an action in the field of rare diseases (2009/C 151/02) (OJ C 151, 3.7.2009, pp. 7-10)

RELATED DOCUMENTS

Communication from the Commission to the European Parliament, the Council, the European Economic and Social Committee and the Committee of the Regions on Rare Diseases - Europe's challenges (COM(2008) 679 final, 11.11.2008)

Report from the Commission to the European Parliament, the Council, the European Economic and Social Committee and the Committee of the Regions: Implementation report on the Commission communication on rare diseases: Europe’s challenges (COM(2008) 679 final) and Council Recommendation of 8 June 2009 on an action in the field of rare diseases (2009/C 151/02) (COM(2014) 548 final of 5.9.2014)

Commission Decision 731/2013/EU of 30 July 2013 setting up a Commission expert group on rare diseases and repealing Decision 2009/872/EC (OJ C 219, 31.7.2013, pp. 4-7)

last update 01.12.2016