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Rare diseases - improving care and quality of life

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Rare diseases - improving care and quality of life

Rare diseases affect millions of people. In 2008 the Commission adopted an overall strategy, followed a few months later by a recommendation from European Union governments on diagnosing, treating and caring for sufferers. The 2014 report assesses the progress made.

ACT

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

SUMMARY

Rare diseases affect millions of people. In 2008 the Commission adopted an overall strategy, followed a few months later by a recommendation from European Union governments on diagnosing, treating and caring for sufferers. The 2014 report assesses the progress made.

WHAT DO THE RECOMMENDATION AND REPORT DO?

The 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.

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.

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 the section on rare diseases on the European Commission’s website.

RELATED ACTS

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 (Official Journal C 219, 31.7.2013, pp. 4-7).

Last updated: 05.01.2015

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