A disease or disorder is defined as rare in Europe when it affects not more than 1 in every 2,000 persons. Yet, because there are so many different rare diseases – between 6,000 and 8,000 – taken altogether they affect a significant share of the population. Today, the Irish Presidency of the Council of the European Union will host an all day conference in Dublin to discuss the key issues in this area.
In the EU, as many as 30 million people suffer from a rare disease, and many of them are children. The European Commission announced that the €144 million is to be distributed between the 26 projects will go towards covering a broad spectrum of rare diseases such as cardiovascular, metabolic and immunological disorders. They will aim at:
- developing substances that may become new or improved therapies for patients
- understanding better the diseases' origins and mechanisms
- better diagnosing rare diseases
- improving the management of rare diseases in hospital and healthcare settings
Teams will work on varied challenges, including: a new 'bioartificial' liver support system to treat acute liver failure; powerful data processing operations to develop novel diagnostic tools, biomarkers and screening strategies for therapeutic agents against rare kidney diseases; and the clinical development of a drug to treat alkaptonuria, a genetic disorder which leads to a severe and early-onset form of arthritis, heart disease and disability for which there is currently no effective treatment.
Many of the new projects will contribute to the International Rare Diseases Research Consortium (IRDiRC), the biggest collective rare diseases research effort world-wide. Initiated by the European Commission, together with national and international partners, its key objective is to deliver, by 2020, 200 new therapies for rare diseases and the means to diagnose most of them. The new projects will bring the number of EU-funded collaborative research projects related to rare diseases to close to 100 over the last six years. Altogether, they represent an investment of almost 500 million euro. This effort can be placed in the larger perspective of EU regulations that are driving the development of novel therapies for treating rare diseases, and at the same time capitalising on advances and discoveries coming out of Europe’s science base.
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