€144m Fund to Provide 200 Rare Disease Therapies by 2020

The European Commission (EC) has announced €144 million in funding to successfully diagnose most rare diseases and provide 200 therapies by 2020.



The fund will be distributed among 26 new rare disease research projects, with the aim of improving the lives of some of the 30 million Europeans suffering from a rare disease.

The projects will involve over 300 participants from 29 countries in Europe, including teams from prestigious academic institutions, SMEs, and patients’ groups. Through pooling resources and working across national borders, researchers hope to gain a better understanding of rare diseases and to develop treatments for those suffering from them.

Máire Geoghegan-Quinn, European Commissioner for Research, Innovation and Science, explained that “Most rare diseases affect children and most of them are devastating genetic disorders resulting in greatly reduced quality of life and premature death. We hope that these new research projects will bring patients, their families and health professionals closer to a cure and support them in their daily battle with disease.”

The projects will address a broad spectrum of rare diseases, including cardiovascular, metabolic and immunological disorders. Their aims are to develop substances that may be useful as therapies; to advance scientific understanding of rare diseases’ origins and mechanisms; to improve diagnosis rates and to improve management of rare diseases in clinical settings. Many of the new projects will contribute to the work of the International Rare Disease Research Consortium (IRDiRC), currently the largest collective rare diseases research effort worldwide, which was launched in April 2011.

A disease is defined as ‘rare’ in Europe if it effects less than 1 in 2,000 of the population; there are thought to be between 6,000 – 8,000 rare diseases, which equate to a significant number of the population when taken together. When these latest projects are taken into account, the total number of rare disease research projects funded by the EC will total close to 100, with the total amount of funding for these projects equalling just under €500 million.

One of the challenges of developing drugs for rare or ‘orphan’ diseases is the difficulty in finding patients for clinical trials, which is why patient advocacy has become such an important tool in recent rare disease research efforts. Patients with rare diseases have often been ahead of the current trend when it comes to the formation of patient communities online, as their scarcity means they have always needed to band together in order to make their voice heard. The EC’s announcement, which took place on Rare Disease Day, represents a step taken by Europe in order to better listen to that voice.


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