This year Rare Disease Day will serve to highlight the commitment of the European Union to enhance research in the field of rare diseases, as stated in the Council Recommendation for Action in the field of Rare Diseases, adopted by all EU Health Ministeres in June 2009.

What is the European added value?
Conducting research at the European level is of utmost importance because it is the only way to increase the number of patients available for each study and to gather the scattered specialists with complementary expertise allowing for the necessary multidisciplinary approaches and the exchange of good practice. 

“Patients are scattered all over Europe which makes setting up cohorts and clinical trials difficult, clinical and scientific data is largely not centralised, there is insufficient funding for research teams, a relative lack of interest from the pharmaceutical industry and a need for a multidisciplinary and horizontal cross-cutting approach. These factors make rare diseases the perfect model for Europe to use to position itself at the forefront of R&D.”

The European Commission has acknowledged the importance of working at the European level in the field of rare diseases by issuing a Council Recommendation  on a European Action in the field of Rare Diseases, adopted in 2009, in which Research has an important place.

The need for more and better Funding
Research on rare diseases requires shared supranational common infrastructures, long-lasting projects and a sustained approach. However, because of the rarity of the diseases, and thus their limited commercial interest, it is very unlikely that a private sponsor would take over the long term funding needed for rare disease research infrastructures.  Therefore research in the field of rare diseases needs long-lasting infrastructures, such as biobanks, databases and registries, to benefit from sustainable public funding.

There must be enough money for long-term research projects on specific rare diseases or groups of diseases, for both fundamental and clinical research projects. This issue is closely linked with the need for sustainable European Reference Networks of Centres of Expertise and European Research Networks, with a view at enhancing translational research.

Shaping the EU Research Agenda
The Framework Programmes for Research and Technological Development, also called Framework Programmes or abbreviated FP, are the European Union’s main instrument for funding research in. In order to shape the EU research agenda today and in the future, the arguments described above, together with the results of this Rare Disease Day campaign will serve as ammunition to convince policy-makers that rare diseases have to be paid attention to and that they should remain high in the Framework Programmes for Research of the European Union. 

Until 2013, funds for research will be distributed under the 7th FP. The specific objectives and actions vary between funding periods, it is therefore very important to start advocating for the inclusion of rare disease research in the 8th FP as of now.

Advocating for more funding in the Framework Programmes cannot be disassociated from political victories such as the Council Recommendation, which include, more and better rare disease research as an important objective.  This issue is closely linked with another main objective of the Recommendation which is to identify Centres of Expertise and foster their participation in European Reference Networks, with a view at enhancing translational research.

In order to shape the EU research agenda today and in the future, rare disease patient groups are mobilising to make sure rare diseases remain high in the public research agenda and that they be given priority in the relevant funding schemes.

To this end, EURORDIS is organising an event in Brussels on March 1st, 2010. The results of the EURORDIS Survey on Patients' involvement in research, will be presented at this event. To know more about the event, click here.
To know more about the survey, click here.