Rare diseases in Europe : tomorrow's vision, a common vision
This year's European Conference on Rare Diseases, held in Luxembourg on 21 and 22 June was, above all, an occasion for reinforcing the importance of partnerships and of sharing experiences at all levels.
Over two days of stimulating presentations, combining personal stories with updates on European initiatives, a wide spectrum of stakeholders answered the key question asked by Terkel Andersen, president of Eurordis, at the beginning of the conference: 'How do we make the most of Europe by combining resources?'.
Fernard Sauer, Director for Public Health at the European Commission, talked of the importance of a global approach to rare diseases in research, information and training, social benefits, hospitalisation and outpatient treatment, and the need for special combined efforts to prevent morbidity, early mortality and the reduction of individuals' quality of life.
Other speakers highlighted many of the existing bottlenecks to progress: lack of epidemiological data, difficulties in coding rare diseases, national legislation, lack of national funding for research, lack of funding continuity, 'postal code lottery' diagnosis... But solutions to these problems were also presented with many local experiences proving that, through partnership, it is possible to improve the quality of life of patients.
Yann Le Cam, Chief Executive of Eurordis, joined many speakers in applauding the success of the EU orphan drugs regulation, this year celebrating its fifth anniversary. But looking beyond this success he highlighted five key tasks for the future :
The take-home messages for professionals in the field, as perceived by Segolene Ayme, leader of the Rare Diseases Task Force, included the following requests:
For those of you who were unable to attend, the presentations from the conference will be accessible on the conference website.
This month's newsletter highlights many of the initiatives already in place, or being planned, to increase collaboration between countries and between stakeholders in Europe: from European clinical trials networks to the efforts of the primary immunodeficiencies community to bring patients together with doctors and researchers. Europe is moving in the right direction.
The next rendez-vous for the rare diseases community in Europe will be in London in October when EPPOSI - the European Platform for Patients' Organisations, Science and Industry - organises its annual workshop on Partnering for Rare Disease Therapy Development, this year in collaboration with US colleagues from NORD and NIH. The workshop theme 'People with Rare Diseases - No Longer Alone in the World' reflects the feelings expressed by many present at the Luxembourg event. Rare diseases are firmly on the public agenda and now we must work together - nationally, internationally - to ensure that they stay there.
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Deadline for the next issue is 1 September 2005.