2007 Commission conference on rare disease research

One of the characteristics of rare diseases is that research is insufficient in regards to the needs of patients, who are without cure today. In this context the initiative of the European Commission to organise a conference on rare disease research at the European Parliament in Brussels on 13 September 2007 came as a sign of maturity of the rare disease community and as a strong political signal.

‘The conference was entitled “Rare Disease Research: Building on Success”,’ says Fabrizia Bignami, Eurordis’ Therapeutic Development Officer and member of the conference Programme Committee. ‘With this event, the Commission wanted to raise awareness at the level of Member States and European Parliament on the needs of research on rare diseases, as well as promote rare diseases as a priority of the European Union’s political research agenda.’ It was a welcomed opportunity for patient representatives to express their needs in terms of research. About twenty patient representatives, identified and selected by Eurordis, had their travel and accommodation expenses reimbursed by the Commission, in coordination with Eurordis. The results of the Eurordis workshop on “Gaining access to rare disease research resources” of 4-5 May 2007 in Paris, which had also been presented at the Commission Conference preparatory workshop on rare disease research on 14 June 2007 in Brussels, were part of a presentation made by John Burn, Medical Director and Head of the Institute of Human Genetics, University of Newcastle. Concluding remarks were made by Christel Nourissier, Eurordis Board Member and Secretary General. ‘The outcomes of this conference were used as a basis for DG Research’s input in the current Commission Communication on Rare Diseases,’ she says.

Along with patient representatives came a wide range of interested parties: members of the EU Parliament, rare disease research funding institutions, national and European health and regulatory authorities, researchers, and representatives of the pharmaceutical industry. The conference was opened by video by Janez Potočnik, European Commissioner for Science and Research. Then came Her Royal Highness Princess Astrid of Belgium, who is particularly interested in medical research. She was followed by Alojz Peterle, MP, who insisted on the inequities in access to care across Europe for rare disease patients, the added value of working at the EU level, and the necessity of EU/US collaboration in the field of rare diseases to avoid duplication of efforts. During the meeting, it was emphasised that discoveries made during research on rare diseases are often a starting point for further discoveries concerning more common diseases, and that rare diseases research plays a crucial role in innovation. It was also singled out that there was a need to:

• Fund rare disease research in the long term
• Conduct research on all levels simultaneously (genomics, diagnostics, therapeutic, etc.)
• Fund clinical trials for designated orphan drugs at their early stages of development (pre-clinical, phase 1 and phase 2)
• Develop closer links between academia (fundamental research) and industry
• Develop closer links between researchers and patient groups
• Increase the number of researchers working on rare diseases (and support them)
• Create European research platforms and make them fully accessible to researchers
• Continue important collaboration initiatives with Member States such as E-RARE, an ERA-NET project

The necessity of a global approach for rare diseases was expressed clearly throughout the conference and served as introduction to ICORD, which took place the following day in Brussels, and in which Eurordis was also involved. ‘The 13 September conference was an official recognition of the EU’s support to rare diseases and the fact they are a public health priority,’ says Fabrizia Bignami. ‘This is excellent news in the context of the 7th Framework Programme for Research and Technological Development. It strengthened the dialogue between the rare disease community and the European Commission.’

Read more:
Programme of the conference
 

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• E-RARE is coordinated by GIS - Institut des Maladies Rares in Paris
• International Conference on Rare Diseases and Orphan Drugs

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This article first appeared in the November 2007 issue of the Eurordis Newsletter.

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Author: Jerome Parisse-Brassens
Photo credits: Programme © Europa.eu; conference © Eurordis/Sylvain Gouraud ; Fabrizia Bignami © Eurordis