Patient groups need access to RD research resources

The Eurordis 10 year anniversary Membership Meeting and the European Workshop on “Gaining access to rare disease research resources” on 4 and 5 May 2007 at the Pasteur Institute in Paris saw 270 participants from 27 countries getting together and discussing issues at the heart of patients’ lives. The audience was made of 75% rare disease patient representatives and 25% researchers, healthcare professionals, industry representatives and public policy makers. The European workshop was part of the Capacity Building for Patient Organisations in Research Activities project (name code: CAPOIRA). ‘It was a milestone in the empowerment of patient groups on the way to playing a greater role in the European research agenda,’ says Fabrizia Bignami, Therapeutic Development Officer at Eurordis who was in charge of the workshop. The workshop, which lasted a day and a half, saw presentations on rare disease research resources and integration of patient groups in research. Two sub-groups were asked to meet and debate on the needs and wishes of patient groups on two different topics: “European tools for rare disease research”, and “getting involved in research”.

The following main points and needs came out of the discussions:
Basic requirements:

• Push for national plans for rare diseases (including research policy) in Member States
• Raise awareness on rare disease research amongst EU decision makers
• Provide more opportunities for patients and EU decision makers to work together
• Establish EU-wide Committees for rare diseases rather than national ones
• Keep providing information in national languages.

European project applications:

• Simplify the procedure for getting support during the preparatory phase;
• Implement a two step approach: expression of interest first, and full application when selected;
• Create a permanent advisory/support desk at EU level within Eurordis, dedicated to rare disease project development; Publish a who’s who of the European Commission, with phone numbers and email addresses
• Organise training sessions on constraints of research, drug development process, clinical trials and related issues for patient representatives;
• Disseminate information on research resources possibilities in the EU;
• Allow for core funding of patient groups;
• Create longer-term support for research projects with long term aims (eg projects on natural history of the disease)
• Develop more research projects on quality of life of patients
• Calls for proposals: provide help at EU level; develop tools to help patient groups organise their own international calls for proposal

Research tools:

• Rare disease biobanks: use EuroBioBank as a starting point for the creation of new high-quality biobanks; make researchers using biobanks understand that they need to share results with patients
• Patient databases and registries: organise training sessions for patient representatives; write and disseminate EU guidelines on the creation of databases; develop databases linking genotypes and phenotypes that can be operated or supervised by patient groups with the support of specialists
• Clinical trials: create an EU centralised registry of clinical trials and publish anonymised results; organise training sessions on protocols, methodology and analysis for patient representatives; ensure results of clinical trials are fully used by the research community

The results of this Eurordis workshop were presented at another European policy development workshop on rare disease research organised by the European Commission in Brussels on 14 June 2007. ‘It was an opportunity to further stress the role of patients in EU research,’ says Fabrizia Bignami, who participated in this meeting. The outcomes of the 14 June discussions will in turn be presented at a conference entitled ‘Rare Diseases Research: Building on Success’, which will take place in Brussels on 13 September 2007, with members of the EU Parliament, representatives of rare disease research funding institutions, national and European health and regulatory authorities, researchers, and patient organisations. Another opportunity for the rare disease community to express its needs in term of research, and another step towards full participation of rare disease patients in the European research agenda and decision making process - something Eurordis has long fought for, and always will.

This article was first published in the July 2007 issue of the Eurordis Newsletter

Author: Jerome Parisse-Brassens
Photo credits: Fabrizia Bignami © Eurordis; Eurobiobank © Eurobiobank; Conference © Sylvain Gouraud