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Platform of information services for the coordination of rare disease research with various stakeholders from research, SMEs and patient organisations and the coordination of early clinical trials

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Winning the fight against rare diseases

Enhanced research and networking regarding rare diseases can help develop drugs for children suffering from challenging illnesses such as leukaemia and rare forms of cancer.


Medical authorities in different parts of Europe and the world are often not adequately prepared to tackle rare diseases. The EU-funded project Orphanplatform aimed to address these shortcomings. It looked at developing an online information service (Orphanet) concerning research on rare diseases and orphan medicinal products per EU Member State and on an EU-wide level as well. The project also encouraged enrolment of patients in clinical research, formulated a database of relevant research projects, supported scientists in the field and organised a stakeholder workshop. In its initial phase, Orphanplatform looked at increasing services in 11 European countries beyond the EU-25. It rendered research in the field more accessible to stakeholders, including databases of research projects and funding agencies in the Orphanet directory. In addition, the project established an online mechanism to enable patients to find out about clinical research projects related to their disease so they could participate in it if desired. This multilingual site lets patients register as volunteers to take part in clinical trials and has the potential to save lives. Another achievement was identifying research projects that needed the help of an industrial company or start-up company to develop relevant new products or techniques. This was made possible through a dedicated portal, OrphanXchange, that was linked to the Orphanet database. Lastly, Orphanplatform organised a workshop in 2005 in London, the United Kingdom, to exchange views, explore development of therapies and identify research projects in advanced stages. The project is set to support development of paediatric drugs and cancer drugs, important prospects considering that 80 % of rare diseases appear at an early age.

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