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Around 30 million Europeans suffer from rare diseases such as cystic fibrosis, Duchenne muscular dystrophy or Hunter syndrome. As there are more than 6,000 known rare diseases and therapies exist for only 400 or so, there remains very high unmet need in rare diseases.

Because 80% of rare diseases have a genetic component, patients cannot influence or cope with their disease through lifestyle changes or using other drugs, as there are often no alternative therapies. Furthermore, more than half of rare disease patients are children; of those, 30% will die before their fifth birthday.

Even where treatments exist, patients are often not getting access to them because of budget pressures, bureaucratic obstacles to investment or problems of scale, panellists told a Friends of Europe ‘Café Crossfire’ lunch debate on Wednesday 21 June. They called for more collaboration between doctors, industry, insurers, patients and EU governments to solve this issue.

The European Commission has already introduced incentives for pharmaceutical companies to develop new drugs. This has increased the number of therapies available to treat rare diseases from eight to over 130 in the last 15 years. A series of European Reference Networks (ERNs) have also been set up to bring patients and specialists together to help speed up and aid treatment. But more ‘soft incentives’ are needed to encourage more evidence-gathering, to invest in prevention, and to train doctors to diagnose rare diseases.

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