Development of medicines for rare diseases

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Development of medicines for rare diseases

An overview of the EU’s orphan designation programme

A disease is considered rare if fewer than five in 10,000 people have it. Around 30 million people in the European Union (EU) suffer from a debilitating rare disease, which means one in 17 people. Finding effective treatment for these rare diseases is a huge challenge.

In 2000, the EU’s orphan designation programme was launched to encourage companies to research and develop medicines for rare diseases. By the end of 2017, over 1,900 medicines had been granted orphan status that gives access to specific incentives that make it more attractive for companies to develop these treatments. To date, over 140 orphan medicines are marketed in the EU providing new treatment options for patients.

To find out more about the EU programme and the incentives available to developers, check out our infographic.

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Factsheet: Orphan medicines in the EU