Orphan medicines in the EU – leaving no-one behind

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21/12/2017

Orphan medicines in the EU – leaving no-one behind

An overview of the EU’s orphan designation programme

There are over 6,000 rare diseases and it is estimated that around 30 million people in the European Union (EU) suffer from one of them. Treating these patients can be very difficult, because there are only few medicines available. This represents a huge unmet medical need and a significant public health challenge.

The EU’s orphan designation programme was launched in the year 2000 to encourage companies to research and develop medicines for rare diseases.

To date, over 1,900 medicines have been designated as orphan medicines, giving access to specific incentives that make it more attractive for companies to develop them. By the end of 2017 over 140 of these medicines were on the market, providing treatment options for patients who previously often had none.

The new factsheet published by European Medicines Agency today explains what a rare disease is, how the EU programme works and what incentives are made available to developers.

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Factsheet: Orphan medicines in the EU