- It must be intended for the treatment, prevention or diagnosis of a disease that is life-threatening or chronically debilitating;
- The prevalence of the condition in the EU must not be more than 5 in 10,000 or it must be unlikely that marketing of the medicine would generate sufficient returns to justify the investment needed for its development;
- No satisfactory method of diagnosis, prevention or treatment of the condition concerned can be authorised, or, if such a method exists, the medicine must be of significant benefit to those affected by the condition.
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.