The MHRA will be responsible for reviewing applications from companies for orphan designation at the time of a marketing authorisation application (MAA). There is no pre-marketing authorisation orphan designation. To qualify for orphan designation in an orphan condition, a medicine must meet the following criteria:
- 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 UK 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 exists in the UK, or, if such a method exists, the medicine must be of significant benefit to those affected by the condition
- Satisfactory methods may include authorised medicinal products, medical devices or other methods of diagnosis, prevention or treatment which are used in the UK.