From 1 January 2020 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 Great Britain (GB) 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 GB, or, if such a method exists, the medicine must be of significant benefit to those affected by the condition
The MHRA will offer incentives in the form of market exclusivity and full or partial refunds for marketing authorisation fees to encourage the development of medicines in rare diseases. Waiver from scientific advice fees will also be available for UK based SMEs.
To read the guidance in full, click here.
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