The UK has designed an independent replacement for present system for officially designating a medicine as ‘orphan’, i.e. for rare diseases, administered by the European Medicines Agency (EMA).
The Medicines and Healthcare Products Regulatory Agency (MHRA) will manage orphan designation alongside other aspects of marketing authorisation. Criteria for designation as an orphan drug are hereby defined, from 1st January 2021, as:
- “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
- Satisfactory methods may include authorised medicinal products, medical devices or other methods of diagnosis, prevention or treatment which are used in GB.”
Applicants for orphan designation should submit a new, additional form with their application through module 1.2 of the eCTD. MHRA asks that attention is specifically drawn to the orphan application in the application cover letter.
The UK approach appears to closely align with the current EMA approach, for example in market exclusivity:
“On grant of a marketing authorisation with orphan status, the medicinal product will benefit from up to 10 years of market exclusivity from similar products in the approved orphan indication.
“The start of this market exclusivity period will be set from the date of first approval of the product in GB or EU/EEA.”
Read the full guidance for more information.