This guidance has been updated. It gives an overview of Orphan medicinal products to support development in rare disease, marketing exclusivity for the products, and how to apply.
The authorisation of orphan medicinal products will change following implementation of the Windsor Framework on 1 January 2025.
Reference should be made to the Human Medicines Regulations 2012 (HMRs), as amended. The HMRs have been amended by Human Medicines (Amendments relating to the Windsor Framework) Regulations 2024 (S.I. 2024/832) which come into force on 1 January 2025.
The table below shows only those sections of the guidance that have changed, in a present/proposed format.
| Prior to 1 January 2025 | From 1 January 2025 |
|---|---|
| If a medicinal product fulfils the criteria to be designated and authorised as an orphan in the EU under Regulation (EC) 141/2000, a Great Britain (GB) orphan MAA can be made under regulation 50G of the Human Medicines Regulation 2012 (as amended). A UK-wide orphan MAA can only be considered in the absence of an active EU orphan designation. If, prior to EU authorisation, a UK-wide orphan marketing authorisation is granted and the medicinal product subsequently receives EU orphan designation, the market authorisation holder (MAH) would need to submit a variation to amend their UK MA to a Great Britain orphan MA. | Following implementation of the Windsor Framework on 1 January 2025 Marketing Authorisations granted for products that fulfil UK orphan criteria will be valid UK-wide regardless of whether there is an EU orphan designation or EU authorisation as an orphan medicinal product. |
| Criteria for UK orphan designation | |
| To qualify for orphan designation in an orphan condition, a medicine must meet the following criteria. These will be considered in relation to Great Britain only until 1 January 2025: • 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 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 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 UK. |
Source: MHRA