MHRA issues post-transition orphan medicine guidance

The Medicines and Healthcare product Regulatory Agency (MHRA) explains that it will provide incentives to encourage the development of products to treat rare diseases, including market exclusivity, refunds for marketing authorisation fees and scientific advice fee waivers for UK-based small and medium enterprises (SME).

The MHRA will be responsible for reviewing applications from companies for orphan designation at the time of a marketing authorisation application (MAA). There will be no pre-marketing authorisation orphan designation.

For orphan drug designation, 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.
  • Satisfactory methods may include authorised medicinal products, medical devices or other methods of diagnosis, prevention or treatment which are used in GB.

To apply for orphan designation, applicants must submit a GB orphan drug designation application form at the same time as their MAA. A decision on the orphan status of the medicine and approval of the MAA will be made simultaneously.

As with the EU, the UK will offer marketing authorisation holders 10 years of marketing exclusivity that will begin from the date of first approval in GB or EU. The UK will also recognise market exclusivity for centrally authorised medicines that are converted to UK marketing authorisations. Furthermore, the UK will offer the same two years of additional exclusivity for medicines authorised with the results of studies from paediatric investigation plans (PIPs) included in the product information.

All medicines that gain a GB orphan marketing authorisation will be listed on the GB Orphan Register.

To view the full guidance, click here.