Brexit: How the MHRA Will Manage Orphan Medicinal Products From January 1, 2021

The MHRA has published new guidance on ‘How the MHRA will manage orphan medicinal products from 1 January 2021 in Great Britain (GB)’ to help stakeholders prepare for the end of the transition period. 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 to treat rare diseases.

The new guidance covers the following matters:

• Marketing authorisation application process for orphan designation: The MHRA will be responsible for reviewing applications for orphan designation at the time of a marketing authorisation application. The following conditions must be satisfied: 1. Intended for the treatment, prevention, or diagnosis of a disease that is life-threatening or chronically debilitating; 2. Prevalence of the condition in Great Britain (GB) must not be more than 5 in 10,000; and 3. No satisfactory method of diagnosis, prevention, or treatment of the condition concerned exists in GB; satisfactory methods may include authorised medicinal products, medical devices, or other methods of diagnosis, prevention, or treatment which are used in GB
• How to apply: Applicants must send an application form, with their marketing authorisation application, specifically indicating in the cover letter their intention to seek an orphan designation
• Decision on orphan status (as well as a decision of the marketing authorisation): Made by the MHRA’s advisory committee
• Appeal: The applicant has the opportunity to appeal the decision to the CHM before the marketing authorisation is granted; the applicant should inform the MHRA of the intention to appeal as soon as possible 
• Market exclusivity: Up to 10 years from the date of first approval of the product in GB or EU/EEA 
• Paediatric indications: Orphan medicines authorised in GB with the results of studies from a paediatric investigation plan (PIP) included in the product information are eligible for an additional two years of market exclusivity
• Variation applications (section 4.1 of the Summary of Products Characteristics): The orphan criteria will be assessed in parallel to the approval of the new indication; a new period of market exclusivity is only given if the applied for therapeutic indication falls within a new orphan condition 
• Fees: No additional fees for orphan designation application
• SME status: Waiver from scientific advice fees will also be available for UK based SMEs; companies who have, or intend to seek, SME status should ensure that they have the relevant documentation in place if an SME fee refund is to be applied for
• Orphan register: All medicines that gain a GB orphan marketing authorisation will be listed on the GB Orphan Register (active and then withdrawn, suspended or expired)