Orphan designations in the post-Brexit world

The UK government guidance How the MHRA will manage orphan medicinal products from 1 January 2021 in Great Britain (1 October 2020) confirms that the MHRA will offer incentives in the form of market exclusivity and refunds of MA fees for medicines to treat rare diseases.

Application for orphan designation

The application for an orphan designation should be made using the UK government’s specific form along with the Marketing Authorisation Application module 1.2 of the eCTD. Further, the applicant’s cover letter should indicate the intention to seek an orphan designation. No additional fees apply for orphan designation.

A medicine must meet the following criteria in for UK orphan designation status:

• 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 GB must not be more than 5 in 10,000; and
• 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’s advisory committee, the Commission on Human Medicines (CHM) will examine the application for orphan designation. Examination of the application for orphan designation and for the marketing authorisation takes place in parallel and a decision on both will be given at the same time.

Medicines with a GB orphan marketing authorisation will be listed on the GB Orphan Register.

Market exclusivity

A medicinal product with orphan status will benefit from up to 10 years of market exclusivity from similar products on the approved orphan indication. The period will start from the date of first approval in the UK. Orphan medicines authorised in GB with the results of studies from a PIP in the product information are eligible for an additional 2 years of market exclusivity. These periods of market exclusivity are the same as what is currently in place under the EU framework.

For centrally authorised orphan medicine MAs that are converted to UK MAs, the applicable market exclusivity period will continue to apply.

Variations to orphan designation

For applications for new or extensions to orphan therapeutic indications, the relevant form for orphan designations will need to be submitted alongside the variation MA application. A new, separate period of market exclusivity is given only if the therapeutic indication falls within a new orphan condition.

For non-orphan designations, a new MA application (rather than a variation) is required.

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