Snapshot

The Orphan medicinal products pathway has been developed by the Medicines and Healthcare products Regulatory Agency (MHRA) of United Kingdom.

This pathway can be used where To qualify for orphan designation in an orphan condition, 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 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; and no satisfactory method of diagnosis, prevention or treatment of the condition concerned exists in Great Britain, 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 Great Britain..

This pathway accelerates the regulatory review process.

It is an abridged review (a reliance pathway).

Marketing Authorisation Applicants should submit the Great Britain Orphan Drug Designation Application Form (https://assets.publishing.service.gov.uk/government/uploads/system/uploads/attachment_data/file/949023/GB_Orphan_Drug_Application_Form.docx) with their MAA in module 1.2 of the eCTD, specifically indicating in the cover letter their intention to seek an orphan designation. Applicants should use MHRA's eAF and cover letter tool to determine what information they need to include in their application. If applicants do not include the correct information, their application will not be validated. Applications for orphan designation will be examined by the MHRA’s advisory committee, the Commission on Human Medicines (CHM). A decision on fulfilment of the orphan criteria runs in parallel with the assessment of the marketing authorisation procedure. Following the validation of the MAA, a decision on orphan status will be made at the time of the decision on approval of the marketing authorisation (see guidance on new assessment procedures: https://www.gov.uk/guidance/apply-for-a-licence-to-market-a-medicine-in-the-uk). Any questions concerning the fulfilment of the orphan designation criteria will be raised with the company during the evaluation of the MAA. If the MHRA concludes that the criteria for orphan designation are not met, there will be an opportunity to appeal the decision to the CHM before the MA is granted. The applicant should inform the MHRA of the intention to appeal as soon as possible. Market exclusivity period: 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 Great Britain. The remaining market exclusivity periods for centrally authorised orphan medicine marketing authorisations granted prior to 1st January 2021 that have been converted to Great Britain marketing authorisations will continue to apply. It is not necessary to submit orphan maintenance reports to the MHRA, but they can be provided as additional information. In the UK/Great Britain there is no orphan designation issued separately from the MA. Therefore, if a Change of Ownership application is submitted, the orphan designation will automatically transfer. Regulation 58D of the Human Medicines Regulation 2012 (as amended) establishes the possibility for the UK Licensing Authority to request that the market exclusivity be reduced from 10 to 6 years, under certain circumstances, if the orphan criteria are no longer met in relation to the medicinal product. Paediatric indications: Orphan medicines authorised in Great Britain with the results of studies from a paediatric investigation plan (PIP) included in the product information are eligible for an additional 2 years of market exclusivity.