Orphan medicines refer to drugs designed to treat diseases so rare that securing funding for their development under standard marketing conditions is challenging. In Great Britain, orphan medicines are those already granted EU Marketing Authorization (MA) and are subject to a GB orphan Manufacturing Authorization Application (MAA) under regulation 50G of the Human Medicines Regulation 2012.
All medicines granted an orphan MA by the Medicines and Healthcare products Regulatory Agency (MHRA) are listed on the Orphan Register, a compilation of authorized orphan medicinal products. This register includes EU MAs converted into GB MAs as per paragraph 6(7) of Schedule 33A of the Human Medicines Regulation 2012.
Upon receiving orphan status and inclusion in the Orphan Register, a medicinal product is granted up to 10 years of market exclusivity from similar products listed therein.
For a medicinal product to qualify for orphan registration and an orphan MAA, it must meet stringent criteria. It must be intended for treating, preventing, or diagnosing life-threatening or chronically debilitating diseases. The disease’s prevalence in GB must be equal to or less than 5 cases per 10,000 people, or it must be improbable that marketing the medicine would generate sufficient profit to cover development costs. Additionally, there must be no satisfactory method of diagnosing, preventing, or treating the condition already licensed. If valid medicinal treatment is already licensed in GB, the potential orphan medicine must offer significant benefits to those affected by the condition.
Examples of orphan drugs licensed for GB use include Fintepla (fenfluramine), used to treat seizures associated with Dravet syndrome, a rare and severe form of epilepsy, and Imnovid (pomalidomide), used to treat patients with multiple myeloma (bone marrow cancer) who have not responded to two previous treatment attempts.
For further information, please refer to the following links:
Orphan medicinal products: https://www.gov.uk/guidance/orphan-medicinal-products-in-great-britain
Orphan registered medicinal products: https://www.gov.uk/government/publications/orphan-registered-medicinal-products