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Orphan Drugs: Registration

Asked by Mr Joshua ReynoldsLiberal DemocratDepartment of Health and Social CareTabled Answered 21 October 2025UIN 81023

The question

To ask the Secretary of State for Health and Social Care, what steps the Medicines and Healthcare products Regulatory Agency is taking to ensure transparency in the (a) operation and (b) maintenance of the UK orphan medicines register.

Answered by Dr Zubir Ahmed

The Medicines and Healthcare Products Regulatory Agency (MHRA) is an executive agency of the Department of Health and Social Care and regulates medicine, medical devices and blood components for transfusion in the UK, with responsibility for ensuring medicines meet appropriate standards of safety, quality and efficacy.

The United Kingdom Orphan Register is publicly available at the following link:

https://www.gov.uk/government/publications/orphan-registered-medicinal-products/orphan-register

The register is updated by the MHRA when a new orphan medicinal product or new orphan indication is authorised or their market protection expires. A process of checks to be carried out on the register on a periodic basis has been implemented to ensure that accuracy of the website is maintained.

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