UK MHRA Guidance: Advanced Therapy Medicinal Products – Regulation and Licensing
Advanced therapy medicinal products (ATMPs) are regulated medicinal products that include gene therapy medicinal products, somatic cell therapy medicinal products, and tissue-engineered products. The Advanced Therapy Medicinal Products: Regulation and Licensing in the UK guidance explains the regulatory and licensing arrangements for ATMPs in the UK, including product classification, regulatory advice, clinical trials, marketing authorisation, combination ATMPs, manufacturing licences, and the arrangements for unlicensed ATMPs.
Classification and Regulatory Advice
Where there is uncertainty about whether a product is an ATMP or which type of ATMP it represents, a classification flowchart can be used. Applicants can also seek assistance from the MHRA Innovation Office for classification-related queries. The guidance also identifies EMA ATMP classification resources as useful information for understanding ATMP classification, including examples of classified products and the scientific and regulatory principles applied to classification.
Regulatory Queries on Regenerative Medicines
Regulatory enquiries concerning regenerative medicines can be made through the MHRA Innovation Office, which provides a single point of contact for the regulators involved in regenerative medicines.
The relevant regulatory bodies include the Human Tissue Authority, Human Fertilisation and Embryology Authority, Health Research Authority, and MHRA.
Scientific Advice
Scientific advice from the MHRA is available at any stage of ATMP development. The service provides an opportunity to obtain regulatory and scientific advice during development, and the service usually involves a fee.
Clinical Trials
ATMPs must undergo clinical trials in the same way as other medicines. Applications for approval to conduct clinical trials for ATMPs in the UK should be submitted to the MHRA in accordance with the applicable clinical trial requirements.
Sponsors should ensure that the appropriate requirements are addressed before conducting a clinical trial involving an ATMP.
Marketing Authorisation
ATMPs intended to be placed on the UK market must have a marketing authorisation. The MHRA is responsible for assessing the quality, safety, and efficacy of ATMPs requiring a marketing authorisation.
Following implementation of the Windsor Framework on 1 January 2025, marketing authorisation applications covering Great Britain only are no longer possible. Applications for ATMP marketing authorisations must be UK-wide.
Combination ATMPs
A combination ATMP contains one or more medical devices or active implantable medical devices as integral
components of the product. For a marketing authorisation application involving a combination ATMP containing a medical device, the application should include evidence that the device meets the applicable essential requirements under the UK Medical Devices Regulations.
Where available, the results of an assessment by an approved body or an EU notified body should also be included.
Manufacturer’s Licence
Manufacturers seeking to obtain a manufacturer's licence for an ATMP should refer to the relevant MHRA licensing requirements. Advice can also be obtained from the MHRA's GMP Inspectorate where appropriate.
Manufacture of Unlicensed ATMPs in the UK
The guidance identifies two routes through which unlicensed ATMPs can be made available in the UK: the hospital exemption and the specials scheme.
The hospital exemption applies to ATMPs prepared on a non-routine basis and used in a hospital under a prescription for an individual patient. The specials scheme applies to medicinal products supplied in accordance with the applicable requirements.
Manufacturers require an appropriate manufacturer's licence from the MHRA to use either scheme.
Human Tissues and Cells in ATMPs
Where human tissues and cells are used as starting materials for a medicinal product, the donation, procurement, and testing of the cells are covered by the Human Tissue (Quality and Safety for Human Application) Regulations 2007. The Human Fertilisation and Embryology Authority is the competent authority for gametes and embryos, while the Human Tissue Authority is responsible for licensing and inspection of other tissues and cells.
Once the starting materials have been made available, medicines legislation applies and is regulated by the MHRA.
Blood and Blood Components in Medicinal Products
Where blood and blood components are used as starting materials in a medicinal product, their donation and testing are covered by the Blood Safety and Quality Regulations 2005. The MHRA is the competent authority for blood product regulation in the UK.
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