Regulation and Licensing of Advanced Therapy Medicinal Products in the UK

1. What is an ATMP

An advanced therapy medicinal product (ATMP) is a type of medicinal product that includes gene therapy, somatic cell therapy, or tissue engineered products, often involving biological materials intended to treat or modify human disease. The definition reflects classifications in UK law and is the starting point for regulatory requirements. Source: Advanced therapy medicinal products: regulation and licensing ([GOV.UK][1])

2. Regulatory authority and scope

The Medicines and Healthcare products Regulatory Agency (MHRA) is the UK competent authority responsible for regulating ATMPs. It oversees all aspects of ATMP regulation including classification, clinical trial approvals, marketing authorisations, manufacturing licensing, and oversight of combined products. Source: Advanced therapy medicinal products: regulation and licensing ([GOV.UK][1])

3. Classification and advice

If developers are uncertain whether a product qualifies as an ATMP or what category it falls into, they can seek a classification opinion or regulatory advice from MHRA. This helps clarify which regulatory pathway applies. Source: Advanced therapy medicinal products: regulation and licensing ([GOV.UK][1])

4. Clinical trial requirements

Before an ATMP can be authorised for use, it must undergo clinical trials similar to other medicinal products. Applications for clinical trial authorisation are submitted to the MHRA, which assesses the quality, safety, and design of the proposed trial. Source: Advanced therapy medicinal products: regulation and licensing ([GOV.UK][1])

5. Marketing authorisation process

All ATMPs must obtain a marketing authorisation (MA) from the MHRA before being sold or supplied in the UK. The MHRA evaluates clinical data for quality, safety, and efficacy as part of the MA application. With recent changes to UK regulation, marketing authorisation applications must cover the entire UK market rather than only Great Britain. Source: Advanced therapy medicinal products: regulation and licensing ([GOV.UK][1])

6. Combination ATMPs

Products that include a medical device component as part of an ATMP (combination ATMPs) require evidence that the device constituent meets the relevant regulatory standards under the UK medical devices regime as part of the marketing authorisation application. Source: Advanced therapy medicinal products: regulation and licensing ([GOV.UK][1])

7. Manufacturing and unlicensed use

Manufacturers must obtain a manufacturer’s licence from the MHRA for ATMP production. There are special provisions for the manufacture of unlicensed ATMPs, such as hospital exemption and the ‘specials’ scheme, allowing use under defined circumstances without a full marketing authorisation if certain conditions are met. Source: Advanced therapy medicinal products: regulation and licensing ([GOV.UK][1])

8. Human tissues and cells

ATMPs often use human tissues and cells as starting materials. The Human Tissue Authority (HTA) and the MHRA collaborate to regulate donation, procurement, and testing under the relevant human tissue regulations, ensuring that tissues and cells used in ATMP manufacture meet safety and quality standards. Source: Advanced therapy medicinal products: regulation and licensing ([GOV.UK][1])

9. Scientific and regulatory support

The MHRA offers scientific advice to developers at various stages of ATMP development, and regulatory queries about regenerative medicines can be coordinated through the MHRA Innovation Office, serving as a one-stop shop for advancing development strategies and compliance planning. Source: Advanced therapy medicinal products: regulation and licensing ([GOV.UK][1])