GovWire

Overview

An advanced therapy medicinal product (ATMP) is a medicinal product which is either:

• a gene therapy medicinal product
• a somatic cell therapy medicinal product
• a tissue engineered product

The definition of ATMPs is found in the Human Medicines Regulations 2012and includes combination ATMPs.

In the UK, MHRA is the competent authority:

• forall medicinal products, including ATMPs
• for UK manufacturers or importers of ATMPs

Getting a classification opinion or advice about ATMPs

If you are not sure if your product is an ATMP or which type of ATMP it is, please look at thisflowchart(PDF,32.1KB,5 pages). If you are still uncertain, please contact the MHRA Innovation Office.

MHRA will classify your product as an ATMP where appropriate.

The European Medicines Agency (EMA) ATMP classification website provides useful information, with examples of products which have been classified. The web site also contains the Reflection paper on classification of advanced therapy medicinal products which outlines the scientific and regulatory principles as applied to the classification of ATMPs.

Regulatory queries on regenerative medicines (one-stop shop)

All regulatory enquiries about regenerative medicines should be made through theMHRA Innovation Office.

The Innovation Office is the single point of contact for all the regulators involved in regenerative medicines:

• the Human Tissue Authority (HTA)
• the Human Fertilisation and Embryology Authority (HFEA)
• Health Research Authority (HRA)
• MHRA

To contact the innovation office either fill out theinnovation office enquiry formor emailinnovationoffice@mhra.gov.uk.

Scientific advice

You can getscientific advice from MHRAduring any stage of ATMP development. There is usually a fee for this service.

Apply to conduct a clinical trial for an advanced therapy medicinal product

All advanced therapy medicinal products must undergo clinical trialsin the same way as all other medicines. Applications for clinical trial approvals in the UK should be made to MHRA.

Apply for a marketing authorisation for an advanced therapy medicinal product

All ATMPs to be placed on the market in the UK must have a marketing authorisation. The MHRA is responsible for assessing the quality, safety and efficacy of each ATMP that needs a marketing authorisation in GB. Please see our guidance on the application process in the UK for further information.

Combination ATMPs

A combination ATMP includes 1 or more medical devices or active implantable medical devices in the product, as well as cells or a tissue component. MHRA evaluates combined ATMPs for marketing authorisations, and UK clinical trials.

The application for a marketing authorisation for a combined ATMP which contains a medical device should include evidence that the device meets the essential requirements set out in the UK Medical Devices Regulations (SI 2002 No 618, as amended) and, where available, the results of an assessment by an approved body. Please see our guidance on the regulation of medical devices in the UK for further information.

Applying for a manufacturers licence for an advanced therapy medicinal product

If you need advice about getting a manufacturers licence from MHRA emailgmpinspectorate@mhra.gov.ukor go to our information about manufacturers licences.

Manufacture of unlicensed ATMPs in the UK

There are 2 ways in which unlicensed ATMPs can be made available in the UK:

• hospital exemption
• the specials scheme

You need a manufacturers licence from MHRA to use either scheme.

To apply under the specials scheme fill out the application form and email it topcl@mhra.gov.uk.

To apply for a hospital exemption fill out theapplication formand email it topcl@mhra.gov.uk.

The UK hospital exemption applies for ATMPs which are prepared on a non-routine basis and are used in a hospital with a prescription for a specific patient.

Seeguidance on the UKs arrangements under the hospital exemption scheme(PDF,67.5KB,10 pages).

Seenon-routine guidance on ATMPs(PDF,661KB,3 pages).

Human tissues and cells in ATMPs

If tissues and cells are being used as starting materials in a medicinal product, the donation, procurement and testing of the cells are covered by theThe Human Tissue (Quality and Safety for Human application) Regulations 2007. Under this regulation there are two competent authorities.

TheHuman Fertilisation and Embryology Authority (HFEA)is the competent authority for the use of gametes and embryos, which may be used in the derivation (development) of cells in the manufacture of ATMP.

TheHuman Tissues Authority (HTA)is the competent authority for the licensing and inspection for all other tissues and cells. If a human embryonic stem cell line is derived in the UK, a sample of the resulting cell line must be deposited at the UK Stem Cell Bank.

Once the starting materials have been made available, medicines legislation applies and is regulated by MHRA. Advice on the regulatory routes is available from the Innovation Office.

MHRA and Human Tissues Authority (HTA) work closely together in this area.

HTA and MHRA have agreed that the collection of blood as a starting material for an ATMP can be carried out under either a tissues and cells licence or a blood establishment licence. See thejoint HTA and MHRA position.

Blood and blood components in medicinal products

If blood and blood components are being used as starting materials in a medicinal product, the donation and testing are covered by the Blood Safety and Quality Regulations 2005. The competent authority for blood product regulation in the UK is MHRA.