Medicines, medical devices and blood regulation and safety – guidance

Advanced therapy medicinal products: regulation and licensing

How to get a marketing authorisation for a regenerative medicine so it can be sold and supplied in the UK and Europe.

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 Directive 2001/83/EC as amended by the ATMP Regulation 1394/2007 and includes combination ATMPs.

In the UK, MHRA is the competent authority:

Get 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 this flowchart (PDF, 32.1KB, 5 pages) or go to European Medicines Agency (EMA) website.

If you are still uncertain fill out the ATMP advice form or go to EMA website for information about classification and to apply to get an opinion from the EMA’s Committee for Advanced Therapies (CAT) .

MHRA or EMA will classify your product as an ATMP if appropriate.

Regulatory queries on regenerative medicines (one-stop shop)

All regulatory enquiries about regenerative medicines should go through the MHRA 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 the innovation office enquiry form or email innovationoffice@mhra.gsi.gov.uk.

Scientific advice

You can get scientific advice from MHRA or EMA during any stage of ATMP development. There is a fee for this service.

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

All advanced therapy medicinal products must go through clinical trials in the same way as all other medicines.

Apply for a marketing authorisation for an advanced therapy medicinal product

All ATMPs to be placed on the market in the EU must have a marketing authorisation and are regulated through the centralised authorisation procedure.

The Committee for Advanced Therapies (CAT), established by the European Medicines Agency (EMA), is responsible for preparing a draft opinion on the quality, safety and efficacy of each ATMP that needs a marketing authorisation.

The CAT’s opinion goes to EMA’s Committee for Medicinal Products for Human Use (CHMP) (link) for final approval.

Further information about the CAT, EMA’s role in the regulation of ATMPs and guidelines specific to ATMPs is available on EMA’s website.

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. CAT evaluates combined ATMPs for marketing authorisations, and MHRA for 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 medical devices legislation and, where available, the results of an assessment by a notified body.

If the application doesn’t include the results of a notified body’s assessment, EMA will get a notified body’s assessment unless the CAT decides that the involvement of a notified body is not needed.

Apply to get a manufacturer licence for an advanced therapy medicinal product

If you need advice about getting a manufacturer licence from MHRA email gmpinspectorate@mhra.gsi.gov.uk or go to our information about manufacturer licences.

Manufacture 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 manufacturer licence from MHRA to use either scheme.

To apply under the ‘specials’ scheme fill out the flowchart (PDF, 32.1KB, 5 pages) and email it to pcl@mhra.gsi.gov.uk.

See application form (MS Word Document, 871KB) .

To apply for a hospital exemption fill out the application form (MS Word Document, 1.13MB) and email it to pcl@mhra.gsi.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.

See guidance on the UK’s arrangements under the hospital exemption scheme (PDF, 67.5KB, 10 pages) .

See non-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 the Tissues and Cells Directive (2004/23/EC). Under this directive there are two competent authorities.

The Human 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.

The Human 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 under the Tissues and Cells Directive, medicines legislation applies and is regulated by MHRA and EMA. Advice on the regulatory routes is available using the UK regulatory route map for stem cell research and manufacture.

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 the joint 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 Directive 2002/98/EC. The competent authority for blood in the UK is MHRA.