Advanced therapy medicinal products: regulation and licensing
- Medicines and Healthcare products Regulatory Agency
- Part of:
- Patient safety and Marketing authorisations, variations and licensing guidance
- 26 January 2015
How to get a marketing authorisation for a regenerative medicine so it can be sold and supplied in the UK and Europe.
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
In the UK, MHRA is the competent authority:
- for clinical trial authorisation for all medicinal products, including ATMPs
- for UK manufacturers or importers of ATMPs
Get a classification opinion or advice about ATMPs
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)
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.
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 firstname.lastname@example.org 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.
See application form (MS Word Document, 871KB) .
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.
Published: 26 January 2015