This section of the site provides advice and guidance on the regulatory arrangements that apply to advanced therapy medicinal products (ATMPs).
This section provides general advice on:
- the different regulatory requirements for ATMPs that are authorised under the Regulation on advanced therapy medicinal products
(104Kb) - the requirements under the hospital exemption scheme under Article 28 (2) of that Regulation.
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On 13 April 2011, the MHRA hosted a meeting of the London Regenerative Medicines Network to discuss advanced therapy medicinal products. A copy of the presentations which were delivered on the day is below. |
Guidance on the UK hospital exemption
ATMPs guidance
(69Kb)Annex A - Flowchart for determining the regulatory status of tissue and cell-based products
(25Kb)Annex B - non-routine guidance
(33Kb)Application for the UK hospital exemption licence
Applying for a Hospital Exemption licence: Manufacturer’s Licence - Exempt Advanced Therapy Medicinal Products (MeAT) 
(1273Kb)
Getting advice about ATMPs
Companies, researchers and others who wish to get advice from the MHRA on the regulatory classification of a product that may come into the ATMP category should complete the advice form. For this kind of product, this form will replace existing arrangements for those who want to get regulatory classification advice.
The frequently asked questions section provides clarification of the specific areas where the MHRA can provide advice and where advice should be sought from the European Medicines Agency (EMA).
When contacting the MHRA for advice please use the contact points below. If you are not sure who to contact with a query, please email info@mhra.gsi.gov.uk
What is an ATMP?
An ATMP is a medicinal product as defined in Directive 2001/83/EC as amended (the Directive). Specifically, an ATMP is a medicinal product which is either:
- a gene therapy medicinal product as defined in Part IV of Annex 1 to Directive 2001/83/EC
- a somatic cell therapy medicinal product as defined in Part IV of Annex 1 to Directive 2001/83/EC
- a tissue engineered product as defined in Article 2 1 (b) of the ATMP Regulation.
How are ATMPs regulated?
Under the ATMP Regulation, the centralised authorisation procedure will apply to ATMPs which are intended to be placed on the market in the Community. A new committee, the committee for advanced therapies (CAT) has been established at the European Medicines Agency (EMA).
The CAT is responsible for preparing a draft opinion on the quality safety and efficacy of each ATMP for which a marketing authorisation application is submitted. The CAT’s opinion will be submitted to the EMA’s Committee for Medicinal Products for Human Use (CHMP) for final approval.
Further information is available on the EMA’s website (external link)
In the UK, the MHRA is the supervisory authority for UK manufacturers or importers of centrally authorised ATMPs, as well as the competent authority for ATMPs which are prepared and used under the hospital exemption and made and supplied under the ‘Specials’ scheme.
The MHRA is also the competent authority for the assessment of applications for clinical trial authorisations and the associated manufacturer’s licence for investigational ATMPs. Further information is available in the guidance and FAQ sections. The documents also provide links to more specific and detailed information about the regulation of ATMPs.
How are combination ATMPs regulated?
A combination ATMP is defined as an ATMP that fulfils the following conditions:
- it must incorporate, as an integral part of the product, one or more medical devices within the meaning of Article 1 (2) (a) of Directive 93/42/EEC or one or more active implantable medical devices within the meaning of Article 1 (2) (c) of Directive 90/385/EEC
- its cellular tissue or tissue part must contain viable cells or tissues
- its cellular or tissue part containing non-viable cells or tissues must be liable to act upon the human body with action that can be considered as primary to that of the devices referred to.
Combined ATMPs will be evaluated by the EMA. 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 laid down in the medical devices legislation and where available the results of an assessment by a notified body in accordance with the medical devices legislation.
If the application does not include the results of a notified body’s assessment, the EMA will seek a notified body’s assessment unless the CAT, advised by its experts for medical devices, decides that the involvement of a notified body is not required.
The MHRA can provide advice for combination products in circumstances where a product may be supplied without a marketing authorisation under the hospital exemption scheme in Article 28 (2) of the ATMP Regulation. Further guidance is available in the frequently asked questions section.
Who is responsible for providing advice about the regulatory arrangements that apply to products containing or derived from stem cells?
In cases where stem cells are to be used in a medicinal product the donation, procurement and testing of the cells are covered by the Tissues and Cells Directive (2004/23/EC).
The Human Tissue Authority (HTA) (external link) is the competent authority for the Directive in England, Wales and Northern Ireland. The processing activities (such as work in derivation laboratories) leading up to the point where a Master Cell Bank has been established with a reasonable expectation of clinical utility in a medicinal product will also be covered under the Tissues and Cells Directive. Thereafter, medicines legislation applies with the MHRA and the EMA as the national and European regulators.
The MHRA participated in the production of a UK stem cell tool kit for stem cell research and manufacture which has been developed by the Department of Health, with the support of regulatory bodies and the Gene Therapy Advisory Committee.
The UK regulatory route map is intended to be a reference tool for those who wish to develop a programme of stem cell research and manufacture ultimately leading to clinical application and was launched on 15 December 2009.
UK Regulatory Route Map for Stem Cell Research and Manufacture (external link)
Background to the ATMP Regulation
The ATMP Regulation (No 1394/2007) came into force on 30 December 2007. The provisions of the Regulation applied from 30 December 2008. The UK’s legislation for implementing the Regulation and the requirements that will apply under the hospital exemption scheme will be made and laid in Parliament later in 2010.
Contacts at the MHRA
Contact points for specific enquiries in relation to ATMPs are as follows:
Borderline/product classification - ATMPadviceform@mhra.gsi.gov.uk
Manufacturing and exemptions - gmpinspectorate@mhra.gsi.gov.uk
Pre licensing and scientific guidance - biologicalsandbiotechnology@mhra.gsi.gov.uk
Clinical trials -
clintrialhelpline@mhra.gsi.gov.uk
Patient information - patient.information@mhra.gsi.gov.uk
Pharmacovigilance - bridget.king@mhra.gsi.gov.uk or alison.shaw@mhra.gsi.gov.uk
Medical device related aspects of combination ATMPs - neil.ebenezer@mhra.gsi.gov.uk
Coordination of UK legislation /guidance - caroline.brennan@mhra.gsi.gov.uk
For queries not covered by other MHRA contact points - info@mhra.gsi.gov.uk
