For the purposes of the information provided on this page we have adopted the definition of the term “regenerative medicine” that was used in the House of Lords Regenerative Medicine Report (see below). This was:
“regenerative medicine” is used to refer to methods to replace or regenerate human cells, tissues or organs in order to restore or establish normal function. This includes cell therapies, tissue engineering, gene therapy and biomedical engineering techniques, as well as more traditional treatments involving pharmaceuticals, biologics and devices.
Roles and remits of the regulators in regenerative medicine
Each regulator has a clear remit and regulates distinct areas of the regenerative medicine process. However, we work closely together to provide effective advice and guidance to support establishments through the regulatory requirements. Each regulator has a core set of standards that apply depending on where you are in the process, from cell derivation to treatment. We are all focused on ensuring that the standards that are applied at one stage of the process do not act as a barrier at another.
The role of each of the regulators in regenerative medicine is set out below:
Health Research Authority (HRA) – has a remit to provide an ethics opinion on clinical trials. Those involving gene therapy regenerative medicines are reviewed through the Gene Therapy Advisory Committee (GTAC). Other regenerative medicine studies may be reviewed by other appropriately flagged RECs. It also provides the Integrated Research Application System (IRAS) through which applications and approvals from GTAC/RECs and MHRA for clinical trials involving regenerative medicines can be made.
Human Fertilisation and Embryology Authority (HFEA)[external link] – regulates the use of human embryos or human admixed (human-animal) embryos to derive stem cells for use in the treatment of patients.
Human Tissue Authority (HTA) [external link] – remit includes regulation of organisations that remove, store and use of human tissue or cells; this includes where they are used as starting materials for Advanced Therapy Medicinal Products (ATMPs). Under the European Union Tissues and Cells Directives (EUTCD), it licenses establishments that remove, test, process, store, and distribute tissues or cells that will (or may) be used to treat patients.
Medicines and Healthcare products Regulatory Agency (MHRA) [external link] – remit includes responsibility for granting the appropriate authorisation for the manufacturing site of ATMPs, which are prepared and used under the hospital exemption, and for ATMPs made and supplied under the specials scheme under the relevant provisions in medicines legislation. In the area of clinical trials, the MHRA’s remit includes assessment of applications for clinical trial authorisation and the associated manufacturer’s licence for investigational ATMPs. The National Institute for Biological Standards and Control (NIBSC) [external link], which houses the UK National Stem Cell bank, is part of the MHRA.
More information about research approvals:
Please refer to the Research Community area of the website for information about the approvals for research studies and how to apply to individual review bodies. Further information about GTAC is also provided on this site. Additionally the Stem Cell Toolkit [external link] provides regulatory routemaps that are specific to individual stem cell projects.
Other relevant agencies:
Department for the Environment Food and Rural Affairs (DEFRA) [external link] has an Advisory Committee on Releases to the Environment (ACRE) [external link], which advises government on requests for permission to release genetically modified organisms (GMO) into the environment. In 2013, this committee published advice on gene therapy clinical trial for heart disease.
Health and Safety Executive (HSE) [external link] has the Scientific Advisory Committee on Genetically Modified Organisms (Contained Use) – SACGM (CU) [external link]. This committee provides technical and scientific advice to the UK Competent Authorities on all aspects of the human and environmental risks, and is responsible for maintaining guidance on the contained use of GMOs.
Regulatory advice service for regenerative medicine
From 13 October 2014, the MHRA’s Innovation Office is the portal for all regulatory queries concerning regenerative medicines. A “one stop shop” service provides a single point of access from the four regulators in the field, the Human Tissue Authority (HTA), the Human Fertilisation and Embryology Authority (HFEA), Health Research Authority (HRA) and the Medicines and Healthcare products Regulatory Agency (MHRA), who will provide a co-ordinated single response service for free regulatory advice.
Any query relating to the regulation of regenerative medicines, including Advanced Therapeutic Medicinal Products (ATMPs) can be submitted to the MHRA’s Innovation Office and will be answered by the relevant experts from the four regulatory bodies.
Individuals or companies who have regulatory questions concerning regenerative medicines and who are unsure which agency to direct their inquiry to, or have a query that impacts several regulators, should use the Innovation Office advice form.
Working in partnership:
The HRA and others work closely together and will continue to engage with those involved in regenerative medicine, including researchers, the British Society for Gene and Stem Cell Therapy [external link], and the Cell Therapy Catapult [external link] to help clarify the regulatory requirements that apply.
The HRA recently held a regenerative medicine event hosted by the Cell Therapy Catapult to look at changes and discuss issues with the sector, regulators and representative bodies. Additionally in 2012, the MHRA hosted an event on the regulation of regenerative medicine [external link].
As set out in the Government Response to the House of Lords Inquiry [external link] a Regenerative Medicine Expert Group (RMEG) is being established to develop an NHS regenerative medicine delivery readiness strategy and action plan. This group will build on existing initiatives so that the NHS is fully prepared to deliver these innovative treatments. The group will be supported by the Department of Health; members will be drawn from a number of groups and organisations, including the HRA. The remit of the Regenerative Medicine Expert Group will include a role to monitor the effect of regulation on the development of regenerative medicines in the UK.
More generally, the HRA is working in partnership with a range of organisations to improve the environment for research in the UK. Please refer to our projects and plans pages for more information.
House of Lords Inquiry into Regenerative Medicine
During 2012-13, the House of Lords Science and Technology Committee held an inquiry into regenerative medicine in the UK. For more information about the inquiry, the resulting report and the HRA’s responses please use the links below:
- Government response to House of Lords Regenerative Medicine Report, published 1 October 2013 [external link]
- HRA Response to House of Lords Regenerative Medicine Report, 1 July 2013
- House of Lords Science and Technology Committee Regenerative Medicine Report, published 26 June 2013 [external link] and associated Press Release, published 26 June 2013 [external link]
- House of Lords Science and Technology Committee Inquiry into Regenerative Medicine – written and oral evidence [external link]; refer to pages 311 – 316 for oral evidence given by HRA and supplementary written evidence.
- HRA, HTA, MHRA and HFEA joint supplementary submission (‘role of the regulators’) to call for evidence for House of Lords Inquiry into regenerative medicine [external link]
- House of Lords Inquiry – call for evidence; published 26 July 2012 [external link]
UK Stem Cell Toolkit [external link]
This toolkit is intended to be a reference tool for those who wish to develop a programme of human stem cell research and manufacture, including clinical applications. It applies only to the regulation of human stem cells and their use in the laboratory and clinical settings. The toolkit provides regulatory routemaps that are specific to individual stem cell projects. It does this by using your responses to questions when you start using the toolkit.
Clinical Trials Toolkit [external link]
This toolkit provides practical advice to researchers in designing and conducting publicly funded clinical trials in the UK. It provides information on best practice and outlines the current legal and practical requirements for conducting clinical trials. The toolkit is primarily focused on Clinical Trials of Investigational Medicinal Products (CTIMPs) and the regulatory environment and requirements associated with these. However researchers and R&D staff working on trials in other areas will also find useful information and guidance of relevance to the wider trials environment.
Cell Therapy Catapult [external link]
The Cell Therapy Catapult was established in 2012 to grow the UK cell therapy industry. It was set up to help businesses take innovative ideas through to commercialisation. The website has specific regulatory resource pages, which include an overview of the relevant regulations for cell therapy.
MHRA Innovation Office [external link]
The MHRA Innovation Office helps organisations that are developing innovative medicines, medical devices or using novel manufacturing processes to navigate the regulatory processes in order to be able to progress their products or technologies. Examples of innovative products include Advanced Therapy Medicinal Products (ATMPs), nanotechnology, stratified medicines, novel drug/device combinations, and advanced manufacturing.
UK Regenerative Medicine Platform (UKRMP) [external link]
The Medical Research Council (MRC), Biotechnology and Biological Sciences Research Council (BBSRC) and the Engineering and Physical Science Research Council (EPSRC) have established the UKRMP to address the challenges associated with translating scientific discoveries towards clinical impact.
UK Stem Cell Bank [external link]
The UK Stem Cell Bank was established to provide a repository of human embryonic, foetal and adult stem cell lines as part of the UK governance for the use of human embryos for research. Its role is to provide quality controlled stocks of these cells that researchers worldwide can rely on to facilitate high quality and standardised research. It also prepares stocks of EUTCD-Grade cell lines for use as starting materials for the development of cellular therapies. The UK Stem Cell Bank is hosted by NIBSC [external link], which is part of the MHRA.
UK Trade & Investment (UKTI) Life Science Investment Organisation (LSIO) [external link]
This dedicated unit within UKTI is intended to support overseas companies to invest and expand in the UK from the earliest research and development collaborations through to clinical trials, commercial operations and partnerships.
Knowledge Transfer Network (KTN) Regenerative Medicine Priority Area [external link]
This is an official group within the Healthtechnologies and Medicine Knowledge Transfer Network (KTN). Knowledge Transfer Networks have been set up by the Technology Strategy Board (TSB) to facilitate collaboration and stimulate innovation by bringing together people from a range of organisations with a variety of expertise.
DEFRA Advisory Committee on Releases to the Environment (ACRE) [external link]
Regenerative Medicine Expert Group [external link]