For the purposes of the content provided within this area we have adopted the definition of the term “regenerative medicine” that was used in the House of Lords Regenerative Medicine Report:
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) have 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 Research Ethics Committees (RECs). We also provide the Integrated Research Application System (IRAS) through which applications and approvals from GTAC/RECs and the Medicines and Healthcare products Regulatory Agency (MHRA) for clinical trials involving regenerative medicines can be made.
Human Fertilisation and Embryology Authority (HFEA) 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) has a remit to regulate organisations that remove, store and use 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)are responsible 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), houses the UK National Stem Cell bank and is part of the MHRA.
More information about research approvals
Please refer to the Approvals and Amendments area on our website for information about the approvals for research studies, or alternatively please visit the guidance on IRAS to find out more information about how to apply to individual review bodies. Further information about GTAC is also provided on this website.
Additionally the Stem Cell Toolkit provides regulatory route maps that are specific to individual stem cell projects.
Other relevant agencies
Department for the Environment Food and Rural Affairs (DEFRA)has an Advisory Committee on Releases to the Environment (ACRE), 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) has the Scientific Advisory Committee on Genetically Modified Organisms (Contained Use) – SACGM (CU). 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 became 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, HTA, HFEA, HRA and MHRA, who provide a co-ordinated, single response service for free regulatory advice.
Any query relating to the regulation of regenerative medicines, including 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
We work closely with others and will continue to engage with those involved in regenerative medicine, including researchers, the British Society for Gene and Stem Cell Therapy, and the Cell Therapy Catapult to help clarify the regulatory requirements that apply.
In the past we have 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.
A Regenerative Medicine Expert Group (RMEG)has been established to develop an NHS regenerative medicine delivery readiness strategy and action plan. The HRA is represented on this group.
More generally, we work in partnership with a range of organisations to improve the environment for research in the UK.
Please refer to our partnerships page for more information.
- UK Stem Cell Toolkit - 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 - 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 - 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 – 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) – 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 – 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 which is part of the MHRA.
- UK Trade & Investment (UKTI) Life Science Investment Organisation (LSIO) – 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 – this is an official group within the health technologies 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.