Clinical trials regulation – know the basics
Trials within the scope of the clinical trials regulations are called clinical trials of investigational medicinal products (CTIMPs): MHRA tend to refer to them just as ‘clinical trials’.
UK clinical trials regulations apply to trials that test the safety or efficacy of medicinal products, including medicines, vaccines, gene and cell therapies, etc. If you are planning an experimental medicine study, exploring mechanisms of action, the regulations may not apply. Our experimental medicine toolkit has more on these.
It isn’t always easy to determine if your trial is within scope of the UK clinical trial regulations. See MHRA guidance When a clinical trial authorisation is needed.
It is a legal requirement to have a sponsor for a CTIMP. In academic trials this is generally the employer of the chief investigator (it’s unlikely to be the funder). Sponsors have legal obligations and corporate systems in place to manage these.
Your trial must get approval from the relevant department in the sponsoring organisation. In Universities this could be the university ‘research governance’, ‘research integrity’, ‘research ethics’ or ‘sponsor’s’ office. In the NHS, it is usually the NHS ‘research and development’ or ‘R and D’ department.
Other approvals include the clinical trial authorisation (CTA) from the MHRA and NHS research ethics committee (REC) approval, obtained as a combined review from a new part of IRAS.
If your trial involves the NHS, or HSC in Northern Ireland, then you will need appropriate approvals or permissions for this. Further guidance is provided in the ‘approvals for clinical trials’ section.
It is a legal requirement to work to the principles of good clinical practice (GCP). If you are conducting a trial with commercial partners and the data will be used to support a marketing authorisation, you are likely to meet the more prescriptive demands of ICH GCP.
You must register your CTIMP and publish your results.
See the Clinical trials toolkit for guidance on the legal requirements for CTIMPs.
UK clinical trials regulation
Overview of MHRA requirements for UK clinical trials. Certain aspects are highlighted below. MHRA scientific advice for medicines, including clinical trial advice.
Is it a clinical trial of an investigational medicinal product?
Please read:
- MHRA guidance: When a clinical trial authorisation is needed
- MHRA guide to what is a medicinal product (PDF)
- Borderline products: how to tell if your product is a medicine
- Borderline between medical devices and medicinal products in Great Britain if it is unclear whether a product is a medical device or a medicine.
Approvals for clinical trials
Guidance on MHRA and HRA approvals can be found here:
- MHRA guidance on clinical trial applications in the UK
- HRA guidance on HRA approval (for trials led from England or Wales)
- guidance for trials led from Scotland
- guidance for trials led from Northern Ireland.
The combined review service brings together a single CTIMP application for both clinical trial authorisation and research ethics committee opinion obtained from a new part of IRAS.
The MHRA has published common problems with clinical trial applications and guidance on how to avoid them: Common issues identified during clinical trial applications.
Your clinical trial sponsor is responsible for submitting your clinical trial application to the MHRA. (Clinical trial sponsors must register to make submissions to the MHRA.)
Amendments and reporting
If you make changes to your trial after approval, an ‘amendment’, then the MHRA (and others) may need to know about this. For further guidance on amendments, see:
Change of principal investigator at an NHS site (or HSC site in Northern Ireland) or the addition of an NHS or HSC site are no longer substantial amendments see Key changes to UK amendment process.
Your clinical trial sponsor is responsible for submitting substantial amendments to the MHRA.
There are various reporting requirements for clinical trials (for example safety reports, progress reports and end of trial notifications). For further guidance, see HRA managing your approval.
Your clinical trial sponsor is responsible for submitting end of trial notifications and developmental safety update reports (DSURs) to the MHRA.
If you are a clinical trial sponsor MHRA guidance for sponsors on submitting clinical trial safety reports is useful, this includes information on the transition between reporting to EU and UK systems.
Importing and exporting
If you need to import or export clinical trial supplies for your trial, you should contact your clinical trial sponsor.
If you are a clinical trial sponsor you may find the following resources useful:
- MHRA guidance list for import and export the headline page with full list of guidance
- importing IMP to Great Britain from approved countries
- supplying IMP to Northern Ireland.
Trials with sites in the EU
For clinical trials in the EU, your clinical trial sponsor will need to make submissions on the Clinical Trials Information System went live on 31 January 2022. The EMA is providing training for the new system.
Additional information specific to advanced therapies
MHRA provides guidance on advanced therapy medicinal products regulation and licensing, including how to get Regulatory advice for regenerative medicines.
The Human Tissue Authority (HTA) human application pages will be useful for those developing cell and tissue-based therapies. HTA regulates those undertaking procurement, testing, processing, storage, distribution, import and export of tissues and cells for human application.
If using human embryonic stem cells for human application, please read joint guidance from the HTA, HFEA and MHRA.
Information around advanced therapies in the EU can be found on the EMA website. A good overview of the EU (also US and Japan) regulatory framework can be found in the following resources:
- Taking advanced therapy medicinal products (ATMPs) to market by Daniel Rabbie 2018
- Regulatory framework for advanced therapy medicinal products in Europe and United States, by Carolina Iglesias-Lopez and others, 2019.