The three projects aim to improve how patients can access life-changing treatments in clinical trials, find a way to introduce complex AI safely into front line clinical settings and make the UK the place to launch advanced microbiome products that can support the development of personalised medicine.

These projects support one of the Agency’s core principles, of unlocking scientific and digital innovation that keeps pace with the changing needs of patients, to ensure it delivers world-leading research innovation that focuses on protecting and improving patient health.

Synthetic data in clinical trials

The first project has been awarded £750,387 to tackle the challenges of finding control groups for clinical trials, through the development of alternative synthetic datasets.

Currently, patients run the risk of being randomly assigned to control groups and therefore are denied access to treatments that could improve their symptoms or extend their life, and clinical trials for rare diseases can often find it difficult to recruit enough patients for their trials altogether.

However, the MHRA have recently made great strides in creating artificial patients, with similar health information to real patients, that could both boost the numbers for small trials and better reflect groups in society that are less well represented in clinical trials, such as different age groups and ethnicities.

This project therefore aims to create a successful, entirely artificial control group to help change the way clinical trials are performed in common and rare diseases, lower their cost and improve how new treatments are tested before they are applied in the NHS. It is thought that this project will take between 12-18 months to complete.

Dr Puja Myles, Director of the Clinical Practice Research Datalink at the MHRA, said:

This funding will allow us to build upon the innovative work we have developed here at CPRD, both before and during the pandemic, creating synthetic datasets that mimic real patient data for use in clinical trials.

This provides researchers with high-fidelity data that replicates the complex clinical relationships in real primary care data while protecting patient privacy, as they are wholly synthetic.

This funding could revolutionise how clinical trials are conducted, giving patients access to potentially life-changing treatments, saving money that could be better spent on more advanced research while providing highly reliable results for the clinical researchers.

Complex AI algorithm interpretability for clinicians

The second project aims to address the issue of how to safely introduce complex ‘black-box’ AI products into clinical settings, so that clinicians can be confident that the decision from the AI device is appropriate and suitable in that specific context.

£167,863 has been awarded to produce a workable methodology that can help to provide clinicians with an understanding of what factors the algorithm considered and the weight it assigned them so that they can see how the algorithm reached its decision and therefore whether to overrule it or not.

This project will accelerate key parts of the recently published roadmap for the Software and AI as a Medical Device change programme, the MHRA’s ambitious programme of reform to ensure that medical device regulation is fit for purpose for software, including AI.

Prof Johan Ordish, Head of Software and AI at the MHRA, said:

AI as a medical device is crucial in supporting the development of next-gen healthcare.

However, the transparency and explainability of AI has a key relationship with the safety as well as usability of these devices, impacting whether clinicians adopt and trust AI as a medical device.

Patient safety is at the heart of everything we do, and we’re determined to be well prepared to get AI technology to the bedside safely, effectively and with confidence in this assurance.

By producing a methodology that supports the regulation of both transparent and complex AI models, it will help us to make this vision a reality.

Bringing microbiome expertise into the UK

The third project has been awarded £52,438 to explore the development of guidelines around regulating microbiome therapeutics and diagnostics, a rapidly emerging field that poses a challenge for regulators and companies due to its novel and complex nature.

Therefore, appropriate internal and external guidance based on expert scientific knowledge are needed to facilitate the regulatory process, bring confidence to the microbiome field and ensure the safety of patients.

Dr Nicola Rose, Deputy Director for Research and Development at the MHRA, said:

The study of microbes associated with the human body has the potential to greatly advance our understanding of human health and disease, providing new solutions for public health and allowing us to move towards a new era of personalised medicines.

However, the absence of specific guidelines can delay the approval process for companies looking to bring their expertise and their pioneering treatments to the UK.

We want to change that and make the UK the home for innovation in microbiome therapeutics and diagnostics, and will work with our extended network of scientists, clinicians, companies and regulators, both nationally and internationally, to capture the requirements needed for effective guidance.

Further information

Notes to editors

• The Medicines and Healthcare products Regulatory Agency (MHRA) is responsible for regulating all medicines and medical devices in the UK by ensuring they work and are acceptably safe.  All our work is underpinned by robust and fact-based judgements to ensure that the benefits justify any risks.

• The MHRA is an executive agency of the Department of Health and Social Care.

• These projects have been made possible by a grant from the £12 million Regulators’ Pioneer Fund launched by the Department for Business, Energy and Industrial Strategy (BEIS). The fund enables UK regulators and local authorities to help create a UK regulatory environment that unleashes innovation and makes the UK the best place to start and grow a business.