£12M research centre will improve efficiency of rare disease trials to unlock tests and treatments

Researchers from The University of Birmingham have created a new £12M research centre to improve clinical trials for rare diseases.

Globally, there are more than 300 million people living with rare diseases. However, clinical trials for rare diseases are challenging and can be a major limiting step in getting new treatments to patients. The research can be fragmented, and researchers sometimes lack access to specialist facilities, as well as advice on regulation, trial designs, preclinical regulatory requirements, and translational project management.

The LifeArc Centre for Acceleration of Rare Disease Trials brings together a consortium of three universities from across the UK. Newcastle University, Queen’s University Belfast, and University of Birmingham are pooling their expertise in a partnership coordinated by Professor David Jones, Professor of Liver Immunology at Newcastle University.

The £12M Centre will focus on improving the efficiency of rare disease trials and increasing the number of opportunities for patients to take part. Researchers will develop a UK ’4 nations’ approach to deliver trials of new treatments using ’one stop’, patient friendly models.

The team will do this by creating a rare disease trial recruitment portal and will design and deliver trials in partnership with patients. This will speed up the delivery of clinical trials for people with rare diseases and enable more rapid approval of new therapies for use in the NHS.

Professor Timothy Barrett, Director of the Centre for Rare Disease Studies at the University of Birmingham said:

"Birmingham is justly proud of its hospital services and scientific research for people living with rare conditions, which build on our partnership between hospitals and University and reflects the cosmopolitan nature of our region.

"This award will represent a stepping stone in our ambition for patients in Birmingham to get more treatments to more people with rare diseases, faster. It also allows us to expand capacity for rare disease clinical trials for the whole of the UK."

This award will represent a stepping stone in our ambition for patients in Birmingham to get more treatments to more people with rare diseases, faster. It also allows us to expand capacity for rare disease clinical trials for the whole of the UK.

Professor Timothy Barrett, Director, Centre for Rare Disease Studies, University of Birmingham
Kerry Leeson-Beevers is the parent of a child with the rare genetic condition, Alström Syndrome, which often causes loss of vision and hearing, and can lead to serious life-threatening problems with the heart, liver and kidneys.

Kerry, who is also CEO of Alström Syndrome UK, said:

"We have no specific treatment for Alström Syndrome and when my son, Kion, was a baby, I was told it could take around 10 years for any treatment to be developed. 20 years later, we are still waiting. People living with rare conditions don’t have the luxury of time and the mainstream way of delivering healthcare and drug development rarely works for people with rare conditions.

"As a mum and the Chief Executive of Alström Syndrome UK, having a centre that will deliver a coordinated, inclusive and supportive approach to accelerate clinical trials gives me great hope."

The LifeArc Centre for Acceleration of Rare Disease Trials, along with the the LifeArc Centre for Rare Respiratory Diseases, LifeArc Centre for Rare Kidney Diseases, and LifeArc Centre for Rare Mitochondrial Diseases, has been awarded a share of nearly £40M over five years from the not-for-profit medical research charity, LifeArc.

Each centre will tackle an area of unmet need, to unlock science, accelerate medical progress and have the greatest impact for patients.

Dr Catriona Crombie, Head of Rare Disease at LifeArc, said:

"We’re extremely proud to be launching four new LifeArc Translational Centres for Rare Diseases. Each centre has been awarded funding because it holds real promise for delivering change for people living with rare diseases. These centres also have the potential to create a blueprint for accelerating improvements across other disease areas, including common diseases."

Professor Timothy Barrett

Leonard Parsons Professor of Paediatrics and Child Health

Dr Martin R. Higgs

Associate Professor for Genomics and Rare Disease


    For media enquiries please contact Tim Mayo , Press Office, University of Birmingham, (0)7815 607157.

    The University of Birmingham is ranked amongst the world’s top 100 institutions. Its work brings people from across the world to Birmingham, including researchers, teachers and more than 8,000 international students from over 150 countries.

    The University of Birmingham is a founding member of Birmingham Health Partners (BHP), a strategic alliance which transcends organisational boundaries to rapidly translate healthcare research findings into new diagnostics, drugs and devices for patients. Birmingham Health Partners is a strategic alliance between seven organisations who collaborate to bring healthcare innovations through to clinical application:
    • University of Birmingham
    • University Hospitals Birmingham NHS Foundation Trust
    • Birmingham Women’s and Children’s Hospitals NHS Foundation Trust
    • Aston University
    • The Royal Orthopaedic Hospital NHS Foundation Trust
    • Sandwell and West Birmingham Hospitals NHS Trust
    • West Midlands Academic Health Science Network
    • Birmingham and Solihull Mental Health NHS Foundation Trust




About LifeArc

LifeArc is a self-funded, non-profit medical research organisation. We take science ideas out of the lab and help turn them into medical breakthroughs that can be life-changing for patients. We have been doing this for more than 25 years and our work has resulted in five licensed medicines and a diagnostic for antibiotic resistance.

Our teams are experts in drug and diagnostics discovery, technology transfer, and intellectual property. Our work is in translational science - bridging the gap between academic research and clinical development, providing funding, research and expert knowledge, all with a clear and unwavering commitment to having a positive impact on patient lives.

LifeArc, a charity registered in England and Wales under 1015243 and in Scotland under SC037861.

Professor Timothy Barrett

Leonard Parsons Professor of Paediatrics and Child Health

Dr Martin R. Higgs

Associate Professor for Genomics and Rare Disease

Rare disease trials to develop playbook for testing new treatments

MRC and NIHR funded project to develop new 'one-stop-study' method for running clinical trials with all phases included.
  • Edgbaston
  • Birmingham, B15 2TT
  • United Kingdom