By Violet Brown
A NEW groundbreaking £12m research centre to improve the efficiency of rare disease trials to unlock more tests and treatments has been created at Queen’s University Belfast (QUB) with people from the Ards peninsula at the heart of the project.
More than 300 million people are living with rare diseases across the world, but clinical trials for rare diseases are challenging and limit the ability to get new treatments to patients.
This £12m centre brings together experts from QUB, Newcastle University and the University of Birmingham who will focus on improving the efficiency of rare disease trials and increase the number of opportunities for patients to take part.
Researchers will develop a United Kingdom ‘4 nations’ approach to deliver trials of new treatments using patient friendly models, enabling more rapid approval of new therapies for use in the NHS.
Kircubbin woman, Professor Amy Jayne McKnight, from the School of Medicine, Dentistry and Biomedical Sciences at QUB, who is co-leading the project, explained there were more than 110,000 people living with rare conditions in Northern Ireland.
“Less than five per cent of rare diseases have an approved therapy, so more trials with innovative designs are urgently required and I’m looking forward to sharing expertise with colleagues as we establish and develop this crucial rare disease initiative,” she said.
The centre in Northern Ireland will establish a collaborative Northern Ireland rare disease trials hub that will support building local infrastructure and skills and allow capacity for trial design and delivery.
It will also aid in the development of practical tools to support patients and doctors participating in research, whilst ensuring the voices of patients and families remain at the centre of this progress.
“Lived experience from patients and their families across Northern Ireland was crucial in helping shape the design of this centre to be of maximum benefit,” Ms McKnight said.
Ms McKnight’s husband Sam Robinson, a patient with a rare disease, is co-ordinating community input into the project.
“It’s great to have funding for Northern Ireland to help get better drugs to more patients faster,” he said. “This means so much to patients and families living with rare diseases who cannot travel across the water to get treatment.”
Greyabbey woman, Christine Collins MBE, chair of the Regulation and Quality Improvement Authority (RQIA) is leading the patient and public engagement component of the project.
“This £12m programme gives everyone affected by a rare disease the chance to contribute to getting better awareness, quicker and more accurate diagnosis and develop improved treatment pathways,” she said.
Ms Collins said the centre would build Northern Ireland’s ability to tackle these challenges, including by training the next generation of rare disease researchers through a training programme for rare disease.
“It is wonderful that people from the Ards peninsula are front and centre in making this happen,” she said. “I am really looking forward to working with the clinical experts and families to make the most of this exciting opportunity”.
