About us
We are committed to accelerating the development of therapies for rare diseases. Through a collaborative effort between patients, researchers, clinicians, and industry partners, we are working towards overcoming the barriers that limit rare disease research.
A rare disease affects fewer than 1 in 2,000 people. While each condition is uncommon, there are over 7,000 rare diseases worldwide, impacting millions
These diseases can often be difficult to diagnose, have limited treatment options, and pose significant challenges for patients and their families.
At the core of our work is the understanding that rare diseases often do not receive the attention they deserve. Many of these conditions are severe and life-limiting, yet clinical research into new treatments is scarce. This project seeks to accelerate the development of therapies for rare diseases, ensuring that these conditions no longer remain overlooked.
By collaborating with researchers, patients, and industry partners, we are working to overcome the unique barriers to conducting clinical trials for rare diseases. We aim to speed up the development of therapies by creating innovative, patient-centred solutions that address the specific challenges faced by rare disease communities.
Together, we can make significant progress toward improving the lives of those affected by rare diseases and paving the way for more effective treatments.
Meet our Leadership Team
Centre Co-ordinator
Professor David Jones, OBE
Professor Dave Jones is an internationally recognised expert in rare disease research and clinical trial design, with a particular focus on autoimmune liver diseases. His research is dedicated to understanding the impact of these diseases on patients and improving outcomes through novel therapeutic strategies. He is the Principal Investigator for the UK-PBC national research consortium and serves as the founding Director of the Newcastle University Centre for Research Excellence in Rare Disease. Additionally, as the NIHR Dean for Faculty Trainees, Professor Jones supports the next generation of clinical researchers, fostering the growth of talent in the field of rare disease research.
In his role as Centre Lead for the LifeArc Centre for Acceleration of Rare Disease Trials, Professor Jones leads the strategic direction and central coordination of the Centre’s activities. He ensures effective collaboration between researchers, clinicians, and patients to streamline the clinical trial process and accelerate the development of new treatments for rare conditions. His leadership ensures that the Centre’s projects are well-coordinated, with a focus on improving patient outcomes and driving forward innovations in rare disease trials.
Newcastle University
Professor Volker Straub
Professor Volker Straub is the Director of the John Walton Muscular Dystrophy Research Centre and Deputy Dean for Newcastle University’s Translational and Clinical Research Institute. He is a Consultant in Neuromuscular Genetics and Paediatrics, and an NIHR Senior Investigator.
Volker has a long-standing interest in the pathogenesis of genetic muscle diseases, with research involving disease models, the application of imaging technologies, next generation sequencing, and machine learning. He is the current president of the World Muscle Society and an author on over 450 peer-reviewed publications.
University of Birmingham
Professor Timothy Barrett
Timothy Barrett is Leonard Parsons Professor of Paediatrics and Child Health in the Department of Cancer and Genomic Sciences. He is an Honorary Consultant in Paediatric Endocrinology and Director of the Centre for Rare Disease Studies (CRDS) Birmingham, an NIHR Senior Investigator, co-leads the LifeArc Centre for Acceleration of Rare Disease Trials (LifeArc ARDT), and the Birmingham contribution to the GOSH BRC Paediatric Research Network of Excellence.
Timothy has published over 200 research papers in scientific journals as well as reviews and book chapters in the fields of paediatrics, diabetes and genetics of childhood diabetes syndromes. He has received major grants from The Medical Research Council, The Wellcome Trust, UK National Institute for Health Research, European Union Directorate General for Health and Consumer Affairs, Diabetes UK and Wellchild. His research interests include functional genetics of rare diabetes syndromes, translational research to early phase clinical trials in rare disease, and complex interventions to reduce health inequalities in childhood diabetes.
Queens University Belfast
Professor AJ McKnight
As a professor of molecular epidemiology and public health, Prof. McKnight’s research is focused on molecular factors that influence human disease and promote health ageing. She runs the NI rare disease research centre and with colleagues, she has helped develop powerful approaches to data analysis and data integration to identify novel biological pathways for disease, discovering many risk factors and biological networks for rare diseases.
As an award-winning researcher, her interests are around translating high quality research into tangible benefits from the NHS, industry and academia collaborations. Her efforts in the All-Ireland Rare disease Interdisciplinary research Network (RAiN), and the RD-IMAG and RD research landscape groups will be critical in the success of the centre.
Our Centre Aims
The Accelerating Rare Disease Trials (ARDT) Centre aims to support and enhance rare disease research across the four nations, ensuring that clinical trials rapidly translate into real-world impact for people living with rare diseases. By bringing together experts from Newcastle University, Queen’s University Belfast, and the University of Birmingham, we are developing a national trial platform that streamlines recruitment, enhances trial methodologies, and accelerates the approval of life-changing treatments. Our work is guided by patient-centric approaches, innovative trial design, and collaboration across data platforms to ensure that rare disease trials are efficient, inclusive, and accessible to all patients.
- Developing a novel rare disease trial recruitment platform, leveraging secure NHS data to create trial-ready cohorts and enable patient referral regardless of location.
- Establishing a distinct trial design framework, with enhanced methodological approaches to overcome the common barriers associated with rare disease trials and improve trial efficiency.
Through these initiatives, the ARDT Centre is building a sustainable, UK-wide infrastructure that enables faster trial delivery, better patient engagement, and the rapid approval of new therapies. By working in close partnership with researchers, clinicians, and patient communities, we aim to remove traditional barriers to rare disease trials and bring effective treatments to patients sooner.
