Rare disease trials to develop playbook for testing new treatments

Patients with rare diseases could benefit from a “revolution” in clinical trials that could see one-stop studies designed that provide robust results even with small numbers of participants.

The CAPTIVATE node is part of the recently introduced UK Rare Disease Research Platform established as part of a £14 million investment over five years by the Medical Research Council (MRC) and the National Institute for Health and Care Research (NIHR).

Led by the University of Birmingham along with collaborators at the Universities of Sheffield and Liverpool, the CAPTIVATE node will be developing a methodology to run a ‘one-stop-study’, which would encompass all phases of clinical trials to enable the efficient evaluation of treatments for rare diseases.

The CAPTIVATE node will bring together the UK's leading trial experts with hospital researchers experienced in rare diseases, industry partners, policy makers and patient partners, and the resulting designs could speed up the approval of medicines for use in rare diseases.

“Added to this fact, in the UK three quarters of rare diseases affect children and more than 30% of children with a rare disease die before their fifth birthday.

“The CAPTIVATE node is looking to scale a revolutionary model of clinical trial that runs all the phases together and can get clinically significant results even with very small numbers of patients. We want to find ways that need as few people to take part as possible, that are quicker and that provide all the information needed for the authorities to approve a new medicine to be prescribed to people with rare diseases from one single trial.”

Bringing together strengths in rare diseases

The MRC-NIHR UK Rare Disease Research Platform is now getting up and running and will bring together UK strengths in rare diseases research to improve diagnosis and treatment through better understanding of the disease.

It is made up of a central coordination and administrative hub and 11 specialist nodes based at universities across the UK, including the CAPTIVATE node at the University of Birmingham.

The aim of the platform is to bring together expertise from across the UK rare disease research system to foster new and innovative treatments for those directly and indirectly impacted by rare conditions.

“The NIHR continues to lead essential ongoing research into rare diseases, including through our Biomedical Research Centres, and we are making it easier for people with rare diseases to take part in research opportunities via our Be Part of Research Service, which can now be accessed through the NHS app in England. We look forward to working with our partners further to accelerate our understanding and treatment of rare diseases affecting millions of people across the UK.”