NIHR and MRC Establish UK Rare Disease Research Platform

Rare Daily Staff

The National Institute for Health and Care Research and the Medical Research Council are co-funding a new UK Rare Disease Research Platform to overcome some of the most common challenges in the diagnosis, management, and treatment of rare diseases.

Established with an $18 million (£14 million) investment over five years from the NIHR and the MRC, the platform will bring together research teams across the UK with patients, stakeholders, and technologies to deliver rare disease research with greater impact.

The platform marks part of significant NIHR investment for rare disease research announced in the Rare Disease Action Plan for England in February 2023. This wider investment includes support for a range of rare diseases research through NIHR’s Biomedical Research Centers.

“The UK Rare Disease Platform marks a significant advance in accelerating rare disease research, supported through NIHR funding and our partners the Medical Research Council. The platform will enable greater collaboration between patients and those working across academic, clinical and industry research,” Lucy Chappell, chief executive of the NIHR. “By bringing the right people and expertise together, we will be able to provide better care more quickly to those living with rare diseases.”

NIHR leads essential ongoing research into rare diseases, including through its Biomedical Research Centers, and makes it easier for people with rare diseases to take part in research opportunities via its “Be Part of Research Service,” which can be accessed through the NHS app in England.

The UK Rare Disease Research Platform is a network which connects experts across the country. It is made up of a central coordinating hub and 11 specialist nodes based at universities across the UK. The hub will bring these specialist nodes together and deliver wider activities to support rare disease research. The 11 nodes each involve several universities and organizations working together.

The platform will provide a point of entry for industry and international partners. NIHC says it will aim to attract investment into UK research, proactively engage industry partners, and also support a set of new projects that will identify and solve particular challenges and unblock progress across many rare diseases or groups of rare diseases.

“The UK has great strengths in rare disease research. However, the rarity of these diseases means it can often be difficult to bring the right people, technologies and tools together. Although these diseases are diverse and have different causes, they often bring common challenges for research into disease mechanisms, diagnosis, management and treatment,” said John Iredale, MRC executive chair. “The platform will bring people together, link participants to resources and activities in the UK and internationally, and support projects that deliver advances in rare disease research.”

Photo: John Iredale, MRC executive chair