A new UK Rare Disease Research Platform is being co-funded by the NIHR and the MRC, which will “bring together research teams across the UK with patients, stakeholders and technologies to deliver rare disease research with greater impact”.
Established with a £14 million investment over five years, it is hoped that the platform will “overcome some of the most common challenges in the diagnosis, management and treatment of rare diseases”. It will also constitute a part of the NIHR’s wider investment in rare disease research as outlined in the Rare Disease Action Plan for England, published in February of this year.
As a “network which connects experts across the country”; the platform will consist of a “central coordinating hub and 11 specialist nodes based at universities across the UK”. Included in these 11 nodes are the Epigenomics Rare Diseases Node led by the University of Exeter and the University of Manchester; the Early Assessment, Diagnosis and Treatment of Parkinson’s Plus Related Syndromes (ExPRESS) led by University College London; and Cardiovascular Rare Disease led by Imperial College London.
It is hoped that the new platform will help to attract investment into UK research, and that it can be used to “identify and solve particular challenges and unblock progress across many rare diseases or groups of rare diseases”.
Professor Lucy Chappell, chief executive of the NIHR, said: “The platform will enable greater collaboration between patients and those working across academic, clinical and industry research. By bringing the right people and expertise together, we will be able to provide better care more quickly to those living with rare diseases.”
Professor John Iredale, MRC Executive Chair, is also quoted commenting on the “great strengths” of the UK in rare disease research, along with the “common challenges” for research into disease mechanisms, diagnosis, management and treatment. Ultimately, he is quoted as saying that he believes “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”.
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