Here we are, a year into Rare Disease Research UK, the MRC (and NIHR) national platform in rare disease. It feels like a good time to look at what we have achieved, and to think about where we are going. The platform is up and running with all 11 of the nodes really pushing on with their research programmes. It is a good time to thank the hub team here in Newcastle for all their hard work, the node teams without whom there wouldn’t be a platform and, of course, the MRC team whose support allowed this to happen. A particular thank you goes to Richard Evans whose hard work and vision were instrumental in getting us to this point. Richard has now moved on to greater things as the Head of Population Health Sciences at the MRC and we all wish him well in his new role. The support of MRC and NIHR for rare diseases goes on, of course. I couldn’t help noticing that the incoming Executive Chair of the council is himself a world-renowned rare disease researcher.
Key parts of the Rare Disease Research UK mission are to bring together researchers in rare disease and to increase the visibility of this important area and we are making real progress. The programme of events to bring together the node researchers is well underway and will develop further over the next year. We are also keen to bring in other researchers with MRC and NIHR funding in rare disease to join the academic programme broadening it further.
An important aspect of what we do is to act as a “front door” for rare disease in the UK. There is growing interest in rare disease around the world, including in the industrial sector. Sometimes companies can find it difficult to find the clinical and research collaborators that they need. Taking a national perspective as we do we believe that the RDR UK platform can play an important role in connecting potential collaborators in all sectors. If you get an e-mail from us about potential partners, do answer it, it could be just the opportunity you are looking for. In addition, patient groups are essential in strengthening the rare disease community, and RDR UK is committed to involving them in our platform. In collaboration with Genetic Alliance UK, we have exciting plans for the coming year to engage with these groups and actively promote PPIE within the nodes’ research.
It has certainly been an exciting year in RDRUK, but that just mirrors a year of real progress and opportunity more generally in the rare disease field. The Rare Disease Framework continues in its implementation and offers the hope of real progress in care delivery for PBC patients in the NHS system. We also welcome the significant investment made by LifeArc (a real friend and supporter of the rare disease community) in their Rare Disease Translational Research Centre. We look forward to working with LifeArc in further developing the rare disease eco-system over the next week years.
We look forward to meeting as many of you as possible over the next year at our RDR UK events. Remember, it is your platform. Use it!
Prof David Jones
Lead, RDR UK Hub
