EUROPLAN II: The next step in the UK’s Strategy for Rare Diseases (2)

EUROPLAN II: The next step in the UK’s Strategy for Rare Diseases


Earlier this year I wrote about the publishing of the UK strategy for Rare Diseases. This week, I was fortunate to attend EUROPLAN II, an event hosted by EUROCERD and Rare Disease UK. The event aims to provide insight and gather opinion on how the strategy for Rare Diseases should be implemented in each of the four countries of the UK. This isn’t an easy task. There are four countries in the UK, each with a devolved healthcare system, with vast differences in population sizes, social issues and geography, meaning we will have four similar but uniquely tailored approaches to Rare Disease care and its funding in each country.

The idea of ensuring unity and collaboration was echoed throughout the day on topics from research databases, cross centre-collaboration and disease management. In some situations, the case for a unified approach was identified as needing a solution beyond the UK and an EU-wide approach was recommended. A clear example how this approach would be useful are patient databases, where from a patient perspective centralising patient information in such small populations will allow identification of patients who then have the option to access trials, research opportunities and support from patient organisations. From a research perspective, this unified database would not only help with all of the above, but also collect enough meaningful controlled data to potentially allow new products and techniques to be developed, improve market access and reimbursement challenges and hopefully deliver better care to those needing it.

This is going to be a huge challenge for the UK’s National Health Service (NHS) and the Rare Disease community across Europe, one which will take time to accomplish. By being a large and diverse population of people, there is also the responsibility to ensure nobody is left behind in getting access to this new system. This will mean there will be a place for wide reaching communications in this new approach, something I do not want to dwell on too much today. I instead want to reiterate that without the unified approach, which is being created to solve the challenges of Rare Diseases, those needing help will continue to struggle to access the diagnosis and care they need.