UK: New £14 million investment targets rare diseases

[Andy]

A new platform bringing together UK strengths in rare diseases research to improve diagnosis and treatment through better understanding of the disease.

The UK Rare Disease Research Platform has been established with a £14 million investment over five years by the Medical Research Council (MRC) and the National Institute for Health and Care Research (NIHR).

It is made up of a central coordination and administrative hub and 11 specialist nodes based at universities across the UK.

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.

https://www.ukri.org/news/new-14-million-investment-targets-rare-diseases/

 

[InitialConditions]

Suddenly ME/CFS is no longer 'a rare disease'.

 

[Shadrach Loom]

Suddenly ME/CFS is no longer 'a rare disease'.

Was it ever? Prevalence figures vary, but never reach the 1 in 2000 threshold.

 

[InitialConditions]

Was it ever? Prevalence figures vary, but never reach the 1 in 2000 threshold.

No, that's why I used quotation marks!

 

[Shadrach Loom]

No, that's why I used quotation marks!

It’s a reasonable threshold though, isn’t it? ME wasn’t really rare, I’d have thought, even before long covid. I know several people IRL who have it or have had it.

 

[CRG]

Was it ever? Prevalence figures vary, but never reach the 1 in 2000 threshold.

For at least 30 years it's been a cornerstone of ME advocacy that there are large numbers of patients which demands high levels of investment - UK quoted figures usually 125,00 - 250,00, so at least 3.5 x the maximum for a rare disease.

 

[Shadrach Loom]

For at least 30 years it's been a cornerstone of ME advocacy that there are large numbers of patients which demands high levels of investment - UK quoted figures usually 125,00 - 250,00, so at least 3.5 x the maximum for a rare disease.

By the way, this is all very well when advocating for research funding, but it strikes me as a counterproductive argument when aimed at insurers and benefits policymakers. We really want those people to be doing their sums based on a small, manageable cohort of “moderate plus” pwME and pwLC. It will take them years to realise that they have underestimated demand and spent the equivalent of a new aircraft carrier on keeping us decently looked after.