NHS warned over rare disease funding

The All Party Parliamentary Group for Muscular Dystrophy calls on Ministers to set up ring-fenced funding for high-cost rare disease medicines. It also urges the National Institute for Clinical Excellence (Nice) to take a new approach to assessing treatments for rare conditions.

Mark Creswick, of High Green, Sheffield, whose son Harley has the life-shortening condition Duchenne’s muscular dystrophy, gave evidence for the report. 
He said: “Unless we find a treatment to stop it, his muscles will weaken and waste as he grows, affecting first his legs, then his arms and eventually his heart and lungs. We are racing against the clock.”

Potential treatments are being trialled, but the report warns convoluted assessment procedures, funding problems and a lack of specialist care could delay or even prevent them reaching children.

They raise concerns funds for rare diseases have been merged into overall budgets for NHS services, leaving them competing with funding for common ailments.

Robert Meadowcroft, of the Muscular Dystrophy Campaign, said: “We are concerned that as breakthrough treatments emerge, poor planning at this stage could stall - or prevent entirely - successful treatments from reaching those who need them. The NHS must also work to ensure that all patients have access to the right specialist care before treatments become available, or risk families enduring long delays before they can be administered.”