Clark Doyle: Mum of Duchenne sufferer calls for special NHS budget to fund rare disease treatments (From St Helens Star)
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Clark Doyle: Mum of Duchenne sufferer calls for special NHS budget to fund rare disease treatments
THE mother of a six-year-old boy with a rare muscle-wasting condition has raised fears in a Parliamentary report that the NHS may refuse to fund breakthrough treatments.
Katy Doyle, whose son Clark has Duchenne muscular dystrophy, gave evidence to a document that was handed to the health minster Norman Lamb this week.
She tells how her family face a “race against the clock” for rare disease therapies to be licensed and fears local NHS boards may not earmark cash to fund them.
The report is the result of a six-month parliamentary inquiry into how the newly shaped NHS will deliver expensive rare disease medicines as more devastating genetic conditions become treatable in the future.
Compiled by the All Party Parliamentary Group (APPG) for Muscular Dystrophy, it reveals potential treatments for Duchenne are at clinical trials.
However, MPs fear that convoluted drug assessment processes, funding issues and lack of specialist care could delay, or even prevent entirely, new therapies from reaching children and young people, such as Clark.
A special budget for treating rare diseases was merged into the overall NHS budget.
It is believed potential genetic treatments for Duchenne, known as exon skipping, are on the horizon. However, they could cost as much as £100,000 per patient and the MPs are calling for a ring-fenced fund to pay for drugs for rare conditions.
They warn the way drugs are funded and assessed could lead to hundreds of children with life-shortening conditions being denied rapid access to new therapies.
Clark, whose family has raised thousands for charity after his story featured in the Star this year, was diagnosed with Duchenne muscular dystrophy two years ago.
The disease – which four-old Jack Johnson, son of ex-Wigan rugby league player Andy, also suffers from – causes muscles to weaken and waste over time, leading to disability and significantly shortened life-expectancy.
Katy, 30, from Waterfield Village, off Lowfield Lane, said: “Clark is only six so the condition hasn’t had a big affect on him yet, although he struggles with running and climbing stairs.
“The earlier we are able to treat Clark, the more likely we are to be able to stop further damage to his muscles. Having to wait while the NHS figures out all the funding and care issues around exon skipping is a real worry.”
The cost and clinical advisory body, the National Institute for Health and Care Excellence (NICE), told the BBC it accepts it needs to develop a different approach for looking at rare conditions.
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