FAMILY’S PLEA TO FUND LIFE-ENHANCING DRUG
Funding would help sufferers of muscular distrophy
A FAMILY from Gerrards Cross face an anxious wait for a decision that could help improve their lives.
Louisa and Gary Hill, parents to 10-yearold muscular dystrophy sufferer Archie, spoke of their fears ahead of a lifechanging meeting on Tuesday night.
The family fear a decision to fund the drug Translarna – which would enhance Archie’s condition – would be rejected by government body NICE.
Translarna is said to be the first drug to target the cause of this life-limiting condition, and is said to keep boys like Archie walking for longer.
NICE will meet to review whether the drug should be funded on the NHS. The Hills will hear before Christmas about NICE’s decision and whether the drug will be made available for Archie and 50 other boys like him.
Last month the family were left devastated after being told Translarna would not be recommended on the NHS without clarification of the costs involved.
Mr Hill said: “Our greatest fear is that NICE will decide that Translarna is too expensive, and Archie and the other boys will miss out. This drug could give them longer to do the things that are incredibly important for a child.
“All Archie wants to do is play football and keep up with his friends. It’s not like there are lots of other therapies out there for Duchenne.
“At the moment steroids and physiotherapy are about all there is to keep children mobile. It’s been such a tough wait for all the families involved.”
The drug has been made available in countries including Germany, France, Spain and Italy, but families across the UK have faced waits for news on funding.
Robert Meadowcroft, chief executive of Muscular Dystrophy UK, said: “When NICE meets, we urge them to do all within their power to find a workable solution for delivering on this long-awaited breakthrough. Archie’s childhood, and those of many other eligible young people across the UK, could be transformed.
“It is deeply encouraging that today, more and more therapies for Duchenne muscular dystrophy are reaching clinical trials. As Duchenne is progressive, we cannot afford to go through this convoluted and painful process each and every time a new therapy is in sight. The emotional cost for families simply cannot be justified.”