DECISION IS A BITTER BLOW FOR FAMILY
Parents slam organisations ‘prioritising cost over health of children’
A FAMILY has been left devastated following a government decision to not recommend funding for a breakthrough drug.
Louisa Hill – who’s son Archie suffers from muscular dystrophy – slammed a recent decision made by the National Institute for Health and Care Excellence (NICE) over the Duchenne muscular dystrophy drug Translana (ataluren).
She said: “We are absolutely devastated.
“How do we tell Archie he is not allowed a drug that will keep him walking and living for longer because NHS England and drug companies cannot agree on a price?
“What sort of an example are these people setting for society if they are prioritising cost over the health of our children? We will not be giving up. This fight is far from over.”
The National Institute for Health and Care Excellence (NICE) announced in its draft guidance earlier this month it will not recommend funding on the NHS for Translana.
But NICE have asked PTC Therapeutics, manufacturers, to provide further justification for the cost of the drug and clarification of its benefits ahead of confirming its final position in the coming months and stress this is not their final decision.
Professor Carole Longson, NICE Health Technology Evaluation Centre director, said: “We are disappointed not to be able to recommend ataluren in this draft guidance. DMD is one of the most common and severe forms of muscular dystrophy. It reduces life expectancy and causes debilitating symptoms that severely affect the quality of life of people with the condition, and their families and carers.”
She added: “However, the Committee was not convinced that the proposed cost of ataluren was justified by the evidence presented on the additional health benefits associated with ataluren over standard therapy.
“Therefore, on the basis of the current evidence, the Committee was minded not to recommend ataluren for treating nonsense mutation DMD.”
In August 2014, Translarna became the first drug addressing the cause of Duchenne muscular dystrophy to be approved in the EU, and since August last year has been available in several EU countries.
It is said to keep those who suffer from Duchenne muscular dystrophy walking for longer.
Recently a local NHS Trust in Scotland approved the drug for one local boy, following an individual funding requst.
Robert Meadowcroft, chief executive of Muscular Dystrophy UK, said: “The news is extremely disappointing, and a bitter blow for all of us awaiting the news.
“Having spoken to some of the families affected, it really is impossible to describe their heartbreak, particularly as just last week Lothian NHS Trust approved the drug for a family in Scotland.
“Many parents across the rest of the UK are watching their child weaken and lose mobility day by day, as this really is a race against time. If we are not careful, it will be too late for many of these boys to even be eligible to take Translarna.
“Duchenne is a lifelimiting condition, and we must take every opportunity to protect children and their quality of life.
“The chance to be able to walk for longer can be immeasurably precious. We may not yet be able to halt the difficult physical and emotional challenges these children face, but with this drug, we have the chance to delay them.
“PTC Therapeutics needs to work with NICE and NHS England to find an urgent solution to approving this breakthrough therapy. We will not give up on these boys who are eligible for Translarna – they must have access to this life-changing drug.”
The next committee meeting is scheduled for November 17, following which the next draft guidance will be issued.
A final recommendation is expected to signed off early next year. For MP Cheryl Gillan’s thoughts about this issue see page 15
Devestated: Archie Hill with his parents Louisa and Gary and elder brother Leyton