Drugs blow for boy
Family speaks out as treatment rejected for routine use
CHILDREN with a rare muscle-wasting disorder have been denied access to a breakthrough drug shown to extend the time they are able to walk after watchdog Scottish Medicines Consortium (SMC) ruled it was not cost effective for the NHS.
Campaigners expressed “bitter disappointment” after ataluren – also known by the brand name Translarna – was rejected for routine use on the health service to treat children suffering from Duchenne muscular dystrophy.
The family of nine-year-old Michael Young, who has benefited from Translarna through a drug trial, said they would petition the SMC to reverse the “devastating” ruling. His mother Michelle Young, from Larbert in Stirlingshire, said: “Translarna is already available in many European countries. Yet here, boys like Michael will continue to lose the ability to walk before the age of 10, to rely on steroids with their severe side effects, go through painful surgery, loss of muscle function, ventilation, without any reprieve.
“How can we possibly accept that, despite medicine being available, nothing will be done?” Patients with the incurable genetic disorder experience muscle weakness from the age of three which progressively deteriorates until they become dependent on a wheelchair, typically between the ages of eight and 10. By their late teens, patients are usually unable to breathe on their own as their lungs deteriorate and most sufferers die from respiratory complications or heart failure in their 20s or 30s.
Translarna is the first therapy available which slows DMD’s progress, but only children who are still able to walk would be consid- ered for the treatment. There are currently five children in Scotland thought to be eligible for the drug, but their families have already been waiting 18 months for the Scottish Medicines Consortium to reach a decision on whether the drug would be made routinely available on the NHS.
Yesterday, the SMC announced it did not believe the treatment’s cost, which ranges from £154,000 to £739,344 per patient per year, was justified in relation to its health benefits. Parents will now be forced to appeal on a case-by-case basis to their local health board to request individual funding for the drug.
In November, five-year-old Cormac Fegan became the first and only child in the UK to temporarily receive the drug through the NHS, following a successful Individual Patient Funding Request (IPFR) to NHS Lothian NHS.
Robert Meadowcroft, chief executive of Muscular Dystrophy UK, added: “We are bitterly disappointed by today’s announcement.
“Our thoughts are with the Scottish families whose every hope has been pinned on this decision. They have endured an 18-month wait for news about a drug that could transform their sons’ childhoods, only to receive this blow.”
Translarna is not yet available on the NHS anywhere in the UK, but NICE, which rejected the drug for England and Wales in October last year, is currently considering whether to approve it on the basis of new evidence.
Professor Jonathan Fox, chairman of SMC, said: “It is disappointing that we were unable to recommend ataluren for Duchenne muscular dystrophy. We know that DMD significantly reduces life expectancy and the committee heard about its profound impact on people with the condition.”
‘‘ Our thoughts are with the Scottish families whose every hope has been pinned on this decision