THE parents of a five-year-old boy with a rare muscle-wasting condition have told of their joy after health bosses agreed to fund another six months of “life-changing” medication.
Cormac Fegan, of Longniddry, became the first child in the UK to get access to the Duchenne muscular dystrophy (DMD) drug Translarna on the NHS, after a funding request last year.
We can go to the cinema as a family and go out in public without the worry that he is going to attack another person.Gary Fegan
His family has reported a “huge” improvement in his quality of life since taking the medication – but they faced an anxious wait to find out whether NHS Lothian would pay for a second round of the drug, which costs around £200,000 a year.
Dad Gary Fegan previously revealed his family would move to England if their last-ditch bid to get the drug failed, but doctors have now told him that a second round is a “done deal”.
The 43-year-old said there had been a “remarkable” change in his son’s fitness, behaviour and wellbeing.
A recent assessment for DMD patients saw Cormac walk 600 metres when previously he could manage just 375m in a given time.
“That’s a huge increase”, said Mr Fegan. “He was pretty much running. His scores have increased massively. This is not just a slight difference. It’s measurable. When he was first diagnosed we also had issues with his behaviour. He was prone to biting but now it has almost stopped.
“It has given him the ability to control himself. We can go to the cinema as a family and go out in public without the worry that he is going to attack another person. He can be in the garden unsupervised and he can play normally with his brother. Before one of us had to be permanently on guard. It has brought down tension in the house considerably.”
The drug, which can slow the progression of the condition, was rejected by the Scottish Medicines Consortium (SMC) last month and the family had to apply to NHS Lothian for further treatment.
Robert Meadowcroft, chief executive of Muscular Dystrophy UK, urged the SMC to come up with a long-term solution to help families.
He said: “It seems unbelievably cruel that the SMC would leave families in fear and uncertainty about their child’s future. Families living in Scotland, who could benefit from Translarna, may now have to consider uprooting their children to England, where the drug has been approved.”
Mr Fegan wants the SMC to overturn their original decision amid fears his family may face the same anxious wait in six months’ time.
He said: “While it is a great relief to get that second round of funding, it’s disappointing that it’s only for six months when it has been shown there is an active benefit – and it hasn’t been given indefinitely or for a longer period of time.
“I can’t see the SMC making any changes in the next few months and I expect us to have to go through this whole thing again – coming cap in hand, begging for help.”
An SMC spokesman said he recognised its decision was “disappointing” but added: “Ataluren [Translarna] is an extremely expensive medicine and NHS Scotland has finite funding. Where a medicine is not recommended by SMC, health boards have procedures in place to consider individual requests.”
A Scottish Government spokeswoman said there was a need for a “long-term solution” at a “fair price” to the NHS.
“We are pleased to note that the pharmaceutical company has already committed to going back to the SMC with a resubmission,” she added.