I’ll move to England for my sick son’s vital treatment
Dad’s vow as NHS refuses drug
A SCOTS father has told how he is planning to move to England after health chiefs refused to approve the £200,000-a-year cost of a drug that has transformed his son’s life.
Cormac Fegan was born with Duchenne Muscular Dystrophy (DMD) – a rare, life-shortening condition which causes muscle degeneration.
For the past four months the five-yearold has been receiving Translarna – a new drug which has drastically improved his behaviour and physical strength.
But this week the Scottish Medicines Consortium (SMC) confirmed it would not approve NHS funding for the treatment. Now Cormac’s family have vowed to move to England if the treatment is accepted there.
The youngster, from Longniddry, East Lothian, frequently used to bite others and had trouble walking – common side effects of DMD.
Earlier this year NHS Lothian agreed to fund his treatment for six months at a cost of £100,000.
His behaviour improved drastically and both his balance and speed have increased.
But he will not be able to continue taking the life-changing medicine as the SMC was ‘unable to recommend ataluren (Translarna) for routine use’.
His parents hope than an appeal will overturn the decision, or that Translarna will become available on NHS boards in England and Wales following a National Institute for Health and Care Excellence announcement on Monday morning.
Cormac’s father, 43-year-old Gary, said: ‘I feel absolutely devastated at this decision, which at the end of the day came down to economics, not whether or not the treatment works. Two other families in Scotland have boys currently using Translarna and we know that it is effective.
‘In the case of Cormac it has transformed his disruptive behaviour... as well as helping make him physically stronger.
‘If it is approved in England we would definitely move, there’s no question. It would be the moral thing to do.’
He added: ‘The SMC has not grasped the importance of allowing these children to remain walking and independent.
‘With life-expectancy of Duchenne children being just their mid-20s, and some children losing their lives long before that, you can well imagine the difference extra years walking – during a child’s most formative years -can make.’
Cormac has approximately 30-days left of pills to take – and is set to run out mid-May. Mr Fegan said: ‘We’ll have to start gathering more information to send another funding request to NHS Lothian, though there is no certainty we’ll have it granted again.’
It is understood US-based manufacturers PTC Therapeutics will be appealing the SMC decision.
Paul Lenihan MBE, CEO of charity Action Duchenne, said: ‘We are absolutely appalled by the SMC’s decision and method for evaluating this groundbreaking medicine.
‘It is clear that Scotland has no framework for fairly appraising and funding drugs for rarer conditions.’
An SMC statement said: ‘The committee was unable to recommend ataluren (Translarna) for routine use in NHS Scotland for patients with Duchenne Muscular Dystrophy as there was too much uncertainty about the overall clinical benefits it might bring in relation to its cost.’
‘Appalled by this decision’