English children get £200k drug
... as ill Scots youngster forced to move south
ENGLISH health chiefs have approved a ‘miracle drug’ denied to Scots children.
The £200,000-a-year treatment for Duchenne muscular dystrophy has worked wonders for patients, including five-year-old Cormac Fegan.
But his father Gary says he may have to move the family from Longniddry, East Lothian, south of the Border so his son can continue to receive Translarna.
The Scottish Medicines Consortium (SMC) said the drug is not cost-effective – but counterpart the National Institute for Health and Care Excellence (NICE) yesterday approved it for patients in England, Wales and Northern Ireland.
Cormac received six months of treatment at the discretion of his local health board. From a young age, he had trouble walking and was constantly biting other children. But after NHS Lothian confirmed individual funding, his behaviour improved drastically.
On Monday, he was told he will not be able to continue taking the drug. His father said: ‘It’s ridiculous. If we don’t get individual funding, we will be moving. It would be morally bankrupt if we didn’t make that decision.
‘There would be a lot to sort out and we would have to find a new house and schools, but we would have to do it.’
NICE said: ‘Translarna represents an important development in treatment. Potential benefits associated with it are great enough to justify its cost to the NHS. It is therefore recommended for treating Duchenne muscular dystrophy.’
Robert Meadowcroft, chief executive of Muscular Dystrophy UK, said: ‘We welcome the decision that Translarna will be funded across England. But we cannot forget that families in Scotland haven’t been given the same opportunity. It is simply unacceptable.’
Diana Ribeiro, director of research at Action Duchenne, said: ‘We were bitterly disappointed with the announcement from the SMC. In evaluating the evidence, we believe the actual route in which they made this assessment isn’t robust enough.
‘The way it’s structured in Scotland is that very little time is designated for any drug – it’s not given the same time and evidence as down South.’
She also said the charity would appeal the decision
An SMC spokesman said: ‘We were unable to accept Translarna as we were not satisfied the evidence on the benefits was strong enough to justify its very high cost.
‘We recognise how disappointing this decision is for patients and carers.
‘But where a medicine is not recommended and a doctor feels it is right for a particular patient, health boards have procedures in place to consider individual requests.’
‘It is simply unacceptable’