Parents said they were “flabbergasted” that the Scottish Medicines Consortium (SMC) rejected the Duchenne muscular dystrophy (DMD) drug Translarna on Monday, before it was approved for use south of the border.
The National Institute for Health and Care Excellence (Nice) announced yesterday it had secured an agreement with the manufacturer to offer the medication in England, Wales and Northern Ireland.
Cormac Fegan, 5, became the first boy in the UK to get access to Translarna on the NHS, after a successful individual funding request to NHS Lothian last year, but his medication will only last until June.
His father Gary Fegan said his family would consider moving to England if they failed in a last-ditch bid to get the drug in the wake of the Nice decision.
Mr Fegan, of Longniddry, East Lothian, said: “I was excited to hear the news but it is bittersweet. I know what other parents are going through and we have had two years of waiting for the decision.
“I am flabbergasted that the SMC did not put the brakes on their decision though. It just seems backwards and unfair.
“They are playing with people’s emotions and lives.”
Mr Fegan, 43, said the family would have to move south of the border if their request to NHS Lothian was unsuccessful.
He said: “This news gives us hope that we can get a decision from the board. We’re against the clock and everything is a bit uncertain.
“But if we can’t get it then we will have to move. I can’t see any other option.”
Campaigners spoke of their disappointment that Scottish patients could miss out on the promising treatment.
Robert Meadowcroft, chief executive of Muscular Dystrophy UK, said: “It is simply unacceptable that eligible boys in Scotland won’t have access to Translarna, which could make such a valuable difference to their condition and lives.”
An SMC spokesman said: “We recognise how disappointing this decision is for patients and carers, as Duchenne Muscular Dystrophy is a devastating condition. Ataluren is, however, an extremely expensive medicine and NHS Scotland has finite funding.
“Where a medicine is not recommended by SMC, and a doctor feels that treatment is right for a particular patient, Health Boards have procedures in place to consider individual requests.”
The SMC has met with the company and it has indicated it plans to make another submission, the spokesman said.