Family considers England move to access vital drug

From left, mum Ella Fegan, brother Ronan, Cormac and dad Gary Fegan. Picture: HEMEDIA

From left, mum Ella Fegan, brother Ronan, Cormac and dad Gary Fegan. Picture: HEMEDIA

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PARENTS of a five-year-old boy with a rare muscle wasting condition will consider moving to England after vital medication was approved for use south of the border.

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 successful individual funding request to NHS Lothian last year.

I am flabbergasted the SMC did not put the brakes on their decision. They are playing with people’s lives

GARY FEGAN

The drug can slow the progression of the condition, which causes muscles to waste over time, meaning sufferers can be reliant on a wheelchair from a young age.

Bosses at National Institute for Health and Care Excellence (Nice) announced yesterday that they had secured an agreement with the manufacturer to offer the drug in England, Wales and Northern Ireland – just days after it was rejected in Scotland.

Dad Gary Fegan said his family would consider moving to England if they failed in a last-ditch bid to get the drug from NHS Lothian, as Cormac’s medicine will only last until June.

Gary, 43, 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.”

Translarna is the first available drug to address the underlying cause of DMD and can help sufferers to walk for longer.

Gary said he was excited for other parents in England as the medication has transformed Cormac’s life.

He said: “I was excited to hear the news but it is bittersweet. I know what other parents are going through and we have had over two years of waiting for the decision. I am flabbergasted that the SMC did not put the brakes on their decision though. They are playing with people’s emotions and lives.”

Campaigners spoke of their disappointment that Scottish patients could miss out.

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.”

A Scottish Medicines Consortium (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, which has indicated it plans to make another submission, the spokesman said.

lizzy.buchan@jpress.co.uk