Angus mum Kirstin Adam says a new cystic fibrosis drug deal for Scotland means her son may no longer face a premature death.
Kirstin, 32, and partner Andy Ashfield, 33, from Brechin, were devastated when they found out Arlo, had cystic fibrosis (CF) when he was just three-and-a-half weeks old.
Cystic fibrosis is a condition that causes sticky mucus to build up in the lungs and digestive system.
This means it can lead to a significantly shorter life expectancy for many sufferers.
The couple hoped their son would be prescribed new medication which they were told could prolong Arlo’s life into adulthood.
Regulators said that this new medication – which includes Kaftrio and two other drugs, Orkambi and Symkevi – was too costly to be prescribed on the NHS.
One of the drugs, Kaftrio, is licensed for children over the age of two.
But it can cost £100,000 a year.
Since last year, health bosses had been consulting on whether it was too expensive to offer to new patients.
Welcoming the decision on the new cystic fibrosis drugs
But last week it emerged that an agreement had been reached with the drugs manufacturer Vertex, the Scottish Medicines Consortium (SMC) and NHS Scotland.
It means that all eligible existing and future CF patients will now have extended long-term access to the new cystic fibrosis (CF) medicines.
Kirstin, whose son is now 13 months old, has welcomed the decision.
“This is fantastic news and the only outcome which made sense,” she says.
“As Arlo turned one on June 11 while the consultation period was still ongoing, he got the all-clear to start Orkambi last month.
“As much as this was great news for our family, it was always in the back of my head that other families might not be so lucky if the decision went the other way.”
She says Arlo will continue to get Orkambi on prescription until he turns two.
And then hopefully he will be able to change over to Kaftrio.
Kaftrio has been found to significantly improve lung function, helping people with cystic fibrosis to breathe more easily.
Drugs offer potential for long life
Last year a number of parents across the country, whose children have cystic fibrosis, had urged health bosses to reconsider their initial decision, which was based on cost.
And a petition – which had over 72,000 signatures – was launched.
Its aim was to help fund continued access to these drugs which could be a lifeline for people with cystic fibrosis.
Kirstin hopes the petition was taken into consideration.
“Newborns being diagnosed with CF are now in a generation like no other before them,” she says.
“The knowledge we now have of CF and the availability of drugs like these allows children with cystic fibrosis the best possible outcome of a long life.”
Kirstin added: “We are over the moon with the decision.
“We hope these drugs continue to transform life for many people with CF.
“Hopefully Arlo will be able to continue taking it for the rest of his life.”
‘Important moment for those with cystic fibrosis’
Manufacturer Vertex says: “We are pleased to confirm that we have finalised the reimbursement agreement with NHS Scotland for extended long-term access to cystic fibrosis (CF) medicines Kaftrio, Symkevi and Orkambi.
“The agreement is comparable to the NHS England agreement (reached last month).
“And provides access to these medicines for all existing and future eligible CF patients in Scotland.”
David Ramsden is chief executive of the Cystic Fibrosis Trust.
He said: “This is a really important moment for people with cystic fibrosis in Scotland.
“And it follows many years of campaigning.
“These treatments have transformed the lives of many people with CF.
“Yet they don’t work for all and are not a cure.”
He added: “But we won’t stop until everyone with CF can live a life unlimited.”
Conversation