Cystic fibrosis patients in Dundee have criticised a decision not to make two potentially life saving drugs available on NHS Scotland.
Orkambi is a precision medicine that would tackle the underlying cause of the condition and could benefit about 50% of patients with the condition.
But the Scottish Medicines Consortium (SMC) said there was “significant uncertainty” around the long term benefits of the drugs, which cost £100,000 a year per patient.
Morag Beckett of the Cystic Fibrosis Tayside group, whose son Greg has the condition, said: “It is heartbreaking as a parent to listen from another room as your child does physio, coughing so hard they are sick, struggling to breathe.
“You would do anything to make it better for them, to allow them to live a life like their peers.
“We are all aware these drugs won’t help everyone, but surely those who it is thought will benefit should be given the chance, regardless of cost. How can you put a cost on saving a young life?
“I feel like we keep getting a carrot dangled in front of us only for it to be pulled away time and time again.
“I have friends who have suffered the loss of a child due to this cruel disease. How many more families need to suffer?
“The people who make the decisions need to live a day in the life of someone with CF to see what it takes to make them function.
“These drugs have been produced and are sitting on a shelf somewhere slowly going out of date when they could potentially save a young life. How can anyone be denied the chance of a better life because of cost?”
Greg, who has the dd508 gene, said: “It’s not fair that this stuff does exist and it does work but we are not getting it.”
Another patient, 19-year-old Adam Lyon from Fairmuir, who also has the gene mutation dd508, said: “It’s very frustrating because it is something that could help a lot of people yet it still doesn’t seem to be rolled out. It’s not fair.”
David Ramsden, chief executive at the Cystic Fibrosis Trust, said: “The SMC’s advice will come as a shock to people with cystic fibrosis and their families in Scotland. While talk of further negotiation between Vertex Pharmaceuticals and the Scottish Government is positive, people living in Scotland need these life-saving drugs now.”