The Press and Journal (Aberdeen and Aberdeenshire)
‘Unlicensed drug could save my life’
24-year-old battling cystic fibrosis in plea for treatment
Aged just 24, Hailey Mann has been told she will soon die if she doesn’t receive a “miracle drug” not yet fully available in Scotland.
The cystic fibrosis (CF) sufferer has made a harrowing plea for urgent access to the medication which could save her life, telling medics: “I will soon be dead. I don’t need to be.”
Miss Mann, from Westhill, says her hopes rest on life-transforming
Kaftrio, which is yet to be fully licenced in Scotland, after being told she should start planning her end-oflife care.
Though the Scottish Government announced yesterday that a pricing agreement had been reached with manufacturer Vertex Pharmaceuticals, availability is restricted to “compassionate grounds”, pending the granting of a licence by the European Medicines Agency.
Diagnosed with CF at birth, Miss Mann learned to live with the debilitating condition, even running her own beauty business until January, having managed to keep her condition relatively stable until about a year ago.
Now an “end stage” CF sufferer, she says she has been left frustrated by “red tape” as she waits to receive good news from NHS Grampian, which submitted an application for the drug on her behalf earlier this year.
She said: “There are certain boxes you need to tick to get Kaftrio on compassionate grounds, and as far as I am aware I tick all the boxes. I see no reason why I haven’t been administered it.
“My health has deteriorated drastically this year and the truth is, without this drug I am going to die.
“I have done my research and I know for a fact that if I took Kaftrio today, within hours of the first dose I would feel like a new person. It really is a miracle drug. It’s the closest thing there is to a cure.
“Without it, the morbid fact is that I’ll be planning for the end of my life.
“While I’m delighted for those who will receive the drug as a result of the Scottish Government’s latest announcement, it doesn’t help me. I need it now.”
An NHS Grampian spokesman said it could not comment on individual cases, but added: “Speaking generally, when a clinician makes a request to use an unlicensed drug on compassionate grounds, we make every effort to urgently evaluate the patient’s suitability for the medication and progress any application to the manufacturer as quickly as possible.
“The manufacturer ultimately makes the decision on whether or not to grant the drug to the patient.”