I’m 24, I don’t have to die, but red tape is about to kill me
Hailey makes urgent plea for cystic fibrosis wonder drug that could save her life
A YOUNG cystic fibrosis sufferer has begged for access to a wonder drug which she believes can save her life.
Hailey Mann, 24, said: “I will soon be dead. I don’t need to be.”
I need this drug now. Not next week, not next month
A YOUNG cystic fibrosis sufferer has made a harrowing plea for urgent access to a wonder drug which could save her life, telling medics: “I will soon be dead. I don’t need to be.”
Hailey Mann, 24, is pinning her hopes on life-transforming Kaftrio, which is yet to be fully licensed in Scotland, after being told she should start planning her end-of-life care.
In a heartbreaking open letter shared with the Daily Record, Hailey, from Aberdeenshire, who is living with just 14 per cent lung function, today begs medics to give her “hope for a future”.
Deals have been struck in England, Wales and Northern Ireland with Vertex Pharmaceuticals in recent weeks to make the triple gene therapy drug available for patients there as soon as the European Commission grants a licence.
The Scottish Government is expected to secure its own deal here in the near future – but Hailey says she needs the drug now to save her life.
She says she was told she could be granted use of the drug on compassionate grounds in January and an application has been made by medics at Aberdeen Royal Infirmary to drug firm Vertex.
But she is still waiting seven months on, due to what she describes as “red tape and bureaucracy”.
She said: “In January, I felt like I was getting somewhere, then Covid happened and I feel like since then it has just been excuses.
“Last week, a doctor told me they were very worried about me and I need to think about whether I want to pass away in hospital or at home.
“I felt like they were giving up on the Kaftrio and weren’t fighting to get it any more. I’ve been assured they are but I just don’t feel the urgency. I need this drug now. Not next week, not next month.”
Cystic fibrosis is an inherited condition in which the lungs and digestive system can become clogged with thick mucus.
Hailey, who ran her own hairdressing business until January, had managed to keep her condition relatively stable until about a year ago.
Her best chance of survival is a double lung transplant but the complexities of the condition mean she has struggled to maintain enough weight to allow her to have the procedure.
She receives daily treatment at home in Westhill, including oxygen, intravenous therapy and antibiotics. But she says lockdown has caused a decline in her condition, which could prove fatal at any time.
Health experts say nine in 10 people with the genetic condition could benefit from Kaftrio – known as Trikafta in the US. The therapy combines three drugs to tackle the underlying causes of the disease, by helping the lungs work effectively.
The Record has highlighted the plight of Scots whose lives depend on the treatment and campaigned for pioneering drugs to be granted to cystic fibrosis patients here in recent years.
Hailey told us she had been left with no option but to pen an open letter about her situation, which she hopes will reach First Minister Nicola Sturgeon.
She said: “Kaftrio has been called a miracle drug. My lungs are badly damaged and I’m not expecting it to do all these wonderful things for me that it can for others, but if I can even use it to get some extra weight on then I would be eligible for a transplant. That would be
HAILEY MANN ON HER URGENT NEED FOR KAFTRIO
enough to give me a chance at life, rather than just discussing my end-of-life plans.”
Hailey’s mum Alison Gauld said: “Her lung function and health is deteriorating and we’re still waiting.
“We were very low last week when she was asked where she would want to pass away. She’s 24 and wasn’t expecting to have that conversation yet.
“With every breath in my body I will keep fighting to get her this drug. It’s all I can do. We’re desperate.”
A Vertex spokeswoman said: “Making decisions about requests for compassionate use of an unlicensed medicine is always difficult and complex. We endeavour to make these decisions as ethically, fairly and quickly as possible, in line with the various medically driven clinical criteria, the available information about the potential medicine as well as the patient’s specific overall medical circumstances once we receive an application from a patient’s doctor.
“There are currently no delays
to review and approve requests for the compassionate use programme by Vertex due to Covid-19. We cannot lawfully comment on a specific patient’s circumstances.”
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.”
A spokesman for the Scottish Government said: “We are very pleased that NHS England has negotiated a pricing deal on Kaftrio, which they have led on as part of a four-nations approach.
“The full details of an arrangement for NHS Scotland are currently being finalised and we are planning to make an announcement on this shortly.”