'Life-changing' cystic fibrosis drug approved for use on the NHS

Hailey Mann and her mother Alison Gauld

A "life-changing" cystic fibrosis drug has been approved by the Scottish government.

Health Secretary Jeane Freeman said Kaftrio could lead to patients "living longer, healthier lives".

But Hailey Mann, who is making end-of-life preparations, fears the breakthrough may have come too late.

The 24-year-old is pinning her remaining hopes on the unlicensed drug and is in the process of applying to use it on compassionate grounds.

Miss Mann told BBC Reporting Scotland: "If anyone is listening that can help, then you would literally be changing my life. You would be giving me a life."

On Tuesday Ms Freeman announced a deal had been agreed with Vertex Pharmaceuticals to make the triple therapy treatment available through the NHS.

Following approval from the European Medicines Agency, it is expected a licence will be granted in the coming weeks.

Hailey during a family holiday to Mexico last year

Cystic fibrosis currently affects about 900 people in Scotland.

Miss Mann, from Aberdeenshire, said: "We still don't have the European licence, so until that comes through we don't have the drug.

"It could be weeks or months and I don't have the time.

"I need it now and I do not believe there's not a way to do that."

'Absolutely devastating'

The delay in receiving the drug has been stressful for Miss Mann and her family, who are still coming to terms with the deterioration in her condition.

Her mother, Alison Gauld, said: "Hearing that news last week was absolutely devastating but it also gave us the kick that we needed to pursue this.

"We need to fight to get this drug. I can't just sit here and watch her be a statistic.

"I've got to do everything in my power to make her better and have a life again."

Vertex Pharmaceuticals said it made decisions about requests for compassionate use in line with various clinical criteria as quickly as possible.

NHS Grampian said requests on compassionate grounds were urgently processed and evaluated, but it was the manufacturer who made decisions on whether to grant the drug to the patient.

'Potentially transformational'

Ms Freeman said: "Cystic fibrosis is an inherited condition, which tragically shortens lives and affects around 900 people in Scotland.

"This is a potentially transformational treatment for cystic fibrosis, and I am delighted that patients in Scotland will be among the first in Europe to benefit and lead longer, healthier lives."

The health secretary said clinicians working in cystic fibrosis treatment centres will be able to prescribe the medicine to patients whom they consider will benefit from its use as soon as the licence was granted "in the coming weeks".

But for Ms Mann, the breakthrough can't come soon enough.

"To have these plans that I've got in my head (such as watching my sister grow up), to be able to fulfil them would be amazing."