Plea from cystic fibrosis sufferer

Published: Sunday 15th February 2015 by The News Editor

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A cystic fibrosis sufferer says he feels like there is a “carrot dangling in front of me” due to being denied a life-changing drug on the NHS.

Mike Boyle, 30, is one of 44 people who it is believed could be helped by the drug Ivacaftor, which is commonly known as Kalydeco.

But although it is currently available to 360 people with the G551D mutation of cystic fibrosis, it is not being offered to others who carry one of eight additional gating mutations found to be affected by the drug.

Mr Boyle, from Pontypridd in south Wales, said he had seen first-hand the life-changing results that Kalydeco has on people that suffer from the condition.

“The thought of not being able to receive it now that I know I am suitable is beyond comprehension,” he said.

“It feels like there is a carrot dangling in front of me.

“My life would transform if I had Kalydeco, but my fate is in the hands of those making the decisions at the NHS – whether they will agree to fund it for the rest of us.”

Those who have received the oral drug who could barely walk, are now able to run marathons, a spokesman for the Cystic Fibrosis Trust, which is campaigning for more widespread distribution of the medication, said.

Those taking it in Wales have seen an 87% reduction in the number of days spent in hospital on intravenous antibiotics, while it is expected to allow a much greater life expectancy.

He said the drug would work as well in the new patient group, but funding is being refused because they have a different genetic make-up.

Ed Owen, chief executive of the Cystic Fibrosis Trust, said: “It is indefensible that cystic fibrosis patients who stand to benefit from the only treatment which provides them with both a long-term quality of life and survival benefit are being denied it.

“With personalised medication being hailed as the future, Kalydeco is leading the way in creating life-saving treatment for people living with cystic fibrosis.

“For patients who are already in receipt of the treatment we have seen remarkable clinical benefits.

“The data shows that this drug could normalise life expectancy, meaning that for the first time patients face the prospect of dying from old age, not cystic fibrosis, where the median age of death currently stands at 27.

“Every day a decision on funding is delayed and access prevented is another day of misery for the outstanding 44 people and their families who stand to benefit from this life-saving treatment.”

Jason McCartney MP, chairman of the All-Party Parliamentary Group on Cystic Fibrosis, said: ” I urge NHS England and Vertex to find a way to extend the gift of life to these 44 people, for who time and hope is running out.

“Equity is the foundation of healthcare in this country. Let us maintain that tradition and find a solution without delay.”

Kalydeco is the first drug to correct the underlying genetic defect which causes cystic fibrosis. This year, the Cystic Fibrosis Trust is making sure all sufferers have the opportunity to know their genotype to allow them to take a more proactive role in their own care and access new medicines.

An NHS England spokeswoman said: “Patient groups have asked NHS England to consult with patients and the wider public about how we make decisions on which specialised services and treatments to invest in, in future.

“This will ensure the principles and process we follow are well informed, evidence-led and in line with the expectations of patients and the public. The consultation runs until the end of April.

“Until then, any decision that is urgent on clinical grounds will be dealt with quickly though our existing procedures.”

Published: Sunday 15th February 2015 by The News Editor

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