A life-changing drug that could help a desperately sick 14-year-old boy has been declined to him on the NHS – because it costs too much.
Joe Barnes suffers with Cystic Fibrosis (CF), an inherited condition that causes sticky mucus to build up in the lungs and digestive system.
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Tragically, he also has an older brother with the same disease.
The condition means that his lung function has now declined to 38-42% meaning that he struggles to breath, can’t eat and spends a lot of time in hospital.
He also weighs only four and a half stone and has to be fed through a tube.
The schoolboy, who has diabetes and liver disease, had to have an operation to stop him from being sick as he was refluxing food into his lungs.
His mum Lorraine told the Mirror: ‘The coughing and retching are so extreme.
‘He can’t breathe, he feels sick, he rarely makes it through a school day now. His quality of life is almost zero.’
Joe and his family have been campaigning for the past two years to get Orkambi made available on the NHS.
It is currently not available as it costs £104,000 per patient every year.
The drug targets a mutation that around 50% of people with cystic fibrosis in the UK have and has been shown to improve lung health and reduce hospitalisations, according to the Cystic Fibrosis Trust.
What is Cystic Fibrosis?
Cystic fibrosis is an inherited condition that causes sticky mucus to build up in the lungs and digestive system. This causes lung infections and problems with digesting food.
Treatments are available to help reduce the problems caused by the condition and make it easier to live with, but sadly life expectancy is shortened.
Cystic fibrosis is a genetic condition. It’s caused by a faulty gene that affects the movement of salt and water in and out of cells.
This, along with recurrent infections, can result in a build-up of thick, sticky mucus in the body’s tubes and passageways – particularly the lungs and digestive system.
Cystic fibrosis tends to get worse over time and can be fatal if it leads to a serious infection or the lungs stop working properly.
Currently, about half of people with cystic fibrosis will live past the age of 40.
According to the 2014 UK Cystic Fibrosis Registry Report, there are 3,296 people in the UK who could benefit from the drug.
The National Institute of Clinical Excellence (NICE) said it was unable to recommend the drug for use within the NHS ‘based on the evidence and price proposal provided by Vertex Pharmaceuticals’.
Lorraine added: ‘I wish they could see Joe, the people who make these decisions. They think it’s about money and resources, but if they could just see Joe lying here, or in hospital on a machine.
‘He’s so ill, he’s like a skeleton. What if it was their child? When you know one small, pink tablet could make such a difference.’
Now, a petition to get Orkambi made available on the NHS has reached over 115,000 signatures and CF families have won a debate in Parliament.
Vertex have announced that they are currently working on a new drug that could treat up to 90% of the CF population, meaning a deal is vital for the survival of people suffering with CF.
NICE have said they ‘remain open to a further approach from the company’.
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