Benat Broderick argued his life has been transformed since Irish officials granted him access to Orkambi
A 14-year-old boy dying from cystic fibrosis today begged the NHS to approve his life-extending drug.
Benat Broderick argued his life has been transformed since Irish officials granted him access to Orkambi – hailed as a ‘wonder drug’.
But health watchdogs in the UK have repeatedly denied patients access to the £100,000 medication, on the grounds that it is too expensive.
Experts believe using the tablets could stop the illness, which is expected to kill half of current sufferers before they reach their 40s, from becoming severe later in life.
In an emotional plea, Benat, from Dublin, revealed the dramatic effects Orkambi has had on his quality of life, from helping him put on weight, giving him more energy and improving his lung function.
Both he and his mother, Caroline, argued that it is so ‘unfair’ that thousands of cystic fibrosis patients in the UK are unable to get their hands on Orkambi.
In an interview with MailOnline, Benat said: ‘I want patients in the UK to have access to Orkambi.
‘I know the difference it has done to me and to my health and I don’t think it’s fair that the people in the UK can’t experience this amazing drug that I have experienced.’
‘In my opinion, I don’t think that it’s fair for the UK government to be putting a price on the CF Communities lives.
‘CF Patients are dying while this drug is on the shelves and that’s just not acceptable. The UK needs to have access to Orkambi now.’
The teenager, who was on a six-month trial of Orkambi before it was approved in Ireland, gained 1st 8lbs (10kg) since he started taking the drug.
And his lung function – considered a key indicator of how badly affected a cystic fibrosis patient is – has improved by five per cent.
In an emotional plea, Benat, from Dublin, revealed the dramatic effects Orkambi has had on his quality of life, from helping him put on weight to giving him more energy
Talking to MailOnline, Benat said: ‘I want patients in the UK to have access to Orkambi’
Mrs Broderick added that her son just ‘really wants’ people in the UK to have access to Orkambi as well.
Speaking to MailOnline, she said: ‘[He’s] seen how life-changing it is for them. It’s so unfair, seeing as how the two countries are so close together.’
Approved in Ireland
Ireland’s Health Service Executive approved the use of Orkambi in April 2017 after fierce negotiations with Orkambi manufacturer Vertex.
But outraged sufferers and charities in England and Wales have battled round-the-clock for two years to get the drug funded by Nice for use on the NHS.
It comes as Health and Social Care Secretary Jeremy Hunt earlier this week renewed calls for Vertex to cut the price of the life-extending drug.
Mr Hunt, speaking in the House of Commons, called on the manufacturer to ‘waive commercial confidentiality’ so the price can be reviewed.
The 51-year-old made the comment in response to Labour former minister Ian Austin, who told ministers they needed to ‘get a grip’ of the situation.
Protests for the drug
Dozens of protesters stormed Downing Street wearing oxygen masks in the summer to make Orkambi available on NHS.
And a petition to approve the drug on the health service gained more than 100,000 signatures in March and led to a debate in the Commons.
Vertex has made financial deals in other EU countries that have led to the drug being given the green light. And campaigners hope the same will now happen here.
NHS England and Nice are currently in deadlock with the Massachusetts-based firm over a price cut on Orkambi. Discussions are ongoing.
The teenager, who was on a six-month trial of Orkambi before it was approved in Ireland, gained 1st 8lbs (10kg) since started taking the drug
And his lung function – considered a key indicator of how badly affected a cystic fibrosis patient is – has improved by five per cent
‘End the deadlock’
Lynsey Beswick, of the Cystic Fibrosis Trust, said: ‘We welcome Benat’s comments.
‘Orkambi could potentially benefit up to half the 10,000 people in Britain who live with the life-threatening condition cystic fibrosis.
‘But – even though the treatment has been licensed for three years now – NHS England and the drug manufacturer Vertex have been unable to agree on the price.
She added: ‘These discussions have t
aken far too long and every day is another that people with cystic fibrosis are denied access to this life-changing medicine.
‘We urge both sides to reach a deal and end the deadlock. It is vital that people with cystic fibrosis have access to this drug as soon as possible.’
Cystic fibrosis: The facts
Cystic fibrosis, which strikes nearly 11,000 people in the UK, is caused by a faulty gene that a child inherits from both carrier parents.
The gene, known as the cystic fibrosis transmembrane conductance regulator (CFTR), is responsible for controlling the movement of water in and out of cells.
The fault leads to the mucus produced throughout the body becoming thick and building up in the lungs and digestive system.
Classic complications of the condition, which tend to present in infancy, include chronic infections, breathlessness, digestive problems and even infertility.
How does Orkambi work?
Orkambi works by correcting the gene fault, keeping a healthy balance of salt and water in mucus so it can function normally.
The drug – a combination of lumacaftor and ivacaftorkeeps – keeps patients’ lungs clear and free of infection and the scarring that eventually kills.
Research has shown it reduces the incidence of infection and can cut the number of hospital admissions by more than 60 per cent.
What is the ‘wonder drug’ Orkambi?
Orkambi is a medication licensed for use by people in the UK with cystic fibrosis, but it is not offered on the NHS except in extreme circumstances.
The drug targets the F508del gene mutation, which affects about 50 per cent of all people with cystic fibrosis.
It is made of a combination of drugs – lumacaftor and ivacaftor – which work together to keep a healthy balance of salt and water in the lungs.
There may be more than 3,000 people with life-limiting cystic fibrosis in the UK who could benefit from the drug.
But the National Institute for Health and Care Excellence (Nice) said in 2016 may not be cost-effective enough, at £104,000 per patient per year, to be offered on the NHS.
More than 117,000 people have signed a petition calling on the Government to make Orkambi available on the NHS, and Parliament discussed the petition in March this year.
Nice is expected to review its stance on the drug in July 2019.
Daniel Bodio, an engineer from Swindon in his mid-30s, was prescribed the drug on compassionate grounds.
He said it improved his lung function from 25 per cent – when he was facing having to have a transplant – to 39 per cent within three weeks.
He told MailOnline last year: ‘The drug should be available to everyone – it brings normality to what is otherwise a difficult existence.’