Emotional moment girl, 13, with life-limiting cystic fibrosis breaks down in tears of joy as her mother reveals she will FINALLY get the drug that will change her life after a landmark NHS deal for Orkambi
- Up to 5,000 patients in England will benefit from the life-extending medication
- It slow the decline of lung function and will be available to them within 30 days
- Beth Finn yesterday recorded her daughter Isabelle’s jubilation at the NHS deal
- Upon hearing the life-changing news, Isabelle instantly broke into floods of tears
- More than 4,000 Twitter users had ‘liked’ the emotional clip this morning
Heartwarming footage has captured the moment a 13-year-old girl with cystic fibrosis found out she will be able to get a life-extending drug.
Beth Finn yesterday recorded her daughter Isabelle’s jubilation at NHS England agreeing a deal for Orkambi after four years of deadlock.
Upon hearing the life-changing news that she will be able to get the drug on prescription next month, Isabelle instantly broke into floods of tears.
More than 4,000 Twitter users had ‘liked’ the clip this morning, which was posted yesterday in the aftermath of the landmark decision.
It begins by Ms Finn, from Staffordshire, asking her daughter: ‘So guess what has been approved today?’
Puzzled, Isabelle – sitting in the passenger seat of a car – replies: ‘What has?’ She then continues moments later: ‘Stop, you’re getting me worried, what?’
It begins by Ms Finn, from Staffordshire, asking her daughter: ‘So guess what has been approved today?’ Puzzled, Isabelle – sitting in the passenger seat of a car – replies: ‘What has?’ She then continues moments later: ‘Stop, you’re getting me worried, what?’
Her eyes then light up and her smile widens when her mother jogs her memory by saying: ‘What have we been fighting for?’ Fighting back the tears, a thrilled Isabella asks twice: ‘Has it?’
Ms Finn, a senior sister in an NHS A&E unit, asks again: ‘Guess what’s been approved by NHS England today?’
Isabelle, still clueless as to the news that the NHS finally agreed a deal with US-based pharmaceutical giant Vertex, responds: ‘I don’t know.’
Her eyes then light up and her smile widens when her mother jogs her memory by saying: ‘What have we been fighting for?’
Fighting back the tears, a thrilled Isabella asks twice: ‘Has it?’
Ms Finn, also clearly emotional by her daughter’s reaction, says: ‘Yeah, so in thirty days we’ll be able to get a prescription.’
Continuing to cry out of sheer happiness, Isabelle responds: ‘Really? That’s so good.’
Her cousin – whose name is unknown – then approaches Isabella, opening her door and giving her a cuddle.
Isabelle apologises to him for getting tears on his top – but he responds: ‘It’s alright, I know what it is for.’
Her cousin – whose name is unknown – then approaches Isabella, opening her door and giving her a cuddle. Isabelle apologises to him for getting tears on his top – but he responds: ‘It’s alright, I know what it is for’
Ms Finn’s original tweet – to which the clip was attached – read: ‘This is my 13-year-old Isabelle. ‘She knows cystic fibrosis is life-limiting and she has struggled not just physically but also emotionally this year’
Up to 4,000 patients in England will benefit from the £104,000-a-year cystic fibrosis drug Orkambi after a deal was struck with the NHS
Isabella adds: ‘It’s [the news] is so good, I was not expecting that.’ And when asked how she feels by Ms Finn, she says: ‘I’m so happy.’
The one-minute video, which has left scores of Twitter users in tears, finishes with Isabelle and her mother telling each other they love them.
Ms Finn’s original tweet – to which the clip was attached – read: ‘This is my 13-year-old Isabelle.
‘She knows cystic fibrosis is life-limiting and she has struggled not just physically but also emotionally this year.
Families ‘hugging in hope’
Luis Walker has spent his whole life in and out of hospital with painful lung infections and other symptoms of cystic fibrosis.
But the nine-year-old has now received the ‘best Christmas present ever’ – the promise of a longer and fuller life.
Luis, from East Sussex, currently needs at least four hours of treatment and more than 20 tablets per day.
Luis Walker has spent his whole life in and out of hospital with painful lung infections and other symptoms of cystic fibrosis
But he will start taking Orkambi within the next few weeks, helping his breathing by reducing the build-up of mucus in his lungs and giving him more energy to play with friends and go to school.
His mother Christina said yesterday: ‘There are families across the country now hugging their children with renewed hope.
‘I’m really hoping to see a fairly quick easing in his ability to breathe. We’ve waited four years for this drug. Very sadly people have died during that time. I just urge that lessons are learned.’
‘Her reaction speaks a thousand words. Thank you.’ She tagged Health Secretary Matt Hancock, NHS England, The Cystic Fibrosis Trust and Vertex.
Up to 5,000 patients in England will benefit from three £104,000-a-year pill after the deal with Boston-based maker Vertex.
The deal is thought to be in excess of half a billion pounds and gives NHS England access to the medication for five years.
Orkambi and two other drugs that were also approved – Symkevi and Kalydeco – slow the decline of lung function, the main cause of death in cystic fibrosis.
Mr Hancock hailed the news as ‘wonderful’ and said the drugs will ‘improve thousands of lives’, adding the deal was ‘great value for money’.
He said: ‘This deal – on the back of several others this summer – shows why we get some of the best value drugs in the world, and is another reason to be so proud of our NHS.’
Cystic fibrosis charities described it as a ‘very special day’ for patients and their families who have campaigned for years for the drugs.
Health Secretary Matt Hancock hailed the news as ‘wonderful’ and said the drugs will ‘improve thousands of lives’
WHAT IS CYSTIC FIBROSIS?
Cystic fibrosis is an incurable genetic disease that affects around 70,000 people worldwide.
A defective gene causes a build-up of mucus in the airways, making it increasingly difficult to breathe over time.
Mucus also blocks the natural release of digestive enzymes, meaning the body does not break down food as it should.
Signs and symptoms:
- A persistent cough that produces thick mucus (sputum)
- Exercise intolerance
- Repeated lung infections
- Inflamed nasal passages or a stuffy nose
While healthy people cough naturally, that does not happen for people with CF.
Eventually, lung function depletes to the point that sufferers will need a double lung transplant to survive.
Source: Cystic Fibrosis Foundation
David Ramsden, chief executive at the Cystic Fibrosis Trust, added: ‘We know it doesn’t end here.
‘We will continue to fight for similar access in Wales and Northern Ireland so that all people with cystic fibrosis in the UK are able to benefit from these life-saving drugs.’
Dr Caroline Elston, chair of the UK Cystic Fibrosis Medical Association, said: ‘We welcome this amazing progress in England, and look forward to receiving further details on how this will be rolled out by NHS England, and what this means for people with CF in Northern Ireland and Wales.’
Last month NHS bosses accused the US pharmaceutical firm of being greedy with its ‘monopoly pricing’.
Negotiations had been at a standstill since Vertex this year rejected an offer of £500million over five years for access to the drug.
The argument flared up again when the Scottish government announced it had managed to reach a deal last month – enabling the supply of the drugs to 350 cystic fibrosis patients north of the border.
Cystic fibrosis is a debilitating illness, which creates a mucus build-up that causes chronic lung infections and progressive lung damage.
The incurable disease affects around 70,000 people worldwide, including 3,000 in the UK, according to figures.
NHS England chief executive Simon Stevens described the deal as a ‘long hoped-for moment’.
He added: ‘The UK has the second highest prevalence of cystic fibrosis of any country in the world, so today is an important and long hoped for moment for children and adults living with cystic fibrosis.
‘That fact also means that any drug company wanting to succeed commercially in this field needs to work constructively with the NHS.
‘I’m pleased that Vertex has now agreed a deal that is good for our patients and fair to British taxpayers.’
Orkambi, which was licensed in Europe in November 2015, is used to treat cystic fibrosis in patients aged two and older while Symkevi treats those aged 12 and up.
The medications target the F508del gene mutation, which affects about 50 per cent of all people with cystic fibrosis.
Labour leader Jeremy Corbyn said the deal was a victory for campaigners who ‘stand together and take on the powerful’.
He tweeted: ‘This is life changing for everyone with cystic fibrosis in our country, they’ve waited years for this drug.
‘A victory for all the campaigners who have taken on big pharmaceutical companies’ greed and Government inaction. When we stand together and take on the powerful, we win.’
It comes after more than 117,000 people signed a petition calling on the Government to make Orkambi available on the NHS.
WHAT IS 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 4,000 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 signed a petition calling on the Government to make Orkambi available on the NHS, and Parliament discussed the petition in March 2019.
It was finally made available on the NHS in England on October 24 after four-years of negotiations.
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