Cystic fibrosis: Joy as NHS seals deal with US firm for medicine to treat condition

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    Matt Hancock

    Health Secretary Matt Hancock (Image: PA Video/PA Wire/PA Images)

    Health Secretary Matt Hancock was in tears as he told Daily Express columnist Carlie Pleasant that Kaftrio will be available next month. The announcement that the ultimate CF treatment, previously known as Trikafta, is coming marks a stunning victory for the Daily Express’s award-winning crusade to help sufferers get key drugs. Mr Hancock unveiled the deal in a video call to CF sufferer Carlie at her home in Kent. Mr Hancock said: “I’ve been bowled over by the incredible bravery of the families fighting for the latest CF treatments.

    “I’m absolutely thrilled we have now secured Kaftrio, a cutting-edge medicine which gives hope to families and patients for a longer, healthier and happier future.

    “I will always fight for families and loved ones to have access to the best possible treatments so that everyone has the opportunity to flourish.”

    The news was greeted with delight by Carlie, who spoke to an emotional Health Secretary.

    The married mother of one, 30, said: “I cried and thanked him and then he cried!

    “It was very emotional. I told him I’ll remember this moment forever.

    “Only recently I was tired, poorly and almost defeated in hospital after being admitted for losing half a litre of lung function.

    “Today the clouds have lifted, the sun of hope is warming my face and I finally face the possibility of it being my last admission for some time.

    “That is all thanks to Kaftrio. This is the drug that I have dreamed about since being diagnosed with CF as a child.

    Carlie with Matt Hancock

    Carlie with Matt Hancock, and a copy of the Daily Express (Image: Tim Clarke)

    “It is a momentous moment for patients and families alike, a true lifeline to thousands that live a life battling a progressive disease.

    “Our lives suffering and battling a war with CF are about to change for the better.

    “That’s hugely overwhelming and thrilling to think that a future has just opened up to me – one not darkened by the gloom of CF.

    “The feeling of suffocation has been lifted, thanks to the Daily Express and our relentless campaigning. Evidence from patients on Kaftrio in America, and those on trials in the UK, prove this remarkable drug works.

    “From being given the life expectancy of just 15, to being handed a prescription for a lifesaving drug that could give me a near-normal life is a miracle.

    “It is the moment my parents never thought would come.

    “Kaftrio means happiness, hope, health and a future, an absolute dream come true and a moment I will neither forget nor take for granted.” Last October, the NHS successfully secured US pharmaceutical firm Vertex’s current CF drugs Orkambi and Symkevi.

    The Daily Express had campaigned tirelessly for the deals. But the next day we began a new battle for Vertex’s upcoming drug Kaftrio, dubbed “almost a cure” for the cruel genetic condition.

    Earlier this year we secured lifesaving Kaftrio compassionately for a number of near-death patients, who have now made miraculous recoveries. Now it will be made available to all CF patients who need it aged over 12 in England from next month onwards.

    Incredible stories from those already on Kaftrio show why patients and doctors have been thrilled by its results.

    Some sufferers taking it have even said they no longer show CF symptoms. One unnamed UK patient on a trial told us that their lung function has rocketed from 30 percent to 90 percent.

    Although this is an NHS England deal, the contract allows for Wales, Scotland and Northern Ireland to negotiate the same terms.

    Last night Vaughan Gething, minister for health in Wales, said they now hope to “reach an agreement to be taken forward urgently” for Kaftrio.

    Stephen Powis

    National Medical Director at NHS England Stephen Powis (Image: PA Video/PA Wire/PA Images)

    CF is a life-limiting genetic condition that destroys the lungs with sticky mucus and affects other vital body organs.

    There are more than 80,000 sufferers in the world and 10,500 in the UK. David Ramsden, chief executive of the CF Trust, said: “It is fantastic Kaftrio will now be available to thousands of people across England in the coming weeks.

    “This will truly save lives.We will not stop until everyone with CF has access to life-saving drugs.”

    NHS Chief Executive Sir Simon Stevens said: “This is a potentially transformational treatment for CF.

    “I am proud that, thanks to this landmark deal, NHS patients will be among the first in Europe to benefit.” Ludovic Fenaux, senior vice president of Vertex, said: “This is an important day for patients with CF in England.

    “Establishing this agreement illustrates the collaboration between Vertex, NHS England and NICE.

    “It shows just what is possible when the biotech industry and the NHS partner together for the benefit of patients.”

    Sir Simon Stevens

    NHS Chief Executive Sir Simon Stevens (Image: Victoria Jones/PA Wire/PA Images)

    Comment by Professor Stephen Powis, NHS England Medical Director

    Thank you to the whole CF community and the Daily Express for working tirelessly alongside the NHS for access to this drug.

    As the country comes together to mark the NHS’s anniversary this weekend, there can be no better present for patients with CF than this landmark deal.

    Patients will shortly be among the first to benefit from the treatment described as truly life-transforming.

    Sufferers will know from powerful social media images and videos just how much difference it potentially makes.

    And this treatment can’t come soon enough. Since the start of the pandemic, people with CF have been shielding as they are thought to be at high risk from the effects of Covid-19.

    It’s therefore a matter of pride NHSE has been able to do this deal with the support of the medicines regulator NICE.

    Together with existing drugs, more than 90 percent of CF patients will soon have access to a potentially life-transforming treatment.

    NHSE has ensured Vertex will offer equivalent terms in Wales, Northern Ireland and Scotland.

    Comment by Matt Hancock, Health Secretary

    Every family dreams of seeing their children reach their full potential and I am bowled over by the incredible bravery of families living with CF.

    I have been so inspired by their determination to fight for the future of their loved ones, and make sure they have access to the best possible treatments.

    In October, after months of tough negotiations, we reached a deal with Vertex to offer patients the revolutionary drug Orkambi.

    This was one of my happiest days as Health Secretary, when we were able to make a difference to the lives of so many people.

    While Orkambi works for some, it does not work for all, and this redoubled my determination to get access to the next generation CF drugs. I am thrilled we have now secured a deal for Kaftrio, a cutting-edge “triple combination” treatment, which tackles the underlying causes of CF, helping the lungs to work more effectively. NHS patients in England will be among the first in Europe to benefit from this groundbreaking treatment. Scotland, Wales and Northern Ireland can also benefit on equivalent terms, if devolved administrations would like to adopt them.

    Carlie Pleasant receiving treatment for her cystic fibrosis

    Carlie Pleasant receiving treatment for her cystic fibrosis (Image: Supplied)

    Yesterday I broke the news to campaigner Carlie Pleasant, whose courage is a real inspiration.

    She once asked me to do everything I can so she lives to see her young son grow up and I have always been determined to do whatever I could to help her and others.

    Carlie was delighted with this exciting new advance, and is one of thousands with the condition who now have renewed hope for a longer, healthier and happier future.

    People with CF have found coronavirus particularly tough, and many have had to shield from loved ones.

    We will continue to strain every sinew to protect you.

    I will always fight for the latest and most cutting-edge treatments for all of our NHS patients because everyone deserves the best possible chance to flourish.



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