I lost my daughter... don’t deny life-saving drug to other children
A MOTHER whose daughter died in her arms at 11 while waiting for lifesaving cystic fibrosis drugs has attacked plans to deny the treatment to new patients as a “disgrace”.
Since 2019 the 11,000 people in the UK with the disease have benefitted from US firm Vertex’s CF medicines such as Kaftrio – but recently NHS drug approval officials decided they were too pricey for new patients.
Elle Morris – for years the face of a campaign to access these drugs on the NHS – died in 2018 after a double lung transplant.
Her mum Becky Whitfield is to host ElleWood – a celebration of Elle’s life – at Peckforton Castle near Nantwich, Cheshire, on February 2, what should have been her 18th birthday, to raise funds for charities including the CF Trust.
But Becky hit out at the provisional ruling by the National Institute for Health and Care Excellence (Nice) that the drugs Elle fought for are too costly for new patients.
The 37-year-old said: “When I heard that new generations of CF patients could be denied these proven, life-saving treatments I was absolutely appalled.
Sense
“Not only is it frankly cruel to deny little toddlers drugs that older children and adults are already taking but it could create a two-tier health service.
“It makes no financial sense. For those not on Vertex’s drugs the NHS would end up spending more when they are older as they get increasingly sick and require weeks at a time on specialist care.
“It also upsets me as Elle was unable to get Vertex’s Orkambi drug as it was not available on the NHS at the time she desperately needed it.” She added: “To think Orkambi and the even better Kaftrio are now on the NHS but bean-counters are thinking about restricting who can have them is a disgrace.”
CF is caused by a fault in a gene which controls the movement of salt and water so mucus clogs up the lungs and bowels, making it hard to breathe and to digest food.
For years the Daily Express fought for patients to have Vertex’s “miracle” therapy and in 2020 families celebrated as then Health Secretary Matt Hancock agreed an NHS deal for Kaftrio. But last autumn Nice
said that, despite Orkambi, Symkevi and Kaftrio being hugely effective, their list prices were too high.
There are 8,000 UK patients on Vertex drugs and they will not have them withdrawn, while around 1,000 have such rare CF mutations that no existing drug works.
So 2,000 toddlers and babies are in limbo – either too young to begin on Kaftrio or awaiting approval to start.
A ruling is due in a few months. It is understood that if the drugs are ruled out NHS England would try to negotiate direct withVertex.
Helen Knight, director of medicines evaluation at Nice, said: “We are evaluating the cost-effectiveness of these cystic fibrosis medicines to ensure taxpayers continue to get value for money.”
John Stewart, NHS England’s national director for specialised commissioning, said it “remains committed to ensuring that these life-changing CF medicines are available to everyone in a way that is fair to patients and taxpayers”.
Vertex said it was “committed to working collaboratively with the NHS and Nice to secure long-term access for any new patients who may benefit” from their drugs.
Elle succeeded in getting stars such as TV talent-spotter Simon Cowell, Jurassic World actor Chris Pratt and presenter Dermot O’Leary to raise awareness of CF. Despite often being very ill, she launched Elle’s Wishes campaign in 2016, making a music video and pushing organ donation.
Legacy
Becky, daughter Cara, 16, fiance Jonny Walker, 29, and Elle’s dad Ian Morris are hosting the charity fundraiser to help the CF Trust and Great Ormond Street Children’s Hospital.
Becky said it would have “music, dinner, entertainment and dancing to celebrate Elle’s incredible legacy”, adding: “It gives me a purpose to carry on.” The £60 tickets can be bought at elleswishes.co.uk