'Appalled' that NHS cannot afford cystic fibrosis wonder drug

A mum whose courageous daughter died in her arms aged 11 waiting for life-saving cystic fibrosis drugs has blasted plans to deny the miracle pills to any new patients is a “disgrace”.

Since 2019 the UK’s 11,000 CF sufferers have benefitted from US firm Vertex’s range of CF wonder drugs like Kaftrio but recently NHS drug approvers dropped the bombshell that they are too costly to give to any new patients.

In 2018 brave Elle Morris, for years the face of the campaign to get these drugs on the NHS, died in her mother’s arms after a double lung transplant having seen her health plunge waiting for the treatments.

On February 2nd mum Becky Whitfield, 37, hosts ‘ElleWood’ at Peckforton Castle in Nantwich, Cheshire – on what should have been Elle’s 18th birthday – to fundraise for charities including the CF Trust.

But ahead of the celebration of Elle’s remarkable, campaigning life, Becky’s raged at the provisional ruling by the National Institute for Health and Care Excellence (NICE) that the drugs Elle fought for are too costly for any new patients.

Becky told the Daily Express: “When I heard that new generations of CF patients could be denied these proven, life-saving treatments I was absolutely appalled.

“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 – those on lifesaving pills and those being denied them.

“There is also the moral factor of those doctors put in a terrible decision of knowing there are treatments being used on the NHS to prolong some sufferers’ lives – but they cannot prescribe them to new patients.

“Lastly it makes no financial sense. For those not on Vertex’s drugs the NHS would end up spending more when they are olde as they get increasingly sick and require weeks at a time on specialist care in hospital.

“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 desperatel;y needed it – so she ended up having a double lung transplant.

“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 faulty gene which controls the movement of salt and water in and out of your cells, meaning sticky mucus clogs up your lungs and bowels making hard to breathe and digest food.

For years we fought for UK patients to access Vertex’s miracle tablets and in the summer of 2020 families celebrated when the then Health Secretary Matt Hancock agreeing an NHS deal for wonder drug Kaftrio.

But last autumn NICE dropped their bombshell draft guidance saying despite the three treatments – Orkambi, Symkevi and Kaftrio – being hugely effective, their ‘list price’ was too high.

NHSE confirm that regardless of NICE’s decision, any CF patient already currently taking Orkambi or Symkevi can move on to Kaftrio when aged two – the minimum age you must be to take the wonder drug.

There are currently around 8,000 UK sufferers on Vertex’s drugs and they will not have them withdrawn regardless of NICE’s final decision – while around 1,000 patients have such rare CF mutations no existing drug works for them.

That leaves around 2,000 toddlers and babies either too young to yet start on wonder pill Kaftrio, or awaiting approval to start, who currently remain in limbo.

ElleWood’ at Peckforton Castle in Nantwich, Cheshire – on what should have been Elle’s 18th birthday – to fundraise for charities including the CF Trust.

Elle Grace Morris lived a tragically short but remarkable life getting stars to raise awareness of CF like Simon Cowell, Hollywood’s Chris Pratt and Dermot O’Leary.

Yet despite being often very poorly, she launched her Elle’s Wishes campaign in 2016, meeting stars like magician Dynamo, Lorraine Kelly, Olly Murs and Rita Ora, making a music video and promoting organ donation.

In February 2017, after seven months waiting in vain for access to Orkambi, poorly Elle had a double lung transplant but after a series of infections, in January 2018 her family revealed she had died in Becky’s arms.

Now next month Becky, her daughter Cara, 16, fiance Jonny Walker, 29, and Elle’s dad Ian Morris are hosting a charity fundraiser to mark what would have been the weekend of her 18th birthday.

It will take place at Peckforton Castle near Nantwich to raise money for the Cystic Fibrosis Trust and Great Ormond Street Children’s Hospital.

Becky explained: “It will be a spectacular evening of music, dinner, entertainment and dancing to celebrate Elle’s incredible legacy. I think that’s the thing that’s keeping me going is the positivity and inspiration from Elle.

“Her wish was to raise awareness for organ donation and raise funds to find a cure for CF. People were in awe of her – she was a very contagious personality.

“The day she passed away was a relief to see her out of pain. For me it’s living on for her wishes – it gives me a purpose to carry on going.”

Tickets are £60 and can be bought – or a charity donation made – by visiting https://elleswishes.co.uk/