Parents fear their children will die young without life-saving Cystic Fibrosis drug deemed 'too expensive' for NHS

14 November 2023, 09:40

By Charlotte Lynch

@charlotterlynch

Hundreds of children could be denied access to a life-saving drug to treat cystic fibrosis, because it's "too expensive" to provide on the NHS.

It's thought there are around 1,000 children under six suffering from the genetic condition, who could benefit from Kaftrio, which increases lung function and prolongs patients lives.

It's currently available on the NHS for patients aged six and older, but the UK's healthcare advice body says it may be too expensive to fund, and has begun evaluating the cost-effectiveness of the drug.

NICE (the National Institute for Health and Care Excellence) is also evaluating whether two other medications, Orkambi and Symkevi, are providing taxpayers with "value for money".

Parents Rose and Scott Blatchford, from Plymouth, were hoping their four-year-old daughter Matilda would be able to begin the treatment, saying she could live to the average life expectancy if she were allowed it.

Rose Blatchford told LBC: "You can't put a price on a child - you can't say if their life is worth X or Y. You don't know what my child is capable of or who she will grow up to be when she's older."

The heartbroken parents described the "horrible" thought of burying Matilda early, saying: "It's not something we've ever prepared for - now we're having to realise it could be a reality for us and Matilda. No parent should have to think about having to bury their children."

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Scott and Rose told LBC they expected the treatment to be approved for younger children, and dreamed of Matilda to be able to live in to old age and have children of her own.

According to the Cystic Fibrosis Trust, the average age of patients at death last year was 32.

According to the 2022 UK Cystic Fibrosis Registry Report, 1,660 patients aged between 0-7 were suffering with the disease in 2022.

Lisa Gooday, the mum of three-year-old Nolah from Braintree in Essex, told LBC the news of the consultation "felt like getting that diagnosis all over again".

"It's taken us back to those days in the beginning where we felt completely helpless," she said.

"It came as a complete shock, no one expected this. We've been led to believe from our team at Great Ormond Street that the next phone call we get is going to be it's been approved, and we'd be going in to start all of the baseline tests to start her on Kaftrio."

Nolah has yearly scans on her lungs, and her father Lloyd described watching them "get worse every year".

Cystic Fibrosis is an inherited condition which affects the movement of salt and water in and out of cells, resulting in recurring infections, and the build up of mucus on the lungs.

Patients with the condition are highly susceptible to infection, and suffer with long coughs which are difficult to shift, causing further damage to their lungs.

Matilda, Nolah and other CF patients must undertake hours of daily physio, use a breathing aid, and take regular medication to move the mucus around their lungs to prevent build-up.

David Ramsden, CEO of Cystic Fibrosis Trust, said: “NICE’s initial recommendation today that the modulator drugs Kaftrio, Orkambi and Symkevi are highly effective for people with cystic fibrosis, but just too expensive to be available on the NHS is disappointing news.

"It is important to emphasise that those already taking any of the modulator drugs are not affected by the NICE process because of the agreements already in place but this update creates uncertainty for those not yet on treatment.

"Vertex, NICE, and the NHS must now urgently work together to find a solution to make these treatments available for all those who could potentially benefit.

"We must never return to a situation where people with CF die far too young, knowing there’s a treatment that could change that."

Helen Knight, director of medicines evaluation at NICE said: "We are evaluating the cost-effectiveness of these cystic fibrosis medicines to ensure that taxpayers continue to get value for money after interim access where further data was collected.

"The committee want to hear from stakeholders through consultation on important aspects of its draft conclusions. This is to ensure that we have all the relevant information to accurately capture the value of these effective medicines when the committee makes its final decision.

"We are continuing to work collaboratively with the company, NHS England and other stakeholders including the Cystic Fibrosis Trust to deliver the best outcome both for people with cystic fibrosis and for the wider NHS. 

"Existing patients and new patients who are started on treatment while the NICE evaluation is ongoing will continue to have access to the treatments after NICE has issued its final recommendations irrespective of the outcome."

A Department of Health and Social Care spokesperson said: “Cystic fibrosis can be a devastating illness, which is why we have invested over £1.1 billion of funding into research of rare diseases.

“It is vital that patients have access to have access to new and innovative medicines like these but the NHS must use its budget fairly and for the good of all patients.

“NICE is the independent expert body responsible for developing authoritative, evidence-based guidance for the NHS on whether drugs and treatments represent an effective use of resources.”