The parents of a little girl living with cystic fibrosis say they are "heartbroken" NHS funding could be withdrawn for a 'miracle' drug. Annie Davies, four, was diagnosed with the life-shortening genetic condition (CF) at just three weeks old.
However mum Nicola and dad Tom, both 37, were holding out hope that their daughter's life would be changed when she turned six. It is at that age children qualify for Kaftrio, which has been hailed as a wonder drug which can transform lives.
But it was announced on Friday (November 3) that the treatment could be removed from the NHS due to its cost. Although a decision has not been made, it has left Tom and Nicola, both sales managers from Kingsclere, Hants., fearful for the future.
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Nicola said: "Kaftrio was this glimmer of hope that Annie would lead a full healthy life. Hearing the news, we feel unimaginable heartbreak. We were hoping they'd bring the age down that treatment could start, not stop it altogether - the entire CF community is blindsided.
"It's just knowing there's something out there that can help her and she can't have it - but a child 18 months older still can? Without this drug, her life will be cut short. Since the news came out, I've hugged her a little bit tighter."
Tom and Nicola had no idea they were carriers of the recessive genetic condition until Annie was diagnosed after blood spot screening. They opted to have IVF when they wanted a second child, so they could screen for the condition, and as a result, their son Freddie, two, doesn't have it.
Annie is already on a different drug called Orkambi, which is suitable for younger children, meaning she'd still be entitled to that even if the NHS withdraw it for future patients. But the parents had been waiting hopefully for her sixth birthday so she could move onto Kaftrio, which has been considered "life-changing" for many patients.
Annie currently needs to do daily physiotherapy as well as using a nebuliser, which helps her breathe, up to three times a day. NHS patients in England were some of the first in Europe to benefit from Kaftrio when a deal was secured in June 2020.
The drug significantly improves lung function, helping people with CF to breathe more easily and enhancing their overall quality of life. However, last Friday's announcement has put its future on the NHS in jeopardy.
The National Institute for Health and Care Excellence (NICE), which advises NHS England on the cost effectiveness of treatments, published an update on the drug. It said that, although experts accept it is clinically effective, its high cost could mean it has to be axed.
The Cystic Fibrosis Trust said this is not a final decision, and there will now be a four-week consultation period followed by a second committee meeting in December to discuss the consultation responses. Nicola said: "Without this drug, her life will be cut short.
"Our first reaction to the news was total shock and frustration - then upset and grief. If people making these decisions had anyone with CF in their family, they wouldn't do it. If this decision comes into play, there are so many things wrong with it.
"Why should my child who has an inherited condition she never asked for not have a medication that's currently available? Children who are already six can have it but because she was born later she might not have access. It feels like discrimination. It's sick."
Helen Knight is director of medicines evaluation at NICE. She 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.
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"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."
