An Ipswich mum says it’s “cruel” that a life-saving drug may be taken away from newly diagnosed people with cystic fibrosis, after guidance suggested it may be dropped due to cost.
Laura Wright, whose three-year-old son Chester was diagnosed after being born, is lobbying to not have the Kaftrio “wonder drug” taken off the NHS list for new patients.
Draft guidance from the National Institute for Health and Care Excellence (NICE) this month said the drug is not considered “an acceptable use of NHS resources” and so is not recommended for use.
This is even though those with cystic fibrosis have a shortened life expectancy and a greatly reduced quality of life.
The inherited condition causes sticky mucus to build up in the lungs and digestive system, causing a host of symptoms and complications.
The family was told Chester’s life expectancy was between 30 and 40 years old, but Kaftrio offered a “glimmer of hope”.
While it has most recently been approved for children aged two to five, and current patients would not be taken off the medication, Ms Wright is campaigning on behalf of new parents.
The mum-of-two said: “I am thinking of anyone with a child under two now, any new families experiencing the diagnosis. If a child is being diagnosed now, I do not even know how you would cope as a parent.
"It would feel worse as Kaftrio is out there and accessible but not for your child, as they are born a few years too late. It's awful."
Around half of people with cystic fibrosis will live past the age of 40, according to the NHS, but one study shows that use of Kaftrio may help people with the condition to live to an average age of nearly 72, if treatment is started in adolescence.
The drug costs more than £8,000 per 56-tablet packet, according to NICE.
Ms Wright said: "Kaftrio is close to a cure, and it is incredible with regards to life expectancy. It would literally save his life and in the meantime give him a life.
“I understand the NHS is struggling but as a parent you feel as though your child's life is not valued as much as someone with cancer or diabetes. They are never going to take away chemo or insulin.”
NICE’s remit is to recommend whether a medicine should be made available on the NHS, but if it is not recommended a private company may still choose to provide it.
NHS England says the licensed treatments for cystic fibrosis are provided as part of a commercial agreement between Vertex and NHS England, with the "full support of NICE".
John Stewart, national director for specialised commissioning at NHS England said: “Within the agreement between NHS England and Vertex there is a flexible commercial mechanism to ensure continued access for patients already receiving any of the licensed treatments following the conclusion of a full NICE evaluation.
“The draft publication is an initial step in the review of these medicines and does not affect patients’ continued access to these drugs on the NHS in any way.
"Eligible children and adults with cystic fibrosis can continue to receive ongoing treatment and be initiated onto treatment with these drugs, as clinically appropriate.
“NHS England remains committed to ensuring that these life-changing cystic fibrosis medicines are available to everyone who may benefit, now and in the future, and will continue to work with Vertex and NICE through the next stages of the appraisal process to make this possible in a way that is fair to patients, and fair to taxpayers”.
NICE said it is evaluating the cost-effectiveness of the medications "to ensure that taxpayers continue to get value for money".
Helen Knight, NICE director of medicines evaluation, said: "The committee want to hear from stakeholders through consultation on important aspects of its draft conclusions. This is so 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."
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