Mother of 10-year-old from Beds with cystic fibrosis speaks of vital importance of medication
The mother of a 10-year-old girl living with cystic fibrosis says the medication is vital for patients who depend on it
Last updated 24th Nov 2023
A four-week consultation on cystic fibrosis is due to end today, after the drugs were deemed 'too expensive' by the watchdog NICE.
Those living with the illness, which affects the lungs, and in some cases can require a lung transplant, depend on the three types of medication available through the NHS.
The licensed treatments are firstly Orkambi® and Symkevi® and then Kaftrio®, following its marketing authorisation in 2020.
The medication can help patients with their everyday life, including eating and drinking, which can often prove painful for those suffering from the illness.
David Ramsden, CEO of Cystic Fibrosis Trust, said: “NICE’s initial recommendation 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."
If the drug is indeed deemed too expensive and access through the NHS prescription is being removed for new patients, it could affect people like 10-year-old Layla from Bedfordshire, who does not yet need the medication but could end up requiring it in the future.
"Even in schools, we always have to ask 'is there a child with cystic fibrosis' and if there is my child can't attend that school..."
Her mum, Shushma Jain, spoke to Greatest Hits Radio about the daily challenges her daughter faces.
She said: "People don't realise because if you see Layla she looks like she's okay, but when she was at school she wasn't allowed to play in mud, go to the swimming pool or step in puddles in the rain."
"What might be okay for you or me and may just be a cold, they pinpoint what bug is growing on the lungs and give the specific antibiotics for that, and the reason for that is to stop scarring on the lungs which in the future can mean a lung transplant, and that is why they treat it straight away."
Cystic fibrosis exposes patients to higher risk in case of common cold or similar passing illnesses, including bugs found in water.
Her mum spoke of the isolating aspect of the illness, as two patients cannot be in the same room due to cross-infection.
She said: "Even in schools, we always have to ask 'is there a child with cystic fibrosis' and if there is my child can't attend that school, because of the risk of cross infection."
The result of the consultation will determine whether patients like Layla, with a mild condition who could one day need the medication, will be able to access it.
Mr Ramsden from the Cystic Fibrosis Trust said: "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.”