- Written by Anne-Marie Tasker & Chloe Loversach
- BBC Look North Health Correspondent
image source, Anne Marie Tasker
Nicole asked that the drug Kaftrio, which is used to treat her baby daughter Charlie, not be stopped.
The mother of a baby born with cystic fibrosis has appealed to the NHS not to stop taking a drug she was told could extend her daughter’s life by 40 years.
Nicole, from North Lincolnshire, was told she could start taking Kaftrio when her four-month-old baby, Charlie, turned two.
The National Institute for Health and Care Excellence (NICE) is currently investigating the drug’s ‘cost-effectiveness’.
Nicole said if it was withdrawn, Charlie “would have a hard time and may not be able to grow up.”
NICE said patients already taking the drug would be able to continue receiving treatment after it decided whether the drug could continue to be available on the NHS.
Kaftrio is the brand name for ivacaftor, tezacaftor, and elexacaftor combination therapy.
Nicole said that when she was told the drug might not be approved for new patients, “any hope we had was dashed in an instant.”
image source, Anne-Marie Tasker/BBC
Four-month-old Charlie was diagnosed with cystic fibrosis after birth.
The family was told that due to the type of cystic fibrosis Charlie was diagnosed with, he might not live to adulthood.
But doctors said treatment with Kaftrio could add decades to her life.
“It’s a big difference, when she talks about what she wants to do with her life, whether she wants to be a mother, whether she wants to go to college, whether she wants to travel,” Nicole said.
“Everyone deserves a chance.”
image source, Anne-Marie Tasker/BBC
Nicole and her family were told that Kaftrio treatment could add 40 years to Charlie’s life.
Nicole said she had always been in awe of people who were willing to involve themselves and their families in drug experiments, but now she was trying to get her baby into the program. .
“Now I realize that these people are not brave, they are just scared and desperate, and that is where we are now.”
image source, Anne-Marie Tasker/BBC
Charlie requires regular medication to treat an infection he suffers from as a result of cystic fibrosis
NICE’s director of medicines evaluation, Helen Knight, said a consultation on the use of the drug had begun.
The health authority said a packet of 56 tablets costs £8,346.
This means the cost of treatment to the NHS could reach between £100,000 and £200,000 per patient per year.
image source, Anne-Marie Tasker/BBC
Nicole said her family would not be able to personally afford to pay for the medication.
“We are evaluating the cost-effectiveness of these cystic fibrosis drugs to ensure that taxpayers continue to receive value for money following interim access as further data is collected,” Knight said. Ms.
“We will continue to work collaboratively with us, NHS England and other stakeholders, including the Cystic Fibrosis Trust, to deliver the best outcomes for both cystic fibrosis patients and the wider NHS.”
NICE said no decision had yet been made on the use of the drug.
