- By Anne-Marie Tasker and Chloé Laversuch
- Health Correspondent, BBC Look North
Image source, Anne-Marie Tasker
Nicolle called for the drug Kaftrio, which could be used to treat her baby daughter Charlie, not to be withdrawn.
The mother of a baby born with cystic fibrosis has urged the NHS not to withdraw a drug she fears could extend her 40-year-old daughter’s life.
Nicolle, from North Lincolnshire, was told that four-month-old Charlie could start taking Kaftrio when he was two years old.
The National Institute for Health and Care Excellence (NICE) is currently reviewing the “cost-effectiveness” of the drug.
Nicolle said that if it was removed, Charlie “would struggle and may not reach adulthood.”
NICE said patients already taking the drug would continue to have access to the treatment after deciding whether the drug should continue to be available on the NHS.
Kaftrio is the brand name for the combination therapy ivacaftor, tezacaftor and elexacaftor.
Nicolle said that when she was told the drug might not be approved for new patients, “every ounce of hope we had been given was ripped away in a second.”
Image source, Anne-Marie Tasker/BBC
Four-month-old Charlie was diagnosed with cystic fibrosis after birth
The family was told that the type of cystic fibrosis Charlie had been diagnosed with meant she might not live to adulthood.
But doctors said treatment with Kaftrio could extend his life expectancy by decades.
“It’s huge, it’s a difference to talk about what she wants to do with her life, if she wants to be a mom, if she wants to go to college, if she wants to travel,” Nicolle said.
“Everyone deserves a chance.”
Image source, Anne-Marie Tasker/BBC
Nicolle and her family learned that the drug Kaftrio could extend baby Charlie’s life expectancy by 40 years.
Nicolle said she had always been impressed by people willing to put themselves or their families through experimental drug trials, but was now trying to get her baby accepted into a program.
“Now I realize these people aren’t brave, they’re just terrified and desperate. And that’s where we are.”
Image source, Anne-Marie Tasker/BBC
Charlie needs regular medication to treat an infection she has from cystic fibrosis.
Helen Knight, director of medicines evaluation at NICE, said a consultation had been launched into the use of the drug.
It costs 8,346 for a pack of 56 tablets, according to the health organization.
This means that NHS treatment costs can be between 100,000 and 200,000 per year for each patient.
Image source, Anne-Marie Tasker/BBC
Nicolle said the family would not be able to pay for the drug privately.
“We are evaluating the cost-effectiveness of these cystic fibrosis drugs to ensure taxpayers continue to get value for money after interim access during which additional data has been collected,” Ms Knight said.
“We continue to work collaboratively with the business, NHS England and other stakeholders, including the Cystic Fibrosis Trust, to deliver the best outcomes for both people with cystic fibrosis and the NHS as a whole. »
NICE said no decisions had yet been made on the use of these drugs.
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