Lincolnshire mum's plea for NHS cystic fibrosis drug to extend baby's life

The mother of a baby born with cystic fibrosis has urged the NHS not to withdraw a drug that she was told could extend her daughter's life by 40 years.

Nicolle, from North Lincolnshire, was told four-month-old Charlie could start taking Kaftrio when she turned two.

The National Institute for Health and Care Excellence (NICE) is now reviewing the "cost-effectiveness" of the drug.

Nicolle said if it was withdrawn Charlie would "struggle and potentially not make it into adulthood".

NICE has said patients already taking the medication would continue to have access to the treatment after it made a decision on whether the drug should continue to be available on the NHS.

Kaftrio is the brand name for ivacaftor, tezacaftor and elexacaftor combination therapy.

Nicolle said when she was told the medication might not be approved for new patients "every single ounce of hope that we had been given just got torn away in a second".

The family were told the type of cystic fibrosis Charlie had been diagnosed with meant she might not live into adulthood.

But doctors said being treated with Kaftrio could add decades to her life expectancy.

"That's huge, that's a difference in talking about what she wants to do with her life, if she wants to be a mum, if she wants to go to university, if she wants to travel," Nicolle said.

"Everyone deserves a chance."

Nicolle said she had always been in awe of people who were willing to put themselves or their families through experimental drug trials, but that she was now trying to get her baby accepted on a programme.

"Now I realise these people are not brave, they are just terrified and desperate. And that's where we are."

Helen Knight, director of medicines evaluation at NICE, said a consultation had been launched on the use of the drug.

It costs £8,346 for a packet of 56 tablets, according to the health body.

It means NHS treatment costs can amount to between £100,000 and £200,000 per year for each patient.

"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," Ms Knight said.

"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."

NICE said a decision had not yet been made on the use of the drugs.

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