Family pleas for NHS cystic fibrosis drug to save baby's life

  • Parents Hannah and Matthew Bennett

The family of a little girl living with cystic fibrosis have issued a desperate plea for her to have access to a life saving drug.

Isabelle Bennett, who is just 11 months old, was diagnosed with the genetic disorder three weeks after she was born.

The youngster has been undergoing treatment at Manchester Children's Hospital ever since, and requires daily physiotherapy and rounds of medication.

She was due to start a life saving modulator drug in just a few weeks, but the treatment could be taken away from her after care bosses claimed it is 'too expensive' for new patients.

Isabelle was diagnosed with the genetic disorder three weeks after she was born. Credit: MEN Media

Parents Hannah and Matthew, from Bury, had hoped she would be given the chance to live a 'full and healthy life' through these drugs.

But the family has been left terrified for her future, saying the draft guidance has left thousands of infants, including Isabelle, in limbo.

The National Institute for Health and Care Excellence (NICE) said they are 'evaluating the cost-effectiveness' of the medicines, and are working alongside the NHS and the Cystic Fibrosis Trust to 'ensure the best outcome both for people with cystic fibrosis and for the wider NHS'.

"It's a heart-crushing decision," Hannah said. "We want Isabelle to have a future, and if these drugs aren't approved, that will be taken away."

Cystic fibrosis is a life-limiting condition that slowly clogs patients' lungs.

Isabelle has been given treatment at Manchester Children's Hospital. Credit: ITV Granada Reports

Drugs Orkambi, Symkevi, and Kaftrio are the first effective treatments on the health service for the condition, and are produced by US pharmaceutical giant Vertex.

Isabelle has physiotherapy sessions twice a day, rounds of medications and enzymes before she eats, to help her manage the condition.

She was due to start Orkambi on her first birthday in December, but could now have the opportunity snatched away from her.

The family had also hoped NICE would approve Kaftrio, which has been dubbed "almost a cure for CF", for use in 2-5 year olds.

But they were left "shocked" when the draft guidance said both life-saving drugs could be too expensive to be given to any new patients.

Hannah and Matthew had hoped she would be given the chance to live a 'full and healthy life' through the drugs. Credit: ITV Granada Reports

Now, they are appealing for people to submit comments to NICE's online consultation and sign a petition to keep the drugs available on the NHS.

"Our only hope is to shout from the rooftops to help children like my daughter get the life saving medication they deserve," Hannah said.

"Please help give my daughter chance to allow her to be a child and grow up planning her future."

Helen Knight, director of medicines evaluation at NICE said: "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. The committee want to hear from stakeholders through consultation on important aspects of its draft conclusions.

The draft guidance from NICE is under consultation until November 24.

Hannah and Matthew's petition can be found here.

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