Cystic fibrosis: mother ‘petrified’ by her daughter’s access to medication – BBC News

Sarah’s four-year-old daughter Lucy suffers from the condition and her family thought she would be eligible for the Kaftrio drug when she was six.

However, NICE’s draft guidance says it would not recommend three treatments, including Kaftrio, to new patients.

In a statement, it said it had “evaluated the cost-effectiveness” of “these effective drugs” and that consultation with stakeholders would help it make its final decision.

Sarah, who lives in Wingham, Kent, said: “I’m completely heartbroken at the moment, I’ve always been scared and worried about Lucy’s future because of her cystic fibrosis, but now I’m petrified.

“It feels like a lifeline that was given to 90% of the CF community has just been ripped away. Hearing the news that they will never get Kaftrio is horrible.”

Sarah called health officials’ decision “incomprehensible.”

Children with CF have a reduced life expectancy.

The draft NICE guidelines are open for consultation until 24 November.

This would not affect those already taking Kaftrio or Symkevi, which are currently recommended for patients aged six and over, or Orkambi, which can be used from one year of age.

If adopted, young children or babies who have not yet received these therapies would not be guaranteed NHS care, despite NICE’s view that there are major benefits .

Cystic fibrosis is an inherited disease that causes sticky mucus to build up in the lungs and digestive system. This causes lung infections and problems digesting food.

In the UK, most cases are detected at birth using a newborn heel test.

Symptoms are present from childhood and worsen over time as the lungs and digestive system become increasingly damaged.

David Ramsden, CEO of Cystic Fibrosis Trust, said this was disappointing news which creates uncertainty for those not yet on treatment.

He said urgent work was needed to find a way to make the drugs accessible to everyone who could benefit from them.

We must never return to a situation where people with cystic fibrosis die far too young, knowing that there is treatment that could change that,” he said.

In a statement, Helen Knight, director of medicines evaluation at NICE, said the body “evaluates the cost-effectiveness” of medicines “to ensure taxpayers continue to get value for money”.

She said NICE wanted to hear from stakeholders through consultation “to ensure we have all the relevant information to accurately capture the value of these effective medicines when the committee makes its final decision”.

The Ministry of Health has also been contacted for comment.