Mother’s anxious wait over daughter’s CF drug-funding decision

The mother of a baby with cystic fibrosis is waiting anxiously to find out whether the NHS will fund drugs to help treat the condition.

Georgia, from Nuneaton, said the treatment could double 15-month-old Gia’s lifespan.

NICE, the medicines review agency that advises the NHS, said it was considering whether to recommend funding for three cystic fibrosis drugs.

The final decision is expected to be announced next year.

Georgia described the drugs Orkambi, Shinkevi and Kaftrio as “lifesaving” and believes they can give her daughter a chance at a normal life.

Treatment is estimated to cost more than £100,000 per patient per year, potentially prohibitive for the NHS.

Georgia’s daughter will soon begin a course of Orkambi, but she cannot begin treatment with Kaftrio until she is two years old.

At this stage, she said, there is no guarantee that her daughter will be able to transition from one drug to another.

“This is important for Gia and other children because this is a life-changing drug. It will extend her life by more than 40 years and improve her quality of life,” she said.

“It might give her the more normal life she deserves.”

Georgia has to carefully calculate the fat content of everything she eats every day because her pancreas is not functioning.

The toddler is also receiving a morning and evening menstrual routine and twice-daily antibiotics to protect against airborne infections.

“Without this medication and physical therapy, she would have been a very sick baby,” she said.

“I want her to be able to go out and live like a normal child.

“We are fortunate to have a great team around us: nurses, doctors, specialists, nutritionists, physical therapists.”

Kaftrio, which can treat this condition, was approved for adults several years ago and earlier this month for infants.

However, a NICE panel decided that although they were clinically effective treatments, they were too expensive to recommend for use in the NHS.

We will discuss whether we can continue the prescription.

Helen Knight, director of drug evaluation at NICE, said NICE wanted to have all relevant information before making a decision.

Ms Georgia said she had contacted the Cystic Fibrosis Trust about the situation as part of the consultation process and was seeking feedback from people.