'Our children's lives are worth the cost'- Calls for better access to cystic fibrosis 'wonder drug'

Mia McCarty, Entitled to cystic fibrosis drug, Kaftrio
Two-year-old Mia McCarty is now able to be treated with the cystic fibrosis drug, Kaftrio.

A family from Plymouth is campaigning for a so-called 'wonder drug' to be offered to all children with cystic fibrosis to help increase their life expectancy and their quality of life.

Two-year-old Mia McCarty had been refused the drug Kaftrio on the grounds that it would be too expensive, but a change in the rules means she is now eligible to get the medication.

However, for children under the age of two, it's not known if they will ever be offered the drug due to cost.

Mia's mum Danielle Tout said: "We will always feel very appreciative and grateful that Mia has been offered this drug but we will always feel sad that there are so many children out there that aren't.

"They have to live continuously with cystic fibrosis day in and out watching their child progressively get worse - it breaks my heart for those parents."

Up until recently children with the genetic condition, which affects the lungs and digestive system, were often moved onto Kaftrio at the age of six.

The family from Plymouth is calling for the drug to be made available to all children with cystic fibrosis.

In November 2023 the Medicines and Healthcare Products Regulatory Agency (MHRA) extended the licence for children aged two to five years old. However, due to its cost, a decision is yet to be made about whether it is prescribed to future patients, meaning children younger than Mia may be denied it.

Danielle continued to say: "This is our child that you are putting numbers on and you are basically saying 'they're not worth it, they're not worth all of this' and that is devastating for parents."

More than 10,000 people in the UK have cystic fibrosis, a condition which causes sticky mucus to build up in the lungs and digestive system.

Kaftrio reduces that mucus but it's estimated to cost between £100,000-£200,000 a year per patient.

Mia's dad, Josh McCarty, said: "For me, it's not just about increasing Mia's life expectancy it's also about the quality of her life, keeping her out of hospital with bacterial lung infections.

"She'll be able to go to school and have a more normal childhood. Kaftrio isn't a cure for cystic fibrosis but it is the next best thing."

The National Institute for Health and Care Excellence (NICE), which guides the NHS in England, is now consulting on using Kaftrico for future patients and its potential cost.

In a statement, they 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.

"Existing patients and new patients who are started on treatment while the NICE evaluation is ongoing will continue to have access to the treatments after NICE has issued its final recommendations irrespective of the outcome."