Matilda will live longer and suffer fewer infections, her mother said.
- author, John Ayers
- role, BBC Spotlight
- Parents of a four-year-old with cystic fibrosis say they are ‘excited’ as ‘life-changing’ medicine becomes available on the NHS
- Matilda Blatchford, from Devon, is expected to live longer and have fewer infections on Kaftrio.
- It is approved for children over 2 years of age, but may not be available to new patients in the future due to cost.
A four-year-old Devon girl with cystic fibrosis will live a longer, healthier and happier life now that the drug is available on the NHS, her parents have said.
Kaftrio is approved for children over the age of two, so Matilda Balchford will soon be able to get a prescription.
However, the National Institute for Clinical Excellence (NICE), which guides the NHS, is consulting on the use of Kaftrio in future patients because of the cost.
NICE said existing patients would continue to receive treatment regardless of the decision.
Rose Blatchford said it was “disgusting” that her future children may not have access to the drug because of cost.
Matilda’s mother, Rose Blatchford, said: “The medications she is currently taking or may be taking generally only treat the symptoms and are working fine.” Ta.
“Kaftrio completely eliminates what’s in her lungs. It eliminates any infections, helps her gain weight, and allows her to live a longer, freer, happier, healthier life.” I’ll give it to her.”
She continued: She said: “You’ll have a better quality of life, fewer hospitalizations…You’ll be able to have children when you’re older, and you’ll be able to retire.
“It’s going to be wonderful, it’s going to be the best Christmas present ever.”
But she said she would “keep fighting” for children who may not have access in the future, depending on NICE’s decision.
“It’s disgusting. We know there are medicines that can prolong their lives, but it’s a mistake to think they won’t have access to them,” she said.
Scott Blatchford said: “This is a life-changing drug and it’s proven to work, so I’m hopeful we can get an agreement.”
‘Cost-effectiveness’
NICE says the drug costs £8,346 for a pack of 56 tablets.
This means the cost of treatment to the NHS could reach between £100,000 and £200,000 per patient per year.
Helen Knight, director of drug evaluation at NICE, said: “We are assessing the cost-effectiveness of these cystic fibrosis drugs to ensure taxpayers continue to get value for money.” Stated.
A Department of Health and Social Care spokesperson said: “Cystic fibrosis can be a devastating disease, which is why we are investing more than £1.1 billion into research into rare diseases.
“It is vital that patients have access to new and innovative medicines like this, but the NHS must use its budget fairly and to the benefit of all patients.”
David Lumsden, CEO of the Cystic Fibrosis Trust, said: “We must never go back to a situation where people with cystic fibrosis die too young and we are very grateful that there is a treatment that could change that. Because I know.”
