Parents hail decision as lifesaving for young children
By Julie Waters
TWO beautiful Bangor sisters have been given the ‘gift of life’ after their access to new cystic fibrosis (CF) drugs was guaranteed.
Four year-old Ava and two year-old Mia Jones both live with the degenerative lung and digestive condition that could cut short their lives without lifesaving medication.
Just six months ago their parents, Steven Jones and Aimee Chambers, were given the heartbreaking news that due to a ‘birthday lottery’ only their older daughter Ava would get the ‘life transforming’ medication she needed and their youngest daughter Mia would not.
After the National Institute for Health and Care Excellence (NICE) deemed the drugs ‘too expensive’ the couple issued an urgent plea for a ‘price tag’ not to be placed on their daughter’s life.
The guidance from NICE stated patients already taking Kaftrio, and other drugs, would be kept on them but the drug would not be prescribed for new CF patients, including children.
Steven and Aimee became part of a massive public campaign, raising awareness and appealing to elected representatives for support. After a ‘worrying’ seven months, the manufacturing company Vertex has now confirmed that the medication will be made available to NHS patients in NI.
The company has finalised a reimbursement agreement to allow for ‘extended long-term access’ to the medication for all eligible existing and future patients.
Dad Steven said the fear that he would outlive his children had been taken away after his daughters’ medication was guaranteed.
“Without the medication they may have reached their 20s and now they will reach their 60s and over,” he said. “It has taken away every parents’ worry that they will have to bury their own child.”
Steven said: “We knew that Ava would get Katrio but we were unsure about Mia. It was a worrying few months, everything was up in the air. As the time went on Mia’s health started to go downhill, her lungs started to become damaged.
“We were up and down to the hospital several times and we thought Mia might have to have a bronchoscopy, a procedure where she is put to sleep and they put a tube down her throat to look at her lungs.”
Such was the deterioration of his daughter’s condition that clinicians decided to begin her on the Kaftrio medication.
Said Steven: “As soon as they started Mia on the medication the lung damage started to reverse and her lungs started to clear.”
Aimee said Mia has been on the medication for almost two months and the x-rays show a significant difference in her lungs.
“She hasn’t coughed with it either and she was always coughing,” she said.
There is also good news for her big sister Ava who has also recently started taking the same medication.
Said Steven: “Ava is thriving as well, her height and weight have balanced, her appetite has increased. When she has had any infections they have cleared up quickly.”
Aimee agreed saying: “You can just see the difference in Ava’s face, she is so full of life. If that is what we can see on the outside then what is it doing on the inside? She is trampolining, dancing, and doing gymnastics, she never stops.”
The Bangor couple believe that campaigning by local families has helped ‘transform the lives’ of many people living with cystic fibrosis who rely on this medication.
Said Steven: “We contacted a number of MLAs and sent letters to MPs at Westminster. I think (by sharing our stories) it really brought it home to people and made it more known.
“The medication has transformed our daughters’ lives and will go on to transform the lives of many others. It is such a relief. Long term it has increased people’s life expectancy.”
Aimee poignantly described one noticeable change in their daughters’ lives since taking the medication. “When you walk past their bedrooms at night they are silent. There was always wheezing, coughing and all sorts of breathlessness. It is strange. If you take away their medication they are just like every other child.”
Said Steven: “They will be on their medication for life but we can now look forward to the future.”
