CF drug delay causing 'unnecessary and irreversible damage'

Sisters Fiadh (6) and Caoimhe Ní Luing (8) from Maynooth attended the protest over the life-changing drug for Cystic Fibrosis patients, Kaftrio, on Kildare Street in Dublin. Pictures: Gareth Chaney/Collins Photos

The mother of a 10-year-old girl denied access to a revolutionary drug for Cystic Fibrosis has called on the health minister to intervene in a dispute between the manufacturer and the HSE.

The drug, Kaftrio, can halt the effects of cystic fibrosis, and patients who have used it say it changed their lives. 

A group of 35 children with Cystic Fibrosis was told in May they do not qualify under current pricing arrangements.

Speaking during a protest held by Cystic Fibrosis Ireland, Julie Bernard said her daughter Aisling was devastated by that news.

“We were told in May she would get the drug, then she couldn’t. For us, it is now day 137 of completely unnecessary and irreversible damage to her body. That is unacceptable,” she said.

Julie Bernard and her daughter Aisling (10) both  during a protest by Cystic Fibrosis Ireland.

“We are calling on the minister, on the HSE and on Vertex to be fair and to give this drug to our children.” 

Aisling needs nebulisers, medication, and physiotherapy every day, among other supports.

“You worry about the now, you worry about the future,” she said.

That is why Kaftrio would make such a difference, this disease is progressive. It is relentless.” 

She praised the medical team in CHI at Temple Street and said they were also shocked to discover her daughter could not be given this drug.

“To me, this feels like wilful, deliberate psychological harm,” she said.

“This disease is doing damage to her body every single day. With Kaftrio, it stops that but it can’t reverse the damage that has been done.” 

Aisling Bernard is from Raheny and needs nebulisers, medication, and physiotherapy every day.

Cystic Fibrosis Ireland chief executive Philip Watt said the issue centres on a 2017 price deal between the HSE and manufacturer Vertex which families and doctors had understood would cover all affected patients.

“If the children are not included in that deal, then we need to see a new deal at a fair price,” he said. 

It is now four months the 35 children have been denied, it raises major human rights issues.” 

He said the group will shortly meet with health minister Stephen Donnelly.

“We think if it can’t be sorted between the two parties, and there is a Mexican standoff really, then an independent arbitrator should be appointed,” he urged.

A HSE spokeswoman said the application for pricing for this group remains under consideration.

“The HSE cannot make any comment on possible outcomes from the ongoing process,” she said.

“The HSE is committed to providing access to as many medicines as possible, in as timely a fashion as possible, from the resources available provided to it.” 

Campaigner Jillian McNulty was also at the protest. She started using Kaftrio just over two years ago.

Jillian McNulty started using Kaftrio over two years ago and says she 'wouldn't be here if it wasn't for Kaftrio'.

“When I started it I’d been in hospital for three months, I was on continual oxygen and in a wheelchair,” she said.

“I started it and walked out of hospital a week later.” 

She added: “I wouldn’t be here if it wasn’t for Kaftrio. I would have been dead in a month if I hadn’t started it.”

Ms Bernard also stressed that Aisling and the other 34 children frequently miss school due to illness, sometimes up to 60 days during the year.

“You can’t pay that back,” she said.

Exercise and activity are crucial for children with Cystic Fibrosis to support their lung function.

“She likes doing dancing, she likes running, she cycles, she scoots,” Ms Bernard said.

“But when she is ill, she misses out on camps and activities. You constantly have to judge if she is too ill to go, but then on the other hand she really needs the exercise. It is a constant balance, it is relentless.” 

She said her sons worry about Aisling when she is sick and said these unseen impacts on a family are not often discussed.

Julie Bernard said being denied Kaftrio feels like 'wilful, deliberate psychological harm'.

“We have to decide where to go, when to go, and should we go,” she said.

Working life for her and her husband is also impacted, she has taken leave at times and changed the days she works to be there for her daughter.

“We do get a lot of support, but I often say to him, ‘It’s you and me in the trenches’,” she said.

Julie Bernard said her sons also worry about their sister's health.

Regular access to food is another consideration, as children with CF need between 150% and 200% more calories every day than children of a similar age, she explained.

This sometimes leaves Aisling having to explain to her friends why she is allowed to have high-calorie foods at school when they are restricted by their parents.

“You can never do catch-up, so you have to do that every single day,” her mother said. 

“You have to map the day out, it is relentless. You can understand why children are asking, they’re curious but she doesn’t always want to say she has [Cystic Fibrosis].”