SOME 35 children with cystic fibrosis (CF) in Ireland, including two being treated in Cork University Hospital (CUH), cannot access life-changing drug therapy because of a pricing dispute between pharma company Vertex and the HSE.
The children, aged between six and 11 years, cannot access CF drug therapy Kaftrio because they have a specific genotype not included in a pricing agreement signed by the HSE and Boston-based Vertex.
Under that agreement, 140 other children with CF in Ireland in the same age group, but with a different genotype, have already gained access to Kaftrio at an agreed price.
Vertex is seeking an enhanced price for the 35 children concerned because of their genotype, a price that the HSE has been unable to accept. It has instead referred the matter to the National Centre for Pharmacoeconomics.
Éadaoin Ní Chadhla, 11, from Waterford is receiving treatment for CF in CUH.
Her mother Bríd said the news that Éadaoin will not be able to access Kaftrio because of her genotype, is like the “rug being pulled out from under their feet”.
“We were told she was going to be able to avail of it, so it was a complete shock when at the end of April we were told those with rare genotypes aren’t included in the deal,” Ms Uí Chadhla said.
The drug therapy would help Éadaoin to gain weight and strength to fight infections, increase lung function, and overall increase the length of her life. While the wait continues for a cure for CF, Ms Uí Chadhla said Kaftrio was the “best she can hope for” right now.
“The company that manufactures Kaftrio are looking for more money for a drug that’s sitting there on a shelf, that is being made available to other CF patients,” she said. “It’s just heartbreaking.
“You’re looking at something that can help your child and it just has to be handed to you, and you can’t get it.”
Cystic Fibrosis Ireland has expressed “deep concern” that these children “are the pawns caught up in a pricing dispute between pharma and the HSE”.
Cork senator Jerry Buttimer raised the issue in the Seanad this week, calling on the Minister for Health to meet with representatives of Cystic Fibrosis Ireland to address the issue.
“This new medication Kaftrio allows cystic fibrosis patients to live a fairly normal life. I know of a number of young children who have it and have benefited enormously from this drug therapy. Their quality of life has been enhanced beyond words,” he said.
“On behalf of the children, their families, and Cystic Fibrosis Ireland, I implore those concerned to find a resolution to this matter and to allow access to the Kaftrio drug therapy.”
Mary Butler, the junior minister at the Department of Health, said the HSE “robustly assesses” pricing applications to make sure it delivers the best value and “ultimately makes more medicines available to Irish citizens who need them”.
She said that a full assessment of the clinical and cost effectiveness of a pricing application relating to Kaftrio was commissioned by the HSE on May 16.
Ms Butler said that “a lot of work is underway behind the scenes” regarding pricing of Kaftrio for children with CF.
“Four meetings have taken place, one of which was held as recently as May 16. I am hopeful and confident that because this drug has been made available to some children who have cystic fibrosis, more progress will be made for the families to whom he referred,” she responded to Mr Buttimer.