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Minister for Health Stephen Donnelly said the approval was a "very welcome development". Leah Farrell
Cystic Fibrosis

HSE approves use of life-changing Cystic Fibrosis drug for 35 children previously excluded

Minister for Health Stephen Donnelly said it was a “very welcome development”.

THE HSE HAS approved the use of cystic fibrosis (CF) drug Kaftrio for children aged 6-11 who were previously excluded from accessing the medication. 

The health service said the approval follows “robust and lengthy engagements with the drug manufacturer Vertex”.

The approval comes after Cystic Fibrosis Ireland expressed concern last year that 35 children with CF had been excluded from accessing the drug due to a pricing dispute between the Boston-based pharmaceutical company and the HSE.

The charity said it believed it was unfair because 140 other children with CF in Ireland in the same age group, but with a different genotype, had already gained access the drug.

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

“HSE decisions on which medicines are reimbursed by the taxpayer are made on objective, scientific and economic grounds,” it said.

“The HSE robustly assesses applications for reimbursement to make sure that it can stretch available resources as far as possible and to deliver the best value in relation to each medicine and ultimately more medicines to Irish citizens and patients.”

Health Minister Stephen Donnelly said it was a very welcome development.

“Kaftrio has been a life-changing drug for Cystic Fibrosis sufferers and I wanted it made available to everyone who needs it. I would like to commend the HSE and the National Centre for Pharmacoeconomics for working so hard to conclude this agreement with the manufacturer,” he said.

“This is good news for the young children with Cystic Fibrosis who can now access this therapy. It is another important milestone that will help us to continue to improve outcomes for patients with Cystic Fibrosis in this country.”

‘Future-proofing’ 

Cystic Fibrosis Ireland has also welcomed the news, but is urging the Minister to ensure “future-proofing” to avoid “stressful disputes” in the future when the drug is extended to younger children.

CEO Philip Watt said: “In quietly celebrating with all who have contributed to this positive outcome today, we particularly think of, and thank, all of the parents, friends and families of the 35 impacted children directly affected by this dispute.

“Their voice, their dignity and their strength over the past year has been the key factor in providing the necessary momentum for resolving a crisis that should never have occurred in the first instance. It is an honour for Cystic Fibrosis Ireland to continue to work with, and support, the Kaftrio 35 as best we can.”

Watt said the impacted parents have been without Kaftrio “for far too long”.

“We would urge that wider lessons can be discussed and learned for the drug reimbursement process itself in Ireland. The recent statements by Minister Donnelly in this context are very much welcomed.

“In thanking the Minister, Cystic Fibrosis Ireland also takes this opportunity to urge him to ‘future-proof’ Kaftrio for the relatively small number of children who are predicted to gain access on age or eligible genotype extensions in the future.

“We do not want to see this dispute happening again in 12 months’ time for 2-5-year-olds with cystic fibrosis when the European Medicines Agency is likely to approve such an extension.”

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