Cystic fibrosis drug for 35 children approved after lengthy delay caused by price dispute

At least 35 children with cystic fibrosis are to get access to a life-changing new treatment following resolution of a lengthy price row between the Health Service Executive and a drug manufacturer.

The HSE says it has approved Kaftrio for use by children aged six to 11 who were previously deemed ineligible for the drug, following “robust and lengthy engagements” with the manufacturer, Vertex Pharmaceuticals.

Last year, 140 children with cystic fibrosis were given access to Kaftrio, which can greatly improve quality of life. However, the 35 other children, aged between six and 11 but with a different genotype, were forced to wait for access due to a price dispute between the HSE and Vertex.

The impasse continued despite protests by Cystic Fibrosis Ireland outside Leinster House and a call by the charity for the appointment of an independent arbitrator to resolve differences. Doctors treating the children accused Vertex of treating them as “pawns for greater profit” for failing to make the drug available.

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HSE and Vertex officials held 13 meetings on the issue in 2021. Kaftrio has been approved for use in this group in the EU since January 2022. The HSE says it told Vertex to submit a dossier on its price claim in May 2022 but that was only submitted in December, after which the application could be processed.

On Tuesday, the HSE said it was 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. It said it robustly assesses applications for reimbursement to make sure resources can be stretched as far as possible.

“We are delighted today to announce that Kaftrio is now to be made available to those children previously ineligible. We have finalised the negotiated agreement with the manufacturer and are pleased to announce that the HSE has now approved this drug, which will improve the lives of the children who can now avail of it,” said HSE chief clinical officer Dr Colm Henry.

Minister for Health Stephen Donnelly described the announcement as “good news” for young people with CF. “This is 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.”

Cystic Fibrosis Ireland said it “warmly welcomed” the announcement. “In thanking the HSE and 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,” said the organisation’s chief executive Philip Watt.