Calls for arbitrator to resolve row over cystic fibrosis drug price

Cystic Fibrosis Ireland has called for the appointment of an independent arbitrator to resolve a price row that is depriving 35 children with the disease of access to a life-changing treatment.

Earlier this year, 140 children with cystic fibrosis were given access to Kaftrio, which can greatly improve their quality of life. However, a further 35 children, aged between six and 11 but with a different genotype, are still waiting for access due to a price dispute between the Health Service Executive and the manufacturer, Vertex Pharmaceuticals.

Families affected by the row protested outside Leinster House on Wednesday as the Dáil resumed after the summer break. Cystic Fibrosis Ireland called on Minister for Health Stephen Donnelly to intervene and break the deadlock between the parties. The charity said the Minister and Vertex should agree to enter an arbitration process if there is little prospect of a resolution.

A failure to provide Kaftrio, which can improve lung function and reduce hospitalisations, could have implications for affected children’s health, it warned.

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“Until there is a resolution to this stand-off, the healthcare of the ‘Kaftrio 35′ remains compromised in a dispute not of their making and which is increasingly raising major ethical and human rights concerns,” said the organisation’s chief executive Philip Watt. “Healthcare delayed is healthcare denied.”

He added: “It raises the unwelcome prospect of parents being forced to consider taking legal action in future because their children are being denied access to healthcare available to all other children and adults with CF, including those in the same age group in Ireland.”

Cystic fibrosis is the most common life-threatening inherited disease in Ireland. One in 19 Irish people carry the cystic fibrosis gene, but both parents need to have it to pass on the disease to a child. It affects the absorption of salt and water in the lungs, sweat glands, pancreas and gastrointestinal tract. This results in the build up of a thick sticky mucous which can clog airways and harbour harmful bacteria, with recurrent chest infections resulting in lung damage the most common symptom. There are about 1,400 people with the disease living in Ireland.

The HSE said the pricing of the drug for the 35 children “remains under consideration”.

“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,” it said. “The HSE robustly assesses applications for pricing and 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.”

HSE and Vertex officials held 10 meetings on the issue in the first half of this year. The National Centre for Pharmacoeconomics, which assesses the price effectiveness of drugs, has recommended a full health technology assessment, which could take months to complete.

Vertex said it was committed to seeking a rapid resolution for the 35 children and was keen to “accelerate” discussions on the issue with the HSE.