The decision by the HSE to approve use of a key cystic fibrosis (CF) drug in young children has been welcomed by Cystic Fibrosis Ireland.

Kaftrio is used to treat CF which is an inherited condition that primarily affects the lungs and digestive system. The treatment has been found to significantly improve lung function.

However until now, a pricing dispute between the drug’s manufacturer Vertex and the HSE had meant that 35 children with CF who were aged between six and 11 years were unable to access it.

The reason these specific children had been excluded was because of their genotype and because they were outside the 10-year portfolio agreement that had been signed by the HSE and Vertex in 2017.

The 35 children have a particular genotype combination that includes a minimal or unclassified gene, which was not included in the original agreement. Vertex had been seeking an enhanced price for these 35 children, which was different to the price charged for all other children and adults under the portfolio agreement.

The HSE was unwilling to accept this price and had referred the matter to the National Centre for Pharmacoeconomics.

Cystic Fibrosis Ireland had been campaigning on this issue since, highlighting that 140 other children in the same age group with cystic fibrosis in Ireland had already gained access to Kaftrio.

However, the HSE has now approved use of the drug by these 35 children “following robust and lengthy engagements with Vertex”.

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

“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,” it noted.

Welcoming the news, Cystic Fibrosis Ireland CEO, Philip Watt, thanked all of the families and friends of the 35 impacted children who had been 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,” he commented.

Meanwhile, Mr Watt urged the Minister for Health, Stephen Donnelly, to take this opportunity “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 children aged two-to-five years with cystic fibrosis when the European Medicines Agency is likely to approve such an extension,” he insisted.