Drug price row ‘crushing news’ for children with cystic fibrosis

THIRTY-FIVE children with cystic fibrosis are unable to access a drug therapy that would be ‘life-changing’ for them and their families.

The children, aged between six and 11, have been excluded from accessing Kaftrio drug therapy due to a pricing dispute between Boston-based pharmaceutical company, Vertex, and the HSE.

Cystic Fibrosis Ireland has said that 140 children with CF in Ireland in the same age group, but with a different genotype, have gained access to Kaftrio.

Philip Watt, of Cystic Fibrosis Ireland, said this is ‘crushing news’ for the families affected.

He explained that the 35 children excluded have a particular genotype combination that includes a minimal or unclassified gene which was not included in a ten-year Portfolio Agreement that was signed by the HSE and Vertex in 2017.

Gráinne Uí Lúing is mother to Caoimhe, eight and Fiadh, six, both of whom are among the 35 children denied the drug.

‘We were hoping that within the early parts of this year we would be starting this life-saving drug for the girls. To find out that we were two of the 35 that were being denied this drug was just devastating for our whole family,’ Ms Uí Lúing, in Maynooth, Co. Kildare, told the Irish Daily Mail.

‘The girls are generally well but we’re still talking about regular hospital visits, daily antibiotics, pro-biotics, inhalers, nebulisers, it’s just constant. We try to prevent any illnesses as opposed to treating them when they come upon then.’

Ms Uí Lúing, who has previously campaigned for the Orkambi drug to be made available to her daughters, said being forced to fight again is devastating.

‘This drug would be life-changing for the girls… the changes for the people who are already on Kaftrio are unbelievable. People who weren’t able to do a flight of stairs are now running marathons,’ she said.

Mr Watt implored the HSE and Vertex ‘to resolve this issue quickly and to extend the portfolio agreement to all children with CF.’