Cystic Fibrosis Ireland (CFI) has expressed serious concern about a pricing dispute which has left 35 children with cystic fibrosis unable to access the life-changing drug therapy, Kaftrio.

The children are all aged between six and 11 years.

The pricing dispute is between the Boston-based pharmaceutical company Vertex and the HSE. CFI understands that the reason these children have been excluded is because of their genotype and because they are outside the 10-year portfolio agreement that was signed by the HSE and Vertex in 2017.

“Specifically, the 35 children excluded have a particular genotype combination that includes a minimal or unclassified gene which was not included in the original agreement. CFI understands that Vertex is currently seeking an enhanced price for the 35 children concerned, which is different to the price charged for all other children and adults under the portfolio agreement.

“As a result, we understand that the HSE has been unwilling to accept this price and has instead referred the matter to the National Centre for Pharmacoeconomics,” explained CFI chief executive, Philip Watt.

The charity said that this development is particularly unfair because 140 other children in the same age group with cystic fibrosis in Ireland have already gained access to Kaftrio.

“The portfolio agreement fixed the price of a suite of drug therapies, including extensions based on age or genotype. It has prevented the ongoing disputes that once dominated access to previous cystic fibrosis drugs such as Orkambi. Until this matter is resolved, it means that the children affected will continue to experience significant delays in accessing Kaftrio and the enhanced quality of health and life that it brings,” Mr Watt noted.

He insisted that once again, children with cystic fibrosis in Ireland “are the pawns caught up in a pricing dispute between pharma and the HSE”.

“This is all the more heartbreaking because 140 other children with cystic fibrosis of the same age, but with a different genotype, recently secured access to Kaftrio. Going back to the old ways of doing business in negotiating access to life-changing medicines is simply unacceptable.

“We implore the HSE and Vertex to resolve this issue quickly and to extend the portfolio agreement to all children with cystic fibrosis to ensure that all concerned get immediate access and that this problem does not happen again,” Mr Watt added.

Cystic fibrosis is a multi-organ disease that primarily affects the lungs and digestive system. A defective gene and its protein product cause the body to produce unusually thick, sticky mucus that clogs the lungs and leads to life-threatening lung infections, and obstructs the pancreas and stops natural enzymes from helping the body break down and absorb food.