Children with cystic fibrosis being used as pawns by pharma company, say doctors

Julie Bernard and her daughter Aisling, 10, from Raheny during a protest at Leinster House by cystic fibrosis families over continued exclusion of 35 children with CF from life-changing drug Kaftrio. Picture: Gareth Chaney/ Collins Photos

Sick children are being used like pawns by a pharmaceutical company trying to make more money from the HSE, a senior group of doctors working with cystic fibrosis sufferers said.

The HSE has been in negotiations since May with Vertex about giving access for a therapy called Kaftrio for 35 young children with a specific genetic condition.

This is already available to other Irish patients under an existing deal.

Doctors with the National Clinical Programme for Cystic Fibrosis said progress on fighting disease can only happen when everyone works together.

“This partnership is under threat, because of the approach of Vertex who we believe are effectively using this group of vulnerable children as pawns in negotiations for greater profit,” they said.

Delays around this drug are now causing “unnecessary suffering and direct, preventable harm”.

They said the deal, in place since 2017, is worth some hundreds of millions of euro.

“We find it difficult to accept why Vertex sees fit to accept the inclusion of children aged 12 and over with the same mutations in this pipeline deal, and then insist that children with these mutations aged 6-11 should be excluded from the deal,” they said.

It is understood Health Minister Stephen Donnelly is now considering writing to EU Commissioner for Health Stella Kyriakides on this issue, and also intends to share the doctors’ letter with her. It is believed the Irish deal with Vertex was used as a template by other countries.

Cystic Fibrosis Ireland CEO Philip Watt echoed the consultants’ concerns.

“The exclusion of these 35 children has been cruel and remains a matter of utmost concern to Cystic Fibrosis Ireland and to the parents of the children impacted by this dispute,” he said.

He pointed out 140 other children also aged 6-11 are already accessing Kaftrio in Ireland.

“They just happen to have a different genotype, with the HSE and Vertex arguing over which genotype was included in the original deal,” he said.

Mr Watt met with the health minister on Thursday. They asked him to consider allowing immediate access for these children, without prejudice to a final agreement on pricing.

“We further urged the possibility of seeking international arbitration to resolve the present impasse,” he said.

Julie Bernard, the mother of a young girl in this group, previously told the Irish Examiner: “This disease is doing damage to her body every single day. With Kaftrio, it stops that but it can’t reverse the damage that has been done.”