'It all comes down to money': 35 children in need of CF drug 'left in limbo'

'The girls are on high-tech drugs, they are on machines that have to be replaced,' said Gráinne Uí Luing of her daughters Fiadh and Caoimhe. File picture: Shane O'Neill 

Cystic Fibrosis Ireland said they are no longer sure the HSE can negotiate fairly with a pharmaceutical company on the cost of revolutionary drugs for young children.

Some 35 children in need of a specialised drug Kaftrio have been in limbo since May as the HSE and pharmaceutical company Vertex argue over extending a drug-price deal to include more health conditions.

Now CF Ireland have called for a public debate following comments made by the director of the health body advising the HSE on value for money in medications.

CF Ireland CEO Philip Watt said: “Our confidence is very shaken that the National Centre for Pharmacoeconomics [NCPE] has the capacity to act as fair judges in the pricing/reimbursement of Kaftrio to 35 children with cystic fibrosis.” 

He said the approach being taken is “problematic”:   

We would challenge the NCPE to take part in a public debate with Cystic Fibrosis Ireland on their recent statements (and omissions).  

Mr Watt said his organisation is fearful that the financial focus means the children could miss out.

“There is no recognition from the NCPE in their statements that these drugs, known as CFTR modulators, have revolutionised CF care in Ireland,” he said.

Gráinne Uí Luing, whose two daughters  Fiadh and Caoimhe are affected, said the HSE are not taking the cost of all the care they need into account, including a bronchoscopy just last week.

“It all comes down to money," she said. “I understand that but they didn’t look at the costing of a cystic fibrosis patient to be in hospital for a week, to be peg-fed, to be on medication for years. The girls are on high-tech drugs, they are on machines that have to be replaced.” 

On Monday, director of the NCPE Professor Micheal Barry wrote to affected families who had contacted him following his comments around finite health budgets in a Sunday newspaper.

“When we learned that Vertex decided that the portfolio deal did not extend to the 35 patients, our reaction was one of disbelief,” he wrote, in an email seen by the Irish Examiner.

I can assure you we will do everything we can to facilitate Kaftrio for all who would benefit from it. 

A Vertex spokeswoman previously defended their decision to ask for increased money.