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Sam Boal
Cystic Fibrosis

'Intensive work' ongoing to give 35 children with Cystic Fibrosis access to life-changing drug

Donnelly said it is important to him ‘that children get access to the drug that they need’.

HEALTH MINISTER STEPHEN Donnelly has said he wants to see the 35 children with cystic fibrosis (CF) aged between six and 11 years old, who have been excluded from accessing a life-changing drug, Kaftrio, get the medication they need.

The minister said there is “intensive work” ongoing between the Department of Health, the HSE and Vertex, the manufacturers of the drug, on the issue. 

Cystic Fibrosis Ireland recently expressed its deep concern that 35 children with CF have been excluded from accessing the drug due to a new pricing dispute between the Boston-based pharmaceutical company and the HSE.

It believes that this development is all the more unfair because 140 other children with CF in Ireland in that exact same age group, but with a different genotype, have already gained access the drug. 

Philip Watt, CEO of Cystic Fibrosis Ireland, has said the dispute has been crushing news to the parents of the 35 children concerned. 

Cystic Fibrosis Ireland understands that the reason these children have been excluded is because of their genotype, and that they are outside the 10-year Portfolio Agreement that was signed by the HSE and Vertex in 2017.

Watt said: “Once again children with CF in Ireland are the pawns caught up in a pricing dispute between pharma and the HSE.”

When asked about the dispute in the Dáil today, Donnelly said it is important to him “that children get access to the drug that they need”.  

He acknowledged that is of huge concern to the families involved.

The minister said the HSE has statutory responsibility for decisions on pricing and reimbursement, but said all parties are working to resolve the issues. 

Professor Paul McNally, a consultant in paediatric respiratory and sleep medicine at Our Ladys Children’s Hospital Crumlin and Beacon Hospital, told RTÉ’s Claire Byrne programme this morning “that the clock is ticking”, stating they are “desperate to get these drugs into children as soon as we can”.  

He said the delay is causing “unnecessary stress and anxiety” for patients and their families. 

A statement from Vertex to the programme stated that children with with a certain genotype were not included in the original reimbursement deal. However, it went on to state that Vertex is looking to extend the agreement to include the 35 children. 

Vertex said it stands ready to resolve the issues as quickly as possible and remains committed to progressing the discussions with urgency. 

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