Denial of access to CF drug is ‘cruel’, says mum of ill boy
PARENTS of children with Cystic Fibrosis are pleading with the HSE to allow them access to a life-changing drug.
A total of 35 children with cystic fibrosis (CF) aged between six and 11 have been excluded from accessing Kaftrio due to a new pricing dispute between the HSE and Boston pharmaceutical company Vertex.
Meanwhile, 140 other children with CF in Ireland in that same age group have already gained access to Kaftrio because they have a different genotype.
However, parents of the children affected have called for an urgent resolution to the dispute, as it is having a ‘cruel’ impact on their families.
Sarah O’Keeffe, who lives in Sligo, said she feels the health services ‘don’t care’ that this drug could change her son Charlie’s life. Charlie, seven, has to be tubefed as a result of CF, as well as having physiotherapy and using a nebuliser.
Sarah explained how this drug would have a major positive impact on his condition.
‘Tube feeding has helped him but eating and weight gain is very, very stressful for our family,’ she said. ‘When we heard of Kaftrio in November 2021, the team advised that this might help him so he wouldn’t need a feeding tube and he would gain weight more normally.’
However, just days after having pre-drug testing for Kaftrio, the family was informed that he did not qualify for the drug.
‘We accepted that this was just our life, but then the hope came that Charlie might be more of a normal kid and he can do sports. It’s cruel. We should never have been told if that was going to be the case,’ Sarah said.
Luke Kennedy’s son, Matthew, 10, from Tallaght in Dublin, has been in hospital with CF frequently since he was born. He has problems with his lungs and bowels and requires physio, medication and at-home treatments every day for his symptoms. Kaftrio would largely prevent CF from causing these symptoms.
‘It’s very stressful and disheartening on the entire family because you see what he’s going through day to day and you know there’s a drug out there and you know it’s just caught in the middle of this dispute situation,’ Luke said. ‘It looks like we have a lifeline with this drug and we just can’t get it.’
Cystic Fibrosis Ireland described the situation as alarming and said it will write to Vertex outlining its concerns.
Philip Watt, head of Cystic Fibrosis Ireland, said: ‘The absence of this life-prolonging medication for the Kaftrio 35 should raise major ethical and human rights questions for both the Government and Vertex. These differences need to be resolved with urgency for the sake of the 35 sick children.’
A spokeswoman for the Department of Health said the Minister has been ‘advised by the HSE that engagement with Vertex Pharmaceuticals is ongoing’.
‘It’s stressful and disheartening’