Father’s plea for life-changing cystic fibrosis drug for son
Warning over complications as 35 children denied drug in price war
THE father of a 10-year-old boy with cystic fibrosis who is awaiting access to a lifechanging drug has warned that further hold ups could lead to major health complications or worse for 35 children caught up in a price war over the drug.
He was speaking to the Irish Mail on Sunday ahead of a protest outside the Dáil this Wednesday organised by Cystic Fibrosis Ireland, as the HSE remains locked in a pricing stalemate with drug firm Vertex.
‘Some of the damage done can’t be reversed’
Luke Kennedy, of Tallaght, Dublin, whose son Matthew has CF, said: ‘Some of the damage that’s done [because of CF] you can’t reverse – scarring of the lung, pieces of the bowel having to be removed. This new drug, it’s not an antibiotic to try and stop an infection, it will stop infections occurring.
‘You see all the positive stories of people getting these drugs. Like when Matthew laughs he generally coughs. You see the stories where people are now saying how they can laugh without coughing.
‘Matthew would not be able to have children due to his CF, because it clogs up bodily organs. But with this, he most likely will be able to.
‘Another big thing is the hospitalisations will all stop. He should be able to get a cold or the flu and shake it off just like any of us.’
The drug, Kaftrio, is already taken in Ireland by people over 12 and children aged six to 11 with particular genotypes.
But it was only approved by the European Medicines Agency for children aged six to 11 with the
genotypes found in the ‘Kaftrio 35’ in January this year, meaning they were not included in a pipeline agreement between the HSE and Vertex made in 2017.
The contract between Vertex and the HSE does not cover children under 12 with a rare genotype, despite covering children over 12 with the same genotype and the same condition.
Mr Kennedy said Matthew is ‘very active’ and plays for the local football club. However, he is on an array of treatments and daily physiotherapy to bring him to a ‘normal’ level, and can be hospitalised for weeks after picking up a cold.
He said: ‘Day to day you do all these things and that will keep him at a certain level, but then as soon as he gets sick, he gets really sick.’
Mr Kennedy’s niece died three years ago aged 11, over complications relating to CF.
He said: ‘It’s not like all these kids are going to make it to 12,’ referring to the fact that the 35 children will be eligible for the drug on reaching that age.
Kaftrio has been described as ‘game-changing’ and was made available to 140 other children here with CF in May 2022, at the same time as the 35 children were excluded. Rather than treating symptoms of CF, it works by modifying proteins at a genetic level.
Mr Kennedy said: ‘Matthew should be able to attend school more regularly too with Kaftrio. Right now he’s probably missing anywhere between 20 to 40 days every year.’
In a statement provided to the MoS, Vertex said it was ‘committed to seeking a rapid resolution for the 35 children who currently do not have access to Kaftrio, in addition to the other populations of CF patients who could be eligible in the future.
The statement said: ‘We remain in discussions with the HSE and are keen to accelerate this.’
A HSE spokeswoman said negotiations with Vertex are ongoing.
‘The HSE is committed to continuing
to communicate and follow up with Vertex Pharmaceuticals in a proactive manner regarding this patient sub-population with a view to addressing the significant additional budget impact to the State proposed by Vertex to enable access to Kaftrio for this patient group.
‘The application for pricing and reimbursement of this subset of the licensed population for Kaftrio remains under consideration with the HSE.
‘The HSE cannot make any comment on possible outcomes from the ongoing process.’
The protest on Wednesday will begin at 12.45pm at Government buildings on Kildare Street in Dublin.
‘As soon as he gets sick, he gets really sick’