Little Charlie O’Keeffe is a born fighter. The seven-year-old has battled cystic fibrosis – Ireland’s most common genetically-inherited disease – since he was a baby.
His body mass index – a marker of the disease’s severity – got so low he needed a feeding tube so he could put on weight.
Parents Sarah and Mark O’Keeffe have to watch him closely all the time.
He can’t play many physical sports or go to summer camps in case he gets injured.
Recently, Charlie, along with 34 other kids who all suffer a rare form of the life threatening respiratory disease, the R347P mutation – were promised the chance to live a normal life.
They were to be prescribed the new wonder-drug Kaftrio produced by Vertex Pharmaceuticals, which costs €100,000 a year per child.
It can reverse the main symptoms – persistent cough, wheezing, infections – and increases lung function.
The CF children had to undergo blood and sweat tests in pre-trial testing at Our Lady’s Children’s Hospital in Crumlin.
Charlie did his on April 22 but then three days later all 35 kids were told they were not getting the drug because of a row over money between Vertex and the HSE.
Sarah, who is from Sligo, said; “ We were devastated. The lives of our children are being put at risk because of a financial dispute.
“I appeal to the Taoiseach Micheal Martin to intervene. He is a family man who adores his children so I know he would understand exactly how we feel.
“Kaftrio is a drug that saves lives and transforms how every CF child lives.
“It makes it easier to breathe and many of the children would not have to use nebulisers any more.
“But more importantly for us, it gave Charlie the chance to live normally, without his feeding tube.
“To be able to do all the things his friends do, like playing football without being at risk.
“To see your only child needing a feeding tube was very difficult to accept.
“But we had no choice because Charlie suffered from severe pancreatic inefficiencies and had severe problems putting on weight.
“We were over the moon every CF child in Ireland from 6 to 11 would be included.” But due to a legal glitch, kids with Charlie’s CF genotype would not be given the life-changing drug. The news was hard to bear.
Sarah added: “I could not believe what I was hearing – my son and all the other kids were being denied the chance to beat this disease.
“You see a light at the end of a tunnel and then suddenly it is all taken away from you.
“The HSE signed a 10-year deal with Vertex to supply this drug to Irish CF children.
“If Charlie was 12 he would get it. However, for some legal glitch, kids aged six to 11 with my son’s genotype were excluded. It is heartbreaking for all 35 families.
“Vertex wants more money at a higher rate for our kids to be included and the HSE are refusing to pay it.
“It is now an ongoing legal dispute and we have no idea how long it will go on for before it is resolved. We had our hands on a drug that could save all these children’s lives and then suddenly it was taken away from us.
“I appeal to both parties to reach an agreement and give these 35 children the chance for a better, normal life.
“I think the dispute came about because of a simple oversight. I know Kaftrio is expensive, but it will mean less hospitalisations, less medication, and less breathing issues for the kids.
“The good news is 140 other CF children who have a different genotype are to get Kaftrio. We are delighted for all of them but I know they won’t be happy until every child in the Irish CF family is included.”
The HSE confirmed it had five meetings with Vertex between January 6 and April 14 to discuss the pricing for the children with Charlie’s genotype.
An internal rapid review assessment in accordance with HSE procedures was also carried out.
It has now invited Vertex to attend a further meeting to discuss issues arising from their Rapid Review assessment report.
It added: “The application for pricing and reimbursement of this further subset of the licensed population remains under consideration.
“We cannot make any additional comment on possible outcomes.”
Get breaking news to your inbox by signing up to our newsletter
