CLARE parents of children with Cystic Fibrosis have told of their fears for their childrens’ future if they cannot access a “miracle” drug at the centre of a dispute between the Health Service Executive and a pharmaceutical company.

Cystic Fibrosis Ireland estimate that around 140 children aged six-years-old to 11-years-old are already are on the drug Kaftrio, but 35 children with a particular genotype are not as they are outside the 10-year Portfolio Agreement that was signed by the HSE and Vertex in 2017.

Emma Fitzpatrick from Sixmilebridge is mother to eight-year-old James and says the drug would mean “everything” to their family, improving her son’s quality of life. She was “devastated” when she received a call from James’ consultant to say that he would not be getting the drug.

“Literally you have your fingertips on this miracle – and it is a miracle to us. It is not a cure, but it’s the nearest we will ever get to a cure within our kids’ lifetimes.

“This is it, our great white hope, and something I certainly didn’t think when James was diagnosed he would ever get. Then suddenly to have that snatched away at the last minute, it’s absolutely devastating.”

She is appealing to the HSE and the pharmaceutical company to come together and resolve the issue for the sake of the children’s health and future.

“Everything we do at the moment is to keep him as well as he possibly can be. But, no matter what we do, CF is progressive. You can make every effort in the world but CF will progress in his little body no matter what we do. Kaftrio can stop that progression, stop damage being done before it is done and we can’t do anything else about it. Every day counts.”

Joining her in her call is Barbara Buckley from Ennis who fears her five and half year old daughter, who had expected to start the drug in January, will join the numbers of those currently excluded.

“At the moment she does an hour treatment in the morning and evenings, and it’s a mixture of nebulizer, physio, air clearances and she does it without complaint.

“Since she was diagnosed this drug was on the horizon for us, it was always a glimmer of hope for the future. We were getting closer and closer to this ‘magic tablet’, as she calls it, then we heard the news two weeks ago.

“It felt like the wind was taken out from underneath us because we always had this hopeful date of January 2023 to look forward to. All of a sudden it is pulled away from us.

“We were devastated and we just knew straight away it was something we were going to have to fight for because our little girl, as well as everyone else in the cohort, are priceless to us and they deserve the same chance as every other child with CF who will be getting the drug straight away.”

Older children who have already benefited from the drug have experienced an “amazing effect” she adds.

They both believe the cost of the drug is a small price to pay for the health of all of the children affected, saying putting them on Kaftrio now would reduce other long term medical costs.

Emma says, “There are kids in every two or three months to get two week IV antibiotics as a matter of course, that’s obviously costing the HSE a fortune, and that would be stopped with Kaftrio.

“You can’t downplay how important this drug is. There is a cost saving, it would be taking away possible future lung transplants, liver transplants, you are literally transforming these children’s lives. By giving them the drug now in the immediate, you are taking away the long term cost of them to the system.”

She concludes, “Time is of the essence. We’ve just gone through two years of Covid and we all know now we just don’t know what’s around the corner. James could wake up in the morning and pick up an infection through no-one’s fault and be in the hospital for weeks without Kaftrio to cushion that.

“As much as he is well now, CF is on a knife-point constantly and you don’t know what’s around the corner. Without having Kaftrio there to stop that progression in his body or stop damage being done you are really leaving them out on a limb.”

Barbara adds, “We are just pleading with them to resolve this and move fast.”

Cystic Fibrosis Ireland have called on the HSE and Vertex to return to the negotiating table saying the dispute has been “crushing” for parents.

Philip Watt, CEO, Cystic Fibrosis Ireland, comments, “Once again children with CF in Ireland are the pawns caught up in a pricing dispute between pharma and the HSE. This is all the more heart-breaking because 140 other children with CF of the same age, but with a different genotype, recently secured access to Kaftrio.

“Going back to the old ways of doing business in negotiating access to life-changing medicines is simply unacceptable. We implore the HSE and Vertex to resolve this issue quickly and to extend the Portfolio Agreement to all children with CF to ensure that the children concerned get immediate access and that this problem does not happen again.”

In a statement the HSE has said it “is committed to providing access to as many medicines as possible, in as timely a fashion as possible, from the resources available (provided) to it”.

“The HSE robustly assesses applications for pricing and reimbursement to make sure that it can stretch available resources as far as possible and to deliver the best value in relation to each medicine and ultimately more medicines to Irish citizens and patients.

“HSE decisions on which medicines are reimbursed by the taxpayer are made on objective, scientific and economic grounds.

“A rapid review assessment was carried out and the HSE have confirmed it had five meetings with Vertex between January 6 and April 14 to discuss the application for pricing and reimbursement of Kaftrio for children with the particular genotype.

“The HSE have invited Vertex to attend a further meeting to discuss issues arising out of the Rapid Review assessment report.

“The application for pricing and reimbursement of this further subset of the licensed population for Kaftrio remains under consideration with the HSE,” the statement read adding, “The HSE cannot make any additional comment on possible outcomes from the ongoing process at this time.”

When contacted by The Clare Champion, a spokesperson for Vertex stated, “The innovative long-term reimbursement agreement between Vertex and the Health Service Executive (HSE) that was completed in 2017 has allowed Irish CF patients to be some of the first in Europe to gain rapid access to the latest approved Vertex medicines. 

“A small number of patients in the 6 to 11-year-old patient population with specific genotypes who are eligible under the EMA licence for Kaftrio, were not included in the original reimbursement agreement as science has moved on and over the years since the initial agreement was signed the knowledge and data regarding which of our medicines demonstrate clinical value to CF patients has evolved considerably.

“Vertex is looking to extend the agreement to include these patients and allow reimbursed access quickly, in a manner consistent with the existing contract. We stand ready to resolve this as quickly as possible and remain committed to progressing these discussions with urgency.”