TWO Clare children with Cystic Fibrosis who have a rare genotype are being “discriminated against” due to their exclusion from availing of a transformative “wonder drug”, a local councillor has claimed.
The HSE has been in negotiations since May with Vertex about giving access for a therapy called Kaftrio for 35 young children with a specific genetic condition.
This is already available to other Irish patients under an existing deal.
Councillor Mary Howard described Kaftrio as a “wonder drug” that stops the progression of Cystic Fibrosis in the body.
She warned that many of the 35 children are not well and have been regularly hospitalised, with more hospital visits expected to increase as the winter progressed.
Speaking at a council meeting on Monday, the Fine Gael Councillor said adults and those 12 and older have been receiving Kaftrio.
Last May, she recalled all children who were older than six were supposed to receive this drug.
“There are 175 children with CF within the six to 12 age bracket, of which 140 have started to receive it.
“However, 35 children with a rare genotype got the news on the May Bank Holiday weekend they wouldn’t be getting it due to a glitch or an omission of this genotype being listed on the contract of the deal for CF medicines, which was created in 2017.
“These 35 children are eligible for this drug and as far as consultants were concerned they were supposed to start it alongside the others of the same age.
“It has been significantly proven this drug works even better for this genotype. The same children have not been suitable for any CF modulator drug to date. Other drugs don’t work for them.”
Quoting one affected parent, Councillor Howard said it seemed as if responsibility for resolving this issue seemed to be passing between the HSE and the drug company, Vertex.
“There is no resolution between the HSE and Vertex. All the time 35 children are waiting and their CF is getting progressively worse. CF doesn’t improve unless you have got a preventative medicine.
“I spoke to one mother whose child couldn’t walk from their sitting room to their front gate without stopping for a breath. He can now play a full game of football as a result of this drug.
“There is nothing more heartbreaking for a parent watching their child struggling to breathe knowing there is a drug out there that would enable them to live like a normal child.”
Quoting a parent who has a second cousin (32) with the exact same genotype has seen her life transformed after receiving the drug.
“Her lung function has gone up from 60 to 90 percentile in a short space of time. She needs no hospitalisation. My little girl turns six in the coming weeks and we are devastated that she will not potentially get a chance to start this drug even though she is eligible for it.
“Her consultant has talked to her about it, her medical team have given her books about it. She was so excited to start knowing it would mean less physio and nebuliser sessions every day and
more freedom to be a child rather than being afraid of picking up every cold that has the potential to land her back in hospital.
“We are trying to protect her from as much as the media hype as we can. We told her there is a little bit of a delay and we are telling the men in the government to hurry. We are just praying that she gets what she calls her “magic tablet”.
Supporting the motion, Councillor Claire Colleran-Molloy recalled she had helped raise funds for national CF organisations having lost her granddaughter, Laura and nephew, Noel to the disease.
She proposed this proposal should be sent to the Cystic Fibrosis Association of Ireland.
Councillor Ann Norton said any young person’s life should not be put at risk due to the fact there is a glitch or whether there is a financial issues between the HSE and a drug company.
“These are children’s lives, they have an opportunity to live a fuller and longer life if they can get this drug. It is an awful shame that parents are suffering at home patiently waiting on whether or not their child will get this drug.
“These parents are continuously wondering how long have the child to live. The HSE shouldn’t be playing with children’s lives.”
East Clare correspondent, Dan Danaher is a journalism graduate of Rathmines and UL. He has won numerous awards for special investigations on health, justice, environment, and reports on news, agriculture, disability, mental health and community.