News that a new treatment for children with Duchenne muscular dystrophy will not be made available by the HSE in Ireland has left families "reeling" at the decision.
Ann Marie Walsh Harte and husband Padraig Harte, who live in Co Mayo, were desperately awaiting approval of the drug treatment, Translarna, for their son Lewis (5) who has Duchenne muscular dystrophy.
“My son is five and the news today that the HSE are not going to fund the medicine is devastating,” said Ms Walsh Harte. “To us, the news comes the same as if it were a death in the family. We are totally devastated.
"This is life-changing medicine we are talking about. Everyone is just devastated as this is life-changing medicine within the muscular dystrophy community. The fact that the drug is available in 20 other EU countries and Northern Ireland makes this decision all the tougher for us."
Ms Walsh Harte said that her son deserves to have access to the drug and said she is appealing to Minister for Health Simon Harris to reverse this decision.
“If this drug was made available, then we would be looking at an extra seven to 10 years of our little boy Lewis being able to walk. The way we are at the moment, we are looking at another two years at best.
Pinned hopes
“Once he loses his ability to walk he will be in a wheelchair but once this happens the entire system begins to fail. The heart, the lungs and everything. The longer he can stay on his feet the longer he can keep going,” added Ms Walsh Harte.
She said that she and her family, like many other families, had pinned their hopes on the HSE and have been let down by the system yet again.
“A lot of people look at Lewis and think he’s okay and dealing, but they don’t see what we see every single day and sadly, we have been let down so badly on this. I am utterly devastated.”
In a statement on Tuesday, the HSE confirmed it “has written to the manufacturer of Ataluren (Translarna) advising them that the HSE will not be funding this drug”.
“The HSE is very much aware that this decision is upsetting to patients who are affected by this condition. It will also be a disappointment to their families and the treating clinicians who support these patients. However, it is of the utmost importance to recognise that the HSE Drugs Group who reviewed the effectiveness of the drug did not consider the evidence for the clinical benefit of Ataluren (Translarna) to be sufficiently strong in the context of the proposed cost and budget impact.
“There is an onus on the HSE to ensure that any new drug is cost effective. New drugs can provide improvements in quality of life, but may not provide a cure. Nevertheless, the prices sought by drug companies for such products can run to hundreds of thousands of euro per patient per annum. The HSE must have regard to its wider obligations to the 4.7 million population it serves and needs to maintain the full range of health services to all of the other patient groups within the finite resources at its disposal.”