Parents of children with an incurable muscle-wasting condition have called on the Minister for Health to accelerate the approval and funding of a new drug which they have described as a "game changer".
It is estimated that over 100 boys are currently living with Duchenne Muscular Dystrophy (DMD), a genetic disorder, mainly affecting males, that causes progressive muscle weakness.
As the life-limiting condition progresses, it affects the respiratory system and over time those with the condition may need the use of a wheelchair.
Families affected are now campaigning for the approval of a new drug, known as Givinostat, to be accelerated.
Among those calling on the medicine to be made available to patients here are the Brogans from Deansgrange in Dublin.
Their nine-year-old son, Fionn, was diagnosed with DMD when he was aged three.
"We were told on the day of the diagnosis that there really wasn’t many options of treatment...and to go off and 'live your best life'," said Meave Brogan.
Ms Brogan said Givinostat is a "massive game changer".
While it does not provide a cure for the condition, clinical trials, she said, have shown it can significantly slow down its progression.
"There's no cure for Duchenne, but Givinostat...gives hope for a real slowdown of the progression."
"We've had to have a lot of really hard conversations, explaining to him why he's struggling more, this is the first time that we could have a conversation about something that could help," she said.
"What makes it so difficult is that we have to explain to him that it's out there, but we just can't access it.
"So the hope feels actually quite painful. It's so close, but it's still really far away because of the process that it has to go through."
The drug received EU conditional marketing authorisation earlier this week, meaning it is on course for fast track approval as it fulfils an unmet medical need.
Campaigners here are concerned that even with full approval, it could take up to two years before it is rolled out.
"If it was anything near that, it's likely that Fionn will not be able to access that drug, and that is absolutely heartbreaking," said his dad, Michael.
"But it's also unacceptable to us, and that's why we're calling on the Minister for Health to intervene."
Mr Brogan said those with the condition do not have time to wait and that every day muscle is lost.
"We’ve a really resilient, really determined boy who wants to get on with life, and he wants to do the best he can do, and we want to give him every opportunity to be able to do everything he wants to do in life," he said.
"There's no cure for Duchenne, but Givinostat gives hope for a real slowdown of the progression.
"There's examples of boys that are still able to take steps in their late teens," he said.
Families affected are gathering outside Leinster House to bring their campaign directly to politicians.
They are also calling for Ireland's health authorities to follow Scotland and England in speeding up approval.
In a statement, the Department of Health said it was aware of the plight of patients with DMD and acknowledges the important of access to medicines.
It said the HSE has statutory responsibility for decisions on medicines, based on comparative clinical and cost effectiveness and engages with patients and clinicians throughout the assessment process.
It also said if the makers of the drug choose to submit a pricing and reimbursement application, the HSE will give "fair consideration" to the application.
The HSE said in a statement that the "national assessment and decision process cannot commence without a pricing and reimbursement application submission to the HSE".
It said the HSE must comply with the "relevant legislation when considering investment decisions around new medicines".
It added that it "robustly assesses applications for pricing and reimbursement to ensure that it can stretch available resources as far as possible and deliver the best value for Irish citizens and patients".
The HSE said that pharmaceutical companies are required to submit formal applications to the HSE if they wish their medicines to be added to the list of reimbursable items or funded via hospitals.
It said the "decision of pharmaceutical companies to market licensed medicines" is "outside the control of the HSE".
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