Campaigners are urging the HSE, the Taoiseach and Health Minister Jennifer Carroll MacNeill to overturn a recommendation from the HSE Drugs Group, issued this week, not to reimburse Skyclarys, the first approved treatment for Friedreich’s ataxia.
The rare disease causes progressive damage to the nervous system and affects around 200 people in Ireland. In 2024, the drug omaveloxolone, branded as Skyclarys, is the first licensed drug approved in Europe for treatment of the disease.
Advocates and patients say that while it is not a cure, it has the ability fo slow the progression of Friederich’s ataxia by up to 55 per cent.
A final decision on whether the State will reimburse the drug is due on 25 August, after a two-year-long campaign led by those living with the disease.
It comes as Independent Ireland released a letter, sent to TD Padraig Rice, Chair of the Health Committee, requesting that the committee be immediately reconvened in light of the HSE recommendation not to approve the drug for patients in Ireland.
Craig Coady is the father of a young boy suffering with the condition. He lost his son Rory last year, aged 13, and has campaigned for the drug to become available for his 16-year-old son Paudie who also lives with the condition.
Speaking this week, he said the news that the HSE Drugs group made a recommendation against the State reimbursing the drug, was crushing.
“We got the worst news we could ever get […] It’s devastating news. Just awful. I really was hoping that it would pass today. The fact that the rare disease committee, two weeks ago, were very positive on this drug, and sent the information back to the Drugs Committee, but unfortunately, they’re not going to reimburse it.
“There is a meeting on the 25th of this month with the HSE senior management team. I really want the people of Ireland to get onto every TD out there and get this overturned, to get this passed. It’s just inhumane,” said Coady through tears.
“It’s the worst news I’ve got since the passing of my little boy, Rory, to this disease. Please get onto every TD. We have to get out there. I’m not going to give up.”
Mr Coady said he “just can’t understand” the rationale behind the HSE’s decision, when the drug is available nationally in ten countries, including nine in Europe. Skyclarys is authorised for use in Europe, and Germany, Slovenia, Luxembourg, Austria, Switzerland, Poland, Italy, Spain, Slovakia, and the United States all have public or State access to the drug.
In February 2023, the drug was approved in the US for treatment of the disease.
The Irish Government’s record of Biogen’s submission confirms this. The Italian medicines regulator, AIFA, recently formally approved the reimbursement of Skyclarys through the Italian National Health Service last December.
28-YEAR-OLD ‘FIGHTING FOR HER SURVIVAL’
The cost of reimbursing the drug in those countries is not publicly available, however the main issue in Ireland appears to be the cost of the drug versus the evidence of benefit.
It has been reported that the cost per patient is approximately €280,000 per year, with the estimated five-year cost of treating all eligible patients said to be around €130 million.
A fortnight ago, 28-year-old Emily Felix, who has Friedreich’s ataxia, secured High Court permission to bring a judicial review challenge against the HSE and the Minister for Health. Ms Felix, who has had the condition since she was 12, is contesting “critical” delays in determining state approval and reimbursement for the drug.
The young woman from Co Kilkenny, who is studying to be a solicitor, is fully reliant on a wheelchair and requires assistance for many aspects of daily life due to the condition.
In the lawsuit, she has asked the High Court to direct the HSE to finalise its decision on the approval, reimbursement and funding of omaveloxolone, a drug capable of slowing the progression of FA. In bringing the case, she ultimately seeks access to the medicine.
Ms Felix says she is “fighting for [her] survival” in bringing the case.
Her lawyers have submitted that delaying her access to the drug has “real and irreversible consequences”.
“She fears that without access to treatment capable of slowing progression now, her condition may deteriorate to such an extent that future treatment opportunities may ultimately be of little practical benefit to her,” her legal team have said.
‘A TRAGIC REFLECTION OF STATE PRIORITIES’
Campaign group Care Champions said this week that the HSE’s rationale was cost, slamming the decision as a “tragic reflection” of the State’s priorities. It pointed to figures provided by the State Claims Agency, NTMA annual reports, and the National Centre for Pharmacoeconomics (NCPE).
“Funding this treatment over 5 years is estimated at €130M to €160M (€26M–€32M a year).
“To understand why this feels so deeply unjust to everybody, we only need to look at where public health money routinely goes when systems fail:
“€80M–€110M plus every year in Legal Fees: The State Claims Agency spends tens of millions annually on legal teams, medical experts, and court costs purely to litigate medical negligence claims. Just 18 months of legal defence costs could cover 5 years of Skyclarys.”
It also pointed out that hundreds of millions had been spent in annual payouts by the Health Service.
“In 2024 alone, clinical negligence damages paid out by the State reached €210.5 million (and over €275 million in clinical care compensation in 2023). A single year of medical failure payouts exceeds the entire 5-year budget requested for Skyclarys.
“The estimated future liability for active clinical negligence claims sitting in the system currently exceeds €5.3 billion,” they added.
“We understand that legal settlements are mandatory court obligations, while drug reimbursement is discretionary budget management. But that is precisely the problem.
“When the healthcare system manages hundreds of millions of euros every single year dealing with the fallout of clinical errors, (often fighting harmed families) but tells 200 people living with a degenerative, progressive disease that €30M a year to slow their physical decline is “unaffordable,” it is a tragic reflection of State priorities.
“European regulators (EMA) approved Skyclarys, and patients in some European countries already have access. Irish patients deserve the exact same chance at a better quality of life.
“Health is not just a line item on a spreadsheet.”
Independent Ireland TD Ken O’Flynn previously said that the Government “found €125 million in a day” to spend on bilateral support to Ukraine, yet Irish patients were waiting over 700 days for a funding decision.
“The argument is about how quickly the State can move when it wants to. And how slowly it moves when the person waiting is Irish and sick,” said O’Flynn last month.