What Skyclarys Does
Skyclarys is not a cure. I want to be honest about that. Friedreich’s Ataxia will still shorten lives. But this drug — the first and only medicine ever approved for FA, both by the FDA and the European Medicines Agency — slows the progression of the disease by approximately 55%.
That 55% is not an abstract number. It is Eva Quinn holding on to her speech. It is Paudie being able to ask for a glass of water. It is Emily Felix, a 28-year-old trainee solicitor from Kilkenny who told Claire Byrne this morning:
“Who wants to survive, living in a body that is failing year by year? You wouldn’t treat an animal that way.”
Emily is recording her voice. She knows it may go. She knows a drug exists that could slow that loss — and she cannot access it.
That is what 510 days in the system looks like in human terms.
What Is Actually Happening
The HSE Drugs Group meets on Wednesday. I don’t know — because the system doesn’t tell us — whether Skyclarys is even on the agenda. These meetings happen quarterly. If it is not on the list this week, families are facing another 100 days of waiting. That would bring the total to over 600 days. Two years. For people losing function every single month.
Here is what I do know: the Drugs Group sought further information from Biogen, the company that makes Skyclarys. Biogen submitted that information on 27 May. Whether it has been fully assessed and is ready for a decision on Wednesday—I genuinely do not know. What I know is that families need timelines. They need transparency. They need to be told what they are working towards.
The National Centre for Pharmacoeconomics has recommended against reimbursement on cost-effectiveness grounds. The cost is over €200,000 per patient per year. I understand the challenge that poses. Professor Michael Barry has a budget and a mandate. But I will say this plainly: the cost of residential care for someone with late-stage Friedreich’s Ataxia far exceeds the cost of this drug. Craig Coady can tell you exactly what that looks like—his wife is in residential care at Bloomfield. Early intervention is not just the compassionate choice; it is the economically rational one. It keeps people in work, in their homes, in their communities.
We cannot keep applying a framework designed for mainstream medicines to the only drug that exists for a rare, progressive, life-shortening disease. The standard cost-per-QALY model was never built for this. It has to change.
What Needs to Happen Now
The Programme for Government commits to two things on this: early access programmes and a review of the reimbursement system. Both are in there. Both need to be actioned — not reviewed, not consulted on, actioned.
Here is what I am calling for:
1. Early Access for Skyclarys — now. While the system processes, patients deteriorate. An early access scheme would allow people to access the drug while the formal reimbursement process concludes. It is in the Programme for Government. Let’s use it.
2. Reform the Drugs Group process. Meeting quarterly is not good enough for urgent rare disease cases. The 180-day legal timeline set by the Oireachtas in 2013 must be enforced — not aspired to. Currently, medicines requiring a full HTA are averaging over 900 days.
3. EU-level collective bargaining on orphan drugs. Ireland is a small country. We have one patient. The drug costs €200,000 a year. Professor Barry’s hands are tied by a budget and a population size. The answer is not to abandon our patients — it is to negotiate collectively across the EU for rare disease medicines. With our EU Presidency platform, we should be leading this conversation in Brussels.
4. Legislate for a fit-for-purpose orphan drug assessment framework. The Bill to amend the 2013 Act and create unique criteria for orphan drugs has been tabled and re-tabled. Pass it.
A Personal Note
I feel privileged to be part of a government that has put these commitments in the Programme for Government. I also feel frustrated — genuinely, deeply frustrated — sitting here on a Monday morning not knowing what will happen on Wednesday, while Craig’s son is losing the ability to speak and Emily Felix is recording her voice for the day it’s gone.
When Craig stood up at our parliamentary party meeting and told us what his family has been through — what it costs, what it looks like, what it feels like — it brought it home. Over 600,000 people have watched his clip online. Ireland heard him. Now Ireland’s institutions need to act.
The drug is made in Cork. The families cannot get it. That is a sentence that should not be possible in this country in 2026.
Let’s make it impossible.
Senator Anne Rabbitte is a Fianna Fáil Senator nominated by the Taoiseach. She is a former Minister of State with responsibility for Disabilities and is currently campaigning for early access to Skyclarys for Irish Friedreich’s Ataxia patients. You can contact her at anne.rabbitte@oireachtas.ie
