Waiting for HSE decision on Skyclarys has been awful — but now I can celebrate

Niamh Ní Hoireabhaird has Friedreich’s ataxia, and has spent years researching the rare disease and campaigning to have the Skyclarys drug reimbursed

Niamh Ní Hoireabhaird: Ireland’s system for reimbursing medicines for rare diseases is not fit for purpose.

When the news came in just after 12pm, I froze with shock before texting a simple “omg” into the groupchat.

After months of uncertainty, the HSE decided on reimbursing Skyclarys. 

It is the first and only approved treatment for my rare disease, Friedreich’s ataxia (FA), a progressive neuromuscular condition that causes nerve damage, muscle weakness, and mobility loss.

Funnily enough, Tuesday is also my 30th birthday.

Instead of making any special plans to go out and celebrate, I spent it waiting anxiously for any word on the decision, fearing the worst.

To say I was surprised is truly an understatement — I’ve spent months preparing myself for bad news.

When I was in college, I became fixated on FA research and the advancements doctors were making in the area. I obsessively followed the development of treatments in the US, and I dreamed of the day that they would become publicly available.

I first read about Skyclarys back then, and I’ve followed its progress from clinical trials to US market authorisation in 2023.

I rejoiced, comfortable in the knowledge that a treatment to slow the progression of FA was on its way. However, I grew more anxious in the days after this.

I was familiar with Ireland’s bad track record with reimbursing drugs for rare diseases, and I knew Ireland’s FA community had a fight on their hands.

I waited anxiously for Skyclarys to make its way to Ireland.

After being approved by the European Medicines Agency in February 2024, Biogen filed in August 2024 — over 750 days ago, at this point.

Now, just a few years later, I am no longer able to live independently. I need help with small things like getting in and out of bed, making a cup of tea, tying my hair up.

With access to Skyclarys, I now have hope that any future progression won’t be quite as dramatic.

Waiting for a decision these past few months has been awful. The constant debates about Ireland paying for Skyclarys have made my mental health spiral. Just last week, I started taking mood stabilisers again after years without needing them.

As I followed these conversations, I could simultaneously hear stories about FA patients in other countries with access to Skyclarys, who were all experiencing huge benefits from the drug.

One girl that I follow in the US reports experiencing less fatigue, more dexterity, and she has audibly clearer speech. I watched her with envy and hope.

Although it’s a celebratory time for other patients and me, I want to take a minute to look at the bigger picture.

Ireland’s system for reimbursing medicines for rare diseases is not fit for purpose.

We’ve seen campaigns like the one for Skyclarys happen over and over again: In the 2010s for Pembro, a drug used to treat rare forms of cancer, and in 2017 for Orkambi, to treat cystic fibrosis.

The system is not protecting some of Ireland’s most vulnerable people.

And the Government’s reasoning is always the same. It always comes down to money.

And then they’ll announce tens of millions for greyhound and horse racing, bike sheds, and printers. This is always money for something, just not for the people who need it. Their priorities are ridiculous.

I dread to think which rare disease group will have to take to the streets to demonstrate for healthcare.

This cannot happen again.

Campaigning and waiting for a decision these past few months has been exhausting, but I’m just incredibly relieved that a positive result came out of it all. I don’t know what I would have done with myself if the decision was negative.

Emily Felix, Emma O’Shea, and Niamh Ní Hoireabhaird join the campaign to protest in Dublin to push for Skyclarys approval in Ireland.

This past Sunday, hundreds gathered at the Garden of Remembrance in Dublin to make a final plea to the HSE to reimburse Skyclarys. It was the most hope I’ve felt throughout these past few months.

I was genuinely shocked to see the turnout. I’ll always be so thankful for the hundreds of people who marched with us.

Yesterday, I celebrated. Both my 30th birthday and the prospect of future birthdays with reduced FA progression.