Protest in Dublin over Skyclarys reimbursment

Protest in Dublin over access to life-changing drug

Demonstrators gathered in Dublin city centre on Sunday in a final push for the Health Service Executive (HSE) to approve reimbursement for Skyclarys, a drug used to treat Friedreich's ataxia, a rare progressive neuromuscular condition that causes nerve damage, muscle weakness and mobility loss. The protest, at the Garden of Remembrance, included patients, families, and their supporters, who marched towards Custom House Quay.

Friedreich's ataxia affects around 200 people in Ireland, according to multiple reports. The condition often begins in childhood and leads to severe movement and coordination issues, and in some cases it can confine people to wheelchairs. While there is no cure, Skyclarys is regarded as a potentially life-changing treatment because it can slow disease progression by up to 50% or even 55%.

The HSE Drugs Group has recommended that the drug should not be covered by the HSE. In a statement to RTÉ, the HSE said the Drugs Group had considered an assessment from the National Centre for Pharmacoeconomics (NCPE) and found that while there is some evidence that the drug may slow disease progression, there remain limitations and uncertainties about its clinical efficacy and its price is substantially above a cost-effective level. The NCPE's assessment last December recommended against reimbursement, stating the drug would cost about €280,000 per patient per year.

Personal stories of urgency

Saturday's protest was personal for many of those involved. Isabella Travers, a 19-year-old from Co Wicklow, was diagnosed at age 12 and became a full-time wheelchair user at 16. She said she is 'scared' about the future and that getting Skyclarys is so important to preserve the abilities she has today for as long as she can, as quoted in The Irish Times.

Isabella's father, Aaron Travers, argues that the €280,000 figure is the published list price and does not necessarily reflect the price the HSE would eventually pay after commercial negotiations. He also called for transparency and accountability in the rare disease drug approval process.

Kiara Lynch, another patient, spoke about losing abilities, saying 'we haven't got time,' while Theresa Kane said that access to the drug 'would stop everything else from progressing.'

Sinéad Maher from Waterford, whose husband Jason has had Friedreich's ataxia for 24 years, told Newstalk she still has hope: 'I suppose once and once only the HSE senior management team has gone against the advice report back in 2019, so a precedent has been set. They have done it before; there's nothing stopping them from doing it again. They have the chance to make the right decision.' She added, 'I don't want to see after Tuesday, we will do x, y, z if this doesn't go our way, because I'm hoping that on Tuesday and Wednesday we're celebrating.'

Jessie Abbey, who was at the protest, shared a plea: 'I don't want to get any worse, I don't want my voice to go, I don't want my hands, my muscles, everything to go. I want to be able to bring my little girl to school. I want to live for my nieces and nephews and my little girl.'

The drug and regulatory history

Skyclarys, the brand name for omaveloxolone, is the first drug specifically designed to treat Friedreich's ataxia. It has been approved in the US since February 2023 and in Europe since 2024. The European Medicines Agency first approved the drug in 2024, and reimbursement evaluation in Ireland began over two years ago. Although approved in a number of European countries, it remains unavailable in Ireland.

The HSE's decision process has been lengthy. The National Centre for Pharmaco-economics issued an assessment in December that recommended against reimbursement. In July, the HSE Drugs Group did not recommend the drug and referred it to the Rare Diseases Technology Review Committee for further input, and in August it recommended against reimbursement.

Dr Suzanne Crowe, speaking on Newstalk's The Hard Shoulder, called the decision a stark reminder of the 'devastating human cost' that can lie behind bureaucratic decisions. She described the process as a 'black box' for families and questioned whether we are doing it the right way.

Campaigners estimate that funding the drug for all eligible patients would cost around €280,000 per patient per year, with a five-year budget impact of around €130 million, as reported by RTÉ. This price remains a sticking point despite debates about the list price.

Political support and final decision pending

The HSE senior management team is expected to make its final decision on Tuesday. In the lead-up to the decision, RTÉ reports there is widespread support across Leinster House, with several opposition parties and some members of Fianna Fáil and Fine Gael calling for approval. However, the HSE's decision remains independent of these political calls.

Some have questioned why a drug approved by the European Medicines Agency in the NAO's assessment course should not be available here, given its ability to significantly slow the progression of a devastating condition. The protest was described as a 'solidarity' event with patients, emphasizing the urgency felt by the community.

Campaigners and families say they will continue to push for access whatever the outcome on Tuesday, but their priority is clear: they want to see the right decision now, as the drug could preserve futures for those living in limbo.