Monaghan woman's fight for life-enhancing drug

A young Monaghan woman with a rare degenerative disease may be forced to leave Ireland for treatment. Aoife Gavin (21) from Castleshane was diagnosed with Friedreich’s Ataxia (FA) age 13 years in 2017: “The worst thing about being diagnosed was being told there was no hope.”

Friedreich’s Ataxia is a rare inherited degenerative neurological condition affecting mobility, co-ordination, speech, vision, hearing and heart health. Around 200 people in Ireland have been diagnosed with the condition. Aoife says her family was told at diagnosis there was “no cure” and she would progressively lose abilities. She currently uses a walker to aid her mobility.

“It’s not as simple as going into a wheelchair. There’s so much in between. I felt like a tennis ball because I was going back and forth between neurology and orthopaedics.”

The young woman is preparing to begin a Master’s degree in Behavioural Analysis soon, having completed an undergraduate degree in English and Psychology.

Skyclarys (omaveloxolone) is the first and only approved drug for the treatment of Friedreich’s Ataxia in adults and adolescents aged 16 years and older. The oral treatment slows the progression of the debilitating disease. Aoife explained why she wants to take the drug: “It’s not a cure, but it gives us time, which is the most important thing. It doesn’t reverse the deterioration, but it slows it down. You’re not just surviving every day.”

However, Skyclarys is not yet available in Ireland: “People all over the world are getting this drug and seeing the benefits from it and we just have to sit on the sidelines and watch. We’re one of the only European countries without access to Skyclarys. It’s just very unfair.”

A key meeting relating to the drug is due to take place next week, July 14: “Basically, Skyclarys was approved by the European Medicines Agency in 2023. The HSE Drugs Group are holding a meeting on July 14. They did have one last month, but Skyclarys, for some reason, wasn’t included in the meeting.”

As a result, Aoife fears that she may be forced to seek treatment for her condition abroad: “If they turn around and say no in two weeks, I will really have to consider moving country. My whole life is here, but realistically, I would have to for my own health,” Aoife explained, adding that the HSE appeared to be “extremely resistant” to funding the drug. She feels there is a serious issue in Ireland with getting approval for medications.

The matter came up at a recent Monaghan Municipal District meeting; it was raised by Cllr Pauric Clerkin (FG). Aoife was grateful to him for shining a light on the issue, adding that she had contacted Deputy David Maxwell’s constituency office in Monaghan Town.

“You know, I think when it comes down to it, it’s not up to the HSE, it’s up to the NCPE,” Aoife commented. The National Centre for Pharmacoeconomics (NCPE) reviews the cost-effectiveness and budget impact of individual drugs in the Irish healthcare setting in response to requests from the HSE Corporate Pharmaceutical Unit (HSE-CPU).

“I don’t really have much faith (that the drug will be approved) after hearing conversations on the radio on how it’s not cost effective, even though it improves life expectancy and daily living,” she said, adding that Skyclarys is relatively new.

Friedreich’s Ataxia has resulted in Aoife having a heart condition, for which she has had to visit A&E three times in the last six months.

Access to Skyclarys would improve her quality of life immeasurably and would have a positive impact on her physical and emotional wellbeing: “I don’t even mind the condition itself. It’s having to fight for the treatment for it, that’s the bit that really gets me. Every day you’re thinking about it. It really affected the end of my college course.”

Speaking on behalf of every person living in Ireland who has Friedreich’s Ataxia (FA) and needs access to Skyclarys, Aoife had this message for decision makers: “Think about yourself in our shoes. You look at the figures, but we’re the people living the real-world situations and the real-world struggles. Skyclarys would maybe not stop the symptoms, but it would put a pause on them and I think that would be so relieving, even mentally.”

Asked about the issue by the Celt, the National Centre for Pharmacoeconomics confirmed that omaveloxolone (Skyclarys) will be considered by the HSE Drugs Group in the near future.

A spokesperson added: “A reimbursement recommendation will be made. This recommendation will then be considered by the HSE Leadership team who will make the final decision as to whether the drug (Skyclarys) will be reimbursed by the HSE.”

The Health Service Executive has confirmed it received an application for pricing and reimbursement of omaveloxolone (Skyclarys) on August 1, 2024, from Biogen Idec (Ireland) Limited for the treatment of Friedreich’s ataxia in those aged 16 years and older.

Following an initial assessment, the HSE commissioned and received a full Health Technology Assessment (HTA) to assess the clinical effectiveness and cost-effectiveness of omaveloxolone compared with the current standard of care. This was received in December 2025.

Following negotiations with the applicant, Biogen (Idec) Ireland Limited made a commercial proposal to the HSE in May 2026. A spokesperson said: “The next steps in the process involve the July 2026 meeting of the HSE’s Drugs Group, the national expert committee, which includes public interest members.”

Considering all evidence and factors, the Drugs Group will then “issue a recommendation to the HSE Senior Leadership Team, which holds final decision-making authority on the reimbursement of a medicine”.

The spokesperson added: “This application remains under consideration with the HSE. The HSE cannot comment on possible outcomes from the ongoing process.”