The Minister of State is most welcome to Seanad Éireann. The first Commencement matter is by an Seanadóir Kyne.

I thank the Cathaoirleach for choosing this Commencement matter this morning. I welcome the Minister of State, Deputy Butler. It is good to have a Minister from the relevant Department, in this case, the Department of Health. I want to talk about a particular case. I met a young girl and her mother on Monday in my office. While she gave me permission to use her full name, I will only use her first name here. Her name is Aoife and she is from Galway. She was diagnosed with Friedreich's ataxia in 2021 during her leaving certificate year. Prior to this, she had been misdiagnosed with apraxia. In October 2020, shortly after she turned 18, a neurologist in Galway took blood tests to investigate Friedreich's ataxia. She said that moment marked the first time she had heard the words "Friedreich's ataxia". From that point on, her life changed completely.

As the Minister of State knows, Friedreich's ataxia is a rare inherited disease. It damages the spinal cord, peripheral nerves and the cerebellum part of the brain. It also leads to heart problems. The disease tends to develop in children in their teens and gets worse over time, with unsteady, awkward moments and a loss of feeling due to nerve injury developing as the disease gets worse. People with the disorder may have other health problems, such as diabetes and heart disease, along with the nervous system symptoms. That is according to Johns Hopkins Medicine. Symptoms can include trouble walking, tiredness, a loss of feeling that starts in the legs and spreads, loss of reflexes, slow or slurred speech, hearing loss, vision loss, chest pain, shortness of breath and heart palpitations. Aoife said that she chose to delay receiving the results of her tests until after her leaving certificate as she wanted to focus on her exams without the added weight of a serious neurological diagnosis, especially during the uncertainty of the Covid pandemic. When the diagnosis was finally confirmed, she said she, her parents and her sisters were thrust into the reality of where her future suddenly seemed frightfully limited. She grieved the life she had envisioned for herself. She is fortunate at this stage that she can still walk, but she lives with severe balance and co-ordination difficulties.

Friedreich's ataxia is relentless and progressive. Every day brings further loss. As the Minister of State knows, a drug called Skyclarys can slow the progression of Friedreich's ataxia. Reimbursement of Skyclarys in Ireland would slow the progression of this disease and give Aoife and others the chance to maintain their strength and independence for as long as possible. She is one of approximately 200 people in Ireland suffering with Friedreich's ataxia. As I said, it is a hereditary genetic condition. Access to this treatment would not only help people like Aoife but also teenagers who are newly diagnosed and who, with timely intervention, may be spared some of the suffering experienced by those before them. It would offer hope not just to patients but to entire families. There was a briefing here recently. The Minister of State's colleague, Deputy Cleere, brought in a group of people, all of whom were in wheelchairs due to progression of the disease and the condition.

What steps are being taken to ensure that the HSE might recognise and respond to the critical nature of Friedreich's ataxia during the current price negotiations with Skyclarys? How are the commitments outlined in the national rare disease strategy, which aims for a 180-day decision timeline, being applied in this case to prevent further delays in addressing this unmet need? Aoife went on to say that Ireland is one of the wealthiest countries in the world but there are issues in terms of access to orphan drugs, with only 20% of rare disease drugs being funded. There has always been talking about using bulk-buying capacity with other countries within the European Union. Is that something that could be looked at? Aoife indicated that if she has to travel and live abroad and move her family to gain access to this drug, that is something that they would have to consider. It should be a right. We should be able to look after people here in our own country.

I thank Senator Kyne for raising this issue today and highlighting Aoife's case in the Seanad. I am taking this Commencement matter on behalf of the Minister for Health, Deputy Carroll MacNeill, and I will make sure she knows from the Senator’s advocacy the impact that Friedreich’s ataxia has on Aoife and her family. I was struck by some of the statements the Senator made about how her life has changed and how every day brings further loss and her future seems frighteningly limited. How brave she was to focus on her exams in 2021 and get them out of the way.

The State acknowledges the importance of access to innovative medicines for patients in Ireland. The Government is committed to providing timely access to new and innovative medicines and has made considerable investments in recent years, with annual expenditure on medicines now exceeding €3 billion. It is actually closer to €4 billion. The level of investment is unprecedented in supporting patients through the availability of new and innovative medicines. Budgets 2021 to 2025 have included dedicated funding for new medicines of €158 million.

This year, we have a record budget for medicines, which has increased by over €200 million, with €30 million ring-fenced to support access to the latest medicines.

The HSE has approved 263 new medicines, or new uses for existing medicines, since 2021, including 107 for treating cancer and 72 for rare diseases. Friedreich's ataxia is a rare, inherited, progressive neurological disorder. It causes progressive ataxia, which is a neurological condition that causes problem with co-ordination, balance and movement, and neuropathy or nerve damage that disrupts normal nerve signalling.

The Government acknowledges the importance of access to medicines and the Minister is particularly aware of the challenging circumstances faced by patients with Friedreich's ataxia. The Minister attended a briefing in Leinster House on Friedreich's ataxia last month. I think that was the briefing to which the Senator referred. It was organised by Deputy Peter 'Chap' Cleere and was led directly by people who had been diagnosed with Friedreich's ataxia and their family members. They spoke with great courage and conviction about their lived experience. I take this chance to acknowledge and thank them, including Aoife, for sharing their perspective with us.

[Omaveloxolone], sold under the brand name Skyclarys, is an orphan medicine used to treat Friedreich's ataxia in adults and adolescents aged 16 years and over. The HSE received a pricing and reimbursement application for the drug in August 2024 for the treatment of Friedrich's ataxia in adults and adolescents aged 16 years and older. The HSE commissioned a health technology assessment in September 2024 and this was submitted by the applicant company to the National Centre for Pharmacoeconomics, NCPE, in July 2025, nine months after it was commissioned. The NCPE concluded its assessment and provided a recommendation to the HSE in December 2025. The HSE corporate pharmaceutical unit, CPU, is the interface between the HSE and the pharmaceutical industry in relation to medicine pricing and reimbursement applications. The CPU recently met with the company to discuss this application and is awaiting a commercial proposal. As the House will appreciate, this is now subject to commercial engagements and I am not in a position to say anything further on this part of the process. Once the commercial proposal is received and engagements conclude, the proposal and the NCPE reports, along with patient group submissions received, will go to the HSE drugs group. The HSE drugs group considers all of the evidence and makes a recommendation to the HSE senior leadership team, which holds final decision-making authority. The HSE has advised that the application remains under consideration and cannot make any comment on possible outcomes from the ongoing process at the moment.

I thank the Minister of State for the clarity in relation to the process of the NCPE and the CPU of the HSE. I appreciate that these are commercially sensitive issues. Aoife has acknowledged that Ireland holds the EU Presidency from 1 July. There may be opportunities for collaboration with other European countries in respect of, as I said, purchasing power or bulk buying, if you like, or co-operation with certain countries, such as the Benelux countries, as was discussed in the past. It is a valid suggestion by her. It is progressive thinking to note the possibilities that come with the EU Presidency. I certainly hope that, notwithstanding whatever the price may be, we, as a State, recognise the possible benefits of slowing the progression of a condition such as Friedreich's ataxia for people such as Aoife and others.

I thank the Senator. I agree wholeheartedly with every word he has said. In the context of rare diseases, we do have a real and active focus on improving how we support people living with these diseases. Since the launch of the National Rare Disease Strategy 2025-2030, an implementation oversight group has been launched. It met in February to begin work planning for 11 recommendations. As implementation of the strategy progresses, it is intended that patient representation, which is important, will be a key feature and will be embedded in all structures. Two new framework agreements have also been reached with the Irish Pharmaceutical Healthcare Association and Medicines for Ireland on the pricing and supply of medicines, which the Senator spoke about earlier. The agreements provide a commitment and a structured process towards achieving a 180-day timeline for completing health technology assessments and reimbursement decisions, accelerating patient access to new treatments. The Minister, Deputy Carroll MacNeill, with whom I work closely, is very keen on having a dedicated timeline that we can manage. This has gone on, as can be seen from the answer I read out, for almost two years. Aoife and others do not have that length of time. As the Senator said, every day is a change.

In addition, the State and the pharmaceutical sector have agreed to develop a further strategic partnership on the development of a sandboxed early access programme for rare diseases proof of concept, having regard to the commitments within the programme for Government. I acknowledge Senator Teresa Costello, who is raising issues about another orphan drug. I am glad to see the Minister's focus on this issue. She has a dedicated focus. I will bring everything the Senator has said back to our team.