Mark Daly (Fianna Fail)
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I welcome the Minister of State, Deputy Grealish, to the House. He is most welcome.

Teresa Costello (Fianna Fail)
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To refresh memories, Duchenne muscular dystrophy, DMD, is a progressive neuromuscular disease that causes the weakening of muscles over time. It almost exclusively affects boys, with symptoms typically appearing between the ages of one and three. In Ireland today, around 110 people are known to be living with DMD and most of them are under 18. A child living with DMD struggles to walk, run or jump like their peers. They have difficulty standing up and need help to climb stairs. These children can also suffer with delayed speech and sometimes face behavioural or learning challenges. As the disease progresses, most of these children will need a wheelchair by the time they are 12. Some will develop scoliosis, and by their mid-teens they may experience heart complications such as dilated cardiomyopathy. Breathing problems often emerge by their late teens or early 20s, making the condition life-threatening.

There is hope, though. A life-changing drug called Givinostat can slow the progression of the disease and keep boys on their feet for longer. Even if a boy is wheelchair-bound, Givinostat can slow the progression of the disease to the organs, providing a better quality of life. This drug is a symbol of hope for these families affected by DMD. I acknowledge the work of the Minister for Health so far on this matter. She has met with families to discuss a pathway to access Givinostat, along Deputy Pádraig O'Sullivan and me. The Minister has also engaged with the HSE and everybody involved to get this medicine to patients in Ireland as quickly as possible.

I held a briefing in Leinster House in June to educate TDs and Senators alike about this illness. At the same time, hundreds of people gathered outside the gates here to raise awareness and show support. Anyone there that day saw the impact this disease has not only on the children living with it but also on their families. That was 105 days ago. While that might not seem that long to me and you, to the families affected by DMD, time is muscle. Their children's abilities and strength are failing every day. Since then, a rapid review was completed on 15 August. It was completed in eight days. This timeline shows there is an ability to move through the reimbursement process at an accelerated rate. The manufacturer tells me it has a scoping meeting with the National Centre for Pharmacoeconomics, NCPE, on 30 September and it will submit a health technology assessment, HTA, in October. The company has not confirmed an exact date, but I was under the impression the NCPE was ready to receive this submission now.

Can I get clarity on this issue so the parents waiting will have a clear picture of what is going on? There is still a long way to go before Givinostat will be available in Ireland, which is why I am seeking early access. Our EU counterpart, Belgium, is providing early access to Givinostat through its compassionate use programme, CUP. This allows eligible boys to receive the drug before a national reimbursement. The CUP is overseen by Belgium's Federal Agency for Medicines and Health Products, FAMHP, ensuring safety while bridging the gap between clinical trials and full market access. There is also talk of approval of an early access programme being signed off on up North. There have been suggestions of getting the drug into Ireland that way. Can the Minister of State clarify if this is a realistic option? We have parents with children who are so sick. They are failing and I do not want unrealistic options being put forward. We cannot follow regular timelines because DMD is not abiding by these timelines. This disease chips away at these boys every day as their families watch while feeling helpless. I cannot express how important it is that we develop a bespoke pathway to early access to Givinostat.

Noel Grealish (Galway West, Independent)
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I thank the Senator for raising this issue. I am answering on behalf of the Minister for Health.

Duchenne muscular dystrophy is a rare disease affecting approximately 200 people in Ireland. Its symptoms can severely impact the lives of those living with the disease and their families. The State acknowledges the importance of access to medicines and is aware of the circumstances of patients with DMD. As the Senator will be aware, the Minister met with the families of children with DMD and their representative body, Muscular Dystrophy Ireland, in July to discuss their plight and provide support. The State has made considerable investments in new medicines in recent years. In 2023, nearly €1 in every €8 of public funding being spent on health was spent on medicines. This level of investment is unprecedented in supporting patients through the availability of new and innovative medicines. Budgets 2021 to 2024 have included dedicated funding for new medicines of €128 million. Budget 2025 allocated €30 million of the funding available for new drugs to be generated by efficiencies to be identified by the HSE. From 2021 up to June 2025, this has enabled the HSE to approve reimbursement for 219 new medicines or new uses for existing medicines, including 91 for the treatment of cancer and 55 for the treatment of rare diseases.

Under the Health (Pricing and Supply of Medical Goods) Act 2013, the HSE has statutory responsibility for decisions on pricing and reimbursement of medicines.The Act provides a rigorous process for the assessment of new medicines for reimbursement. This ensures the right medicines are available and approval is at a sustainable price.

Givinostat, sold under the brand name Duvyzat, is a medication used to treat Duchenne muscular dystrophy, DMD, in ambulant patients aged six years and older. Conditional marketing authorisation was granted for givinostat on 6 June 2025 by the European Commission.

The Minister for Health met with her Italian counterpart at the EPSCO Council meeting in June in Luxembourg to ask him to encourage the company to submit a pricing and reimbursement application to Ireland. The HSE has also proactively engaged with the company to strongly encourage it to submit a pricing and reimbursement application.

On 6 August 2025 a pricing and reimbursement application from the marketing authorisation holder for givinostat was made to the HSE with an indication for the treatment of DMD in ambulant patients aged six years and older. Following the conclusion of a rapid review by the NCPE on 14 August 2025, a full health technology assessment, HTA, was commissioned by the HSE on 26 August 2025. The pricing and reimbursement assessment is ongoing. A HTA is a detailed evaluation of medical technologies, including medicines, to determine their effectiveness, safety, cost-efficiency and broader impact on the health budget and service. A HTA takes time to evaluate as it involves gathering and analysing complex clinical and economic data, consulting stakeholders and aligning with regulatory standards to ensure decisions are evidence-based and transparent.

To support this process, there has been significant investment to enhance the HSE's pricing and reimbursement process for medicines. Thirty-four new staff were hired across the pricing and reimbursement system in 2024 which will support faster access to new and innovative medicines for patients. In addition, to support transparency in this process, an application tracker has also been developed and launched.

Teresa Costello (Fianna Fail)
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I thank the Minister of State for his answer. With regard to the questions that I asked, is the pathway through the North a realistic option? It has been flown, so to speak, as a bespoke pathway. At present, there is a delay somewhere because the NCPE is waiting for a HTA to be submitted while the manufacturer is telling me it has a meeting next week. These are children whose parents sat in the room with the Minister. Those children will not qualify for this drug if this takes the timelines it normally takes. Even with the best will in the world of it moving fast, we are sitting on our laurels for a month here. That is one month gone. That is children failing.

Where is the delay coming from? Why has this HTA not been submitted and why is work not going on? I am reading about extra staff being allocated. I would understand if it takes overtime to get this moved as quickly as possible. To be honest, we do not have until Christmas. There are little children whom I have met and seen and I will not be able to look their parents in the eye if I have been working on this and those children do not qualify for that medication. It needs to be done quickly.

Noel Grealish (Galway West, Independent)
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I thank Senator Costello for raising the issue. I will bring to the attention of the Minister what the Senator has raised. I am not aware of what the hold-up is regarding going through Northern Ireland and what the delay is.

Going on what the Senator is saying, she is very passionate about this and is anxious to get this over the line. Genuinely, I do not have the answers for her; I have the script here from the Department. What I will do is bring it back to the Minister and ask that she respond directly to the Senator as to why we cannot go through Northern Ireland or what the hold-up is and the delay on getting this through. I hear what the Senator says. It is much needed, badly needed and seriously needed for young people for their improvement in life. I will bring that to the attention of the Minister. I thank the Senator.