2026-06-17

I acknowledge that the Minister is here in person. It does not always happen for Topical Issues but I am grateful for the fact that she is here. It is probably a reflection of the fact that she is getting the same emails I am getting from people in our constituency who are affected by Duchenne muscular dystrophy. I am sure the Minister already knows about it but Duchenne muscular dystrophy is a condition that affects muscular restoration and muscular development, mostly in boys although not exclusively. It occurs from about the age of four. It is a degenerative condition. Over time, it restricts the ability of those boys to do things that all our children take for granted, simple things, whether that is getting up after a fall, getting up out of a chair, getting in and out of a car or climbing stairs. Over time, that gets worse and worse until eventually they cannot stand independently or cannot stand up at all. Unfortunately, the nature of this condition is that as it gets worse and worse, boys who suffer from it will often not be able to walk by the age of 12. This is, therefore, a heartbreaking condition for parents as they watch their young children unable to do the things their peers are able to do. That degenerative nature of the disease must be extraordinarily difficult for them. It is a rare disease. I know there are different assessments as to how common it is. Ultimately, it does not affect a huge number of people but those people whom it does affect are deeply affected by it, not only the families and the extended families but also, obviously, the people who suffer from it themselves. The Minister is aware that families were in here last week, a year after they first brought this to our attention, to have a meeting to inform many of the Members of this House about the situation in relation to Duchenne muscular dystrophy and the treatments that are available. Many Members will have attended that briefing in the audiovisual room. The families referred specifically to givinostat, a drug that is available for the treatment of the condition. Givinostat is really important because it is a turning point in the treatment of Duchenne muscular dystrophy. It cancels out some of the enzymes that prevent muscular development and muscular restoration. The muscles of young boys who suffer from Duchenne's are generally wasting away; they cannot restore themselves. Now there is a drug that is available and has been approved, for example, in the United States and provisionally in the European Union and that can treat that specific cause and deal with the enzyme that prevents the muscles from regenerating and being restored. It acts as a pause or a stop on the progress of the disease. There are a couple of difficulties with it, however. The first is that it is not something that can be taken at any stage in a sufferer with Duchenne's. There are boys who are no longer eligible to take givinostat because of the delays in getting the drug approved in this jurisdiction. There are other boys who are approaching the point at which they will not be able to take it. Time is therefore of the essence in this matter. As one parent who wrote to me said, time is muscle when it comes to Duchenne's. Again, I am sure the Minister is familiar with these issues. Those parents who have children who suffer from Duchenne's are therefore incredibly anxious that this drug will progress. I know that was a meeting - last week, I think - of the senior management team or that this is progressing through the Minister's Department. What I would really like her to tell us, though, is the progress in relation to givinostat; whether the families can expect that this drug will be available not only to them but on the same terms that it is available in other parts of the island - for example, in Northern Ireland; and if she can tell parents, conscious that time is muscle and conscious that there are kids who need this drug urgently, if this will progress in such a way that it will be available to them in the short term, or when it will be available to them. This heartbreaking disease can be stopped in its tracks for some patients, and we should be doing everything we can to ensure that happens.

I thank Deputy Ward for raising this issue. This is something I have been working on with families for over a year now, since this drug was approved by the European Medicines Agency. I have met many of those families. I met them when they were here last week, before they had their audiovisual room briefing, I have met them four, five or six times online and I met them past year in person - I have met them so many different times. Together, we have been working to try to get this drug approved within the Irish system. As the Deputy will be aware, when a drug is approved at a European Medicines Agency level, it does not necessarily mean that it will be available in Ireland. Why? It is because it is necessary for the drugs company to make an application to have the drug available in Ireland. It is not as though Ireland simply gets it as a matter of course. The HSE needs an application to be made. The drug company has to choose to want to sell it here. We have been trying to encourage Italco Pharma for some time to make the appropriate application. The drug was approved in June of last year. I immediately met families in relation to the next steps. We did not get an application from the maker of givinostat for some time, so we all engaged in a lobbying campaign in different ways. I met the Italian health minister, Mr. Schillaci, in Luxembourg to encourage him to ask the company to make an application in Ireland. I made the same ask of him again at a later stage, in November, in South Africa, at the G20 meeting. The Taoiseach has been involved in this, asking Prime Minister Meloni to get involved to ask the Italian pharmaceutical company to make the appropriate, complete application here in order that the HSE could respond. I was on my summer holidays in August of last year waiting to see every day with the HSE whether the application had been made. Eventually, on 6 August, the HTA application was first made. I thank Michael Barry and the people in the HSE for the speed with which they processed that part of the application, which was exceptionally quick. The National Centre for Pharmacoeconomics did a rapid review. The application was made on 6 August, the rapid review was completed on 14 August and a full HTA commissioned on 26 August. That is unprecedentedly fast, and I am very grateful to the HSE for its speed, reflecting the seriousness of this. On 30 September the HSE met with Italco Pharma to assess the HTA process. The assessment of the pricing and reimbursement application could not proceed until the full HTA was received from the Italian company. That was eventually received on 15 January 2026. We were updating the families at every stage, discussing and giving them full information and transparency as to what was happening. I am grateful to the HSE for concluding the assessment on 3 March, which was a matter of 26 days or so. That was very fast. The HSE's corporate pharmaceutical element, which is the interface between the HSE and the pharmaceutical industry, held a meeting with Italfarmaco on 27 March 2026. A commercial offer was received from Italfarmaco on 8 May 2026. Again, we were waiting for these applications to be made. The HSE drugs group continued that application at a meeting one month later on 9 June. When we bring together all of the different experts, it is not something that is stood up quickly. We have to review the documentation and have the scientific experts there. The drugs group supported the reimbursement of givinostat. Yesterday, the senior leadership team in the HSE supported that application and the drug will now proceed through a managed access protocol. We have discussed in detail with the families how a managed access protocol works. It is a matter of clinical decision making as to who gets the drug. Of course, as the Deputy has referenced, not every boy is suitable for it. The question of being ambulatory, being able to stand and all of those things are not decisions for the Deputy and I but are decisions for clinicians. We want to make that drug as broadly available as possible but, ultimately, it is a clinical decision as to who should get the drug and who should not. I am so thankful to the HSE and thankful to the families for their constant engagement with me in relation to this, not just in our constituency, but around the country.

I thank the Minister. I join with her in thanking the HSE for the work that has been done and also acknowledge her commitment to this. Anybody who has dealt with families who are suffering with this disease understands both the heartbreaking nature of it and the urgency involved. Listening to what the Minister has said in relation to the progress and the managed access protocol, do we have a timeline for when it will be available? I accept entirely what the Minister has said about the clinical criteria for the application of the drug. That is entirely appropriate and, unfortunately, there will be boys, because of the delay in the application outside our control, who may not be eligible to receive the drug now. For those who are still eligible and those who can still stand up independently or who can stand up, can we give them a timeline for when they might be assessed for that and the conditions under which it will be made available to them? I am conscious of the fact that muscle is time and the fact that this drug is the very best way available to sufferers of Duchenne muscular dystrophy to stop the progress of this disease and to stop the degeneration of their muscles, which is irreversible. For the families the Minister has been dealing with and I have been dealing with, can we give them a particular timeline for when they can hope to be clinically assessed, if that still has to happen, but more importantly to have access to this drug in the same way it is available, for example, to our cousins in the North of Ireland under the National Institute for Health and Care Excellence, NICE, protocols? Will the drug be available under the same conditions as it is available in the UK under the NHS?

It was Úna Ennis who coined the phrase "time is muscle". I am grateful to her for her engagement and advocacy all the way through. The first thing is that we are trying to get the drug physically into the country. My officials have been working on that for some time in anticipation of what we hoped would be a positive response. Of course, it is not just a decision whether to give the drug or not. It is also a decision about whether the drug is effective and for whom it is effective. It is a complicated balance and we must always defer to the scientists on these decisions as well, recognising that Ireland remains a country that believes in science. We are trying to get the drug physically into the country. This is a high-tech drug and not one that is put on the back of a van and sent to a pharmacy. Due to the way in which it is given, we are trying to work out whether this is something we can do more quickly in acute settings and how we can most quickly get this drug to the boys who need it so badly. Our broad approach is one of trying to give maximum access to it but I will defer to clinicians on who the appropriate people are to get it. Yesterday, I was meeting with the European health ministers. We had a working lunch specifically on medicines and the challenge of most favoured nation, MFN, access to medicines. We have been trying to work in different ways on two things. As a small country Ireland, like the Benelux countries, Croatia and other small countries, have a difficulty with access to drugs. If we look at drug companies making applications, they are making it to the likes of France, Germany and other huge markets. We are just not in the same space. We do not have the same visibility. We are trying to partner with other countries to create a bigger market so we can be more attractive and also so we can negotiate on more attractive terms. I feel that the only group with all of the information are the drug companies and that the member states are working somewhat against each other in terms of each of them making individual negotiations because of the way the European treaty is structured and health being a national competence. It is something we need to reflect on. I am working with other European health ministers to find a way to streamline the way the HTA process works, reduce some of the national country administrative burden, make Europe a more powerful block, and rebalance that balance of power, crucially so that patients get access and so the Irish State can spend as much money as possible in getting as many drugs as possible for the broadest range of people. That will not always result in positive decisions by the HSE. I need to say that loudly. We have had difficult days on that, too, but we are desperately trying to take the right approach in every way.