Teresa Costello (Fianna Fail)

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.