2026-05-13

Gabhaim buíochas leis an Teachta as a chuid ama a roinnt liom. Tá dhá nóiméad agam. I raise the urgent access to the drug givinostat for children-----

Dhá nóiméad.

Yes. I want to raise the urgent issue of access to the drug givinostat for children living with Duchenne muscular dystrophy, or DMD, as it is commonly known. Givinostat was approved by the European Medicines Agency last June. Nearly a year has passed, the HSE is still reviewing whether it is going to cover the cost of this drug for children and adults across the State. What makes the delays around givinostat's availability particularly distressing - and I am sure the Minister of State knows this - is that it is only available to children who still have mobility. These children are in a race against time, and their parents are looking on as the HSE and the Government drags their feet while their children are seriously at risk of not being able to have the life-changing support offered by this drug. Matters are particularly urgent for young Aaron Langan. Aaron is a nine-year-old from Laghey in my county, Donegal. His family are fearful that if Aaron's mobility is further impacted by this condition that he will not be able to have this life-changing drug, even if the HSE approves its reimbursement. Aaron's mother and father were here in the Dáil last year campaigning and pleading with Government to get its act together to allow them access to this lifesaving drug. At that time, Aaron was walking. Today, he is in a wheelchair. As a result, the issue of mobility is absolutely crucial. This drug is not just a life game-changer for Aaron. Young Diarmuid O'Sullivan from Donegal is another example. Diarmuid is ten years of age. His older brother Nathan was on a trial in Britain. Nathan is now an adult who is able to avail of this drug and have a fulfilled life, yet Diarmuid, who is ten, is not able to have it. Imagine the distress of a parent knowing that one child has the drug that changes your life and the other child is denied it because the HSE and the Government have not got their act together. I ask the Government for a compassionate intervention to allow these drugs be made available to children who are in a race against time.

I am pleased to have the opportunity to raise this important issue. My colleague Deputy Doherty has outlined how it is affecting two families in Donegal. The European Medicines Agency has approved this drug for DMD last year. Now, together with, I am sure, all colleagues in the House, I am anxious that the Department would exert pressure on the HSE to make this drug available to these children. An example of the success of this drug is that Nathan O'Sullivan, who Deputy Doherty mentioned, has been on this drug for a number of years. His mother took him to the UK. He took part in the trial relating to the drug. The trial was extremely successful, and his life has been prolonged. He leads a full life. He works with the local GAA club as a statistician and has his own car. We would like to think that his brother Diarmuid and the Langan family would have the opportunity of obtaining this drug as well. We know the cost of it can seem rather high but we cannot put a cost on the life of a child. They are suffering and the families are suffering. The mental torture for families with this condition is 24 hours a day, seven days a week, 365 days a year as they care for their children. There are about 100 or so in the country. It is incumbent on the Minister, the HSE and the Department to ensure that this drug is made available as soon as possible. There is a question of being ambulatory. They have to be able to move when they start the drug so the clock is ticking. It is vitally important that a positive decision is taken as soon as possible and that the drugs are made available. I would also say to the drug company involved, which is making exorbitant profits, that it should reduce the prices and work with the Department and the HSE to make the drug available sooner rather than later.

I thank Deputies Gallagher and Doherty for raising this matter. I will be answering on behalf of the Minister for Health, Deputy Jennifer Carroll MacNeill. As stated, Duchenne muscular dystrophy is a rare and progressive disease affecting the muscle strength and function of boys almost exclusively, with approximately ten children diagnosed with the condition each year. The Minister, Deputy Carroll MacNeill, has engaged with representative groups and the parents of children suffering from this condition several times over the past year. The Minister has followed progress closely and will be meeting with patient groups again this week. 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 approaching €4 billion. To move completely to givinostat, it is sold under the brand name Duzyvat, for the treatment of DMD in ambulant patients aged six years or older. It has recently undergone assessment by the European Medicines Agency, EMA. The European Commission granted conditional marketing authorisation for use of givinostat in this indication on 6 June 2025. The Health Service Executive has statutory responsibility for decisions on pricing and reimbursement of medicines, in accordance with the Health (Pricing and Supply of Medical Goods) Act 2013. In line with the Act, a company must submit an application to the HSE to have a new medicine added to the formal reimbursement list. Reimbursement is for licensed indications that have been granted marketing authorisation by the EMA or Ireland’s Health Products Regulatory Authority. This is the importance piece. The Minister met with her Italian counterpart at the Employment, Social Policy, Health and Consumer Affairs Council, EPSCO, meeting in June 2025 in Luxembourg to ask him to encourage the company, Italfarmaco, to submit a timely pricing and reimbursement application to Ireland. On 6 August 2025, an application for reimbursement was received by the HSE and underwent a rapid review by the National Centre for Pharmacoeconomics. Following the conclusion of a rapid review on 14 August 2025, a full health technology assessment, HTA, was commissioned by the HSE on 26 August. The assessment of the pricing and reimbursement application for givinostat could not proceed until the NCPE received the HTA from the company almost four months later, on the 15 January 2026. I need to set out the timelines clearly. The HTA has been completed and confidential commercial negotiations between the HSE’s corporate pharmaceutical unit and Italfarmaco have begun, with the receipt of a commercial offer from Italfarmaco on Friday, 8 May, which was last Friday. The HSE continues to engage with the applicant to progress this application in a timely manner. To be clear, the conclusion of the rapid of review was on 14 August. A full health technology assessment was commissioned by the HSE. The assessment of the pricing and reimbursement application for givinostat could not proceed until the NCPE received a full HTA from the company. That arrived on 15 January this year. The HTA has been completed now and confidential commercial negotiations are under way. A commercial offer was received from the company last Friday. I thank the Deputy for sharing the stories of the two families who will want to understand exactly where the hold-ups are.

With respect, that does not cut it for these families. These parents are looking at their children in a race against time. The case of Diarmuid is an example. His mother said that Diarmuid's brother, Nathan, has the same condition. Nathan has had access to the drug through a UK trial for more than ten years but Diarmuid is approaching the age where he is likely to lose mobility. She asked me to imagine being a mother opening the fridge every day to give her eldest boy this life-changing medicine and not giving it to her other little boy. She asked me to imagine the turmoil that causes her day after day. She said she met the Minister for Health and a promise was made to allow compassionate access that would be fast-tracked for her child. She says time is not on her side. The case of Aaron, a young boy of nine years of age, is the same. He was walking when people were pleading with the Government last year and now he is in a wheelchair, not knowing, even if the drug is approved, whether he will be able to access it. This is not good enough.

Go raibh maith agat, a Theachta.

I am a parent of four young boys. I would go through a wall for them. The Government has to allow compassionate access to these drugs immediately.

What is difficult to understand is that givinostat is available in most EU countries. Whatever is going to be decided now must be fast-tracked. The Minister of State referred to the receipt of a commercial offer on Friday, 8 May. In the next few weeks, a decision must be taken. The ironic thing about this is that Aaron's family lives in Laghy, close to the Border and the O'Sullivan family lives in Ballybofey, close to the Border. The children on the other side of the Border, in Fermanagh, Tyrone or wherever are availing of this. It is vital that there is no discrimination as far as these children are concerned. We are pleading with the Minister of State and with the Minister, who is pragmatic - we have had dealings with her about issues in Donegal. I hope a decision can be taken soon. The question of finance should not be an issue. I am calling on all concerned, including the drug company, the HSE and the Department, to ensure these children can avail of this treatment as soon as possible.

Go raibh maith agat, a Theachta.

Sorry Chair, time is ticking. It is an important issue.

I thank the two Deputies. It is important we understand that negotiations are under way. There is a system and process in place and simply saying that does not cut it from a political perspective is not at all logical. There are children all over Ireland who need this drug. The Minister recognises that. There is a process in place. Italfarmaco, the pharmaceutical company, came with a proposal last Friday and the confidential commercial negotiations are under way. Today is Wednesday. The reason there was a delay between January and now is that the NCPE did not received the full HTA from the company until 15 January. It took four months. I do not know why these delays have happened but there are children who need this drug and I agree with the Deputies that it should not be the situation that a child living in Donegal does not have access and a child living five miles down the road does. We understand that but it could not be clearer. The application relating to the commercial negotiations was received last Friday and it is currently getting the best attention. I will speak to the Minister again later today about this. She knows the two Deputies are raising it. She was not in a position to be here today. The HSE cannot comment on the possible outcomes of the ongoing process, as the Deputies will understand, because of the confidential nature of it. I understand what they said about compassion but I also agree with Deputy Gallagher that the pharmaceutical companies need to come to the table as well. I answered a similar question here last week about Friedreich's Ataxia. It is important now that the negotiations are under way and that we get a positive result.