Maria Byrne (Fine Gael)
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I welcome the Minister of State, Deputy Buttimer, to the House.

Teresa Costello (Fianna Fail)
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I want to be clear I am no expert on Duchenne muscular dystrophy or DMD as it is commonly referred to. However, since a young boy from Tallaght was diagnosed with this condition, I have come to understand the immense challenges faced not only by the patients but by their families as well. From what I gather - and I am open to correction on this - treatment options in Ireland are limited to not much more than steroids and a wheelchair.

Duchenne muscular dystrophy is a progressive neuromuscular disease that causes the weakening of muscles over time. It affects almost exclusively 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. Most of them are under the age of 18. In the UK, approximately 100 boys are born with the condition each year. For a child living with DMD, issues, they say, can include struggling to walk, run or jump like their peers, difficulty standing up, delayed speech, needing help to climb stairs and sometimes facing behavioural or learning challenges. As the disease progresses, most children will need a wheelchair by the time they are 12. Some will develop scoliosis. By their mid-teens, many experience heart complications and, by their late teens or early 20s, breathing problems often emerge, making the condition life-threatening.

There is, however, a glimmer of hope in the form of a new drug called givinostat. It is marketed as Duvyzat and it offers a breakthrough. It has been shown to slow the progression of muscular degeneration in those with DMD. Clinical trials have met both primary and secondary endpoints, demonstrating its effectiveness. In December 2024, the UK's Medicines and Healthcare Products Regulatory Agency approved givinostat for use in boys aged six and over. It is currently being provided through the expanded access programme at no cost to patients via local NHS trusts.

This drug, however, is not yet in Ireland. Givinostat should have its full EU licence by approximately the end of June. The next step would be that the manufacturer will make a submission for a higher technical assessment with the National Centre for Pharmacoeconomics, NCPE. In Scotland, which is not very far away, I have been told - and I am open to correction on this - that, as regards the decision-maker similar to the NCPE in Ireland, the turnaround time from submission to availability is about five months. I would like to push for a similar turnaround time given the impact time has on the eligibility of patients for this treatment. If you are looking at your child who has been diagnosed with this, you are looking at a clock. Givinostat does not cure DMD; it slows its progression. It cannot turn back the clock or undo the damage done but it can slow it down, giving these boys more time on their feet without the need of a wheelchair.

There is a race against time for these children. I read a press release a few days ago, on 25 April, confirming that the European Medicines Agency, EMA, has recommended a conditional marketing authorisation for Duvyzat across the EU. I would like to know the status on getting givinostat to our Irish patients.

Jerry Buttimer (Cork South-Central, Fine Gael)
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I thank Senator Costello for raising this very important matter. I am taking it on behalf of the Minister for Health, Deputy Jennifer Carroll MacNeill. I thank the Senator for highlighting the case of a young boy from Tallaght. She is right that it is a race against time. I am conscious that my reply probably will not give her the answer that she needs and that the young boy deserves. I hope we can all work together to ensure we can progress the matter she has spoken about.

Duchenne muscular dystrophy is, as the Senator said, a rare disease affecting fewer than 200 people in Ireland. The State acknowledges the importance of access to medicines and is aware of the plight of patients with DMD.

The State has made considerable investment in new medicines in recent years. In 2023, the State spent over €3.2 billion on medicines, which represents nearly €1 in every €8 of public funding being spent on health. This level of investment is unprecedented in supporting patients through the availability of new and innovative medicines. Senator Costello is right to highlight the challenges and the issues faced by people with DMD, and I thank her for doing so. The budgets of 2021 to 2024 have included dedicated funding for new medicines of €128 million. This has enabled the HSE to approve reimbursement for 194 new medicines or new uses for existing medicines, including 74 for treating cancer and 49 for treating rare diseases. Last year's budget allocated €30 million of funding for new drugs to be generated by efficiencies to be identified by the HSE.

Givinostat, which is sold under the brand name Duzyvat for the treatment of DMD in patients aged six years or older, has recently undergone assessment by the European Medicines Agency. The EMA is a centralised agency of the European Union responsible for the scientific evaluation, supervision and safety monitoring of medicines in the EU. The EMA plays an integral role in the authorisation of medicines in the EU. The company marketing this treatment applied for marketing authorisation from the EMA and its evaluation concluded with a recommendation that it be granted conditional marketing authorisation. The European Commission will consider this recommendation from the EMA and make a final decision on granting marketing authorisation for the treatment. At this time, givinostat does not have marketing authorisation in the EU.

The Health Service Executive has statutory responsibility for decisions on pricing and reimbursement of medicines and medical items, in accordance with the Health (Pricing and Supply of Medical Goods) Act 2013. In line with that 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 which have been granted marketing authorisation by the EMA or Ireland’s Health Products Regulatory Authority. As givinostat has not been granted marketing authorisation by the Commission, an application for pricing and reimbursement cannot be made to the HSE. If marketing authorisation is granted and the company chooses to apply, the HSE will give fair consideration to the application, with consideration for the criteria detailed in the 2013 Act.

I agree completely with Senator Costello that the process must be expedited. We cannot have this going on forever. There are people like the young boy the Senator mentioned and other families who need help and support. I thank Senator Costello for her advocacy and for raising this matter. She right to say that the clock is ticking. People are racing against the clock and I hope we can all see this matter expedited.

Teresa Costello (Fianna Fail)
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I thank the Minister of State for his response. I appreciate his acknowledgement of the urgency of this matter. When the marketing authorisation is granted, I would really appreciate it if we could work together to ensure that this product gets onto the Irish market quickly. I would like to see it fast-tracked by whatever means necessary. I personally cannot look at a video of a child who is able to walk now and watch the time pass by. We need an all-hands-on-deck approach to make this medication available in the most urgent fashion and I would really appreciate that commitment from the Government.

Jerry Buttimer (Cork South-Central, Fine Gael)
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I thank the Senator and agree that it is urgent. She is right that we all need to work together, including the European Commission, the authorities here and the company in question. As a former Chairman of the health committee and someone who was involved in the passage of the 2013 Act, I assure the Senator that I genuinely appreciate the importance of ensuring that patients with DMD have access to the latest drugs. We are, as a country, very much open to that avenue of new drugs being made available. I assure the Senator that once the drug is granted marketing authorisation, the Department, along with the HSE, will strongly encourage all pharmaceutical companies to apply for reimbursement here. The pharmaceutical company has a critical role to play in that regard.

There have been significant investments to enhance the HSE's pricing and reimbursement process for medicines. A total of 34 new staff were hired across the pricing and reimbursement system last year and will support faster access to new and innovative medicines for patients. To support transparency in this process an application tracker has also been developed and launched. I assure the Senator that the Department of Health will continue to engage with all involved to ensure that all patients have access to new and innovative treatments.