I welcome this opportunity to speak on rare diseases. I spoke on the issue in the previous Dáil. I do not think I have spoken about it in the current Dáil. It has been said many times that, although rare diseases are classified as rare, we all probably know somebody who has one because one in 17 people in the country, a total of 300,000 people, have a rare disease. It is a very difficult issue to talk about. The system to approve medicines in this country has been poor. It has resulted in medicines that are available to sufferers of rare diseases under the public system in other countries, including some medicines I will discuss in a few moments and that others have already mentioned, taking longer to get to the people who need them in Ireland. I know the Minister and her predecessor have worked to change that. We need to make progress to expedite the system and to ensure that we get the best drugs in the world into treatment programmes for people in this country who have rare diseases. I take this opportunity praise the families of those children living with Duchenne muscular dystrophy. Their tenacity in fighting for their children is unbelievably admirable and we are glad to have met so many of them in recent years. I acknowledge the work of Senator Teresa Costello and my colleague Deputy Pádraig O'Sullivan, who has been steadfast in his work and advocacy for sufferers during all his time in the Dáil. I will also mention the former TD, Denis Naughten, who did a great deal of work over many years. I am so glad for those children living with DMD that the HSE drugs group has recommended reimbursement. It is now imperative that those children get access to this drug as soon as is practicable. At the core of this fight was the sheer length of time it took for this medicine to be considered and to go through the assessment process for reimbursement following EMA approval. I am sure that all of us in this House, including the Minister, agree that the process needs to be reformed. Our health spokesperson, Deputy Marie Sherlock, wrote to the Minister more than a year ago about access to this medicine. While it might have been given approval faster than other medicines, it was still too slow, taking far beyond 180 days. This is especially so as it had been made clear that these children simply did not have time on their side. As a result, some young people living with DMD will unfortunately not be able to benefit from this very welcome decision. It would be good to hear from the Minister and the HSE senior leadership team as when the drug will be formally approved - that may have been done; I am not sure of the timelines - and when these families can expect this medicine. I will raise, as others have, Skyclarys and the treatment of Friedreich's ataxia, a rare neurodegenerative disorder that progresses very quickly, resulting in neurological decline, the loss of bodily function, mobility and independence, and ultimately death. Approximately 200 people in Ireland have Friedreich's ataxia. I know the Minister met some of them earlier this year. Skyclarys is the only approved treatment for this condition. It was approved by the EMA in February 2024 and is available in many states across the EU. As yet, it is not publicly available in Ireland. An application was lodged here in August 2024. A constituent of my colleague Deputy Sherlock has told us that she and others are losing hope. People she knows have lost the ability to stand upright, to walk, to feed and dress themselves and to speak clearly. Just like those children with DMD, time is not on the side of those with Friedreich's ataxia. This person has said: I am not asking for special treatment - just the fair treatment that patients in Germany, Italy, and many other European countries already receive. Please help to ensure that the "National Rare Disease Strategy" becomes a reality for those of us who cannot afford to wait any longer and, by doing so, provide access to this life-changing treatment. The HSE drugs group needs to consider this medicine with the utmost urgency. The reality that Irish patients cannot access many novel and innovative medicines that other citizens across the EU can is a let-down coming into our EU Presidency. We believe the EU Presidency presents a clear opportunity for the Minister and for Ireland with regard to the procurement of medicines, particularly orphan and innovative medicines. During the Covid pandemic, the EU proved how effective the collective procurement of treatments can be through its procurement of Covid vaccines. We must work to create a similar process for the procurement of medicines, particularly rare medicines, which can be costly for states with smaller populations to procure. It has been well flagged to the Minister and to many of us here that the value-for-money or quality-adjusted life-year assessment needs to be looked at given the small number of patients with rare diseases, where there are treatments. Many innovative medicines struggle to meet this threshold. This needs to change. I welcome the recent announcement of a review into the reimbursement process. I hope this will consider my point regarding the quality-adjusted life-year assessment. Rare disease patients can wait up to 722 days for decisions to be made on orphan medicines. This is far above the European average. I welcome the plan to a more structured approach of achieving a 180-day reimbursement decision. As the Marie Keating Foundation has stated, we must make the 180-day timeline a dependable standard as opposed to an aspiration. Ireland is now the only country in western Europe without a formal early access programme. We need to see the consideration and introduction of a compassionate early access programme. This is about recognising that for rare diseases, time is often not on the side of those with such diseases. In some cases, the earlier a medication is started, the more effective it can be in slowing illness progression down. The Minister has stated that she is working on this. We will support her in that regard. While prostate cancer is certainly not a rare disease, I want to raise once again issues with accessing innovative medicines, which is certainly an issue that also relates to rare diseases. It comes down to access. It is the case that access to private medical treatment here can, in turn, grant access to medicines that one cannot get in the public system. The Minister will agree that this is utterly unfair. We accept the need for value for money, but we must demand equitable access to treatments. I reference Pluvicto, which is a medicine to treat men with metastasized prostate cancer. This is a matter I have previously raised with the Minister. The drug is not reimbursed by the HSE, yet a patient in, for example, the Mater Private Hospital can get access to it. The inequity is unacceptable. I ask that the Minister might at some stage provide an update on the HSE's engagement with the manufacturer in relation to this medicine. In the context of other issues that we raise in the Dáil, we sometimes can get a sense that a Minister or the Government do not quite get the issue or understand it. I do not think that is the case with rare diseases. The Minister and the Government understand the issue and the difficulties relating to it and are aware of what needs to be done. She will have our support if we are moving in the right direction. I hope that we do so. I hope that we can get the 180 days. I hope that people, especially children, with rare disease can get access to various novel medications that have been approved elsewhere as soon as possible. I hope that we do not have to have statements on rare diseases too often and that we can move forward.

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