2026-06-11

I again raise the issue of rare diseases, which I have raised consistently in the House for the past five years. I have spent the past hour and a half with Senator Costello, who was meeting with parents whose children suffer from Duchenne muscular dystrophy. They received the good news this week that the treatment drug is on its way to the HSE management team for consideration, having been positively recommended for reimbursement. It is a great relief for the families but the victory is bittersweet because they know that every week they lost waiting for the drug over recent years has led to a deterioration in their children's condition. Those parents are not alone in their story. There is a campaign currently under way by parents whose children primarily suffer from Friedreich's ataxia and who urgently require the drug Skyclarys. I understand that matter is at a sensitive stage of negotiations with the HSE and I do not expect the Minister to comment on it. On the overall picture, the Minister made an important point in his interaction with Deputy Paul Murphy. The Minister is a father and, like me, he understands how difficult it is for parents of children who are ill. No child should have to wait for healthcare but we consistently have a system in this country under which children are waiting. On average, children in Ireland wait 800 days for rare disease drugs. That is in contrast with the situation in other EU and EEA countries. I am not talking about the big countries with the big economies and the big patient cohorts. I am talking about smaller countries, some of them with smaller economies than our own, like Iceland, Luxembourg and Denmark. They are reimbursing drugs at twice and three time the rates we are reimbursing them. We are consistently laggards in the league table when it comes to reimbursement of these drugs. I commend Senator Costello and the families on their campaign over the past few months. It came to fruition this week and they have achieved the result they want. However, there must be dozens of families at home looking on enviously while they wait for the drug they need to be assessed for reimbursement. It is a cruel and horrible situation for any family to be in. It is inhumane. I am asking for follow-through on the two commitments on this issue in the programme for Government. One relates to the early access scheme and the other is for a review of the reimbursement process as a whole. To my knowledge, action has not commenced on either recommendation. I am hearing about bits and pieces going on but when I talk to clinicians and consultants, they tell me nothing is happening. The Government is a year and a half into its term. The Minister knows as well as I do that anything involving a review or report will take God knows how long. It could take a year, two years or three years, if we are lucky. I ask the Minister to urgently speak with the Minister for Health to ensure those two commitments in the programme for Government are honoured and prioritised with immediate effect.

I thank Deputy O'Sullivan for his important question. We have about 300,000 people in Ireland who are living with rare diseases - about one in seven of the population - which is very significant. We have made commitments in the programme for Government and we are working with the Minister for Health, Deputy Carroll MacNeill, whom I know is very committed in this area. One of the critical areas we are working on is access to new medicines. That is why, later this year, we will bring forward a life science strategy which, in partnership with the Department of Health and my Department, will work to ensure access to rare medicines and incentivise innovation. We have many life science companies in operation in Ireland, employing over 100,000 people, which are advancing the most clinically innovative drugs. It is critical we get access to those drugs for our population, particularly the cohort the Deputy mentioned. There is a mechanism for approval of rare drugs and resources have increased significantly to the Health Products Regulatory Authority, HPRA, to ensure we get better at approving these key medicines that are so important for society, children and families. As the Deputy rightly pointed out, there is family who are very much suffering because of that. I know Senator Maria Byrne and my Fine Gael colleagues do a huge amount of work with the rare diseases group here in the Oireachtas, and they are very interested in progressing it. I look forward to working with the Deputy in this regard. I will relay the issues he raised to the Minister for Health.

I commend the Minister as she was central to this drug being approved. She met with families, treated them with dignity and gave them hope, and I commend her on that. However, it feels like groundhog day because I know that in three, six or nine months' time, another cohort of patients will be looking for another miracle drug. I have seen children regain their mobility from taking some of these drugs. That is what they do. The innovation the Minister referred to is phenomenal. Every year, corporation tax collected from pharmaceutical companies amounts to between €7 billion and €10 billion. Even more phenomenal is that we cannot get a good price from these pharmaceutical companies. We cannot get these drugs at a quick pace, which we should. If we are facilitating these large corporations being here, the least we can do is get early access to a reasonably priced drug. If we do not listen to the health argument on this issue, which we have not done for over a decade now, I would make the argument that the jobs the Minister referred to are contingent on us keeping up with innovation. Unfortunately, we have an archaic system that has not changed and kept pace with that innovation. I ask the Minister to press home the issue with the Minister for Health to make sure we get an early access scheme as quickly as possible.

I will raise this issue with the Minister of Health. As the Deputy pointed out, she is committed and has been meeting families on this. In budgets between 2021 and 2025, an additional €158 million was made available for new drugs and of the 263 new drugs, 72 were new drugs that were licensed in that regard. We also have the rare diseases strategy from 2025 to 2030, which the Government is working on to ensure we breathe life into its recommendations to support the families the Deputy referenced. That is what the Government is achieving. I will work with the Minister for Health and raise the concerns the Deputy has brought to the House this afternoon.