I join with other speakers in highlighting the issue of rare diseases. In particular, I want to speak about a condition affecting a small number of people in Ireland, which is SMA, a genetic disorder that affects motor neurons in the spinal cord, leading to progressive muscular weaknesses, loss of function and even shortened life expectancy. For many years, families affected by SMA lived with little hope of effective treatment. Thankfully, however, breakthroughs in research mean that there are therapies available today that can dramatically improve the quality of life for many patients. Ireland can be proud of the progress that has been made. The State has agreed reimbursement for innovative SMA treatments for children and young people. These decisions have transformed lives, and this is something we should acknowledge and commend. However, there remains a small group of adults with SMA who have been denied treatment because their dates of birth do not comply with the chosen cut-off date. Approximately 16 adults in Ireland with SMA are currently excluded from reimbursement of treatments that are available to younger patients. Through no fault of their own, they miss an opportunity afforded to those who came after them. As a result, we have a situation where two people with the same condition are treated very differently. A patient diagnosed as a child will continue to receive the treatment into adulthood but another adult with the same diagnosis and the same clinical need may be unable to access reimbursement at all. That is unjust and inequitable. It discriminates against this small group of people because of their age. I appreciate fully that decisions on reimbursement must be evidence based, clinically informed and financially responsible but I ask that, alongside the necessary procedures, we apply a degree of joined-up thinking, practicality and humanity. We need to think outside the box for this small group of adults. Their lives could be improved by treatments that are already recognised, licensed and reimbursed elsewhere for others with SMA in this country. When this group of adults looks to Europe and sees other adults with SMA accessing therapies, and when they look to Northern Ireland and across the Irish Sea where similar patients are receiving treatment, and when they see younger Irish patients benefiting from these medicines, is it any wonder that they feel frustrated, excluded and let down? Rare disease policy requires us to think differently. One size does not fit all. Traditional health technology assessment models are designed around larger patient populations. Rare diseases do not fit neatly into these frameworks. This is why flexibility and innovation are practically essential. I ask that every effort be made to explore practical solutions for this exceptionally small group of patients through management access arrangements, conditional reimbursement measures, special rare disease pathways and other approaches. There is scope for constructive engagement that balances fiscal responsibility and compassion. Our national rare disease strategy recognises that people living with rare disease face many barriers. These 16 adults are experiencing a unique barrier - their age. I know the Minister is committed to improving outcomes for those living with rare diseases. I know that significant work is underway with the Department-----

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----- and I ask that special attention be given to this group-----

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-----and that the Minister engages further with EMA Ireland.

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