Rare Diseases Aren’t Rare — But Treatment Still Is
By Pádraig O’Sullivan
Imagine needing a life-changing treatment that exists, but being told you’ll have to wait 877 days to get it. That’s almost two and a half years. That’s not a worst-case scenario, it’s the current average in Ireland for accessing treatments for rare diseases.
We need to be honest, rare diseases aren’t actually rare. In Ireland, 1 in 17 people live with one. That’s over 300,000 people — children, parents, grandparents. This is not some distant issue affecting a handful of patients in specialist clinics. This is happening in towns, schools and homes across every county. Most of us know someone, whether we realise it or not, who is impacted.
These conditions are often progressive and debilitating. People lose mobility, cognitive function and independence. Not overnight, but day by day. The longer treatment is delayed, the more irreversible the damage becomes.
But here’s what’s really frustrating: the treatments are already out there. These are drugs that have passed EU-wide safety checks and are already in use across Europe. Germany delivers access to patients in under 100 days. Ireland takes almost nine times longer. We’re among the slowest countries in Europe, not because of funding, but because of our process.
And here’s the real kicker, a lot of these delays are completely avoidable.
Our current system treats drugs for rare diseases, often called “orphan drugs”, the same way it treats medicine for common illnesses. That might sound fair, but it doesn’t work. These drugs are usually more expensive and have less data behind them, simply because there are fewer people with these conditions. That doesn’t make them less effective, it just means our approval process isn’t built to handle them.
It’s outdated, too slow, and completely out of step with the rest of Europe.
This isn’t just frustrating, it’s dangerous. These aren’t conditions you can afford to sit on. Every delay makes the situation worse. And in a lot of cases, we’re talking about young people whose lives could be drastically improved if they just got access to treatment sooner.
Other EU countries, including our Nordic neighbours, have already introduced models that fast-track access while still maintaining rigorous oversight.
I’ve spoken to families who have spent years fighting for medication that’s already available elsewhere. I’ve heard from young adults trying to hang onto their independence while their condition slowly chips away at their strength. And I’ve seen parents worn out by a system that seems more focused on paperwork than on people.
It’s heartbreaking. And it’s entirely preventable.
That’s why, with the support of my Fianna Fáil colleagues, I’m reintroducing a bill in the coming weeks that would speed things up. It will create a separate track to assess treatments for rare diseases, one that recognises the urgency and the reality of these conditions.
It doesn’t mean lowering standards. It doesn’t mean spending more money. It just means creating a fairer, faster system that actually works.
Right now, we’re measuring everything by the same yardstick, whether a drug is for a common cold or a life-limiting genetic condition. That’s not good enough.
We’ve got the brains. We’ve got the pharmaceutical industry, many of these drugs are actually made here in Ireland. We export them all over the world. And yet, people here can’t get their hands on them. That’s madness.
We need a smarter, more responsive system. One that says: if a treatment is already being used safely across Europe, why are we still making people in Ireland wait three times longer to access it?
This bill will cut through the red tape. It will make it easier to assess rare disease treatments based on what really matters, their potential to help people.
And let’s be clear, we’re not asking for a blank cheque. We’re not even asking for new funding. Just a bit of common sense. A better process. A way forward that puts people ahead of paperwork.
Because right now, people are losing years of their lives while we “wait and see.”
We can do better. And we must.
Rare diseases might be individually uncommon. But together, they affect a huge number of people across Ireland. This is a national issue, not a niche one. And it’s long past time we treated it that way.
Let’s fix the process. Let’s stop the delays. And let’s make sure no one in Ireland is left waiting for treatment that’s already within reach.
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