There are around 36 million people with rare diseases living in the EU — that’s one-in-every-12 people.
‘Orphan drugs’ is the name given to treatments specifically designed for rare diseases, which often have exorbitantly expensive price tags.
When a drug is approved by the European Medicines Agency (EMA) in Amsterdam, it then falls to individual national governments to perform higher technological assessments (HTA) and cover the cost of the drug,
This leads to unfair, unequal access in Europe.
In countries where HTAs are performed quickly and efficiently, patients can access drugs affordably within months of EMA approval.
Elsewhere, patients often endure years of deteriorating symptoms while they wait.
Only a Common Pharmaceutical Policy will bring all of Europe up to the standard of the best-performing member states.
If you’re Irish, pay for it yourself
Ireland, a country hailed globally as an innovation hub for pharmaceutical products, is failing its citizens with rare diseases.
I say that, not as a policy expert or a professional in Ireland’s booming pharmaceutical sector, I say that as a somebody with a rare disease called Friedreich’s Ataxia (FA)
FA is a rare, progressive neuromuscular condition that causes nerve damage and muscle weakness throughout the body. Sadly, there is no cure.
Niamh Ní Hoireabhaird (Photo: Disabled Women Ireland)
In February 2024, the EMA approved Skyclarys, a drug that has been shown to slow the progression of FA. Also, notably, this is the first and only treatment for patients with FA.
To date, Skyclarys is marketed in 12 member states through various mechanisms, but the drug is still not reimbursed in Ireland.
The hesitancy to reimburse this drug would be bewildering if this were not the standard for orphan drugs in Ireland. This is just one small part of the bigger picture.
Member state lottery
Each year, the European Foundation of Pharmaceutical Industries and Associations conducts a survey offering a comprehensive overview of public drug reimbursement across over 30 countries. The 2024 study noted that the access disparity is 87 percent between the highest and lowest European countries.
The same study reported that, between 2020 and 2023, there were a total of 66 orphan drugs approved in the EU. Some 59 became available in Germany, 13 in Ireland and 9 in Malta.
Additionally, the average time between market approval and time to availability from 2020 to 2023 in EU countries was 611 days. Very impressively, Germany had an average of 97, while Ireland averaged at 695, and Malta struggled with an average of 876.
In Germany, all orphan drugs are initially subject to a limited assessment after market access, with only specific drugs being subjected to a regular HTA.
Considering this, there is no denying that Ireland’s drug reimbursement process has a fundamental flaw.
In a social media post, Irish MP Pádraig O’Sullivan described Ireland as “a laggard” in this context, and that the reimbursement system is “fundamentally broken”.
It feels comforting, in a way, to see my concerns and issues being shared by an elected representative. But then again, it’s disheartening to know the government is aware of this issue, but still hasn’t fixed it.
In its 2026 budget, Ireland allocated a €30m of funding available for new drugs from the overall additional €217m in funding available for medicines.
Speaking about this, Ireland’s minister for health, Jenifer Carroll McNeill said, “A record of over €3bn, around €1 in every €8 of public funding spent on health, is now being spent on medicines.”
An undeniably huge amount.
In February 2026, minister Carroll McNeil announced new framework agreements on the pricing and supply of medicines, to enable cheaper and quicker access to drugs.
With the new framework agreements and billions of euros being spent on medicines, why are hundreds of rare disease patients like me still waiting for EMA-approved orphan drugs?
The orphan drug landscape in Europe is constantly evolving, with multiple new treatments being approved each year – we need new policies and regulations to keep pace.
I was relieved to learn the frustration of unequal access is shared by EU representatives.
Recently, during the European Parliament’s Rare Diseases Forum 2026, former EU commissioner for health, Lithuanian MEP Vytenis Andriukaitis, called for deeper integration of EU member states on the issue of unequal access to orphan drugs: “It’s time now to discuss a Common Pharmaceutical Policy.”
EU-wide cooperation essential for specialist care access and putting an end to unequal access to medicines across the EU.
With national health systems struggling financially, a EU-wide centralised approach to drug reimbursements would enable faster and fairer access to the latest emerging treatments in all EU member states – not just Ireland.
For the lives and futures of EU citizens with rare diseases, I hope these discussions take place and changes are made sooner rather than later.