Ireland's Orphan Drug Access Crisis: Reimbursement Delays Leave Rare Disease Patients Waiting Years
Key Insights
Between 2020 and 2023, only 13 of 66 EU-approved orphan drugs became available in Ireland, compared to an EU average of 28, with Ireland averaging 695 days from market approval to availability versus the EU average of 611 days.
The Irish State and HSE have been in breach of the Health Act 2013's 180-day reimbursement decision deadline for over a decade, with orphan drug assessments averaging 422 days since 2022.
Patient advocates and industry leaders, including Rare Diseases Ireland (search) and the IPHA, are calling for a total system overhaul, citing a reimbursement process that is "not fit for purpose."
Ireland is failing patients with rare diseases. Despite its wealth and a thriving pharmaceutical industry, the country consistently ranks among the worst-performing EU member states in providing timely access to orphan drugs, with only 13 of 66 EU-approved orphan medicinal products becoming available to Irish patients between 2020 and 2023 — less than half the EU average of 28.
The disparity is laid bare in the 2024 European Federation of Pharmaceutical Industries and Associations (search) (EFPIA) study, which tracks public drug reimbursement across more than 30 countries. While the average time from market approval to patient availability across the EU stood at 611 days, Ireland lagged significantly at 695 days. Germany, by contrast, made 59 orphan drugs available in the same period, with an average turnaround of just 97 days.
A System in Breach of Its Own Law
At the heart of the crisis is a reimbursement process that multiple stakeholders describe as fundamentally broken. The Health Act 2013 mandates that reimbursement applications be processed within 180 days, excluding periods during which additional information is requested from pharmaceutical companies. Yet according to Eimear O'Leary, director of communications and advocacy at the Irish Pharmaceutical and Healthcare Alliance (search) (IPHA), the State has been in violation of this legal commitment for well over a decade.
"Since 2022, the average timeframe for the State to assess and decide on an IPHA company orphan medicine treatment, excluding industry response time, has been 422 days. That is more than double the legal requirement," O'Leary stated.
The bottleneck occurs after the European Medicines Agency (search) (EMA) grants marketing authorization. At that point, responsibility splinters to individual national governments, each conducting their own health technology assessment (HTA) and making reimbursement decisions. In Ireland, the National Centre for Pharmacoeconomics (search) (NCPE) evaluates clinical effectiveness, cost-effectiveness, and budget impact before advising the Health Service Executive (search) (HSE). Critics argue the process is disproportionately weighted toward economic considerations rather than patient need.
Manufacturers Deterred, Patients Left Begging
The consequences extend beyond delayed access. Cork North-Central TD Pádraig O'Sullivan, a long-term advocate on rare disease issues, told the Dáil that "many manufacturers do not want to come here not just because it is a small market but because they know they are going to feed into a system that will… take between five and 800 days in going through a HTA predominantly and a reimbursement process that is not fit for purpose."
Vicky McGrath, chief executive of Rare Diseases Ireland (search), underscored the human toll: "It cannot fall to the patient community to keep begging for access to medicines." She called on the State to "show leadership and address the timelines within their systems, address the timelines with industry to make sure that industry is responding in an appropriate and appropriately quick fashion."
A Presidency Moment for Reform
Ireland assumed the Presidency of the Council of the European Union on July 1, holding the position until December 11. The presidency's stated priorities — competitiveness, values, and security — are intended to address Europe's economic standing relative to America and China, but advocates argue they must also apply to Ireland's competitiveness with its European neighbors in healthcare delivery.
O'Leary sees the presidency as a pivotal moment: "During the presidency of the EU Council, Ireland can spotlight the importance of timely patient access to medicines, particularly for rare diseases, and promote best practice in how member states implement reimbursement timelines under EU law."
Recent government actions — including the publication of the long-awaited National Rare Disease Strategy and a framework agreement on the pricing and supply of medicines — signal that orphan drugs and rare diseases are on the policy agenda. Yet stakeholders insist these steps are insufficient without a comprehensive review of the reimbursement architecture.
The orphan drug landscape in Europe continues to evolve rapidly, with multiple new treatments gaining EMA approval each year. For Ireland, a country with both the financial means and a booming pharmaceutical sector, the gap between what is possible and what is delivered has become untenable. The six-month presidency window offers what advocates describe as the perfect moment for an honest reckoning — and, they hope, the political will to close the gap.