Ireland's drug approval system needs major overhaul

Ireland is lagging behind many of its fellow EU member states in providing medicines and specialised orphan drugs used to treat rare diseases — the EU presidency offers a chance for this country to make significant changes

Niamh Ní Hoireabhaird: 'Within the six months Ireland holds the presidency, there is ample opportunity to have an honest conversation about Ireland’s failure regarding orphan drug reimbursements, and what needs to happen to bring us closer to the European average.'

According to a Goodbody report, pharma exports amounted to €139bn in 2025, representing more than half of all goods exports and 41% of gross national income.

With figures like this, you would think Ireland is a haven for people who need specific medicines and drugs. But that is simply not the case.

I’ve previously reported that Ireland is lagging behind many of its fellow EU member states in providing medicines and specialised orphan drugs, which are used to treat rare diseases.

Until December 11 this year, Ireland is hosting the Presidency of the Council of the European Union. During the six months that Ireland will hold this position, the country will sit at the epicentre of European politics.

The role offers a chance for Ireland to make significant change and become the orphan drug haven it should be.

Unfair, unequal access

Following a drug’s approval by the European Medicines Agency (EMA), the decentralised European body that evaluates and supervises the safety of new drugs, the delivery process splinters off. Individual national governments are responsible for performing a higher technological assessment (HTA) and covering the cost of the drug.

In countries where HTAs are performed quickly and efficiently, patients can access drugs affordably within months of EMA approval.

Additionally, many EU countries also offer early access programmes which allow people with rare diseases to access life-changing orphan drugs before they receive full marketing approval.

The splintered-off processes lead to unfair, unequal access to orphan drugs in Europe. This is obvious from yearly studies conducted by the European Foundation of Pharmaceutical Industries and Associations, which offer a comprehensive overview of public drug reimbursement across over 30 countries. 

The 2024 study reported that, between 2020 and 2023, there were a total of 66 orphan drugs approved in the EU, with an average of 28 products available in EU countries. Only 13 became available in Ireland.

Additionally, the average time between market approval and time to availability from 2020 to 2023 in EU countries was 611 days. Ireland averaged at 695.  At the other end of the scale, Germany made 59 drugs available with an average of 97 days between market approval and availability.

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.

In Ireland, drug reimbursement recommendations are made by the National Centre for Pharmacoeconomics (NCPE), a team which evaluates the benefit and costs of medical technologies and provide advice to the HSE.

When looking to recommend reimbursements for a drug, the NCPE looks at clinical effectiveness, cost-effectiveness, and budget impact.

In my opinion, too much of the discussion focuses heavily on the economic aspect, and not the fact that there are people who desperately need these drugs to survive.

While I understand there are budget constraints, I believe Ireland should be moving heaven and earth to make sure its citizens can get the medication they need.

Long delays 

The priorities for Ireland’s presidency are competitiveness, values and security.

In theory, this is meant to refer to Europe’s economic competitiveness with America and China, but it ought also to refer to Ireland’s own competitiveness with its European neighbours.

The EU cannot excel while individual member states are dragging it down. By that measure, Ireland is far from meeting its own priorities.

According to Eimear O’Leary, director of communications and advocacy at the Irish Pharmaceutical and Healthcare Alliance (IPHA): “The Irish State and the HSE, have been in breach for well over a decade of their legal commitments in the Health Act, 2013, on making timely reimbursement decisions for all medicines, not only orphan drugs.” 

The Health Act 2013 set a deadline of 180 days for applications to be processed days, excluding periods during which additional information is requested from applicant pharmaceutical companies.

“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.” 

A major problem of the current system is the timeline from EMA approval to availability to patients in Ireland.

The blame cannot entirely be placed on the shoulders of the Government and the HSE. Cork North-Central TD Pádraig O’Sullivan has been a long-term advocate in this area. In a recent discussion on rare diseases in the Dáil, Mr O’Sullivan said “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”. I also suspect this to be true; it takes two to tango.

System overhaul 

People working across the system agree the reimbursement procedure is not fit for purpose.

“It cannot fall to the patient community to keep begging for access to medicines”, said Vicky McGrath, chief executive of Rare Diseases Ireland.

“I think the State needs 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.” 

I don’t deny that the current Government is doing more than the previous government in regards to orphan drugs and rare diseases. With the publication of the long awaited National Rare Disease Strategy last year, and the framework agreement on the pricing and supply of medicines earlier this year, it’s clear orphan drugs and rare diseases are on the minds of the people with the power to make a change.

But we need more.

We need a total review of the systems that exist in this country. Within the six months Ireland holds the presidency, there is ample opportunity to have an honest conversation about Ireland’s failure regarding orphan drug reimbursements, and what needs to happen to bring us closer to the European average.

Like me, Ms O’Leary hopes the presidency will present Ireland with an opportunity to examine and improve the orphan drug provision in Ireland:

“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.” 

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.

Ireland, with its wealth and its booming pharma industry, needs to shift into gear and get on the same level as its fellow EU member states.

We need a system we can rely on, and now is the perfect time to discuss this.

• Niamh Ní Hoireabhaird is a freelance journalist who lives with Friedrich's Ataxia