Professor Michael Barry of the National Centre for Pharmacoeconomics made a number of unbalanced assertions on RTE Radio 1 last Sunday regarding suppliers of orphan medicines which IPHA believes require important context. We are therefore providing further detail to support informed public debate, so that patients, clinicians, legislators and the wider public can better understand why reform is necessary and where efforts should be focused. The need for reform has been clearly embraced and underlined by the Minister for Health and the Government.
First, the Irish State and the HSE, regardless of which agency acts on their behalf, have been in breach for well over a decade of their legal commitments in the Health Act, 2013, on making timely reimbursement decisions. The law is clear: applications must be processed within 180 days, excluding periods during which additional information is requested from applicant pharmaceutical companies.
The Minister for Health has embarked on an over-due reform process to comply with this law.
The fact that it may take until 2029 for the State to meet its own legal obligations makes clear that the HSE process has not routinely adhered to statutory timelines. Since 2022, the average timeframe for the State to assess and decide on an IPHA company’s orphan medicine treatment, excluding industry response time, has been 422 days. That is more than double the legal requirement. In several cases, the timeframe for NCPE assessors to respond to an application exceeded 180 days. The NCPE plays a key part in the process as a service provider to the HSE but not with independent statutory obligations or separate latitude on timelines. The entire process is envisaged in law to be under the governance of the HSE.
This central fact is not in dispute and is one of the reasons the 2026 Framework Agreement with industry includes a commitment, stated by the Minister, to adhere to the 180-day timeline. If the majority of delays are attributable to industry, as was suggested, it is difficult to understand why achieving this legal standard would be considered challenging for the State. The fact is that the reimbursement system has never heretofore been designed, operated and governed to meet the legal obligation of 180 days passed by the Oireachtas in 2013, transposing European law. Now, it is policy to comply and IPHA welcomes this and has already started to work constructively with the Department of Health and HSE to bring it about as soon as possible.
We note and welcome the State’s processing of reimbursement of a medicine for Duchenne muscular dystrophy, which was completed in approximately 130 days. That should now become the standard for assessing and deciding on orphan treatments. It demonstrates that timely decisions are possible.
IPHA members wish to bring forward medicines for reimbursement and note the keen interest of the Minister is seeing applications made. We agree and want to make the system as attractive as possible. Pharmaceutical companies have a clear incentive to supply their products to as many patients as possible, as quickly as possible. The track record of IPHA members submitting medicines for reimbursement in Ireland that have been newly-authorised by the European Medicines Agency is good: the HSE receives applications for approximately 70% of EMA-licensed medicines from all companies. The number of non-applications of medicines by IPHA members is less than 10% of licensed medicines.
However, the unfortunate experience of some recent years of lack of timely and predictable responsiveness from the reimbursement system made Ireland a higher-risk and less predictable market. Companies then had fewer options but to focus on markets where the risk of decision-making delays was lower. Iceland, Denmark, Cyprus, Luxembourg, Estonia, Slovenia, Lithuania, Latvia and Norway are all making orphan treatments available to patients faster than Ireland. This data point is based on publicly verifiable information and has never been disputed.
It is also important to note that the NCPE’s mandate in assessing medicines is limited to three criteria: clinical effectiveness, cost effectiveness and budget impact (as per its website). The value of a medicine does not end with those three criteria. The Health Act, 2013 lists six additional criteria that are not assessed by the NCPE, including the health needs of the public. A medicines benefit can extend to include mental health benefits associated with successful treatment, benefits for carers and patients’ families from a reduced burden, the economic impact of a healthier and more productive workforce, and the value of scientific progress, as medicines typically improve incrementally over time.
These wider criteria may fall outside the NCPE’s current mandate, but they are highly relevant to patients. Orphan medicines, by their nature, will often have limited data, as large-scale clinical trials can be impossible and, in many cases, unethical. Breaching this evidence gap requires productive dialogue.
We further note that the development of early access schemes is included in the Programme for Government and is a priority for the Minister. Estimates of the costs associated with such schemes should be considered in the context of international experience, where early access arrangements typically involve some form of cost sharing. The process for assessing how, not if, to implement Programme for Government commitments is, IPHA believes, for the Minister for Health and accountability is to the Oireachtas and to the patients’ representatives, who were key drivers of the proposals for early access schemes.
Early access schemes and the health system generally are best served through constructive dialogue between policymakers and pharmaceutical companies. That approach led to the 2026 Framework Agreement with a plan to improve speed of access. IPHA is therefore continuing to engage with the Minister, the Department and all parties on a constructive basis.
