Dáil Éireann Debate, Thursday - 14 May 2026
536. Deputy Robert O'Donoghue asked the Minister for Health if she will outline the pathway available for access to the medication Miglustat for a child diagnosed with Juvenile Batten Disease (CLN3), where the medication is already approved and reimbursed in Ireland for other rare conditions, including Gaucher Disease and Niemann-Pick Type C; and if she will make a statement on the matter. [35902/26]
Amharc ar fhreagra538. Deputy Robert O'Donoghue asked the Minister for Health if she will review the case for access to Miglustat for children diagnosed with Juvenile Batten Disease, particularly where there is significant unmet clinical need and no alternative treatment currently available; and if she will make a statement on the matter. [35904/26]
Amharc ar fhreagraI propose to take Questions Nos. 536 and 538 together.
The State recognises the importance of timely access to innovative medicines for patients in Ireland, particularly patients with rare diseases.
Budgets 2021-2025 allocated an additional €158 million for new drugs, which has facilitated the introduction of 263 new medicines, to date, 72 of these new medicines were for rare diseases. Budget 2026 allocated €30 million of funding available for new drugs to be allocated from the overall additional €217 million in funding allocated for medicines.
The Year 1 cost of a new drug comes from the allocation for new medicines, but it should be noted that once these medicines are approved for reimbursement the full cost of providing them can reach multiples of this initial cost as their uptake increases.
Ireland is among the highest spenders on health across the EU (OECD), with the third-highest rate of State coverage of pharmaceutical expenditure in Europe.
There are formal processes which govern applications for the pricing and reimbursement of medicines, and new uses of existing medicines, to be funded and / or reimbursed. The HSE is the statutory decision maker for the pricing and reimbursement of new medicines under the Health (Pricing and Supply of Medical Goods) Act 2013. The HSE considers the criteria set out in the Act. The Minister for Health has no role in decisions on pricing and reimbursement.
There is no hierarchy of disease and pricing and reimbursement applications are considered in order of application.
The HSE must comply with the relevant legislation when considering investment decisions around new medicines. Pharmaceutical companies are required to submit formal applications to the HSE if they wish their medicines to be added to the list of reimbursable items / funded via hospitals and must continue to engage with the HSE throughout the process. In order to submit a formal application the medicine must hold a marketing authorisation.
The European Medicines Agency (EMA) is a centralised agency of the European Union (EU) responsible for the scientific evaluation, supervision and safety monitoring of medicines in the EU. The EMA plays an integral role in the authorisation of medicines in the EU.
To date, the EMA has not granted a marketing authorisation for Miglustat for the treatment of Juvenile Batten Disease. As outlined above, the national assessment and decision process cannot commence in the absence of a marketing authorisation.
The State has successfully negotiated new Framework Agreements on the Supply and Pricing of Medicines with the Irish Pharmaceutical Healthcare Association and Medicines for Ireland. These Agreements include commitments that will enable faster medicines access for patients in Ireland.
As part of the Agreement talks, the State and the pharmaceutical sector have agreed to establish a strategic partnership on the development of a sandboxed early access programme for rare diseases.
This will be a proof of concept initiative, aligned with the commitments in the Programme for Government. The mechanisms involved are complex, and the potential impacts must be carefully considered to ensure any future system is fair, effective, and sustainable.
