Eoin Ó Broin (Dublin Mid West, Sinn Fein)
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300. To ask the Minister for Health the reason a type of medication (details supplied) is not currently available in Ireland; if consideration will be given to approving the medication urgently in light of the reported benefits to people living with a specific progressive neuromuscular disease; and if she will make a statement on the matter. [30839/25]

Jennifer Carroll MacNeill (Dún Laoghaire, Fine Gael)
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In 2023 a record €3.2 billion, nearly €1 in every 8 of public funding spent on health, was spent on medicines. This is an unprecedented level of investment in supporting patients through the availability of the latest and wide range of medicines.

From January 2021 up to December 2024 the State made an additional €128 million available for new drugs which has seen 194 new medicines or new uses for existing medicines approved for reimbursement by the HSE. This includes 74 medicines for oncology and 49 new drugs for rare diseases. .Budget 2025 includes an allocation of €30m for new medicines, to be generated from efficiencies.

Duchenne Muscular Dystrophy (DMD) is a rare disease affecting fewer than 200 people in Ireland. The State acknowledges the importance of access to medicines and is aware of the plight of patients with Duchenne Muscular Dystrophy (DMD).

Givinostat, sold under the brand name Duvyzat®, is a medication used to treat Duchenne muscular dystrophy (DMD) in patients aged 6 years and older. Conditional marketing authorisation was granted on the 6th June 2025 by the European Commission for this medicine as a treatment for Duchenne muscular dystrophy (DMD) in patients from the age of six who are able to walk.

The Health Service Executive (HSE) has statutory responsibility for decisions on pricing and reimbursement of medicines under the community drugs schemes, in accordance with the Health (Pricing and Supply of Medical Goods) Act 2013.

The National Centre for Pharmacoeconomics (NCPE) conducts the Health Technology Assessment (HTA) of drugs for the health service in Ireland in collaboration with the HSE's Corporate Pharmaceutical Unit (CPU). They assess comparative clinical effectiveness and cost-effectiveness of drugs compared to current standard of care. The NCPE engages with stakeholders, including patients and clinicians, throughout the assessment process.

The HSE’s CPU is the interface between the HSE and the Pharmaceutical Industry in relation to medicine pricing and reimbursement applications.

As conditional marketing authorisation has now been granted to the marketing authorisation holder for Givinostat(Duvyzat®) the company must now apply to the HSE for the treatment to be reimbursed through the public health system.

If the company chooses to submit a pricing and reimbursement application, the HSE will give fair consideration to the application with consideration for the criteria detailed in the 2013 Act.

The State encourages all marketing authorisation holders of new medicines to apply to the HSE for reimbursement once marketing authorisation is granted by the European Medicines Agency.

John Brady (Wicklow, Sinn Fein)
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301. To ask the Minister for Health the actions she is taking to ensure equitable access to treatments for all adults with spinal muscular atrophy, regardless of age; and if she will make a statement on the matter. [30840/25]

John Brady (Wicklow, Sinn Fein)
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302. To ask the Minister for Health the actions she is taking to ensure equitable access to treatments for all adults with spinal muscular atrophy, regardless of age; and if she will make a statement on the matter. [30841/25]

Conor Sheehan (Limerick City, Labour)
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315. To ask the Minister for Health her views on age-based exclusion for adults with SMA and if she believes a fairer criteria is introduced to ensure all adults with SMA are treated equally under the healthcare system; and if she will make a statement on the matter. [30953/25]

Naoise Ó Cearúil (Kildare North, Fianna Fail)
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365. To ask the Minister for Health her plans to review and reform the age-based exclusion criteria for adults living with spinal muscular atrophy to ensure a fairer and more equitable approach; and if she will make a statement on the matter. [31073/25]

Jennifer Carroll MacNeill (Dún Laoghaire, Fine Gael)
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I propose to take Questions Nos. 301, 302, 315 and 365 together.

The HSE has statutory responsibility for decisions on pricing and reimbursement of medicines under the community drugs schemes, in accordance with the Health (Pricing and Supply of Medical Goods) Act 2013. Therefore, I have asked the HSE to provide an update in relation to treatments for SMA patients over 18 years of age.

The HSE has advised that once a medicine is approved by the European Medicines Agency, the HSE is dependent on a pharmaceutical company making an application to Ireland for its drug or new indication to be assessed for reimbursement. All medicines are considered for pricing of reimbursement in order of application.

While many companies engage proactively with the HSE, Ireland, by virtue of its size and market share, may not always be prioritised by some companies in the first stages of marketing a new medicine. Reports describing timelines for reimbursement from EMA approval to HSE reimbursement approval do not consider this important factor, as statutorily the HSE are not able to add a new medicine to the reimbursement list until an application is received.

The HSE has advised that it is committed to providing access to as many medicines as possible, in as timely a fashion as possible, from the resources available (provided) to it.

The HSE must robustly assess applications for pricing and reimbursement to make sure that it can stretch available resources as far as possible and to deliver the best value in relation to each medicine and ultimately more medicines to Irish citizens and patients. HSE decisions on which medicines are reimbursed by the taxpayer are made on objective, scientific and economic grounds. 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.

In terms of the specific details of the application for pricing and reimbursement of Risdiplam (Evrysdi®):

Reimbursement approval is in place for Risdiplam (Evrysdi®) for the treatment of 5q spinal muscular atrophy (SMA) under the High Tech arrangements from the 1st September 2023. Reimbursement support is for a subgroup of the licensed indication.

As a condition of reimbursement, an individual patient approval system has been put in place by the HSE, to enable reimbursement for patients who meet the pre-defined criteria as per a HSE devised managed access protocol.

If the manufacturer of Risdiplam(Evrysdi®) (Roche) submits a de novo application for adult patients with a clinical diagnosis of SMA Type 1, Type 2 or Type 3, that application will be duly progressed through the formal processes governing the pricing and reimbursement of medicines.

In terms of the specific details of the application for pricing and reimbursement of onasemnogene abeparvovec (Zolgensma®):

In October 2021, onasemnogene abeparvovec (Zolgensma®) was approved for reimbursement in Ireland following joint assessment and negotiations under the Beneluxa initiative for the treatment of patients with spinal muscular atrophy (SMA) with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA type 1, or pre-symptomatic patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and up to 3 copies of the SMN2 gene.

As a condition of reimbursement, an individual patient approval system has been put in place by the HSE, to enable reimbursement for patients who meet the pre-defined criteria as per a HSE devised managed access protocol.

Managed Access Protocol – Onasemnogene abeparvovec (Zolgensma®) for the treatment of 5q Spinal Muscular Atrophy (SMA)

In terms of the specific details of the application for pricing and reimbursement of nusinersen (Spinraza®) for adult patients (>18 years):

The HSE approved funding of nusinersen (Spinraza®) effective 1st July 2019 for the treatment of 5q Spinal Muscular Atrophy (SMA) Type I, Type II and Type III in children under 18 years.

The first step in the process is the submission of a Rapid Review dossier (a clinical and economic dossier) to the National Centre for Pharmacoeconomics (NCPE) for assessment. The HSE commissioned the Rapid Review process on the 28th September 2020.

The NCPE Rapid Review assessment report was received by the HSE on the 29th October 2020. The NCPE advised the HSE that a full Health Technology Assessment (HTA) was recommended for nusinersen (Spinraza®) to assess the clinical and cost-effectiveness compared to the current standard of care for adult patients (>18 years).

Nusinersen (Spinraza®). HTA ID: 20044 | National Centre for Pharmacoeconomics

The HSE commissioned a full Health Technology Assessment on the 9th November 2020 as per agreed processes.

The HSE Corporate Pharmaceutical Unit (CPU) is the interface between the HSE and the Pharmaceutical Industry in relation to medicine pricing and reimbursement applications. The CPU met with Biogen regarding their application for Nusinersen (Spinraza®).

The Drugs Group is the national committee which the HSE has in place to make recommendations on the pricing and reimbursement of medicines. The membership of the HSE Drugs Group includes public interest members. The pharmacoeconomic report will be reviewed by the HSE Drugs Group along with the outputs of commercial negotiations, and any patient group submission(s) received. The HSE Drugs Group will consider all of the evidence and make a recommendation to the HSE Senior Leadership Team.

The decision-making authority in the HSE is the HSE Senior Leadership Team. The HSE Senior Leadership Team decides on the basis of all the demands it is faced with (across all services) whether it can fund a new medicine, or new use of an existing medicine, from the resources that have been provided to it in line with the Health (Pricing and Supply of Medical Goods) Act 2013.

A completed HTA is required from the applicant (Biogen) to progress this application, as per the formal processes governing the pricing and reimbursement of medicines. In the absence of a completed HTA the pricing and reimbursement application cannot progress.

The application remains under consideration with the HSE. The HSE cannot make any comment on possible outcomes from the ongoing assessment processes.