Noel Grealish (Galway West, Independent)

I thank the Senator for raising this issue. I am answering on behalf of the Minister for Health.

Duchenne muscular dystrophy is a rare disease affecting approximately 200 people in Ireland. Its symptoms can severely impact the lives of those living with the disease and their families. The State acknowledges the importance of access to medicines and is aware of the circumstances of patients with DMD. As the Senator will be aware, the Minister met with the families of children with DMD and their representative body, Muscular Dystrophy Ireland, in July to discuss their plight and provide support. The State has made considerable investments in new medicines in recent years. In 2023, nearly €1 in every €8 of public funding being spent on health was spent on medicines. This level of investment is unprecedented in supporting patients through the availability of new and innovative medicines. Budgets 2021 to 2024 have included dedicated funding for new medicines of €128 million. Budget 2025 allocated €30 million of the funding available for new drugs to be generated by efficiencies to be identified by the HSE. From 2021 up to June 2025, this has enabled the HSE to approve reimbursement for 219 new medicines or new uses for existing medicines, including 91 for the treatment of cancer and 55 for the treatment of rare diseases.

Under the Health (Pricing and Supply of Medical Goods) Act 2013, the HSE has statutory responsibility for decisions on pricing and reimbursement of medicines.The Act provides a rigorous process for the assessment of new medicines for reimbursement. This ensures the right medicines are available and approval is at a sustainable price.

Givinostat, sold under the brand name Duvyzat, is a medication used to treat Duchenne muscular dystrophy, DMD, in ambulant patients aged six years and older. Conditional marketing authorisation was granted for givinostat on 6 June 2025 by the European Commission.

The Minister for Health met with her Italian counterpart at the EPSCO Council meeting in June in Luxembourg to ask him to encourage the company to submit a pricing and reimbursement application to Ireland. The HSE has also proactively engaged with the company to strongly encourage it to submit a pricing and reimbursement application.

On 6 August 2025 a pricing and reimbursement application from the marketing authorisation holder for givinostat was made to the HSE with an indication for the treatment of DMD in ambulant patients aged six years and older. Following the conclusion of a rapid review by the NCPE on 14 August 2025, a full health technology assessment, HTA, was commissioned by the HSE on 26 August 2025. The pricing and reimbursement assessment is ongoing. A HTA is a detailed evaluation of medical technologies, including medicines, to determine their effectiveness, safety, cost-efficiency and broader impact on the health budget and service. A HTA takes time to evaluate as it involves gathering and analysing complex clinical and economic data, consulting stakeholders and aligning with regulatory standards to ensure decisions are evidence-based and transparent.

To support this process, there has been significant investment to enhance the HSE's pricing and reimbursement process for medicines. Thirty-four new staff were hired across the pricing and reimbursement system in 2024 which will support faster access to new and innovative medicines for patients. In addition, to support transparency in this process, an application tracker has also been developed and launched.