Press release
Minister for Health announces approval of reimbursement for Atidarsagene autotemcel (Libmeldy) through Beneluxa Initiative
From Department of Health
Published on
Last updated on
From Department of Health
Published on
Last updated on
Minister for Health Stephen Donnelly has today announced that the Health Service Executive (HSE) has approved reimbursement for Atidasagene autotemcel, also known by the trade name Libmeldy, for the treatment of metachromatic leukodystrophy (MLD). MLD is a rare disease affecting an estimated 1 in 40,000 – 160,000 people. The Minister is hopeful that access to Libmeldy will make a significant positive impact on the lives of the children with this condition and their families.
Access to this treatment has been made possible through Ireland’s participation in the Beneluxa Initiative, a collaboration between Belgium, the Netherlands, Luxembourg, Austria, and Ireland. On this occasion, Ireland engaged in joint Health Technology Assessment and pricing negotiation for Libmeldy alongside Belgium and the Netherlands. The positive outcome of this procedure is a testament to the potential of collaborative initiatives such as Beneluxa, and the Minister looks forward to future cooperation with Ireland’s Beneluxa partners.
Announcing this positive outcome, the Minister said:
"MLD is a devastating rare disease normally resulting in fatal consequences for the patient – often a child. I am delighted to announce the approval of Libmeldy for reimbursement in Ireland. I believe this offers new hope to families and provides a treatment option that up to now has not been available.
"Partnerships like Beneluxa are critical, particularly when it comes to new medicines for rare diseases. It opens up opportunities to secure greater access to innovative medicines and with the potential to increase the value.
"I want to acknowledge and commend the work of families for tirelessly supporting and advocating for access to this medicine, most especially, Les Martin, who through deep and personal experience understands both the devastating outcome where no treatment is available, and the hope and life changing impact provided by this medicine."
Providing innovative new medicines to patients in Ireland remains a priority for the Minister. Dedicated funding of almost €100 million over the past three years has enabled access to almost 150 new medicines or new uses of existing medicines. This funding is in addition to the record over €3 billion that was spent on medicines in 2023. This represents nearly €1 in every €8 of public funding being spent on health. For 2024, further funding of €20 million has been allocated to broaden the HSE’s access to new medicines. In addition, the Minister has instructed the HSE to realise at least €10 million in efficiencies. This money will then be re-invested in new drugs, including those with an orphan designation, for the benefit of patients with a rare disease.
While individual rare diseases are, by their nature, rare, there are more than 6,000 known rare diseases. The number of people living with a rare disease in Ireland has been estimated at 1 in 15. Innovative treatments for these diseases offer valuable assistance to these people, and 39 such treatments have been made available in Ireland since 2021.
The State also aims to support people living with rare diseases in ways beyond pharmaceutical treatments. In February 2023, the Minister announced the development of a new National Rare Disease Plan, as committed to in the Programme for Government. The National Rare Disease Steering Group to progress this was recently established and has a diverse membership including patient advocates, clinicians, the Health Service Executive, and the Department of Health. The new plan is expected to launch in Autumn 2024.