Irish patients with Friedreich's ataxia fear delays accessing Skyclarys despite reimbursement approval

Concerns have been raised that Irish patients with Friedreich's ataxia could face delays accessing the long awaited drug Skyclarys despite it being approved for reimbursement. This is because specialist services may not have the clinical and administrative capacity needed to begin treatment. The warning comes as neurologists receive guidance on prescribing Skyclarys ahead of its introduction under the HSE's High Tech Arrangement from Thursday next. Ataxia Foundation Ireland (AFI), which has campaigned extensively for access to the treatment, said the reimbursement decision was a major breakthrough — but that the focus must now shift to ensuring eligible patients can actually start treatment without avoidable delays. The organisation is concerned about capacity for patient assessment, pre-treatment checks, prescribing and ongoing monitoring, particularly as Skyclarys requires regular clinical follow-up and monthly liver-function tests during the first three months of treatment. AFI is warning that without additional resources, patients could face delays simply because specialist services are already operating with limited capacity. Tallaght University Hospital's National Ataxia Clinic is the country's specialist service for inherited cerebellar ataxia. The hospital says approximately 150 patients attend the clinic annually, while its combined neurology and cardiac ataxia clinic operates four times a year and provides neurological and cardiac assessment for patients, predominantly those with Friedreich's ataxia. AFI believes the existing service will need additional capacity as eligible patients come forward for assessment and treatment. The organisation is calling for dedicated neurology, diagnostic and administrative resources, alongside effective use of regional services, to ensure that the introduction of Skyclarys does not create a new bottleneck in the health system. It is also calling for a clear national plan explaining how patients will be identified and assessed, how prescriptions will be issued, how monitoring will be organised and how the medicine will be supplied through community pharmacies. The concerns come after a months-long campaign by patients, families and supporters to secure access to Skyclarys in Ireland. The campaign intensified during the summer, with patients and supporters staging a major demonstration in Dublin in August, marching from the Garden of Remembrance to Custom House Quay and calling for the HSE to fund the treatment. The campaign followed a series of setbacks in the reimbursement process. In July, the HSE Drugs Group declined to recommend Skyclarys for reimbursement and referred the application for further consideration. In August, the Drugs Group again recommended against reimbursement following its review of the medicine. Patients and campaigners continued to lobby politicians and the HSE, with public meetings highlighting the impact of Friedreich's ataxia and the importance of gaining access to a treatment that could slow the progression of the condition. The breakthrough came on August 25, when the HSE Senior Management Team approved reimbursement following a revised financial offer from manufacturer Biogen. The HSE said the decision also reflected the substantial unmet need among people with Friedreich's ataxia and the absence of other medicines to treat the disease. At the same time, it acknowledged the limited efficacy demonstrated by the drug, the high cost and uncertainties in the clinical evidence. . ​​​