Overview
- The HSE Drugs Group recommended on Wednesday that Skyclarys should not be reimbursed by the public health service and has sent its advice to the HSE senior management team for a final decision on 25 August.
- The National Centre for PharmacoEconomics says Skyclarys is not cost‑effective at the current price, which is estimated at about €280,000 per patient per year and projects roughly €130–160 million in budget impact over five years.
- The Rare Diseases Technology Review Group recommended introducing the drug because of unmet need and disease severity, but the Drugs Group weighed that alongside the NCPE economic appraisal and evidence on clinical benefit.
- Around 200 people in Ireland live with Friedreich’s Ataxia and campaigners say the recommendation is devastating; one patient, Emily Felix, has been granted High Court permission to challenge the HSE over access delays.
- Biogen says it is still engaging with the HSE, and officials have signalled that only a substantial price reduction or a new commercial agreement would bring the drug within Ireland’s usual reimbursement thresholds, while some countries such as Portugal have already reimbursed it.