Overview
- The HSE Drugs Group did not approve public funding and sent the Skyclarys application to the Rare Diseases Technology Review Committee for further expert advice.
- The HSE says it aims to complete the RDTRC review and return a recommendation within about one month.
- The National Centre for Pharmacoeconomics recommended against reimbursement in December after finding the drug's clinical benefit did not justify the price sought by the manufacturer.
- Patients and families protested outside Leinster House and campaigners said delays are harming people with Friedreich's Ataxia, who number roughly 200 in Ireland and face progressive loss of mobility.
- Skyclarys is the first EU‑authorised treatment for Friedreich's Ataxia, has shown trial evidence of slowing symptom progression, is publicly funded in several European countries, and costs about €280,000 a year which factors heavily in the value‑for‑money debate.