Campaigners make final appeal to HSE over Skyclarys drug

Advocates for those with Friedreich's Ataxia have once more urged the Health Service Executive (HSE) to approve the Skyclarys treatment prior to a final decision expected next week. This rare, progressive neuromuscular condition impacts approximately 200 individuals in Ireland. Jessie Abbey, a 31-year-old, implored the HSE during a Dublin meeting, stating, "I want to live.

I have everything to live for. I yearn to see my young daughter grow up." She expressed distress at publicly revealing her vulnerability, explaining, "Three years ago, I could walk, but now I'm confined to a wheelchair. What will the next three years hold? I need this drug, I want to live, I cherish my daughter."

Earlier this month, the HSE Drugs Group recommended against covering Skyclarys. A meeting of the HSE senior management team will deliver the final verdict on August 25th. Shanice O Reilly, also living with Friedreich's Ataxia, pleaded with the HSE, "They need to grant us a chance. I've seen individuals in America thriving on the drug.

If it doesn't proceed, there'll be significant disappointment." Isabella Travers, diagnosed at age 12, recounted her experience, "I struggled with balance as a teenager and now rely on a wheelchair. It's disheartening how they deem our lives not worth the investment."

Several opposition parties, including Sinn Féin, Independent Ireland, and Labour, have championed Skyclarys approval, alongside substantial support from Fianna Fáil and Fine Gael. The HSE cited an assessment from the National Centre for Pharmacoeconomics, which found Skyclarys may slow disease progression in Friedreich’s Ataxia, yet noted uncertainties and limitations in the available clinical efficacy data.

At its current price of around €280,000 per patient annually, Skyclarys would impose a five-year budget impact of approximately €130 million. Despite Biogen's engagement with the HSE, the drug has already received approval from the European Medicines Agency and the FDA in the US. Campaigners are set to organize a demonstration at Dublin's Garden of Remembrance this Sunday, advocating for Skyclarys reimbursement.