'This cannot happen again' - Friedreich's Ataxia patients

Patients battling Friedreich's Ataxia are urging for fundamental changes in how rare disease treatments are accessed, following their successful campaign to secure funding for the drug Skyclarys. The Health Service Executive (HSE) announced its approval for the reimbursement of Skyclarys, a decision that followed a significantly improved financial proposal from the drug's manufacturer, Biogen.

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This is going to happen time and time again unless something changes.

Niamh Ní Hoireabhard, a patient with Friedreich's Ataxia and a freelance journalist, expressed deep concern that other individuals with rare diseases might face similar arduous public campaigns to obtain necessary treatments. "This is going to happen time and time again unless something changes," she stated, emphasizing the emotional and personal toll such battles exact.

Ní Hoireabhard described the years of campaigning as all-consuming, impacting her social life and requiring constant engagement with politicians and media. "My social life has taken a hit in the past few months," she admitted. "This cannot happen again. The reimbursement system is not fit for purpose." She noted that while Skyclarys is not a cure, it offers valuable time for patients.

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My social life has taken a hit in the past few months. This cannot happen again. The reimbursement system is not fit for purpose.

Craig Coady, whose son Paudie has Friedreich's Ataxia and whose younger son Rory passed away from the disease eight months prior, shared his profound relief and emotional response to the news. He recounted receiving a call from the Minister for Health, Jennifer Carroll MacNeill, with the announcement. "I just couldn't believe it," he said. "When I saw her number on my phone, my stomach just turned and I thought - this is it." His son's reaction, a hug and a "thank you daddy," brought tears of joy.

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I just couldn't believe it. When I saw her number on my phone, my stomach just turned and I thought - this is it.

Coady reflected on the difficulty of campaigning while grieving his son, describing it as "very unfair" and running on "adrenaline." He stressed the immense burden placed on parents who must advocate publicly for their children's medical needs. "No parent should have to go on social media, or on radio stations to talk about what's personal," he stated, highlighting the need for a more compassionate and efficient system.

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I haven't seen a smile like that on his face in a very long time.