Father of boy with Friedreich’s ataxia devastated as HSE recommends against drug funding

A father whose son suffers from Friedreich’s ataxia expressed deep disappointment after the Health Service Executive (HSE) recommended against funding a newly approved treatment for the rare genetic disorder. The recommendation comes despite the drug being approved in both the United States and Europe within the past year. The drug, known as omaveloxolone and marketed under the brand name Skyclarys, was approved in the U.S. in February 2023 and subsequently in Europe 12 months later. While it is not a cure, it has shown potential in slowing the progression of Friedreich’s ataxia by up to 55 percent, according to patient advocacy groups. Around 200 people in Ireland live with the condition, and many have been pushing for the HSE to cover the costs of the medication. The HSE Drugs Group's recommendation will now be reviewed by a senior management team meeting scheduled for August 25. Craig Coady, whose younger son Rory died from Friedreich’s ataxia last September, shared his anguish over the decision. His older son, Paudie, who also has the condition, had hoped to benefit from the drug. Coady described the emotional impact of the news on his son, saying Paudie’s reaction was devastating. “When I told Paudie his face just dropped,” Coady said during an interview on RTÉ Radio 1’s Morning Ireland. “I feel I’ve let him down.” He urged Taoiseach Micheál Martin and Health Minister Jennifer Carroll MacNeill to intervene and overturn the decision, emphasizing that the drug works even though it is not a cure. Michael Barry, clinical director of the National Centre for Pharmacoeconomics, explained that the HSE Drugs Group’s recommendation was based on both economic assessments and evaluations of the drug’s effectiveness. He noted that the group took into account inputs from the Rare Diseases Technology Review Committee, which included perspectives from patients, patient representatives, and clinical experts. Barry highlighted the complexity of the decision-making process, acknowledging the challenges faced by the HSE in allocating resources among various treatments. He mentioned that while the HSE has reimbursed other drugs for rare diseases, the current situation involves balancing the effectiveness of Skyclarys with its cost. “There’s huge demand on the HSE in relation to cancer therapies and other drugs for rare diseases,” Barry said. “It’s trying to balance all these things.” He emphasized that the decision was not solely based on economic grounds but also on the drug’s effectiveness and the lack of alternative treatments. However, Barry pointed out that the HSE does not determine the pricing of medications. That responsibility falls to the manufacturer, Biogen, which sets the price and provides the necessary evidence for approval. Despite the recommendation, some stakeholders remain hopeful that the situation can be resolved. Coady believes there is still hope and is urging political leaders to take action. Meanwhile, the final decision rests with the HSE senior management team, which will meet on August 25 to review the recommendation. The outcome of this meeting could significantly affect the availability of Skyclarys for patients in Ireland, particularly those like Paudie Coady, who stand to benefit from the treatment. The HSE’s decision will likely influence future discussions around funding for similar rare disease treatments.