Families Urge Irish Government to Fund Rare Disease Drugs Amid Reimbursement Delays
Families and clinicians in Ireland are demanding more public funding for rare disease drugs, citing lengthy reimbursement delays by the NCPE. Cases like Archie Ennis and Emily Felix highlight the impact of these delays, while the Martin family's story underscores the life-or-death consequences. The NCPE attributes delays and high costs to pharmaceutical companies.
Families, campaigners, and clinicians in Ireland are calling for increased public funding to access drugs for rare diseases, which affect approximately 300,000 people across 6,000 conditions.
Decisions on drug reimbursement, made by the National Centre for Pharmacoeconomics (NCPE), can take years. Archie Ennis, 7, diagnosed with Duchenne Muscular Dystrophy, is denied Givinostat reimbursement due to cost-effectiveness concerns, despite European approval. His mother, Úna Ennis, stresses, «Time is muscle for our little boys, and we need this medication as soon as possible.»
Emily Felix, 28, with Friedreich's Ataxia, was denied reimbursement for Skyclarys, the only approved treatment, despite its availability in other European countries. She described the NCPE's December recommendation against reimbursement as a «slap in the face.»
The Martin family's experience highlights the urgency: Ciarán, diagnosed with Metachromatic Leukodystrophy (MLD) as an infant, received life-saving treatment in Italy before Irish reimbursement, while his older brother Cathal, whose symptoms had progressed, died at six in 2020. The treatment for MLD was reimbursed in Ireland three years after European approval.
Professor Michael Barry, NCPE Director, stated recommendations are based on clinical benefit versus price, not solely cost. He attributed delays and high prices to pharmaceutical companies, citing Skyclarys's 27-month delay since European approval, with NCPE's assessment taking only five months. He emphasized, «It's the value for money. We're saying that these two drugs are not offering value for money. If you reduce the price, then yes, you improve the value for money.»