OBJECTIVES: aHUS is a rare disease characterized by a dysregulated and overactive complement system. Prior to the introduction of eculizumab, a complement C5-inhibitor, outcomes for patients with aHUS were generally poor. Eculizumab has substantially improved patient prognosis, but requires biweekly infusions that can impose a large treatment burden for patients and caregivers. Ravulizumab was designed via targeted modification of eculizumab to reduce dosing frequency to every 4 to 8 weeks, with an immediate, complete and sustained inhibition of complement C5. The aim of this study was to evaluate the economic impact of patients with aHUS switching from eculizumab to ravulizumab in the Netherlands from a societal perspective.

METHODS: A previously published US cost-minimization model was adjusted to the Dutch setting to compare ravulizumab with eculizumab in patients with aHUS. The model considered adult and paediatric treatment-naïve patients with aHUS. The primary outcomes were the average lifetime medical costs (treatment [drug acquisition, administration], monitoring, treatment of acute kidney injury) and societal costs (costs for patients and family [travel , informal care], productivity losses).

RESULTS: Ravulizumab produced lower lifetime medical and societal costs per patient than eculizumab in adult patients with aHUS (€6,200,521 vs. €8,289,022 respectively), reducing total costs by 25% (€2,088,501). In the paediatric population, ravulizumab cost €2,175,929 (24%) less per patient over a lifetime horizon. The estimated cost-savings were predominantly driven by a reduction in total treatment costs (€2,103,922 and €2,190,676 in adult and paediatric patients respectively). Compared to eculizumab, ravulizumab reduced the societal costs for patients and family in adult patients by 74.4% (€31,504) and in paediatric patients by 75.6% (€45,791). Moreover, in the adult population, ravulizumab lowered societal costs associated with productivity losses by 74.4% (€13,508).

CONCLUSIONS: Ravulizumab is cost-saving compared to eculizumab for long-term treatment of both adult and pediatric patients with aHUS in the Netherlands.