OBJECTIVES

: Ravulizumab, the first FDA-approved long-acting complement inhibitor treating atypical hemolytic uremic syndrome (aHUS), was developed from eculizumab to improve patient outcomes while reducing dosing frequency. This US study compared the cost of infusions of ravulizumab and infusions of eculizumab for the treatment of aHUS.

METHODS

: A cost-minimization model compared direct medical costs (including treatment, administration, monitoring and relapse management) for ravulizumab and eculizumab treatment in patients with aHUS, assuming equivalent efficacy and safety. The model took a US payer perspective and used a lifetime horizon and 3.5% discount rate. Base-case individuals were assumed to be treatment-naïve and 28 years old at treatment initiation. Patient characteristics were based on clinical trial data and treatment patterns (discontinuation, relapse and re-initiation) were derived from long-term follow-up study data for eculizumab. Treatment costs (2019 US$) were based on wholesale drug acquisition costs, fee schedules from Centers for Medicare & Medicaid and published studies on disease management. Deterministic sensitivity analyses were conducted by adjusting variables, including discount rate, time horizon, age at treatment initiation and proportion of treatment-naïve versus experienced patients.

RESULTS

: Base-case analyses demonstrated ravulizumab lowered lifetime per-patient costs by 32.4% (discounted) and 32.6% (undiscounted) versus eculizumab. Discounted total costs for ravulizumab and eculizumab were $12,451,923 and $18,426,888, respectively, and undiscounted costs $29,800,045 and $44,221,747, respectively. Average pre-discontinuation treatment costs (discounted) contributed the largest proportion of total costs for both ravulizumab ($11,175,823; 89.8% of total costs) and eculizumab ($16,538,692; 89.8%). Sensitivity analyses demonstrated that ravulizumab lowered costs versus eculizumab, regardless of patient populations (including pediatric patients) or treatment strategies.

CONCLUSIONS

: In US adults with aHUS, ravulizumab provided cost savings of 32% versus eculizumab, with a reduced dosing frequency for ravulizumab. The magnitude of savings was consistent across sensitivity analyses, including in a pediatric population.