The potential of individualized dosing of ravulizumab to improve patient‐friendliness of paroxysmal nocturnal haemoglobinuria treatment at reduced costs

Ravulizumab is a very expensive complement C5‐inhibitor for the treatment of paroxysmal nocturnal haemoglobinuria, with a fixed‐dosing interval of 8 weeks. For lifelong treatment, a cost‐effective and patient‐friendly dosing strategy is preferred. We therefore explored alternative ravulizumab dosing regimens in silico based on the thorough dose‐finding studies of the manufacturer. Extending the interval to 10 weeks or individually extending the interval to a mean of 12.8 weeks based on pharmacokinetic monitoring resulted in noninferior efficacy in terms of lactate dehydrogenase normalization, with drug cost savings up to 37%. We here show the potential of individualized ravulizumab dosing to improve patient‐friendliness at reduced costs.