A new study compared ravulizumab and eculizumab in terms of quality of life (QoL), the occurrence of breakthrough hemolysis (BTH), and cost-effectiveness in patients with paroxysmal nocturnal hemoglobinuria (PNH) who live in the Netherlands.

The study, published in the European Journal of Health Economics, evaluated adult patients with symptoms of high disease activity or who had been treated with eculizumab for at least 6 months.

“Two pivotal clinical trials . . . have shown that ravulizumab is clinically noninferior compared with eculizumab in eculizumab-naive patients as well as eculizumab-experienced and stable patients,” the authors wrote. “However, the reduced infusion frequency and lower rate of BTH events associated with ravulizumab treatment are expected to lead to a gain in QoL for patients.”

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The research team assessed 84 patients, including 7 complement inhibitor-naïve patients and 77 who had been treated with eculizumab for 6 months. They employed a Markov cohort cost-effectiveness model to assess the economics of the 2 therapies, and the model also compared differences in QoL between patients undergoing ravulizumab and eculizumab therapy.

QoL scores were assessed based on the European Organization for Research and Treatment of Cancer questionnaires.

All patients started the model in a “No BTH” state, meaning they were stable on either therapy without BTH events. If a patient on eculizumab experienced a BTH event, their dose was increased, while the dose was not changed in patients on ravulizumab based on clinical studies showing no need to up-dose patients on this treatment.

The results revealed that the lower rate of infusions of ravulizumab (once every 8 weeks compared with once every 2 weeks) had a considerably positive impact on the quality of life of patients on ravulizumab. Furthermore, there were no IncC5Inhib BTH events reported, which is beneficial not only to patients but also to physicians and caregivers.

The authors also found ravulizumab to be cost-saving and more effective, from a Dutch societal perspective.


Quist SW, Postma AJ, Myrén KJ, et al. Cost-effectiveness of ravulizumab compared with eculizumab for the treatment of paroxysmal nocturnal hemoglobinuria in the Netherlands. Eur J Health Econ. Published online January 12, 2023. doi:10.1007/s10198-022-01556-5