OBJECTIVES Ravulizumab, a treatment for atypical hemolytic uremic syndrome (aHUS), was engineered from eculizumab to leverage the transformative clinical benefits and safety profile provided, while decreasing drug clearance and reducing infusion frequency (every 8 weeks [q8w] vs 2 weeks [q2w], respectively). We estimated utility values for aHUS treatment attributes in Australia, Canada, the Netherlands, Sweden, and the UK for economic modelling.

METHODS A discrete choice experiment (DCE) was developed based on a literature review and discussion with experts. The DCE evaluated preferences for aHUS treatment attributes in the adult general population of each country, including reduction in overall survival, risk of kidney impairment due to aHUS, risk of meningitis, need for hospitalization and administration frequency. Choice sets were constructed using a published orthogonal array. A mixed effects logit regression model estimated preference strength for each attribute. Utilities were estimated using marginal substitution rates between overall survival and other attributes, weighted against average life expectancy.

RESULTS Participants were from Australia (n=477), Canada (n=471), the Netherlands (n=481), Sweden (n=476) and the UK (n=477). Excluding life expectancy, the risk of kidney impairment had the strongest influence on treatment preference across countries. Participants were adverse to risk of kidney impairment ([dis]utility = –0.166 [Australia]; –0.185 [Canada]; –0.176 [Netherlands]; –0.185 [Sweden]; −0.158 [UK]); risk of meningitis (–0.041 [Australia]; –0.032 [Canada]; –0.034 [Netherlands]; –0.035 [Sweden]; −0.034 [UK]); need for hospitalization (–0.050 [Australia]; –0.057 [Canada]; –0.063 [Netherlands]; –0.048 [Sweden]; −0.056 [UK]), and preferred q8w versus q2w infusions (0.020 [Australia]; 0.014 [Canada]; 0.016 [Netherlands]; 0.039 [Sweden]; 0.013 [UK]).

CONCLUSIONS We evaluated public preferences for aHUS treatment attributes, and estimated country-specific utility weights. While participants favoured q8w dosing, life expectancy and risk of kidney impairment were the largest drivers of treatment preference. These utility weights can be used in future cost-utility models supporting the reimbursement of ravulizumab.