Utility Values Associated with Atypical Hemolytic Uremic Syndrome-Related Attributes: A Discrete Choice Experiment in Five Countries

Abstract

Background Atypical hemolytic uremic syndrome is a rare disease caused by complement dysregulation that can lead to progressive kidney damage or death if untreated. Owing to its rarity, the impact of atypical hemolytic uremic syndrome and available therapies (eculizumab and ravulizumab) on patients' health-related quality of life is difficult to describe, but such data are required for an economic evaluation. Objective The objective of this study was to estimate utility values for atypical hemolytic uremic syndrome-related attributes in five countries for an economic evaluation. Methods Using discrete choice experiment surveys, key atypical hemolytic uremic syndrome-related attributes (life expectancy, administration frequency, risk of meningitis, need for hospitalization, and risk of kidney impairment) were evaluated in adult general population samples from Australia, Canada, the Netherlands, Sweden, and the UK. Survey choice sets were constructed using a published orthogonal array. A mixed-effects logit 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 Across all countries (N = 2382), utility weights revealed a consistent pattern: participants were averse to the risk of kidney impairment (disutility/utility weight range: −0.185 to −0.158), risk of meningitis (−0.041 to −0.032), and the need for hospitalization (−0.063 to −0.048), but preferred 8-weekly vs 2-weekly infusions over 1 h (0.013-0.039). Conclusions Although all attributes played a role in determining treatment preferences, the largest drivers were life expectancy and risk of kidney impairment. Participants favored 8-weekly dosing (corresponding to ravulizumab administration frequency) vs 2-weekly dosing. The discrete choice experiment was designed such that estimated (dis)utility weights can be used in future cost-effectiveness models in atypical hemolytic uremic syndrome.

Atypical Hemolytic Uremic Syndrome Treated With Ravulizumab or Eculizumab: A Claims-Based Evaluation of Health Care Resource Utilization and Clinical Outcomes in the United States

“… 16 , 17 The clinical profiles of patients, as indicated by claims for clinical manifestations, showed improvements or were consistent over time, and facility visits were also observed to decrease after the treatment switch, potentially owing to the longer dosing interval of ravulizumab than that of eculizumab, which may have led to fewer visits. Furthermore, a previously published discrete-choice experiment showed that a general population sample preferred ravulizumab dosing regimens over those of eculizumab; in particular, a dosing regimen corresponding to the 100-mg/mL ravulizumab formulation, 32 which confers additional benefits of decreased infusion time and number of vials. 33 Finally, the comparable efficacy of ravulizumab with that of eculizumab shown in an indirect trial comparison and meta-analysis of patients with aHUS 22 , 34 provides important evidence when considering ravulizumab as a treatment option in countries or regions where it has been approved.…”

Estimating utility values for non-alcoholic steatohepatitis health states: a discrete choice experiment

“…They were all aged over 18, had a diagnosis of NASH with their fibrosis stage confirmed by biopsy or fibroscan (participants did not specify which procedure was used for staging), had a fibrosis stage between 1 and 4 (compensated or decompensated) or HCC and lived in the UK or US. No formal sample size estimation was conducted; target sample sizes for each sample were based on sample sizes reported in previous valuation studies [ 24 , 25 ], best practice guidance [ 16 , 26 ] and recruitment feasibility. All eligible participants provided online informed consent.…”

Complement Inhibitors in the Management of Complement-Mediated Hemolytic Uremic Syndrome and Paroxysmal Nocturnal Hemoglobinuria

“…51 Furthermore, a qualitative study regarding patient preference of therapeutic agent found that participants favored administration every 8 weeks compared with every 2 weeks, thus favoring ravulizumab (Table 2). 52…”