CADTH Reimbursement Recommendation - Ravulizumab (Ultomiris) - Indication: For the treatment of adult patients with anti–aquaporin 4 (AQP4)

The proportions of patients with clinically important worsening from baseline through the end of the study period in HAI score were 3.4% (2 patients of 58) in the ravulizumab arm and 23.4% (11 patients of 47) in the placebo arm. [...] The use of ravulizumab may result in a clinically important reduction in the proportion of patients with worsening from baseline in EDSS score at the primary data cut-off compared to placebo; however, the evidence is very uncertain because the CI for the difference between groups includes the possibility of no difference. [...] The clinical experts indicated that loss of function and quality of life is cumulative over time, and that the magnitude of worsening depends on the severity of the relapse; therefore, measurement of these outcomes may be less sensitive to changes in the context of a clinical trial. [...] CADTH’s review of the sponsor’s NMA concluded that ravulizumab may perform better in some contexts compared to other comparators, but the quantification of benefit over the comparators is not clear due to the limitations of the NMA and the wide confidence intervals around the estimates. [...] This disclaimer and any questions or matters of any nature arising from or relating to the content or use (or misuse) of this document will be governed by and interpreted in accordance with the laws of the Province of Ontario and the laws of Canada applicable therein, and all proceedings shall be subject to the exclusive jurisdiction of the courts of the Province of Ontario, Canada.