Economic evaluation of ruxolitinib versus best available therapy for treatment of steroid-refractory acute graft-versus-host disease in Singapore

Abstract

Abstract Background Acute graft-versus-host disease (aGvHD) is a complication of allogeneic hematopoietic stem cell transplantation (allo-HSCT) that typically develops within 100 days of transplantation. Current treatments for steroid-refractory aGvHD (SR-aGvHD) are poorly tolerated and are often ineffective. In the REACH2 open label randomized controlled trial, ruxolitinib was found to lead to significantly higher overall response rates at 28 days than best available therapy (BAT). A cost-effectiveness analysis was conducted to determine the value of ruxolitinib compared with BAT for treatment of patients with SRaGvHD in Singapore. Methods A semi-Markov model was developed in Microsoft Excel® to capture all costs and outcomes associated with ruxolitinib compared with BAT for the treatment of patients with SRaGvHD. The model used the Singaporean healthcare system perspective with a time horizon of 15 years and 3% discount rate in the base case analysis. The efficacy and safety inputs in the model were based on the data from the REACH2 trial. The distribution of comparator treatments within the BAT and local costs were informed by inputs provided by local clinicians in Singapore. Probabilistic sensitivity analysis (PSA), one way sensitivity analysis (OWSA) and scenario analysis were conducted to determine the robustness of the model outcomes. Results In the base case analysis, over a 15-year time horizon, ruxolitinib was found to be more effective (0.18 more life years (LYs) gained, 0.15 more quality adjusted LYs (QALYs) gained) while costing less (SGD 31,079) than BAT. PSA found that ruxolitinib was less costly and more effective than BAT in 93.8% of iterations. OWSA found that ruxolitinib was less costly and less effective when the Weibull shape parameter used to extrapolate overall survival in non-responder (SGD 1,211,562/QALY) and overall survivors (SGD 896,543/QALY) were varied. In all scenarios explored ruxolitinib dominated BAT, in alignment with the base case. Conclusion Overall, this economic evaluation suggests that ruxolitinib is likely to be cost-effective for the treatment of SR-aGvHD in Singapore when compared against BAT.