Cost Effectiveness of Ruxolitinib for Treatment of Steroid Refractory Acute Graft Versus Host Disease in Patients -12 Years of Age from a Singapore Healthcare System Perspective

OBJECTIVES: Acute graft-versus-host disease (aGvHD) is a complication of allogeneic hematopoietic stem cell transplantation (allo-HSCT) which contributes to post transplant morbidity and mortality. In the REACH2 open label randomized controlled trial, treatment of steroid refractory (SR) aGvHD with ruxolitinib led 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 SR-aGvHD from a Singaporean healthcare system perspective.

METHODS: A semi-Markov model was developed in Microsoft Excel® to compare direct costs and outcomes associated with ruxolitinib and BAT for the treatment of SR-aGvHD patients. The model used a 15-year time horizon with half-cycle correction and a 3% discount rate. Efficacy and safety inputs used in the model were informed by the REACH2 trial. Local clinicians’ inputs were sought for distribution of comparator treatments included in BAT and local costs. Probabilistic sensitivity analysis (PSA) and one way sensitivity analysis (OWSA) were conducted to determine the robustness of the model outcomes. Scenario analysis tested modeling assumptions including the use of a societal perspective.

RESULTS: In the base case analysis, over a 15-year time horizon, ruxolitinib dominated BAT, leading to 0.18 more life years (LYs) gained and 0.15 more quality adjusted LYs (QALYs) gained at lower costs (31,079 Singapore Dollars). PSA found that ruxolitinib dominated BAT in 93.8% of iterations. In OWSA, ruxolitinib dominated BAT in most variations, except when the Weibull shape parameter used in survival extrapolation in either non-responders or overall survivors was varied. In all scenarios explored ruxolitinib dominated BAT, in alignment with the base case.

CONCLUSIONS: Compared to BAT, Ruxolitinib is likely to be cost-effective from a Singapore healthcare system perspective for the treatment of SR-aGvHD in Singapore.