OBJECTIVES: Ruxolitinib is a targeted therapy approved for the treatment of intermediate and high-risk myelofibrosis that has been shown to improve survival and quality-of-life. The aim of this study was to assess the cost-effectiveness of ruxolitinib versus the best available therapy (BAT).

METHODS: COMFORT-II trial data was used to compare the survival of ruxolitinib versus BAT treatment. A Markov model with three health states (on-treatment, off-treatment, dead) was created for a ruxolitinib treatment and BAT arm using TreeAge Pro. A U.S. healthcare perspective was adopted with a willingness-to-pay (WTP) threshold of $150,000/QALY. A lifetime horizon of 15 years was used with a 28-day cycle length with costs and utilities discounted by 3%. Data on treatment discontinuation for ruxolitinib and BAT were estimated by calibrating a Gompertz distribution to the proportion of COMFORT-II patients who discontinued treatment at the 3 and 5-year time points and wide ranges were used in the on-to-off treatment transition probabilities in sensitivity analyses. Utility weights derived from a standard gamble approach were adopted for the model and direct medical costs were synthesized from the literature and updated to 2021 USD using the consumer price index medical component. One-way sensitivity analyses and probabilistic sensitivity analyses (PSA) were conducted.

RESULTS: Ruxolitinib treatment was expected to generate 4.71 QALYs at a cost of $1,107,203 while BAT was expected to generate 1.85 QALYs at a cost of $426,355 resulting in an incremental cost effectiveness ratio (ICER) of $238,474/QALY. None of the one-way sensitivity analyses resulted in an ICER < $150,000/QALY and the PSA revealed that ruxolitinib had an ICER < $150,000/QALY in 0.7% of iterations.

CONCLUSIONS: This analysis found that ruxolitinib may extend quality adjusted survival by almost three years but at current prices is unlikely to be a cost-effective option to treat myelofibrosis compared to BAT in the U.S.