OBJECTIVES:

Differences exist in the various treatment options for haemophilia B (HB) in terms of cost and efficacy. An analysis was undertaken to estimate the cost-effectiveness of prophylaxis with extended half-life recombinant factor IX Fc fusion protein (rFIXFc) compared to on-demand treatment with standard half-life recombinant factor IX (rFIX).

METHODS:

A Markov model was designed to compare lifetime costs and health outcomes between rFIXFc prophylaxis (once weekly and individualized interval) and on-demand rFIX. Adolescent and adult males (≥12 years) with severe HB (defined as ≤2 IU/dL) without inhibitors were modeled. Costs were reported from the perspective of the Italian National Health Service and patient characteristics were sourced from the B-LONG trial. Total costs accounted for prophylactic treatment, bleeding management (drugs and procedures), and surgery. Health outcomes were estimated as quality-adjusted life-years (QALYs). One-way deterministic sensitivity analyses and probabilistic sensitivity analyses were performed.

RESULTS:

Prophylaxis with rFIXFc was associated with lower costs and greater number of QALYs. Prophylactic treatment with rFIXFc was also associated with fewer bleeds compared to rFIX given on demand. rFIXFc was the dominant strategy over rFIX across most uncertain parameters. In the probabilistic sensitivity analysis (1,000 simulations), rFIXFc once weekly and individualized interval prophylaxis were the dominant strategies in 61% and 53% of the runs, respectively.

CONCLUSIONS:

In comparison with on-demand rFIX, prophylaxis with rFIXFc was the dominant treatment strategy. Prophylactic rFIXFc is likely to be more effective, cost-saving, and associated with fewer bleeds compared with on-demand rFIX.