Cost-effectiveness of bevacizumab therapy in the care of patients with hereditary hemorrhagic telangiectasia.

No FDA or EMA approved therapies exist for bleeding due to hereditary hemorrhagic telangiectasia (HHT), the second-most-common inherited bleeding disorder worldwide. The current standard-of-care (SOC) includes iron and red cell supplementation, alongside the necessary hemostatic procedures, none of which target underlying disease pathogenesis. Recent evidence has demonstrated that bleeding pathophysiology is amenable to systemic antiangiogenic therapy with the anti-VEGF bevacizumab. Despite its high cost, the addition of longitudinal bevacizumab to the current SOC may reduce overall healthcare resource utilization and improve patient quality-of-life. We conducted the first cost-effectiveness analysis of IV bevacizumab in patients with HHT with the moderate-to-severe phenotype, comparing 1) bevacizumab added to SOC versus 2) SOC alone. The primary outcome was the incremental net monetary benefit (iNMB) reported over a lifetime time horizon and across accepted willingness-to-pay thresholds, in USD per quality-adjusted-life-year (QALY). Bevacizumab therapy accrued 9.3 QALYS while generating $428,000 in costs, compared to 8.3 QALYs and $699,000 in costs accrued in the SOC strategy. The iNMB of bevacizumab therapy versus the standard of care was $433,000. No parameter variation and no scenario analysis, including choice of iron supplementation product, changed the outcome of bevacizumab being a cost-saving strategy. Bevacizumab therapy also saved patients an average of 133 hours spent receiving HHT-specific care per year of life. In probabilistic sensitivity analysis, bevacizumab was favored in 100% of all 10,000 Monte Carlo iterations across base-case and all scenario analyses. Bevacizumab should be considered for more favorable formulary placement in the care of patients with moderate-to-severe HHT.