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Cost-effectiveness of on-demand rFVIIa vs prophylactic emicizumab in hemophilia a with inhibitors: clinical, QoL, and ICER-based field insights
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Abstract<sec> <title>Objective

To evaluate the cost-effectiveness of emicizumab compared to recombinant activated factor VII (rFVIIa) in Iraqi patients with hemophilia A and inhibitors.

Method

A retrospective cost-effectiveness analysis was conducted on 46 male patients with hemophilia A and inhibitors treated at a public children’s hospital in Baghdad. Data collection was conducted between November 2024 and March 2025. Clinical and economic data were retrospectively collected for a 12-month follow-up for each of rFVIIa and then emicizumab, including bleeding episodes, emergency visits, treatment costs, and health-related quality of life assessed via EQ-5D-5L (adults) and EQ-5D-Y-3 L proxy (children). Quality-adjusted life years (QALYs) and incremental cost-effectiveness ratios were calculated.

Results

Emicizumab improved mean utility scores from 0.10 to 1.00 in children and from 0.32 to 0.89 in adults, corresponding to an incremental cost of –$2726.36 and –$4304.78 per QALY, respectively (indicating cost savings). Annual bleeding episodes dropped from 8.70 to 0, saving $282.04 per episode. Hospitalization costs decreased significantly, resulting in a total saving of $286650.41 per hospitalization prevented. Use of emicizumab also reduced overall healthcare resource utilization, eliminating emergency department visits, and minimizing diagnostic procedures.

Conclusion

Emicizumab delivers substantial clinical and economic benefits over rFVIIa. Its capacity to enhance quality of life while reducing treatment costs positions it as a cost-effective option for adults and children with hemophilia A and inhibitors in resource-constrained settings. Health authorities should prioritize emicizumab over rFVIIa for patients with hemophilia A and inhibitors, due to its superior efficacy and cost-effectiveness.

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