Cost-utility analysis of recombinant factor VIIa (NovoSeven((R))) in six children with long-standing inhibitors to factor VIII or IX

The high cost of treating patients with inhibitors in an environment of res tricted budgets warrants consideration of cost-effectiveness. We determined the clinical response, effect on quality of life and the cost-effectivenes s of treatment with rFVIIa in six boys with long-standing inhibitors to fac tors VIII or IX, compared with other treatment regimes previously used in t hese patients. The study used a longitudinal before-and-after design and wa s conducted in three phases. Phase 1 was 6 months preceding the introductio n of rFVIIa, during which patients received on-demand 'usual care' with oth er treatment regimes; phase 2 was 6 months treatment on rFVIIa assessed ret rospectively; and phase 3 was 6 months on rfVIIa treatment assessed prospec tively. Treatment with rFVIIa was reserved for intrarticular, compartment, psoas, mucosal and suspected intracranial bleeding. Treatment outcomes were obtained by interview using structured questionnaires, the quality-of-life instruments CHQ CF-80 and CHQ PF-50, patient self-reporting diary, interro gation of hospital records, and the EuroQoL EQ-5D for utility valuations. O ur results confirm that rFVIIa is clinically effective and resulted in 63-9 2% reductions in the number of re-treatments, duration of painful episodes, delay to initiation of treatment, days requiring wheelchair or crutches, e mergency room visits and lost carer time compared with the patients' other therapies. Quality-of-life improvements were observed in several important areas as perceived by both patients and their families, at an incremental c ost per QALY of A$51 533.