[Prophylactic factor substitution in severe haemophilia A. Economic assessment in adult patients]

Prophylaxis in adults can be necessary and reasonable for clinical reasons. The aim was to evaluate from an economic viewpoint prophylactic factor VIII substitution in adult patients with haemophilia in Germany.

A decision model (time frame: one year; perspective: statutory health insurance; reference patient (RP) and 2 patient profiles) was developed. Calculations are based on data from a structured literature search and a pharmacovigilance study: therapy switch on-demand/prophylaxis (OD/Proph).

RP: 45 years, 20 bleeds p.a. OD, 16 bleeds avoided with 8.5 I.U./kg/d Proph, additional cost Euro 141,113 p.a.; profile 1: 50 years, 55 bleeds p.a. OD, factor consumption per bleed 20 I.U./kg higher than RP, 39 bleeds avoided with 8.5 I.U./kg/d Proph, additional cost Euro 19,134 p.a.; profile 2: 60 years, 35 bleeds p.a. OD, factor consumption per bleed 40-80 I.U./kg higher than RP, 34 bleeds avoided with 11 I.U./kg/d Proph, cost reduction Euro 660 p.a.

Prophylactic factor VIII substitution in adult haemophilia patients is depending on the individual clinical situation not only clinically but also economically reasonable. To evaluate this effects in the future comprehensively, longitudinal real-life data from patient-centered care are needed including clinical outcomes, quality of life and adherence.