Cost-utility analysis of primary prophylaxis versus treatment on-demand for individuals with severe haemophilia

Abstract

Objective: To assess the cost effectiveness of primary prophylaxis with clotting factor instead of treatment following a bleed (on-demand) for individuals with severe haemophilia.

Design: Different data sources on the clinical effects and costs of treatments were combined using a Markov model.

Setting: English treatment centres.

Perspective: UK societal.

Participants: Hypothetical cohorts of 100 individuals with severe haemophilia A or B or severe von Willebrands disease.

Interventions: Primary prophylaxis treatment on-demand with clotting factor.

Outcome measures: Costs, quality-adjusted life-years (QALYs) and incremental cost per QALY in UK pounds ( pound, 1999/2000 values).

Results: The baseline results showed that treating individuals with severe haemophilia A/severe von Willebrands disease or severe haemophilia B with primary prophylaxis instead of treatment on-demand cost an additional pound 46500 and pound 8600 per QALY gained, respectively. However, the results were extremely sensitive to a number of factors including the clotting factor unit cost, the time between prophylactic doses and the discount rate.

Conclusions: Despite the high costs of treatment, primary prophylaxis was cost effective compared with treatment on-demand in some scenarios. Primary prophylaxis is more likely to be cost effective for individuals with severe haemophilia B compared with individuals with severe haemophilia A/severe von Willebrands disease. Further research is required to assess the relationship between methods of clotting factor infusion and health-related quality-of-life.