Cost-effectiveness analysis of alternative factor VIII products in treatment of haemophilia A

Manufactured factor VIII (FVIII) concentrates of varying purity are availab le for managing patients with haemophilia A. This study is a cost-effective ness analysis of ultra-high purity and recombinant (UHP/R) FVIII products r elative to intermediate and very-high purity (IP/VHP) preparations. Because the societal (including research and development) costs of FVIII products are unknown and product prices vary with market conditions, we conducted th e analysis with treatment cost as a variable quantity. We estimated the lar gest price premium that could be paid for a UHP/R concentrate relative to a n IP/VHP concentrate such that the UHP/R product is the more cost-effective preparation. In the analysis haemophilic patients were assumed to be serop ositive for human immunodeficiency virus, seropositive for hepatitis C (HCV ), or at risk for seroconversion of hepatitis A (HAV) or hepatitis B (HBV). The results showed that the maximum cost-effective UHP/R price premium is essentially zero if the patient is only at risk of HAV or HBV infection, po sitive but small for the base-case HCV+ patient, and positive and large for the base-case HIV+ patient having a short life expectancy. Thus UHP/R prep arations are not uniformly more cost-effective than IP/VHP products across the spectrum of haemophilic patients' health problems, and the relative cos t-effectiveness of the two classes of prepared FVIII products is sensitive to product prices. The methodology employed here can be used in other circu mstances where multiple treatments exist for illnesses for which there are significant multiple comorbidities or health risks.