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

Abstract

Manufactured factor VIII (FVIII) concentrates of varying purity are available for managing patients with haemophilia A. This study is a cost-effectiveness analysis of ultra-high purity and recombinant (UHP/R) FVIII products relative 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 the analysis with treatment cost as a variable quantity. We estimated the largest price premium that could be paid for a UHP/R concentrate relative to an IP/VHP concentrate such that the UHP/R product is the more cost-effective preparation. In the analysis haemophilic patients were assumed to be seropositive 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, positive 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 preparations are not uniformly more cost-effective than IP/VHP products across the spectrum of haemophilic patients' health problems, and the relative cost-effectiveness of the two classes of prepared FVIII products is sensitive to product prices. The methodology employed here can be used in other circumstances where multiple treatments exist for illnesses for which there are significant multiple comorbidities or health risks.

Cost‐utility of a six‐month programmed sports therapy (PST) in patients with haemophilia

“…35 Available studies in haemophilia investigated only fragments of the impact of sports therapy on the use of healthcare services or focused on the cost-effectiveness of on-demand factor treatment in comparison with prophylactic therapy. [16][17][18][19] Our results on the use of factor treatment are comparable with the study of Czepa et al, 20 in which an increase in the coagulation factor over a one-year sports therapy was not documented, either. Consequently, both studies independently from each other show, that an individual sports therapy does not lead to a rise in the coagulation factor.…”

“…In addition, it is not possible to compare results with cost‐utility analyses in diseases showing similar symptoms, for example osteoarthritis, because studies apply different time horizons and include older patients . Available studies in haemophilia investigated only fragments of the impact of sports therapy on the use of healthcare services or focused on the cost‐effectiveness of on‐demand factor treatment in comparison with prophylactic therapy . Our results on the use of factor treatment are comparable with the study of Czepa et al, in which an increase in the coagulation factor over a one‐year sports therapy was not documented, either.…”

“…With regard to the limited financial resources, the determination of costs of haemophilia treatment is important, particularly in order to save expenses without a decline of effectiveness. Various health economic studies in PwH are related to the coagulation factor and the medical preparations . To date, investigations of the influence of sports therapeutic options on healthcare costs (medical and nonmedical) as well as productivity losses (indirect costs) in PwH are still lacking.…”

“…Various health economic studies in PwH are related to the coagulation factor and the medical preparations. [16][17][18][19] To date, investigations of the influence of sports therapeutic options on healthcare costs (medical and nonmedical) as well as productivity losses (indirect costs) in PwH are still lacking. In their study on the impact of 1-year sports therapy programme on physical performance in adult haemophilia patients, Czepa et al had a closer look at the association of sports therapy and factor substitution.…”

An adventure in biotechnology: the development of haemophilia A therapeutics – from whole‐blood transfusion to recombinant DNA to gene therapy

“…However, there are still issues with respect to use of concentrates developed with advanced technologies as well as gene therapy. With the use of ultrapure therapeutic Factor VIII concentrates (including those derived from recombinant DNA technology) steadily increasing, it is important to understand that other therapeutic concentrates of Factor VIII can be more cost‐effective, retaining patient safety [166]. Indeed, an article by Jean‐Pierre Allain [167] observes that cryoprecipitation remains an effective therapeutic option.…”