Hemophilia Burden of Disease: A Systematic Review of the Cost-Utility Literature for Hemophilia.

BACKGROUND: Prophylaxis with clotting factor replacement products is recommended by the Medical and Scientific Advisory Council of the National Hemophilia Foundation as the optimal therapy for the prevention of bleeding episodes in individuals with severe hemophilia A or B (< 1 IU per dL endogenous factor VIII or factor IX activity, respectively). Prophylaxis is associated with an improved health-related quality of life and has been shown to be cost-effective compared with on-demand therapy. However, the overall cost of treatment remains high, particularly among patients with a greater propensity to bleed. The overall value of hemophilia treatments and their associated benefits, measured in quality-adjusted life-years (QALYs), and dollar costs compared with other interventions can be evaluated through the use of cost-utility analyses (CUAs). Previous CUA studies in hemophilia have focused primarily on patients with more severe forms of hemophilia and on prophylaxis compared with on-demand treatment. However, to our knowledge, no studies to date have utilized QALYs as a standardized outcome measure to systematically evaluate the relative cost-effectiveness of current hemophilia treatment options.

OBJECTIVE: To systematically review the CUA literature of hemophilia treatments and demonstrate the challenges in producing cost-utility evidence compared with other rare diseases.

METHODS: We conducted a systematic literature review using the Tufts Medical Center Cost-Effectiveness Analysis Registry and the National Health Service Economic Evaluation Database for English-language CUAs published from 2000 through 2015 with the search terms hemophilia, haemophilia, factor VIII, or factor IX. Two trained reviewers independently reviewed every study to extract relevant data. Incremental cost-effectiveness ratios were converted to 2014 U.S. dollars using exchange rates for currency conversion and the Consumer Price Index to adjust for inflation.

RESULTS: Our search yielded 52 studies, 11 of which met our inclusion criteria. The cost-effectiveness of hemophilia treatments varied widely based on variations in the study designs, including differences in time horizon, discount rates, and medical interventions.

CONCLUSIONS: We found the cost-effectiveness of hemophilia treatments to be broadly comparable to that of other orphan drugs. Improved standardization of future CUA studies will be important for further evaluation of the cost-effectiveness of hemophilia treatments.

DISCLOSURES: This research was funded by Biogen, which provided an unrestricted research grant to the Center for the Evaluation of Value and Risk in Health at Tufts Medical Center. Biogen and Sobi reviewed and provided feedback on the manuscript. The authors had full editorial control of the manuscript and provided final approval of all content. The authors report no conflict of interest regarding the material discussed in this article. Neumann and Chambers are employed at the Center for the Evaluation of Value and Risk in Health at Tufts Medical Center. Thorat was an employee of Center for Evaluation Value and Risk in Health, Tufts Medical Center when the analyses were carried out. Chambers has participated on advisory boards for Sanofi and Astellas Pharma.