Non-factor vs. Factor Prophylaxis of Hemophilia A: A Comparison of Cost and Treatment Effectiveness Based on Real-World Data from the USA
In the prophylactic treatment of patients with hemophilia A, particularly its severe form, concentrates of clotting factor VIII (FVIII) with extended half-life (EHL) are increasingly administered, or non-factor treatment, represented in clinical practice by FVIII mimetic antibody emicizumab. However, insights into the direct comparison of effectiveness and economic burden arising from prophylaxis with these modalities are limited, including data from real-world practice. One recently published study therefore attempted to conduct such a retrospective comparison based on data from healthcare-related databases in the USA.
Methodology of Evaluation and Analyzed Data
The study included 101 males with hemophilia A without inhibitors, aged 3–63 years, who were switched to emicizumab in 2018–2019. The data assessed covered at least 6 months before switching to emicizumab. FVIII consumption before switching to emicizumab had to be at least 2340 IU/kg/year (equivalent to an average dose of 45 IU/kg/week, meeting the definition of continuous FVIII prophylaxis). Patients’ weights were not known, so age-specific national averages were used for calculations.
Within the analysis, the so-called billed ABR (ABRb), that is, annualized bleeding rate episodes identified through billing for suspected bleeding, bleeding therapy, and other bleeding-related examinations, was calculated. Another evaluated variable was the total cost of care (TCC), which included all healthcare costs, both related and unrelated to hemophilia. To compare differences in ABRb and TCC before and after switching to emicizumab, Bayesian inferences, especially posterior probabilities of the average change in ABRb or TCC, were used.
Results
According to the cost analysis, ABRb increased from 0.52 to 0.62 (p = 0.83) after transitioning to this treatment. The posterior probability that the average ABRb would increase after switching to emicizumab was 75.4%.
TCC increased from 517 thousand to 627 thousand USD per patient per year. Here, the posterior probability of increased average costs after switching the patient was 99.8%.
Conclusion and Discussion
The authors conclude that they did not find evidence of improved ABRb after switching to emicizumab in the context of prophylaxis, while the cost of care significantly increased.
Nonetheless, this analysis underscores the fact that identifying suitable patients for a given type of treatment is crucial, as it can help optimize both clinical and cost outcomes.
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Source: Escobar M., Agrawal N., Chatterjee S. et al. Impact of switching prophylaxis treatment from factor VIII to emicizumab in hemophilia A patients without inhibitors. J Med Econ 2023; 26(1): 574–580, doi: 10.1080/13696998.2023.2196922.
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