Comparison of EHL Products in the Treatment of Hemophilia A – Pharmacoeconomic Analysis
In the work presented below, whose results were recently published, a model combining pharmacokinetic and pharmacodynamic data was developed. With its help, the authors tried to compare the bleeding rates in hemophiliacs treated with various EHL products. At the same time, they focused on the cost-effectiveness of this treatment.
Treatment Options
The standard care for patients with hemophilia A with bleeding manifestations is prophylaxis, i.e., preventive administration of clotting factor VIII (FVIII) concentrates. Newer FVIII products with an extended half-life (EHL – extended half-life) offer the possibility of less frequent administration compared to the classical FVIII products with a standard half-life (SHL – standard half-life). Due to the relatively short period of use of EHL products, there is currently not much data directly comparing individual products.
Pharmacoeconomic Analysis
Initially, data concerning the population of participants in the phase III Pathfinder 2 study with turoctocog alfa pegol were evaluated, based on which the relationship between plasma FVIII levels and the annual bleeding rate (ABR) in patients with severe hemophilia A was established.
Subsequently, a pharmacokinetic/pharmacodynamic model was developed to estimate FVIII levels for 4 EHL products currently registered by the European Medicines Agency (EMA) and predict comparative ABR. These included the following medicinal products: turoctocog alfa pegol (Esperoct), rurioktocog alfa pegol (Adynovite), efmoroctocog alfa (Elocta), damoctocog alfa pegol (Jivi). FVIII consumption (prophylaxis and bleeding management), treatment costs, and QALY value (quality-adjusted life years; life years gained with consideration to their quality) were estimated with regards to achieving a 70-year horizon and the conditions in the United Kingdom.
Results
Prophylaxis with turoctocog alfa pegol reduced the frequency of bleeding episodes (traumatic and spontaneous) by 8–19% cumulatively compared to the comparators. At the same time, this prophylaxis demonstrated good cost-effectiveness – it reduced bleeding management costs by 9–25% and was associated with the greatest QALY gain compared to other evaluated medications.
Conclusion
Using an analysis based on pharmacokinetic and pharmacodynamic modeling, prophylaxis with turoctocog alfa pegol in adolescent and adult males with hemophilia A was evaluated as more effective and less costly from a pharmacoeconomic perspective compared to other EHL products. For more accurate evaluation, obtaining data for modeling not from clinical trials but from real clinical practice would be advisable.
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Source: Benson G., Morton T., Thomas H., Lee X. Y. Long-term outcomes of previously treated adult and adolescent patients with severe hemophilia A receiving prophylaxis with extended half-life FVIII treatments: an economic analysis from a United Kingdom perspective. Clinicoecon Outcomes Res 2021 Jan 18; 13: 39–51, doi: 10.2147/CEOR.S280574.
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