Cost Effectiveness of FVIII Substitution Versus Non-Factor Therapy for Hemophilia A
With new treatment options for hemophilia A without inhibitors, the question of cost-effectiveness of different treatment approaches arises. The results of a model comparing replacement therapy and emicizumab in terms of cost-effectiveness were presented at a recently held ASH conference.
Introduction
The standard of care for patients with hemophilia A without factor VIII inhibitors is replacement therapy with factor VIII. Recently, therapeutic options have also expanded to include so-called non-factor therapy, represented by emicizumab. It is indicated for the prophylaxis of bleeding in patients with and without inhibitors.
The study presented in December 2019 at the 61st American Society of Hematology (ASH) annual meeting in Orlando modeled the cost-effectiveness of recombinant FVIII replacement therapy (both standard and extended half-life products) administered prophylactically or on-demand versus non-factor therapy (emicizumab) in the USA.
Methodology
A Markov model was utilized, incorporating 5 states related to hemophilia: absence/presence of target joint involvement, absence/presence of arthropathy, and death. Estimated costs and treatment effectiveness were measured using the QALY (quality-adjusted life year) indicator. Data included in the model came from a literature review and a network meta-analysis of clinical trial outcomes. Patient input characteristics, weight change with age, previous treatment (prophylactic/on-demand), annual bleeding incidence, likelihood of developing or regressing target joint involvement or arthropathy, number of infusions to manage one bleed, number of medical examinations, hospitalizations, and mortality were used.
The model operated with patients aged 33 years at model entry, with 72% having target joint involvement and 58% having arthropathy. The model assumed lifelong adherence to the same hemophilia A treatment. The cost of treatment was based on average prices for the year 2018. The model did not include the occurrence of adverse events and inhibitor development.
Results
According to this model, replacement therapy was less financially demanding and more cost-effective than non-factor prophylaxis with emicizumab (lifetime treatment cost of USD 13.6 million versus USD 16.4 million; QALY 17.61 vs. 17.58) with an average life expectancy of 70 years. Prophylactic FVIII substitution was less costly and more cost-effective than on-demand FVIII administration (USD 13.6 million versus USD 13.8 million; QALY 17.6 vs. 12.29).
Conclusion
The results of this model study suggest favorable cost-effectiveness for long-term prophylaxis with FVIII products in patients with severe hemophilia A without inhibitors.
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Source: Sun S. X., Wu Y., McDermott M., van Keep M. Cost-effectiveness model of recombinant FVIII versus emicizumab treatment of patients with severe hemophilia A without inhibitors. Poster 2102. 61st ASH annual meeting, Orlando, Florida, 2019 Dec 7.
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