Takeda has presented new real-world data to support the cost-effectiveness and persistent benefits to patients of its hemophilia A and B therapies: Adynovate [antihemophilic factor (recombinant), PEGylated] and Feiba (anti-inhibitor coagulant complex).
The findings were discussed at the recent 61st American Society of Hematology Annual Meeting in Orlando, Florida.
One study, highlighted in the poster “Real-World Age-Stratified FVIII Consumption and Bleed Outcomes Before and After Switching to Rurioctocog Alfa Pegol in a Retrospective, Observational Study Using US Specialty Pharmacy Data,” showed that people with hemophilia A who switched to preventive treatment with Adynovate experienced a 40% to 50% decrease in their annual bleed rates.
The study analyzed data from 82 patients in the U.S., 36 of whom were children or teenagers under 18, who had been on prophylactic treatment with another FVIII therapy (with a standard or extended half-life) for at least 90 days, before switching to Adynovate.
Compared with any prior FVIII therapy, moving to Adynovate decreased mean frequency of bleeds by 39.5% in patients under 18 and by 50.3% in adult patients. In addition, Adynovate increased FVIII dose per administration, while reducing the frequency of weekly administrations by 21.4% in patients younger than 18 and by 28.1% in adults.
“These findings from real-world data are in agreement with TAK-660 [Adynovate] clinical trial results,” the researchers said.
In the poster “Cost-Effectiveness Model of Recombinant FVIII Versus Emicizumab Treatment of Patients With Severe Hemophilia A Without Inhibitors,” researchers presented a study that compared the cost-effectiveness of replacement therapy with recombinant (lab-made) FVIII products versus non-factor therapy with Roche‘s Hemlibra (emicizumab) for prophylaxis treatment of people with severe hemophilia A without inhibitors (antibodies that block treatment).
Considering a 70-year lifespan, treatment with recombinant FVIII was estimated to be less costly and more effective — corresponding to a total cost of $13,656,238 and yielding 17.61 quality-adjusted life years (QALYs; a measure of health outcome that incorporates the impact on both quantity and quality of life).
In comparison, treatment with Hemlibra had a total cost of $16,447,843 and provided 17.58 QALYs.
Here, total costs include those related to prophylactic treatment ($12,850,894 for rFVIII vs. $15,555,379 for emicizumab) and those with healthcare resources ($805,344 for rFVIII vs. $892,464 for emicizumab).
The data suggest that preventive therapy with rFVIII is a more cost-effective approach for long-term treatment of patients with severe hemophilia without inhibitors.
A different study, titled “Real-World Clinical Management of Patients with Hemophilia and Inhibitors: Effectiveness and Safety of aPCC in Patients with >18 Months’ Follow-up in the FEIBA Global Outcome Study (FEIBA GO),” describes the long-term safety and effectiveness of Feiba‘s use in clinical practice.
The study, called the FEIBA Global Outcome study, is still ongoing and will follow patients on Feiba for four years.
The preliminary data presented refers to 52 patients with congenital hemophilia A and one with congenital hemophilia B who have been followed for more than 18 months. The data was consistent with the experience and “continues to validate the safety and effectiveness” of Feiba, the researchers wrote.
Of 15 patients who received prophylaxis with Feiba, four (26.7%) reported three or fewer bleeding episodes per year, and seven (46.7%) reported yearly joint bleeds of three or less.
A total of 139 non-serious treatment-related adverse events were reported in 29 patients. A total of 40 serious side-effects were reported in 17 patients. No thrombotic microangiopathies, a blood clot obstruction of certain blood vessels, were reported.
“Real-world evidence plays a crucial role in understanding patterns of care that happen in day-to-day medical practice outside of rigorous clinical studies,” Jonathan Roberts, MD, associate medical director of Bleeding & Clotting Disorders Institute, said in a press release.