Preventive treatment may cut hospital costs for acquired hemophilia
Early Hemlibra shortens stays, reduces need for other meds in acquired hem A
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Upfront treatment with Hemlibra (emicizumab-KXWH) may help reduce hospitalization costs associated with treating bleeds in older adults with acquired hemophilia A (AHA), according to a study.
While Hemlibra itself carries a hefty price tag, economic models indicate that using the therapy may help shorten hospital stays and reduce the need for other medications, keeping total costs down while improving patients’ quality of life.
The researchers noted that Hemlibra is not commonly used outside of specialty centers, largely due to its cost. They said their models “provide strong economic and clinical justification for pharmacy and therapeutics (P&T) committees to consider up-front [Hemlibra] use and include it in hospital formularies, regardless of practice setting.”
“We strongly recommend that hospital P&T committees incorporate [Hemlibra] into their formularies, as this represents a cost-effective strategy that offers immediate benefits in [bleeding] control,” the team wrote.
The study, “Cost-effectiveness analysis of emicizumab use in hospitalized older individuals with acquired hemophilia A,” was published in Blood VTH.
Effective but expensive
AHA is a rare autoimmune disease driven by the production of antibodies that attack a clotting protein called factor VIII (FVIII), preventing it from working properly and putting patients at an increased risk of bleeding. Unlike other types of hemophilia that are present from birth, AHA usually develops later in life.
Hemlibra is an antibody-based medication that works by mimicking the action of FVIII in the body, restoring clotting activity. The therapy is approved in the U.S. to prevent or reduce bleeding frequency in people with hemophilia A, a genetic form of hemophilia driven by a deficiency in FVIII. In the U.S., Hemlibra is not indicated for the treatment of AHA, though it is approved for this indication in Japan.
Clinical trials in people with hemophilia A have shown that Hemlibra can help reduce bleeding. However, individual vials of Hemlibra cost thousands of dollars.
“Due to the high cost of [Hemlibra], hospital administrators are hesitant to include it on the hospital formulary, and its cost-effectiveness remains uncertain,” the researchers wrote.
The team of U.S. scientists constructed economic models to test how adding Hemlibra would affect healthcare costs for people with AHA who are hospitalized for bleeding.
The models compared costs in two situations: one in which patients received only standard-of-care therapy, and another in which patients received Hemlibra in addition to standard therapy. The researchers said they designed their models to err on the side of giving an advantage to the standard of care alone.
Roche, the company that sells Hemlibra, was not involved in the analysis.
Results showed that total costs for standard of care alone were $569,038. By comparison, when Hemlibra was used, total costs were less than half of that, at $248,934.
Data indicated that Hemlibra may help shorten hospital stays and reduce the need for other treatments to control bleeding. The addition of Hemlibra also led to more quality-adjusted life days, a measure that combines length and quality of life, suggesting better overall health outcomes for patients given the therapy.
“Our finding is unsurprising, as both clinical trials and real-world studies have consistently shown that [Hemlibra] decreases use of costly hemostatic agents, shortens hospital length of stay, and reduces infection- and bleed-related mortality,” the researchers wrote. “Yet, despite robust evidence of clinical value, its high list price has persisted as a major obstacle to hospital formulary inclusion, primarily owing to its substantial up-front budget implications. Our results therefore support that up-front [Hemlibra] use improves clinical outcome while simultaneously [reducing] overall hospital expenditure.”
The researchers noted that they built their economic models based on available data on the use of Hemlibra in AHA, but because AHA is a very rare disease, there were limits to the data available. The scientists highlighted a need for continued research to assess the economic and clinical impact of Hemlibra in AHA.
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