Rebinyn cost-effective relative to other FIX therapies, study finds

'Robust' drop in yearly treatment costs tied to lesser use of effective therapy

Lindsey Shapiro, PhD avatar

by Lindsey Shapiro, PhD |

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Switching to Rebinyn (nonacog beta pegol) from other standard or extended half-life replacement therapies led to “robust” reductions in annual treatment costs among hemophilia B patients in Canada, a study based on real-world data reported.

Its findings add to an earlier and real-world analysis of patients who moved to Rebinyn, which reported similar or better bleed control relative to their previous factor IX (FIX) therapy and a lower overall use of treatment.

“Cost savings realized with [Rebinyn] are robust,” the scientists wrote, suggesting the therapy will become “a dominant treatment choice … meaning better patient outcomes at a reduced cost to the health care system.”

The study, “Treatment switch to nonacog beta pegol factor IX in hemophilia B: A Canadian cost-consequence analysis based on real-world factor IX consumption and clinical outcomes,” was published in the journal Research and Practice in Thrombosis and Haemostasis.

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Study in hemophilia B patients who switched to Rebinyn

FIX replacement therapy is the mainstay preventative (prophylactic) or on-demand treatment for hemophilia B. It works by supplying patients with a working version of factor IX, the blood clotting protein they lack.

Standard versions of these therapies require into-the-vein infusions once or twice a week when used to prevent bleeding episodes. Newer extended half-life (EHL) versions remain in the bloodstream longer, allowing for less frequent infusions, generally once every one or two weeks.

Rebinyn, available as Refixia in Europe, is an approved EHL-FIX replacement therapy marketed by Novo Nordisk.

Prior to Rebinyn’s emergence on the Canadian market in 2018, many patients there were using standard replacement therapies like BeneFix (nonacog alfa), or an EHL such as Alprolix (eftrenonacog alfa).

Canada Blood Services, which regulates the availability and pricing of blood products and treatments for patients in the country’s health system — except for Quebec, which has its own service — mandated a switch to Rebinyn for people using Alprolix in 2018. A similar switch was optional for those on standard FIX therapies.

The earlier real-world analysis of hemophilia B patients, using data from the Canadian Bleeding Disorders Registry (CBDR) and published in 2022, associated switching from BeneFix or Alprolix to Rebinyn with optimal bleed control and a lower use of FIX therapies.

Specifically, moving from a standard therapy like BeneFix was associated with a significant reduction in annualized bleed rates, from a mean of 7.2 to 3.1. A switch from extended half-life Alprolix showed a nonsignificant trend toward lower annual rates, from a mean of 3.4 to 2.2. In both cases, the overall use of FIX therapy fell significantly after the switch.

In an extension of that 2022 study, researchers at McMaster University in Ontario, joined by scientists with Novo Nordisk, now examined whether lower therapy use led to lower hemophilia-associated annual treatment costs.

Their analysis also used data covering 42 males with hemophilia B, ranging in age from 7 to 72; all had been using Rebinyn for at least six months after switching from another replacement therapy. Their median time of treatment with Rebinyn was 2.3 years, and they had used a previous FIX therapy for a median of 1.4 years.

Twenty-six of these patients previously were taking Alprolix, and 16 had used BeneFix. Among the entire group, 62% had severe hemophilia B, 36% had moderate disease, and the remaining 2% had mild disease.

Annual treatment costs 10.5% lower overall moving from Alprolix to Rebinyn

Reductions in overall FIX usage — taking into account both preventive and on-demand treatment of bleeds — linked with lower treatment costs, according to the analysis.

Among patients switching from BeneFix, there was a 43% reduction in treatment costs associated with breakthrough bleeds and a 3.1% reduction in costs for preventive treatment. This amounted to a 9.4% reduction in overall annual FIX treatment costs, the scientists reported.

Switching from Alprolix to Rebinyn led to a 42.3% reduction in costs of treating breakthrough bleeds, and a 6.5% reduction in the costs of preventive treatment, yielding a 10.5% reduction in overall annual FIX treatment costs.

“In terms of improving bleeding outcomes and reducing treatment costs, the results of the analysis favored [Rebinyn] … suggesting that, from a cost-consequence perspective, [Rebinyn] may be considered a dominant treatment option,” the researchers wrote.

To validate their findings, an additional cost-effectiveness analysis was conducted using the published prices of FIX therapies in Germany, converted to Canadian dollars. The exact pricing of FIX therapies in Canada is not released, so the researchers had worked with “hypothetical, threshold-set costs” for the therapies.

Annual treatment costs calculated using converted German prices fell by 11.5% overall among those switching from BeneFix and by 17% for patients moving from Alprolix.

Researchers also reported that the price of Rebinyn could increase by up to 121% relative to BeneFix, and by 40% relative to Alprolix, before it would no longer offer a comparative cost savings.

At that point, “[Rebinyn] remained the dominant treatment choice because there was cost neutrality but fewer bleeds,” the researchers wrote.

“The results of this study indicate that [Rebinyn] is a treatment option for all patients with hemophilia B,” they concluded, adding that findings of this and the earlier study support both therapy effectiveness and cost being part of policy decisions for treatment approvals.

“Despite likely being well managed with their previous EHL,” analysis of the 26 patients moving from Alprolix to Rebinyn showed “cost savings were still realized in this patient cohort as a result of decreased overall ABR [annualized bleed rate] and overall decreased FIX consumption after the switch.”