Switching to Hemlibra Can Add Significant Costs for Patients in US

Hemlibra may increase annual cost by $100K without 'substantial clinical benefit'

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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Switching to Hemlibra (emicizumab) can add more than $100,000 a year to healthcare costs for people with hemophilia A in the U.S. who were previously using preventive replacement therapies, according to a new analysis.

“Increased use of [Hemlibra] may cause an increase in economic burden for payers without providing a substantial clinical benefit, substantively impacting their ability to manage the health of their patients,” researchers wrote. “This evidence can aid providers and payers to better understand the economic impact of switching to [Hemlibra] and meaningfully engage in shared decision-making process to improve patient care.”

The study, “A real-world study comparing pre-post billed annualized bleed rates and total cost of care among non-inhibitor patients with hemophilia A switching from FVIII prophylaxis to emicizumab,” was published in the journal Current Medical Research and Opinion.

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The study was funded by Takeda Pharmaceuticals, which markets the approved hemophilia A replacement therapies Advate (octocog alfa) and Adynovate (rurioctocog alfa pegol). Hemlibra is marketed by Roche, which was not involved in this analysis.

Hemophilia A is caused by the lack of a clotting protein called factor VIII (FVIII). Factor replacement therapies are a standard treatment that involves administering a working version of this protein. In some patients, however, the effectiveness of these treatments may be reduced by the development of inhibitors, or neutralizing antibodies targeting the delivered FVIII.

How does Hemlibra work?

Hemlibra is an alternative therapy made up of a specially designed antibody that mimics the normal function of FVIII, thereby preventing or lowering the frequency of bleeds in hemophilia A patients. It is approved for hemophilia A patients with or without FVIII inhibitors.

In the study, scientists at Takeda and other institutions conducted an analysis of U.S. insurance data to evaluate the economic impact of switching from replacement therapy to Hemlibra.

“New non-factor replacement therapies, such as [Hemlibra] entering the market merit understanding the depth of clinical benefits relative to the economic burden placed on the healthcare system, payers, providers, and patients,” the researchers wrote.

The team noted that while the economic benefits of Hemlibra are established for patients with inhibitors, they are less clear for those without inhibitors.

Using data from the IQVIA PharMetrics Plus database from 2015 to 2020, the researchers identified 121 people with hemophilia A who switched from factor replacement to Hemlibra for prophylactic treatment. Prior to switching, the mean dose of replacement therapy was 82.8 international units, per kilogram of body weight, per week.

All of the patients were male with no FVIII inhibitors, and their mean age was about 26 years. The mean observation time was 2.5 years before they made the switch to Hemlibra and 1.1 years after. Nearly half (46%) of the patients had joint pain, 28% had soft tissue disorders, 14.1% had hepatitis B or C, and 9.1% were positive for the human immunodeficiency virus.

Using statistical models, the researchers compared yearly rates of billed bleeds, or bleeds that required medical attention that resulted in a bill. This rate decreased slightly after patients switched to Hemlibra, but this difference from rates seen while patients were on factor replacement was not considered statistically significant.

What happened to billed bleeds after patients switched to Hemlibra?

Models indicated the annual rate of billed bleeds had a 21% chance of worsening after patients switched to Hemlibra. There was also a 22% chance that the rate would not change and a 57% chance that it would improve following the switch, though the predicted magnitude of improvement was relatively small (generally less than 0.5 billed bleeds/year).

“The 21.0% potential worsening of the disease with [Hemlibra] needs to be carefully weighed, especially for patients who are well-controlled on FVIII prophylaxis,” the researchers wrote.

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In other analyses, they looked at the total cost of care, which included all medical expenses related and unrelated to hemophilia.

In these models, the probability that the total cost would increase after patients switched from factor replacement to Hemlibra was about 99.9%, and the probability that total cost would increase by more than $100,000 was nearly 90%.

The mean yearly total cost of care increased from $518,151 per patient per year on factor replacement, to $652,679 on Hemlibra. The researchers noted the vast majority of all costs were attributable to hemophilia treatments.

“The 99.9% probability of cost increase after switching to [Hemlibra] is important to understand amidst the increasingly complex treatment landscape of this already very costly disease with restrictive payer environment,” the researchers wrote.

The team added that, based on the relatively modest effects on billed bleeding rates, “the substantial cost increase seen with switching to [Hemlibra] is not offset by a clinical benefit” for these inhibitor-negative patients.

They also noted that this analysis of insurance data is limited because insurance datasets do not contain detailed clinical information, and cannot capture events that are not severe enough to require medical attention. This is particularly noteworthy given that the study included the early stages of the COVID-19 pandemic, when many people were trying to avoid going to the hospital unless absolutely necessary.