In nonsevere hem A, Hemlibra not as cost-effective as standard care
Cost would need to drop by at least 35% to be worth clinical benefits
Preventive treatment with Hemlibra (emicizumab) was not considered cost-effective at its current price compared with standard replacement therapies in adults with mild or moderate hemophilia A without inhibitors, according to a modeling study.
The cost of Hemlibra would need to drop by at least 35% to become cost-effective in these patients, or to be worth its price considering its clinical benefits, calculations showed.
“Our results highlight the need for greater attention to indication-based pricing of novel therapies, such as [Hemlibra], where alternate pricing mechanisms may play a role in achieving cost-effectiveness,” the researchers wrote in “Cost-effectiveness of prophylactic emicizumab versus prophylactic recombinant factor VIII in patients with moderate or mild hemophilia A without inhibitors in the United States,” which was published in the American Journal of Hematology.
Hemlibra is an antibody-based treatment that mimics the action of factor VIII (FVIII), the blood clotting protein that’s either missing or defective in hemophilia A. It’s approved for patients with or without FVIII inhibitors, that is antibodies that may reduce standard hemophilia A replacement therapies‘ effectiveness.
Hemlibra is administered via an injection under the skin, allowing for less frequent and more convenient treatments over replacement therapies that are given as an infusion into the vein.
Its effectiveness with mild or moderate hemophilia A without inhibitors was shown in an ongoing Phase 3 clinical trial called HAVEN 6 (NCT04158648). Over a median follow-up of more than a year, most participants had no bleeds that required treatment.
Comparing cost-effectiveness of Hemlibra, standard of care therapies
Despite its benefits, regular preventative (prophylactic) maintenance dosing with Hemlibra is expensive, costing about $47,000 a month in the U.S. Little is known about its cost-effectiveness with nonsevere hemophilia A without inhibitors, however.
Researchers at Yale School of Medicine in Connecticut constructed a model based on HAVEN 6 participants to compare its cost-effectiveness with prophylactic standard FVIII replacement therapies.
Patients were first assigned one of the two treatments, then entered into the model without bleeding events and no long-term joint damage. From there, they could have no bleeds, experience a newly treated bleeding event with or without joint involvement, undergo joint replacement surgery, or die. Patients received recommended treatment with each bleed, then returned to a no-bleed state.
The primary outcome was the incremental cost-effectiveness ratio (ICER), which compared prophylactic Hemlibra to standard FVIII replacement.
According to the model, those who received Hemlibra saw 62% fewer bleeding events that required treatment over their lifetime than those with standard replacement therapies. Similarly, 64% fewer joint bleeds occurred with Hemlibra versus standard of care.
Hemlibra was associated with a slightly higher number of discounted quality-adjusted life years (QALY) compared with standard of care treatment (18.9 vs. 18.5). QALY is a generic measure of disease burden that considers quality of life and number of years lived, while discounting means future health outcomes are typically valued less than present health outcomes.
We found prophylactic [Hemlibra] to be cost-ineffective compared with standard of care in patients with nonsevere hemophilia A without inhibitors in the United States.
Hemlibra had a higher discounted cost compared with standard therapies ($17.4 million vs. $11.6 million), however.
Together, this resulted in an ICER of $14.5 million per QALY for Hemlibra over standard of care. In a sensitivity analysis, standard FVIII replacement was also favored.
“We found prophylactic [Hemlibra] to be cost-ineffective compared with standard of care in patients with nonsevere hemophilia A without inhibitors in the United States,” the researchers wrote.
This finding remained even if prophylactic Hemlibra were to eliminate chronic joint bleeds. Furthermore, the cost of 1 mg of Hemlibra would need to decrease by about 35% to be considered cost-effective.
“At current pricing, prophylactic [Hemlibra] was found to be a cost-ineffective strategy in patients with moderate or mild hemophilia A without inhibitors over a lifetime time horizon in the United States,” the researchers wrote. “Indication-based pricing across existing expensive medications, such as [Hemlibra] for patients with moderate or mild hemophilia A without inhibitors, would aid the goal of a value-based care transformation of the United States’ health system.”
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