Treatment of hemophilia A with bypassing agents is associated with significantly higher costs than with factor replacement therapy, according to a real-world study in the U.S.
The study, “Healthcare costs among patients with hemophilia A treated with factor replacement or bypassing agents,” appeared in the journal Current Medical Research and Opinion.
Factor replacement therapy in patients with hemophilia A may lead to the development of inhibitor antibodies against factor VIII (FVIII), the protein missing in these patients. Clotting agents that bypass inhibitors, aptly called bypassing agents, are among the treatment options in these cases.
Prior research on hemophilia A-related healthcare costs did not include recently available treatments and may not reflect current practice. These studies also did not focus on female patients, who are less likely to develop hemophilia A than males.
Researchers in this study addressed these issues by analyzing healthcare costs and resource use of males and females with hemophilia A treated with either factor replacement or bypassing therapy from January 2007 to August 2014.
The Genentech-sponsored study analyzed claims data for people with commercial or Medicare Advantage with Part D prescription drug coverage, using the Optum Research Database with information from a large U.S. health insurer.
Treated patients were identified based on one or more medical claims with a diagnosis code for hemophilia A and one or more medical or pharmacy claim for bypassing agents — recombinant factor VIIa and activated prothrombin complex concentrate (brand name Feiba) — or FVIII replacement.
All-cause costs, adjusted to 2014 U.S. dollars, included health plan and patient paid amounts, and calculated the sum of all medical claims — such as ambulatory, emergency services, inpatient and in-home visits, skilled nursing facilities, and long-term care — and all pharmacy claims. Hemophilia-related costs used medical claims with a diagnosis code for hemophilia A, as well as medical or pharmacy claims for hemophilia therapy
A total of 580 patients were included (64.7% adults), 50 of whom had one or more claims for bypassing agents, while the remaining 530 were treated with factor replacement therapy.
Overall, 510 patients were male, 38 were treated with bypassing agents, representing 76% of this group’s total, and 472 with factor replacement, making up 89.1% of the group.
Mean all-cause costs at the beginning of the study period were $19,378 for bypassing therapy and $8,172 for factor replacement. Approximately 90% of the costs in either group were hemophilia-related — $18,129 for bypassing agents and $7,200 for factor replacement therapy. More than 90% of these initial costs were attributed to medical expenses.
Over the study period, the mean total per-patient and per-month costs were $60,062 for bypassing agents and $13,141 for FVIII replacement. In both groups, these costs mostly came from in-home visits (38.7% and 58.5%, respectively), inpatient hospital costs (27.3% and 10.9%), and outpatient pharmacy expenses (20.1% and 14.4%).
Compared with factor replacement, bypassing therapy had a 4.6 times greater all-cause cost and 4.8 times higher hemophilia-related cost. This is in line with previous studies, according to the team. Bypassing agents were also linked to four times higher mean medical costs, 11-12 times higher mean inpatient hospital costs, and 6-7 times higher pharmacy costs.
For patients who were followed for at least one year, all-cause costs were 5.2 times greater among the 30 on bypassing therapy ($804,514) compared with the 376 on FVIII replacement ($155,733). Hemophilia-related costs represented 95.9% of the mean costs in the bypassing therapy group and 88.6% in patients on factor replacement.
“Patients with hemophilia A treated with bypassing agents between 2007 and 2014 incurred substantially higher monthly hemophilia-related medical and pharmacy costs than patients treated only with FVIII replacement,” the scientists wrote.
Of note, three of the study’s authors were employees of Genentech at the time of study.