Patients who have hemophilia with inhibitors showed an elevated disease-related economic burden in a recent retrospective study of claims data. The study results were presented in a poster at the ISPOR 2023 conference by Sanket Shah, MBBS, PhD, of Novo Nordisk Inc. in Plainsboro, NJ, and colleagues.

Patients in this analysis were diagnosed with hemophilia A or B with inhibitors and were identified from a database of medical and prescription claims from the study period of January 1, 2016, to June 30, 2022. Claims data were obtained from Komodo Health’s Healthcare Map.

Prophylactic use of bypassing agents was used in identifying the presence of inhibitors. In this study, for patients with inhibitors, the research team evaluated hemophilia-related costs, with some analyses based on use of prophylactic or on-demand care. Estimates of mean and median costs involved costs for patients who had 1 or more occurrences of the particular cost.

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The analysis included 33 patients with hemophilia A and fewer than 5 patients with hemophilia B, with all included patients having evidence of prophylactic use of bypassing agents. Patients had a mean age of 32 years (SD, 20) and a median age of 29 years (interquartile range, 16-48).

The mean inflation-adjusted, annual pharmacy cost per patient for those with hemophilia A was $893,704 (median, $438,527). For those with hemophilia B, the mean inflation-adjusted, annual pharmacy cost per patient was $1,378,131 (median, $906,982).

For treatment that was considered prophylactic, the mean annual cost per patient was $979,564 (median, $391,247) for patients with hemophilia A. For those with hemophilia B, the mean annual cost of this type of treatment per patient was $1,330,847 (median, $892,868). For treatment that was identified as being on demand, the mean annual cost per patient with hemophilia A was $86,001 (median, $27,203), while it was $47,284 (median, $37,482) for those with hemophilia B.

Dr Shah and colleagues concluded that with patients who have hemophilia and inhibitors, there is a significant economic burden related to treatment. They also noted that the very small patient population having hemophilia B and inhibitors poses a limitation in interpreting these findings.

In their conclusions, the research team also highlighted a need among patients with hemophilia and inhibitors for more treatment options that are efficacious and cost saving.

Disclosures: This research was supported by Novo Nordisk Health Care AG. Please see the original reference for a full list of disclosures.


Shah S, Waller J, Ibiloye E, Dusendang JR, Patel K. Economic burden in patients with hemophilia with inhibitors: results from a real-world cohort study in the United States. Presented at: the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) 2023 conference; May 7-10, 2023. Abstract EE545.