Approvals and Pricing of Factor VIII and Factor IX Concentrates for Hemophilia A and B in the United States

  • Oak B
  • Eguale T
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Objectives: To describe approvals and assess price trends of Factor VIII (FVIII) and Factor IX (FIX) concentrates used in patients with Hemophilia A & B in the United States (US). Methods: Relevant data on US Food and Drug Administration (FDA) approval dates and product information of FVIII and FIX concentrates were obtained from the US FDA website and product labels until December 2017. Earliest and latest available average wholesale prices (AWPs) per International Unit (IU) were obtained from Red Book (Truven Health Analytics) in US dollars ($) and adjusted for inflation. Approval trends were analyzed per decade by type and indication of factor concentrates. Effects of type of product, time of approval, half-life and orphan drug designation on mean earliest AWP/IU were analyzed using non-parametric tests. Results: Seventeen FVIII and ten FIX concentrates were approved in the US as of December 2017. Earliest plasma-derived FVIII concentrate was approved in 1966 and FIX concentrate in 1970. There has been a shift towards recombinant products in the last three decades. Most recent AWP/IU ranged from $1.35-$2.56 for FVIII concentrates and $1.31-$5.28 for FIX concentrates. Mean AWP/IU was significantly lower for plasma-derived factor concentrates as compared to recombinant products (p< 0.05). Mean AWP/IU was significantly higher for FVIII and FIX products approved after 2000 compared to those approved before 2000 (p< 0.05). FIX concentrates with half-life > 30 hours had significantly higher AWP/IU than those with ≤ 30 hours (p< 0.05), while there were no significant differences in AWP/IU of FVIII concentrates based on half-life. Both FVIII and FIX concentrates with orphan status had numerically higher mean AWP/IU. Conclusions: The evidence-based push towards prophylactic treatment and chronic nature of hemophilia poses increased economic burden on patients and payers, and more competitive prices need to be implemented to differentiate between comparable factor concentrates in the market.




Oak, B., & Eguale, T. (2018). Approvals and Pricing of Factor VIII and Factor IX Concentrates for Hemophilia A and B in the United States. Value in Health, 21, S252–S253.

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