Economic Burden in Patients with Hemophilia with Inhibitors: Results from a Real-World Cohort Study in the United States


Shah S1, Waller J2, Ibiloye E3, Dusendang JR4, Patel K4
1Novo Nordisk, Inc., Gaithersburg, MD, USA, 2Novo Nordisk Health Care AG, Zurich, Switzerland, 3Novo Nordisk, Inc., Plainsboro, NJ, USA, 4PicnicHealth, San Francisco, CA, USA

Presentation Documents


Approximately 13.0% and 2.6% of patients with hemophilia A and B respectively are estimated to have inhibitors (HAwI/HBwI) (Puetz J et al. Haemophilia, 2014), a subtype of hemophilia with limited and expensive treatment options and higher disease burden. This study aims to use retrospectively captured claims data to describe the economic burden in these patients.


Patients with ≥2 outpatient or ≥1 inpatient visit claims with a hemophilia diagnosis and closed medical and pharmacy claims for ≥12 continuous months between 1 January 2016 to 30 June 2022 (study period) were identified from Komodo Health's Healthcare Map of de-identified, patient-level medical and prescription claims data. Among these patients, HA/HB patients were identified as those with ≥1 visit claim with a diagnosis of hemophilia A or hemophilia B respectively, and patients with inhibitors were identified as those with prophylactic use of bypassing agents during the study period. Analyses of hemophilia-related costs among HAwI/HBwI patients were performed, and pharmacy claims were considered prophylactic if there were 6 consecutive claims for the same medication after accounting for days supply prescribed and allowing for gaps of <60 days. Any costs that did not fit the definition of prophylaxis (at least 6 consecutive claims) were included in on-demand treatment costs.


Thirty-seven patients with HAwI/HBwI (33 and 4, respectively) were identified. Average, inflation-adjusted, annual pharmacy costs per patient were $893,704 (standard deviation: $1,122,136; median: $438,527) and $1,378,131 ($1,160,404; $906,982) for HAwI and HBwI, respectively. Annual costs averaged $979,564 ($1,165,719; $391,247) and $1,330,847 ($1,119,784; $892,868) for prophylactic treatment, and $86,001 ($123,204; $27,203) and $47,284 ($47,594; $37,482) for on-demand treatment per patient with HAwI and HBwI, respectively.


Patients with HAwI/HBwI experience significant economic burden associated with disease treatment, both in ongoing management and response to bleeding episodes. There is a need for more cost-saving, efficacious treatment options for HAwI/HBwI patients.

Conference/Value in Health Info

2023-05, ISPOR 2023, Boston, MA, USA

Value in Health, Volume 26, Issue 6, S2 (June 2023)




Economic Evaluation, Real World Data & Information Systems, Study Approaches

Topic Subcategory

Health & Insurance Records Systems


No Additional Disease & Conditions/Specialized Treatment Areas

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