Economic Burden of Illness among Persons with Hemophilia B from HUGS Vb: Examining the Association of Severity and Treatment Regimens with Costs and Annual Bleed Rates

Value Health. 2017 Sep;20(8):1074-1082. doi: 10.1016/j.jval.2017.04.017. Epub 2017 Jun 17.


Objectives: To determine US societal burden of illness, including direct and indirect costs and annual bleed rate (ABR), for persons with hemophilia B (HB), a rare and debilitating genetic disorder, and to examine associations of hemophilia severity and treatment regimens with costs and ABR.

Methods: From 2009 to 2014, the Hemophilia Utilization Group Studies Part Vb collected prospective data from 10 US hemophilia treatment centers. Participants with HB completed initial surveys on sociodemographic characteristics, clinical characteristics, and treatment patterns. During the 2-year follow-up, participants reported bleeding episodes, work absenteeism, and caregiver time quarterly. These data were used to calculate ABR and indirect costs. Direct costs were calculated using 1-year clinical chart records and 2-year dispensing records.

Results: Of the 148 participants, 112 with complete medical records and one or more follow-up survey were included. Total mean annual per-person costs were $85,852 (median $20,160) for mild/moderate HB, $198,733 (median $147,891) for severe HB, and $140,240 (median $63,617) for all participants without inhibitors (P < 0.0001). Mean ABR for participants with severe HB on prophylaxis (5.5 ± 7.9 bleeds/y) was almost half that of those treated episodically. Clotting factor and indirect costs accounted for 85% and 9% of total costs, respectively. Compared with episodic treatment, prophylaxis use was associated with 2.5-fold higher clotting factor costs (P < 0.01), low but significantly more missed parental workdays (P < 0.0001) and clinician (P < 0.001) or nursing visits (P < 0.0001), less part-time employment and unemployment, and lower hospitalizations costs (P = 0.17) and ABR (P < 0.0001).

Conclusions: HB is associated with high economic burden, primarily because of clotting factor costs. Nevertheless, prophylaxis treatment leads to clinical benefits and may reduce other nonfactor costs.

Keywords: burden of illness; economic outcome; prospective studies; rare diseases.

Publication types

  • Multicenter Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Absenteeism
  • Adolescent
  • Adult
  • Blood Coagulation Factors / administration & dosage*
  • Blood Coagulation Factors / economics
  • Caregivers / statistics & numerical data
  • Child
  • Child, Preschool
  • Cost of Illness*
  • Employment / statistics & numerical data
  • Female
  • Follow-Up Studies
  • Health Care Costs / statistics & numerical data*
  • Hemophilia B / economics
  • Hemophilia B / physiopathology
  • Hemophilia B / therapy*
  • Hemorrhage / economics
  • Hemorrhage / etiology
  • Hemorrhage / therapy*
  • Hospitalization / economics
  • Hospitalization / statistics & numerical data
  • Humans
  • Male
  • Middle Aged
  • Prospective Studies
  • Severity of Illness Index
  • Surveys and Questionnaires
  • United States
  • Young Adult


  • Blood Coagulation Factors