Objectives

Severe hemophilia A (SHA) is a congenital bleeding disorder characterized by severe and repeated bleeding without prophylactic Factor VIII (FVIII) and associated with high treatment costs. This analysis aims at quantifying societal costs associated with SHA without inhibitors among insured adults in the United States.

Methods

Cross-sectional data on insured adults with SHA without inhibitors from the ‘Cost of Haemophilia across the US: a Socioeconomic Survey’ (CHESS US), a physician-reported database, and a separate patient-reported database (CHESS US+) were used to quantify: 1) Direct medical cost (DMC), including healthcare utilization and FVIII costs based on physician-reported data, 2) Direct non-medical cost (DNMC) (e.g., disability entitlement, formal and informal caregiving) and 3) indirect cost (IC), including labor market outcomes based on patient-reported data. Mean (SD) annual costs are reported in 2017 US$ (year data was collected) and summarized by FVIII therapy regimen (on-demand or prophylaxis) and insurance type (private or government insurance).

Results

DMC (N=281) was $493,152 (±$740,396). Among prophylaxis patients (N=209), DMC was $656,707 (±$795,009) with FVIII cost ($639,895[±$796,876]) accounting for 97% of DMC, and hospitalizations representing the largest non-drug cost. DNMC (N=241) was $5,655 (±$18,573), and primarily driven by disability entitlement ($1,916[±$5,689]) and homecare ($2,970[±$16,116]. IC was $8,832 (±$17,616), predominantly driven by early retirement ($6,047[±$16,384]). Prophylaxis patients (N=193) incurred similar DNMC ($6,213[±$19,583]) and IC ($9,529[±$18,249]). While DMC was similar between patients with private and government insurance, DNMC and IC were fourfold higher in government-insured patients.

Conclusion

FVIII cost, particularly among prophylaxis patients, remains the predominant cost-driver from both healthcare system and societal perspectives. DNMC and IC also represent important sources of societal cost, affecting patients, government programs and society, accounting for approximately $15,000 a year. Improved personalized care, care coordination, and development of new treatment options may help to reduce healthcare system and societal cost burden.