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Economic Burden of Hemophilia B Patients in the United States: A Systematic Literature Review

Speaker(s)

Pathak S1, Solanki GS1, Aggarwal A1, Wadgaonkar M1, Dhone DH1, Goyal R2
1IQVIA, Gurgaon, India, 2IQVIA, Gurugram, India

Presentation Documents

Objectives: Hemophilia B (HB) is a rare X-linked congenital bleeding disorder characterized by a deficiency of coagulation factor IX (FIX). Diagnosed and undiagnosed prevalence rates per 100,000 males are 2.57 and 1.14 for HB in 2020, USA, respectively. Though HB affects only a small portion of population, it imposes high overall cost and financial burden on individuals, payers, and society in general. Current systematic literature review (SLR) aimed to summarize the economic burden reported in patients with HB.

Methodology: An extensive literature search was conducted via Ovid on MEDLINE, EMBASE and Cochrane databases by combination of Emtree/MeSH terms and keywords from inception to December 2021. Bibliography of included studies and supplementary searches were also conducted for additional publications. Publications were screened for eligibility by two reviewers at abstract and full-text level. Observational studies investigating expenditure data for HB patients in USA were included.

Results: Ten studies were included after screening 182 citations. Included publications captured data from claims database (n=9) and population survey (n=1). All the publications studied HB patients without inhibitor. Median FIX expenditure per-patient-per-month (PPPM) were $47,419 and $24,414 for extended half-life (EHL) and short half-life (SHL) product cohort, respectively. Upon switching from the SHL to EHL product, median expenditure increased by $107,303. Median expenditures PPPM for eftrenonacog alfa and albutrepenonacog alfa (EHL) were $48,336 and $42,664 respectively. Average inpatient and outpatient cost of EHL vs SHL was $0 vs $15,110 per-patient-per-year (PPPY) and $3,529 vs $17,709 PPPY, respectively. CHESS US and CHESS US+ study reported mean annual direct medical cost ($614,886), mean annual non-medical direct costs ($2,371), and indirect costs ($6,931).

Conclusion: This SLR provides economic burden of HB patients without inhibitor using FIX treatment. Further analyses incorporating overall cost could provide additional insight into these findings and can be utilized as inputs for the economic modelling.

Code

EE261

Topic

Economic Evaluation, Study Approaches

Topic Subcategory

Cost-comparison, Effectiveness, Utility, Benefit Analysis, Literature Review & Synthesis, Work & Home Productivity - Indirect Costs

Disease

Biologics and Biosimilars, Rare and Orphan Diseases, Systemic Disorders/Conditions