Healthcare costs among patients with hemophilia A treated with factor replacement or bypassing agents

Abstract

Objective: To assess real-world costs for patients with hemophilia A treated with bypassing agents versus factor VIII (FVIII) replacement.

Methods: Claims data from a large US health insurer during 1 January 2006–30 September 2014 were used for analysis. Treated patients with hemophilia A were identified based on ≥1 medical claim with a diagnosis code for hemophilia A (ICD-9-CM 286.0) and ≥1 medical or pharmacy claim for bypassing therapy and/or FVIII replacement during 1 January 2007–31 August 2014. The bypassing therapy cohort comprised patients with ≥1 claim for bypassing therapy; all others were assigned to the factor replacement therapy cohort. Post-index hemophilia-related costs were computed as combined health plan plus patient paid amounts for medical claims with hemophilia A diagnosis code or hemophilia therapy procedure code (bypassing therapy, FVIII replacement therapy, desmopressin, antifibrinolytic therapy), as well as pharmacy claims for hemophilia therapy.

Results: The study sample represented 580 patients: 50 (8.6%) in the bypassing therapy cohort (mean age: 38.5 years; mean post-index period: 2.1 years) and 530 (91.4%) in the factor replacement therapy cohort (mean age: 29.3 years; mean post-index period: 2.7 years). Compared with the factor replacement therapy cohort, mean per-patient-per-month hemophilia-related total costs were 4.8-fold higher in the bypassing therapy cohort ($57,232 vs. $11,899), comprising 4.4-fold higher medical costs ($45,911 vs. $10,352) and 7.3-fold higher outpatient pharmacy costs ($11,321 vs. $1547).

Conclusions: Patients with hemophilia A treated with bypassing agents between 2007 and 2014 incurred substantially higher monthly hemophilia-related medical and pharmacy costs than patients treated only with FVIII replacement.

Keywords:

• Hemophilia A
• bypassing agents
• FVII replacement
• healthcare costs

Transparency

Declaration of funding

This study was sponsored by Genentech Inc., South San Francisco, CA, USA. The study sponsor was involved in study design, interpretation of data, editing manuscript content and had final approval of the manuscript prior to submission.

Author contributions

Y.X., Y.M. and P.G.S. were involved in study design, manuscript preparation and editing, and approving final manuscript content. JPS contributed to study design (including patient selection criteria), data interpretation, manuscript preparation and editing, and approval of final manuscript.

Declaration of financial/other relationships

Y.X., Y.M. and P.G.S. have disclosed that they are former employees of Genentech. J.P.S. has disclosed that he is an employee of Optum, whose health economics and outcomes consultancy was retained to conduct the study on which this manuscript was based.CMRO peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Previous presentation

Portions of this study were presented at the 21st Annual International Meeting, ISPOR; 2016 May 21–25; Washington, DC; and at the Academy of Managed Care Pharmacy; 2016 Oct 3–6; National Harbor, MD.

Acknowledgements

The authors wish to thank Jenifer Wogen, MedMentis Consulting LLC, for medical writing assistance. Ms. Wogen was compensated by Optum for her role in manuscript preparation.