Early real-world experience with emicizumab and concomitant factor VIII replacement products in adult males with Hemophilia A without inhibitors

Abstract

Aims

To assess real-world use of emicizumab in adult people with hemophilia A (PwHA) without inhibitors including healthcare resource utilization (HCRU) and costs.

Materials and methods

Adult, male PwHA without inhibitors initiating emicizumab (index date) were identified using IBM MarketScan after 4 October 2016. Patients were required to have continuous health insurance coverage for ≥180 days prior to and ≥90 days after index date and have ≥90 days of continuous use of emicizumab. Patients were followed until treatment gap, disenrollment, or end of data. Results were reported overall and among a subgroup with prior factor VIII (FVIII) prophylaxis. Emicizumab use, concomitant FVIII treatment use, HCRU, and costs were assessed separately over baseline, the emicizumab induction period, emicizumab maintenance period, and annualized.

Results

Among the 71 emicizumab patients (FVIII prophylaxis subgroup: 52) included in the study, the mean age was 35 (subgroup: 34) years and mean follow-up was 12 (subgroup: 11.1) months. At baseline, the annualized mean total healthcare cost was $532,948 (subgroup: $645,727). After emicizumab initiation, per-patient-per-month (PPPM) HCRU was higher in the emicizumab induction period compared to the maintenance period with higher monthly FVIII fills/in-office administrations (0.37 vs 0.17), non-FVIII outpatient visits (2.23 vs 1.55), and emergency department visits (0.06 vs 0.03). The FVIII prophylaxis subgroup yielded similar HCRU trends. Hemophilia treatment costs accounted for over 95% of total healthcare costs. The annualized mean cost was $50,491 (subgroup: $61,512) for concomitant FVIII treatment and $777,171 (subgroup: $793,168) for emicizumab and concomitant FVIII treatment for the first year of emicizumab treatment.

Conclusion

This study represented experience with emicizumab after the approval for PwHA without inhibitors. The study cohort may not be representative of all PwHA taking emicizumab. The findings highlight the continued burden of treatment and healthcare cost for PwHA without inhibitors despite advances in treatment options.

Keywords:

• Hemophilia
• emicizumab
• factor VIII
• Marketscan
• claims database
• healthcare resource use
• Cost

JEL CLASSIFICATION CODES:

• I11
• I1
• I
• I10
• I1
• I
• I00
• I
• I15

Transparency

Declaration of funding

This study was sponsored by BioMarin Pharmaceuticals, Inc.

Declaration of financial/other interests

LC has no financial disclosures. AE, PP, and JT are employees and options holders in Aetion, Inc, which received funding in connection with this study and preparation of this manuscript. EC, DH, and HM are employees and stockholders in BioMarin Pharmaceuticals, Inc which contributed funding in connection with this study and the preparation of this manuscript. CLK has received honoraria for advisory board participation from Biomarin-Genzyme, Sanofi, Takeda, Spark Therapeutics, Genentech, and Pfizer. All named authors meet the International Committee of Medical Journal Editors (ICMJE) criteria for authorship for this article, take responsibility for the integrity of the work, and have given their approval for this version to be published.

Author contributions

All authors contributed to the conception, study design, protocol development, data analysis, interpretation, and drafting and revising of the manuscript.

Acknowledgements

None reported.

Reviewer disclosures

Peer reviewers on this manuscript have received an honorarium from JME for their review work but have no other relevant financial relationships to disclose.

Previous presentations

Data from this study were previously presented in poster and abstract format at the 2022 ISPOR annual meeting.

Compliance with ethics guidelines

This was a secondary analysis of data and was performed using deidentified data without access to personal identifying information. The MarketScan data are Health Insurance Portability and Accountability Act of 1996 compliant. Institutional review board approval was not required as this study only used deidentified patient records and did not involve the collection or use of individually identifiable data.

Notes

i FVIII medication visits billed through a medical benefit excluded from outpatient visits.

ii Ibid.

iii Ibid.

iv Ibid.

v Ibid.

vi Ibid.