Real-World Amount of Clotting Factor Concentrates Dispensed and Annual Medical Expenditures for Japanese Patients with Hemophilia B

Abstract

Introduction

Until extended half-life (EHL) factor IX (FIX) concentrates became available in Japan in 2010, patients with hemophilia B received intravenous FIX replacement therapy with standard half-life (SHL) FIX concentrates.

Purpose

To investigate the amount of factor dispensed and the associated medical expenditures for the treatment of hemophilia B in the real-world clinical setting in Japan.

Methods

This retrospective study comprised patients with hemophilia B (N=197) who had filled prescriptions for FIX concentrates reported in Japan’s Medical Data Vision database from 2015 to 2019. Patients were included if they had 2 or more prescriptions for the same FIX concentrates within the first 6 months of the study period and the interval between prescriptions was at least 2 weeks.

Results

Since 2015, there was a decrease in the proportion of patients using SHL FIX concentrates and a corresponding increase in international units of dispensed EHL FIX concentrates. Median annualized dispensed dosages (IU/kg body weight) of EHL FIX concentrates were lower than for SHL concentrates for outpatient use only. Annual total health care expenditures per patient and annual expenditures for prescribed FIX concentrates increased each year during the study period. Following a switch from an SHL to an EHL concentrate, the median amount of prescribed FIX concentrate decreased slightly, although median total health care expenditures and FIX concentrate expenditures increased.

Conclusion

In the real-world setting in Japan, medical expenditures and the proportion of patients prescribed EHL FIX concentrates for the treatment of hemophilia B have increased.

Keywords:

• blood coagulation factors
• drug labeling
• factor IX
• health care administrative claims
• health care costs

Data Sharing Statement

All the rights for the database used for the current study are reserved with Medical Data Vision. However, Pfizer Japan Inc. have a contract with Medical Data Vision to use this database and publish the results. The data may be made available upon request and are subject to a license agreement with Medical Data Vision.

Ethics Approval

This study was conducted in accordance with legal and regulatory requirements in Japan, and included anonymized claims data, with no personal patient data. Japanese Ethical Guidelines for Medical and Biological Research Involving Human Subjects do not apply to studies that use anonymized secondary data; consequently, this study was not reviewed by any institutional review board or ethics committee.

Acknowledgments

Medical writing and editorial assistance were provided by Courtney M. Cameron, PhD, of Engage Scientific Solutions and by Mia DeFino, MS, ELS, and Michael Morren, RPh, MBA, of Peloton Advantage, an OPEN Health company, and funded by Pfizer.

The affiliation for Leona E Markson was Pfizer Inc, Collegeville, PA, USA at the time of the study.

Author Contributions

All authors made a significant contribution to the work reported, whether that is in the conception, study design, execution, acquisition of data, analysis and interpretation, or in all these areas; took part in drafting, revising or critically reviewing the article; gave final approval of the version to be published; have agreed on the journal to which the article has been submitted; and agree to be accountable for all aspects of the work.

Disclosure

Katsuyuki Fukutake has received honoraria from and/or served as a member of an advisory board or speakers’ bureau for Bayer Yakuhin, Ltd., Chugai Pharmaceutical Co., Ltd., Fujimoto Pharmaceutical Co., Pfizer Japan Inc., Sanofi S.A., CellGenTech, Inc. and Takeda Co., Ltd. Kanae Togo, Linghua Xu, and Toshiyuki Karumori are employees of Pfizer Japan Inc. and may own stock/options in the company. José Maria Jimenez Alvir is an employee of Pfizer Inc. and may own stock/options in the company. Leona Markson was employed at Pfizer at the time of this study and previously retired from Merck and may hold stock/options or receive other retiree benefits from these companies. Ian Winburn is an employee of Pfizer Ltd. and may own stock/options in the company.

Additional information

Funding

This study was sponsored by Pfizer.