Burden of illness: direct and indirect costs among persons with hemophilia A in the United States

Abstract

Objective:

To examine the direct and indirect costs of hemophilia care among persons with hemophilia A in the US.

Methods:

Observational data were obtained from HUGS-Va, a multi-center study from six federally supported hemophilia treatment centers (HTCs). Eligible individuals completed a standardized initial questionnaire and were followed regularly for 2 years to obtain information on work or school absenteeism, time spent arranging hemophilia care, and unpaid hemophilia-related support from caregivers. Data from 1-year healthcare utilization records and 2-year clotting factor dispensing records measured direct medical costs. Indirect costs were imputed using the human capital approach, which uses wages as a proxy measure of work time output.

Results:

A total of 222 patients with complete data were included in the analysis. Two-thirds had severe hemophilia and the mean age was 21.1 years. The use of prophylaxis in severe hemophilia patients is associated with statistically significant reduction in the numbers of emergency department (ED) visits and bleeding episodes compared with those who were treated episodically. From the societal perspective, mild hemophilia costs $59,101 (median: $7519) annually per person, $84,363 (median: $61,837) for moderate hemophilia, $201,471 (median: $143,431) for severe hemophilia using episodic treatment, and $301,392 (median: $286,198) for severe hemophilia receiving prophylaxis. Clotting factor contributed from 54% of total costs in mild hemophilia to a maximum of 94% for patients with severe hemophilia receiving prophylaxis.

Conclusion:

Hemophilia is a costly disorder not only because of its high medical expenses, but also due to the high indirect costs incurred.

Keywords::

• Hemophilia A
• Burden of illness
• Economic
• Indirect cost

Transparency

Declaration of funding

This project was supported by grants from Baxter Healthcare Corporation, Bayer Foundation, CSL Behring, Novo Nordisk, and Wyeth/Pfizer, with additional financial support from the Federal Hemophilia Treatment Centers/Region IX, Grifols, and CHOC at Home.

Declaration of financial/other relationships

No financial conflict of interest was reported by the authors of this paper.

Acknowledgements

We are very appreciative of all the individuals who have been involved in the Hemophilia Utilization Group Study Part-Va (HUGS-Va) project since 2005: Michael B. Nichol, PhD (Principal Investigator), Kathleen A. Johnson, PharmD, MPH, PhD (late Principal Investigator), Zheng-Yi Zhou, MS, Mimi Lou, MS, Joanne Wu, MS, and Jiat Ling Poon from University of Southern California; Robert Miller, PA (Site Principal Investigator) from Children's Hospital, Los Angeles, Hemostasis and Thrombosis Center (CHLA); Heather Huszti, PhD (Site Principal Investigator), Marie Serrato, RN, Eve N. Fontaine, PhD, Cecilia Dueñas, MA, and Elizabeth Berker from Children's Hospital of Orange County, Hemophilia Treatment Center (CHOC); Brenda Riske, MS, MBA, MPA (Site Principal Investigator), Ruth Ann Kirschman, RN, NP, Jasper Hillhouse, Carissa Smith, Mallory Kinlaw, Deirdre Cooper-Blacketer, and Cassie Ross from University of Colorado Denver (UCD) Hemophilia Treatment Center; Amy Shapiro, MD (Site Principal Investigator), Natalie Duncan, MPH, Brandy Trawinski, and Jayme Harvey from Indiana Hemophilia & Thrombosis Center (IHTC); Ann D. Forsberg, MA, MPH (Site Principal Investigator) and Patricia Forand, RN, from UMASS Memorial Hospital, New England Hemophilia Center (UMASS); Megan Ullman, MA, MPH (Site Principal Investigator) from University of Texas Health Sciences Center at Houston/Gulf States Hemophilia and Thrombophilia Center (GSHTC).

The authors thank the HUGS Steering Committee/Consultants for their advice and contributions: Randall G. Curtis, MBA, Denise R. Globe, PhD, Shelby L. Dietrich, MD, Judith R. Baker, MHSA, DPH, Femida Gwadry-Sridhar, BSPhm, MS, PhD, Marion A. Koerper, MD, Brenda Riske, MS, MBA, MPA, Megan Ullman, MA, MPH, and Kathy L. Parish, PhD.