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Prophylactic use of factor VIII: an economic evaluation
1Program for the Analysis of Clinical Strategies, Brigham and Women's Hospital, Boston, MA 02115, USA. rlbohn@bics.bwh.harvard.edu
Thrombosis and Haemostasis
|June 3, 1998
Summary
Prophylactic factor VIII therapy for hemophilia reduces disability costs but increases overall healthcare expenditures due to high drug prices. Clinical benefits may outweigh economic concerns for some patients.
Area of Science:
- Hematology
- Health Economics
- Pharmacoeconomics
Background:
- Exogenous factor VIII therapy is established for hemophilia treatment.
- Clinical benefits of factor VIII prophylaxis are well-studied.
- Economic aspects of factor VIII prophylaxis require further investigation.
Purpose of the Study:
- To conduct a cost analysis of factor VIII utilization in hemophilia.
- To compare healthcare costs of on-demand versus prophylactic factor VIII use.
- To examine expenditures averted and incurred with different factor VIII regimens.
Main Methods:
- Cost analysis of 831 severe hemophilia patients (aged 1-31) from 19 international centers.
- Patients categorized into three groups based on prophylactic factor VIII regimen frequency.
- Costs estimated for hospitalization, surgery, lost school/work days, and factor VIII utilization; stratified by age and joint score.
Main Results:
- Episodic factor VIII use incurred higher disability-related costs (hospitalization, surgery, lost days) than prophylaxis.
- Hospitalization for hemophilia-related conditions was the primary driver of disability costs.
- Year-round prophylaxis had the highest factor VIII drug cost, exceeding savings from reduced disability.
Conclusions:
- Prophylactic factor VIII significantly reduces non-factor healthcare costs and disability.
- High cost of year-round prophylaxis led to the highest total healthcare expenditures in this group.
- Clinical benefits of prophylaxis may justify its use on medical, rather than purely economic, grounds.