A haemophilia disease management programme targeting cost and utilization of specialty pharmaceuticals

The high cost of clotting factor concentrate (CFC) used to treat haemophilia and von Willebrand disease (VWD) attracts health plans' attention for cost management strategies such as disease management programmes (DMPs). In 2004, Indiana's high risk insurance health plan, the Indiana Comprehensive Health Insurance Association, in partnership with the Indiana Hemophilia and Thrombosis Center developed and implemented a DMP for beneficiaries with bleeding disorders. This report evaluates the effectiveness of the DMP 5 years post implementation, with specific emphasis on the cost of CFC and other medical expenditures by severity of disease. A pre/post analysis was used. The main evaluation measures were total cost, total outpatient CFC IU dispensed and adjusted total outpatient CFC cost. Summary statistics and mean and median plots were calculated. Overall, 1000 non-parametric bootstrap replicates were created and percentile confidence limits for 95% confidence intervals (CI) are reported. Mean emergency department (ED) visits and mean and median duration of hospitalizations are also reported. The DMP was associated with a significant decrease in mean annualized total cost including decreased CFC utilization and cost in most years in the overall group, and specifically in patients with severe haemophilia. Patients with mild and moderate haemophilia contributed little to overall programme expenditures. This specialty health care provider-administered DMP exemplifies the success of targeted interventions developed and implemented through a health care facility expert in the disease state to curb the cost of specialty pharmaceuticals in conditions when their expenditures represent a significant portion of total annual costs of care.