Adverse Events (AEs): Are They Factored into Costs of Hemophilia Care?.

Much of the high cost of hemophilia treatment ignores the cost of care for potential AEs related to treatment. Data have been published regarding the thrombotic AEs associated with the use of therapeutic biologics in the management of hemophilia patients with inhibitors. One objective was to quantify the incremental costs of these thrombotic AEs associated with either the available plasma-derived or recombinant products used in managing these patients. Although these occur at low frequency, myocardial infarction, stroke, and pulmonary emboli are reported.

Methods: A review of the literature revealed that no data exist for the cost of these events in hemophilia patients. Using the PharMetrics longitudinal database, it provided us with the frequency and charges associated with thrombotic AEs in hemophilia patients with and without inhibitors. These changes are not inclusive of total replacement product.

Thrombotic complications of inhibitor therapy have been recognized for many years. Essentially, little data exist for the costs of these events and none for potential requirements for rehabilitation, loss of employment, and impact on the family. Data from clinical trials may well be a future key factor in determining reimbursement for therapeutics. There is a growing need for longitudinal data to assess the long-term safety as well as the associated costs of potential AEs. Knowledge of these costs will aid treaters in making therapeutic decisions. Coupled with the incidence of these events, incremental costs will aid health care planners to better understand the costs of care associated with hemophilia patients with inhibitors.