Consideration in Hemophilia Therapy Selection
Section snippets
Increasing Use of Recombinant Therapies
In 2004, the US Centers for Disease Control and Prevention (CDC) reported on the distribution of clotting factor therapies among the nearly 14,000 persons with clotting disorders who had registered in the CDC’s Universal Data Collection surveillance program, beginning in 1998.2 In the 12 months preceding entry into the surveillance program, the majority of patients with hemophilia A (62.7%) and hemophilia B (56.1%) had used a recombinant therapy (Table 1).2 This is reflective of similar trends
The Healthcare Provider’s Responsibilities in Therapy Selection
The hemophilia community has developed deep beliefs and concerns about its access to treatment options and the appropriate conditions for making therapy selections. Patients with hemophilia have embraced the concept of “patient choice” to allow both their unrestricted access to all therapy choices and their right to full participation in the final decision on therapy choice. The steady trend detected among private third-party payers to reduce patient therapy options through formulary
Overview of Hemophilia Therapy Selection Criteria
In order to comprehensively and appropriately guide patient selection in hemophilia therapy, it is necessary to consider all the following criteria:
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Safety and efficacy
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Potential for inhibitor development
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Convenience
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Consistency of supply
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Company reputation
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Cost-benefit ratio
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Reimbursement
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Patient preference
Safety and efficacy are, as with most therapies, the most important criteria in therapy selection, but the choice in hemophilia therapy is substantially complicated by fear of inhibitor
Selection Criteria: Safety From Blood-Borne Pathogens
Safety and efficacy have long been the most important considerations in selection of factor replacement therapy.5, 6, 10, 11 Safety with regard to infectious pathogen risk has been enhanced through effective and progressively more sensitive pathogen screening of blood products and their derivatives. Most known blood-borne pathogens are identifiable, but a high degree of sensitivity in their identification is less common.
Selection Criteria: Concerns About Inhibitor Development
In addition to issues of product safety and efficacy, patients with hemophilia are concerned about the potential for inhibitor development. Inhibitors that develop in a patient in response to one FVIII clotting therapy will not only make that therapy but all FVIII clotting therapies ineffective. Up to 30% of previously untreated patients33 and approximately 1% of previously treated patients34 develop inhibitors. No particular therapy is associated with increased inhibitor development: ongoing
Conclusion
Patients with hemophilia have expressed that, historically, they have been the “canaries in the coal mine” for HIV in the blood supply. Healthcare providers, government, and industry have an ongoing responsibility to be proactive about threats to patient safety, even when levels of acceptable risk are hard to define. Clinicians should work closely with patients to choose the safest and most appropriate hemophilia therapy, especially when the criteria for selection are complex and competitive.
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