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Supplements Incorporating Emerging Innovation in Hemophilia A and B: Tailoring Prophylaxis and Management Stra

Economic Costs of Hemophilia and the Impact of Prophylactic Treatment on Patient Management

Sheh-Li Chen, PharmD, BCOP
     Ensuring optimal cost-effective care in hemophilia has never been more important. The number of patients with hemophilia who are covered under Medicaid health plans is expected to rise.21 This results from reforms in the healthcare system, including expansion of benefits, and the eligibility of current Medicaid members with hemo-philia who qualify under disability eligibility rules. A larger number of states also are utilizing the services of health plans to cover their disabled and dual-eligible populations. Medicaid health plans have better care-coordination programs and quality-reporting systems than fee-for-service Medicaid. However, the high costs of treating hemophilia may present a challenge to health plans.

    Key drivers in ensuring quality and cost-effective care include optimizing pharmacy management and ensuring patient involvement. Optimizing pharmacy management is crucial to cost management in hemophilia care. Pharmacies need to ensure treatment access, in a timely manner, of safe and effective products and supplies so that patients can adhere to their treatment regimen. Patient involvement entails education on recognizing the signs and symptoms of a bleed, home infusion, care management, and treat-ment adherence, which can ensure that patients and their caregivers are active participants in their care and take responsibility for management decisions.21


    Although a rare condition, congenital hemophilia places a significant economic burden on healthcare payers, patients/caregivers, and society. It results in not only direct costs from hospitalizations, outpatient visits, and drug treatments, but also indirect costs from diminished work productivity and absenteeism. Hemophilia also has intangible costs, including reduced QoL, pain and suffering of the individual and family, and the emotional and physical toll on the patient and caregivers.

     The significant evolution of treatment patterns in hemophilia have transformed it from a fatal disease into a chronic, well-managed condition. This can be seen in the advances in therapeutic agents and the increased use of prophylaxis treatment in persons with hemophilia. However, complications, such as inhibitors, have added to the complexity and cost of its management, as well as the complexity of individualized treatment, especially for patients with inhibitors. This has resulted in a critical need to understand the utilization of health resources in the treatment of hemophilia. Patient education and factor management are key to minimizing waste, ensuring optimal therapy and management, and improving outcomes.

    As both the clinical and economic complexities surrounding hemophilia prophylaxis can be significant, it is imperative that managed care pharmacists, clinicians, and providers be aware of the complications of hemophilia, the role of prophylaxis, and the health-care implications and costs surrounding the disease and its prophylactic management. Cooperation among the key stakeholders—healthcare professionals, patients and their caregivers, and managed care professionals—will help provide individualized treatment strategies for patients with hemophilia, strategies designed to prevent complications and optimize clinical and economic out-comes while enhancing patient QoL.


Author affiliation: University of North Carolina Medical Center, Chapel Hill, NC.
Funding source: This activity is supported by an independent educa-tional grant from Baxalta.
Author disclosure: Dr Chen has no relevant financial relationships with commercial interests to disclose.
Authorship information: Concept and design, critical revision of the manuscript for important intellectual content, and revision per editorial comments.
Address correspondence to:

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