Treatment Considerations

Hemophilia and Bleeding Disorders Toolkit

Overview of Hemophilia: Treatment Considerations


Treatments for hemophilia include episodic/on-demand care which is used to stop a patient’s bleeding episodes and prophylactic care which is used to prevent bleeding episodes from occurring in the first place.

Episodic: On-Demand

  • Mild to moderate patients with low frequency of joint bleeds
  • Drug therapies can be self-administered in the home or at an infusion center (e.g. Hemophilia Treatment Center (HTC), hospital ER or outpatient infusion center)
  • Treatment occurs in response to a clinically evident bleeding event or as a preemptive measure prior to an invasive procedure (e.g. dental tooth extraction, surgical procedures)
  • Challenge: Episodic treatment does not prevent bleeds. One bad bleed can lead to permanent, irreversible debilitating pain or can be life-threatening if the brain or a major organ is involved.

Hemophilia Treatment Centers provide leading expertise and improved outcomes for people with hemophilia. Today, there are over 140 federally recognized HTCs across the US. There are also significant employer and patient benefits offered by using HTCs – from pricing to an annual comprehensive care plan for each patient.

Prophylaxis: Prevention

Prophylactic medication or therapy is used to prevent a disease from occurring. Prophylaxis has been the standard of care since the 1990’s for moderate to severe patients with insufficient clotting factor in their blood who are susceptible to frequent bleeding episodes. The goal of regular and ongoing treatment is to prevent bleeds and minimize risk to major organs and/or permanent joint damage. There are two types of prophylaxis for prevention:

Clotting Factor Replacement Therapy

  • Delivered through intravenous infusion (generally self-infused in the home)
  • Average two to three doses weekly
  • Challenges: Long-term patient adherence to the therapy; potential issues accessing veins can sometimes lead to direct vein implantation (PORT) for infants and small children which can increase risk of infection

Substitution Therapy

  • Delivered through subcutaneous injection (generally self-administered in the home)
  • Weekly, every two or four weeks dosing options; uses same amount of drug per 4 weeks regardless of frequency
  • Challenge: Clotting factor replacement therapy is still needed to treat bleeds, if and when they occur; potential for a drug interaction with some bypassing agents

Both episodic and prophylaxis therapies can be billed under either the medical or pharmacy benefit.

Bleeding Episodes & Inhibitors

People with hemophilia have a better quality of life today than ever before, but medical complications can still occur. Developing an inhibitor is one of the most serious complications of a bleeding disorder. Inhibitors occur when the body develops antibodies (proteins) to the infused clotting factor treatments. The antibodies are seen as a foreign substance needing to be destroyed, rendering the standard treatment ineffective. This results in increased morbidity and mortality and has the potential to substantially increase the financial burden for people with hemophilia.

Inhibitor Occurrence by Type of Bleeding Disorder:

  • Hemophilia A: 20% - 30%
  • Hemophilia B: 1% - 4%
  • Von Willebrand Disease: 5% - 10%


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Employer Action Steps

National Hemophilia Foundation’s Medical and Scientific Advisory Council (MASAC) recommendations establish standard treatment guidelines for Hemophilia A, B and other bleeding disorders.

The Institute for Clinical and Economic Review (ICER) reports provide recommendations for carriers and PBMs on the treatment of Severe Hemophilia A and Hemophilia A with Inhibitors.

Based on MASAC and ICER reviews, employers should consider these recommendations when discussing coverage with carriers, PBMs and partners.

  • Ensure coverage of prophylaxis treatment is at a level adequate to provide bleed protection.
  • If utilization management programs (UM) such as prior authorization or step therapy are in place, ask your vendor what clinical criteria is used:
    • Are patients experiencing a delay to care when stepping through the UM programs?
    • Could there be unintended medical consequences or costs due to the UM programs?
  • Consider comparative clinical and economic evaluation of prophylaxis treatments in the ICER reports.
  • Consider requiring that management of prophylaxis be done by or in consultation with a Hemophilia Treatment Center to better manage costs through their integrated comprehensive care model.
  • Explore innovative approaches to covering single use therapies such as gene therapies for hemophilia.