Economics of Hemophilia

Hemophilia Toolkit

Building the Business Case : Economics of Hemophilia & Addressing Waste

The total cost of care can increase when members with hemophilia are unmanaged or undermanaged, resulting in avoidable use of services and treatments, including emergency room visits and hospitalizations. This can happen when members are treated by health care professionals lacking ongoing training or expertise to effectively treat and manage hemophilia. Medical claims for people with hemophilia A, the most common type, are twenty times higher when compared with the non-hemophilia population. Although cancer is often the number one high-cost claimant condition for employers, the average cost of treatment for hemophilia is more than three times higher than that of cancer, despite it affecting significantly fewer members.

Hemophilia cost drivers can be addressed through a variety of strategies, including use of a benefit plan design that drives members to the highest quality, most cost-effective site of care for treatment and ensuring carriers and PBMs are appropriately managing factor replacement therapy for each patient.

Hemophilia Cost Drivers

Most employers delegate the development of hemophilia cost containment strategies to intermediaries including PBMs, carriers, TPAs, and/or pharmacy and medical consultants. While these vendors may develop appropriate strategies that result in the lowest total cost of care, there are many instances where conflicts can and do exist that increase employer costs unnecessarily. To better manage costs, it is important to gain a basic understanding of key cost drivers:


  1. Vendor Oversight, Waste, and Assay Management: Hemophilia assay management is the process of filling a prescription as closely to the prescribed target dose as possible using factor replacement vials (vials come in a range of unit or assay sizes). Prescriptions for factor replacement are typically written for + or – 10% of the fill. However, intermediaries, including PBMs and specialty pharmacies, often fill the maximum amount each time a prescription is filled. Lack of oversight by vendors on how much factor replacement is sent to the patient and/or consistent over-prescribing can lead to the patient having excess factor replacement on hand. This leads to waste which can significantly increase employer costs.
Real_Life_Example.jpg

Here is a real-life example:

  • Patient is prescribed 2,500 units of clotting factor three times per week
  • Guidelines allow specialty pharmacy to ship additional clotting factor at plus (+) minus (-) 10%; the maximum amount is almost always filled (in this case, 250 extra units per prescription)
  • Patient received an extra 750 units each week (250 units X 3)
  • The financial impact to the employer is substantial:
    • Average cost for clotting factor per unit = $1.30
    • 2,500 units X $1.30 = $3,250 X 3 vials/week X 52 weeks = $507,000 (actual cost of necessary treatment)
    • Additional 750 units prescribed X $1.30 X 52 weeks = $50,700 (unnecessary cost; added “waste”)


  1. Site-of-Care: Hemophilia is a complex disorder requiring a high-touch, individualized approach to patient care. Where care is directed has an important impact on outcomes for members and costs for employers. Optimal management of hemophilia is based on:
    • Early detection and diagnosis
    • Prevention and early treatment of bleeding episodes to avoid complications
    • Detection and management of inhibitors
    • Psychosocial and educational support
    • Monitoring for treatment-related comorbidities
    • Coordination of care with other providers and payers involved in patient management; making sure patients are getting the right care at the right time and the right place

  1. Medication Management: Hemophilia treatment consists of either the infusion of factor replacement or subcutaneous injections of substitution therapy, which can represent up to 90% of the member’s total cost of care. Each treatment has potential for waste. Since these are biologic/specialty drugs, prescriptions are written in a way that does not follow traditional dosing guidelines. This can lead to dispensing excess medication and unnecessary additional cost to the employer, especially related to on-demand use of factor replacement. High out-of-pocket costs can cause patients to avoid or restrict the use of medications. Correct placement of these therapies within the formulary and plan design is essential to ensure adequate coverage and lower out-of-pocket costs.

  1. Prescription/Adherence Management: Prescription non-compliance can contribute to poor outcomes and even death. With poor adherence, frequent and recurrent spontaneous bleeding episodes into soft tissue and joints can occur, leading to joint damage and severe disability. Because the damage is progressive, it can lead to severely limited mobility of joints, muscle deterioration, and chronic pain. Significant cost implications can result from bleeding episodes including unnecessary ER visits, hospitalizations, and complications from bleed-related joint damage. To reduce waste and minimize health care costs, communication and coordination between the medical care provider, pharmacy, and patient are paramount.




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