Hemophilia: Treatment Landscape, Costs, and Disease Management

Hemophilia affects approximately 13 out of every 100,000 lives. “Looking at this through the lens of a payer, we know that hemophilia is a rare disease. It’s also a very expensive disease,” said Sam Leo, PharmD, director, specialty clinical programs, Magellan Rx management, during a session at the Academy of Managed Care Pharmacy’s Managed Care & Specialty Pharmacy Annual Meeting held April 23-26, in Boston, Massachusetts.

He continued: “Even though it’s a rare disease, it’s common enough that most groups are going to be dealing with hemophilia patients in their network.”

Leo began the presentation by explaining that when classifying patients with hemophilia, there are several differentiators. First, you can classify them based on their factor deficiency—whether they have hemophilia A or hemophilia B. He noted that hemophilia A is 4 times more common than hemophilia B. Patients can also be classified on their disease severity, whether it be mild (about 25% of patients), moderate (about 15% of patients), or severe (about 60% of patients).

These factors play a key role in how these patients are treated, said Leo. Patients with severe disease are those who are at the highest risk for spontaneous bleeds, therefore they are utilizing prophylaxis factor replacement therapy and are associated with the highest costs.

The last differentiator is patients with inhibitors. According to Leo, 20%-35% of patients develop antibodies that reduce the efficacy of replacement products, leaving them with less treatment options and thus are more complex to treat.

Leo then moved on to the current treatment landscape for the disease. “One thing we can certainly say is we don’t have a limited number of products to choose from,” said Leo. Currently, there are 48 drugs on the market, compared to just 1 that was available 40 years ago.

“A patient born today with hemophilia, due to the treatment options available, if they’re treated appropriately, can live to a normal life span, and that’s completely reflective of some of these changes in treatment and drugs we have to treat these patients,” said Leo. However, Leo did note that despite the amount of treatment options and competition, treatment costs remain high.

For a patient with hemophilia A, the annual cost of treatment ranges from $59,101 for those with mild disease to $301,392 for patients with severe disease receiving prophylaxis. For a patient with hemophilia B, the cost of treatment ranges from $85,852 to $263,253. According to Leo, factor replacement products represent up to 94% of total costs for patients with severe disease.

In addition to addressing high treatment costs, there are also management challenges that payers face, including:

• Fragmentation of care and lack of uniformity: There is a lack of standardized guidelines or a standardized approach to how patients with hemophilia are treated
• Navigating the need for reinsurance programs for high-cost members
• Pharmacy and medical benefit utilization
• Potential stockpiling and product waste
• Limited transparency: Payers have little insight into clinical data outside of factor product cost and providers have limited insight into product utilization, dispensed amounts, and total healthcare resource utilization
• Lack of individualized treatment
• Robust and complex product selection

Gary Tereso, PharmD, senior clinical pharmacist, Health New England continued the presentation with current management strategies, which include:

• Utilization management: encouraging more frequent patient interaction and defining best practices
• Individualized prophylaxis dosing protocol: ensure that treatments are reassessed and readjusted based on patient needs. There is no one size fits all drug or dosing regimen
• Assay management and standardized dispensing criteria
• Collaboration with local hemophilia treatment centers
• 340B pricing
• Product referencing
• Patient engagement and care management

Tereso concluded the presentation with several future considerations. He underscored the importance of data collection and analysis in order to determine benchmarks and measure relevant outcomes, as well as collaborating with local centers of excellence under pre-agreed upon standards of care and data sharing arrangements to prevent the need for prior authorization. Tereso also highlighted holistic care and coordination of care, citing the need to consider the whole patient, not just the disease.