Understanding Hemophilia

A hemophilia diagnosis is the start of a complex journey for patients and plan sponsors alike, with a range of treatment options, clinical considerations and potentially staggering costs. As therapies continue to evolve, so does the importance of experienced, connected support. 

Below are answers to some of the most important questions about hemophilia—and how Evernorth, through Accredo Specialty Pharmacy, helps bring greater predictability and confidence to this highly specialized therapy class.

What is hemophilia?

Hemophilia is a rare, lifelong condition caused by a deficiency in a clotting protein—known as factor—that helps control bleeding. Because it is a genetic condition, hemophilia is usually inherited at birth and passed down through families.

Hemophilia affects males almost exclusively, with an estimated 30,000–33,000 males living with the condition in the United States. Disease severity ranges from mild to severe. 

Two most common types :

• Hemophilia A, caused by a deficiency of clotting factor VIII
• Hemophilia B, caused by a deficiency of clotting factor IX

Hemophilia A is approximately three to four times more common than hemophilia B. Among all males with hemophilia, just over 40% have severe disease.

When someone without hemophilia experiences an injury, twelve clotting factors interact in a chain reaction to form a blood clot. Because people with hemophilia are missing one of these factors, bleeding lasts longer, and even minor injuries can increase the risk of internal bleeding.

What are some of the challenges presented by hemophilia?

Hemophilia impacts the ways people live, work and play. Minor bumps and bruises can quickly turn into emergency events and internal bleeding remains a serious, potentially life-threatening risk.

Fortunately, with the right treatment plan and support, many people with hemophilia can safely participate in everyday activities. That support often extends beyond the patient to caregivers, schools, employers and health plans—all of whom play a role in managing risk and responding when bleeding events occur.  

How can hemophilia be treated?

Since the late 1960s, factor replacement therapy has been the foundation of hemophilia treatment. These therapies—derived from plasma donations or produced through recombinant technology—are infused through an IV to stop bleeding episodes or administered prophylactically to help prevent them, particularly in patients with severe disease.

For patients with hemophilia A, Hemlibra^® is a monoclonal antibody used for prophylaxis that is administered subcutaneously weekly, every two weeks, or every four weeks. While it reduces bleeding risk, factor therapy may still be required to treat breakthrough bleeding episodes. 

Approximately 30% of patients with hemophilia A and 1-3% of patients with hemophilia B will develop an inhibitor during their lifetime.² Inhibitors are antibodies that interfere with factor replacement therapy, significantly complicating treatments and increasing the total cost of care, as multiple therapies and more frequent treatments are often needed.

What about gene therapy for hemophilia?

Gene therapies work by modifying a patient's genetic code to enable the body to produce its own clotting factor. In hemophilia, this approach aims to increase factor VIII or IX production following a single administration.

Currently FDA-approved gene therapies for hemophilia include Hemgenix^® for hemophilia B (approved 2022) and Roctavian^® for hemophilia A (approved June 2023).

While many gene therapies target ultra-rare diseases with few or no existing treatment options, hemophilia has a number of highly effective therapies available today. Gene therapies may offer another option for some patients, but they come with a significant upfront cost—ranging from approximately $2.7 million to $3.5 million per dose, depending on product and contracting.

Also, gene therapies aren’t always curative, nor are they considered first line treatment for hemophilia. They are typically reserved for carefully selected adult patients due to eligibility requirements, durability considerations, and high upfront cost.

What should plan sponsors know about how to address concerns around drug spend?

There is no one-size-fits-all solution for managing hemophilia spend. Because each patient’s condition presents differently, costs can be incredibly difficult for plan sponsors to predict and manage.

The average annual cost of medication for a patient with severe hemophilia typically ranges from $300,000 to over $500,000, and can exceed $1 million annually in patients with inhibitors. 

Several patient-specific variables drive this high cost:

• Frequency of bleeding episodes and eligibility for prophylactic therapy
• Weight-based dose requirement
• Variability in vial sizes and dosing precision
• Balance between encouraging activity and managing injury-related bleeding risk

This variability can lead to waste and unnecessary spend. It underscores the need for an experienced specialty pharmacy partner to accurately calculate doses and dispense therapies. Greater precision helps improve predictability and lower costs across this complex therapy class.

Supporting plan sponsors with managing hemophilia

Evernorth combines its best-in-class specialty pharmacy, Accredo, with connected management strategies to help deliver greater benefit predictability and lower costs across this complex therapy class. 

Support for plans sponsors include:

• Self-service hemophilia reporting
• Therapy discounts across hemophilia treatment options
• A robust care model for patients and caregivers
• Support navigating benefits coverage and utilization management for gene therapies 

Originally published on 3/15/2023 and updated on 3/2/2026.