Get ready for the highest drug price the world has yet to see

Get ready for the highest drug price the world has yet to see

By Cheryl Levesque, VP, Medical Risk Underwriting, and Kristine Karish, Cost Containment Specialist for the Accident & Health business of QBE North America

Recent FDA approval (11/22/22) of Hemgenix, the latest – and highest priced1  – gene therapy to date at $3.5M, is sending the self-insured community scrambling for cost containment solutions for their adult members with hemophilia B (Factor IX deficiency a.k.a. Christmas disease), a coagulation disorder. 

Hemgenix is approved for the treatment of adults receiving prophylactic (preventative) Factor or who have had life-threatening, repeated or serious spontaneous bleeding. It is a one-time infusion designed to reduce or eliminate the need for regular infusions. In the ongoing HOPE-B global clinical trial, Hemgenix reduced the need for regular Factor in 94% of subjects, and average episodes over a year declined from 4.1 to 1.9 bleeds.2 

The Institute for Clinical and Economic Review (ICER) did a cost and health benefit analysis, indicating a reasonable price for the drug may be $2.93M to $2.96M.3    

Currently, patients being treated for hemophilia B receive regular clotting factor infusions to control their condition. Patients with moderate to severe hemophilia B can cost more than $20M over their lifetime. 

Hemophilia B life expectancy

Present in approximately 1 in 25,000 male births4  and affecting 4,000 individuals in the U.S., hemophilia B can be life-threatening if not treated. With proper care most patients go on to lead relatively normal lives, although the life expectancy for a hemophilia B patient who receives proper treatment is 10 years shorter than the average male population.

Males with severe hemophilia have a life expectancy of 63 years, while mild to moderate hemophilia patients average 75 years. 

The hemophilia gene is passed to the child via the mother. Although primarily found in males, females can have mild hemophilia. 

What happens if hemophilia is left untreated? 

Proper treatment of hemophilia is necessary to avoid life threatening and life altering complications. Spontaneous or traumatic intracranial hemorrhage, bleeding around the airway and hemorrhage in or around internal organs are some of the most life-threatening complications, as well as the leading causes of death for hemophiliacs. Joint disease is the most common complication.5 Bleeding into the joints can cause significant damage and result in severe reductions in range of motion and mobility, deformity, chronic pain and permanent disability.

Inhibitor complications and costs 

Roughly 3%6  of people with hemophilia B will face further complications due to an inhibitor, which is an antibody that develops and works against the product (Factor) to treat hemophilia, making it more difficult to get a bleeding episode under control. 

Inhibitors are more likely to develop during childhood and their incidence is highest in patients with severe hemophilia.

The cost of treatment for patients with inhibitors is much higher than others with hemophilia. They have an increased risk of death and are twice as likely to require inpatient hospitalization for bleeding. According to the CDC, the mean cost of care for a hemophilia patient with inhibitors is $1,000,000 per year compared to nearly $200,000 for those without inhibitors.7  

Factor levels and the type of bleed

Treatment for hemophilia varies widely. Cost factors for any given patient include weight, the presence of inhibitors and the development of complications. The dosage is weight-based so adults cost much more than small children to treat.

The goal is to get factor levels into a therapeutic range that is determined by the type of bleed (e.g., intracranial versus skin cut), the patient’s response, and the strength of any inhibitors.   

Common complications and their treatment costs (billed charges) 8

  • Joint replacement:  $36K – $97K (depending on the joint being replaced and the network/region where the treatment is given).
  • Synovectomy – surgical removal of the lining of a joint:  $21K – $45K 
  • Arthrocentesis – Procedure to remove blood or other fluid from a joint:  $4.6K – $7.3K per episode 
  • Bypassing agent:  $39K – $54K per dose
    ◦ A bypassing agent is a blood product that is used to treat bleeding episodes for those with high levels of inhibitors (antibodies that work against Factor).  Often used instead of Factor to help the body clot blood.
  • Plasmapheresis:  $6K – $12K per treatment
    ◦ This is typically done for patients who have inhibitors when they are experiencing a severe bleed – and can be performed several times a week.

Cost Containment

High-cost gene therapy drugs represent both a breakthrough in improving people’s lives and a financing challenge for the companies that self-fund their employee health plans. While the one-time high-cost treatment may result in lower expenses over the subject’s lifetime, the subject could move to another employer’s plan within a few years.

As the health insurance industry grapples with this dilemma, it is important for employers to work with experts that have deep experience with high-cost treatments. For those who self-insure, choosing a medical stop loss insurer that has a dedicated medical risk management team with deep clinical and case management expertise will help ensure they get continual education and guidance for effective risk planning and mitigation.

Leading providers are also likely to have cost containment resources available through a strong network of vendor partners that can negotiate manufacturer agreements for the lowest rates available. These vendors may also negotiate all-inclusive agreements with facilities who administer the therapy to lower the overall spend in such high-dollar treatment cases.

Developing a long-term relationship with a best-in-class medical stop loss insurer will only grow more important as the number of high-cost treatments like Hemgenix proliferate. 


2 Note: results referenced were as listed on March 10, 2023. Stated results may change over time as the study continues. 
3ICER Publishes Final Evidence Report on Gene Therapies for Hemophilia A and B - ICER
4Hemophilia B - Symptoms, Causes, Treatment | NORD (
5Joint Disease | Introduction to Hemophilia Care | NCBDDD | CDC
8Managed Care Resource Alliance | Welcome (