Will Employers See a $1.5 Million Gene Therapy Cure?

It’s in the news: Hemophilia may be in line for a multi-million dollar cure.

Hemophilia is a rare but potentially life threatening bleeding disorder. A genetic mutation causes clotting factor proteins to be missing from the blood. Hemophilia can lead to permanent disability due to bleeding into the joints, such as ankles, knees and elbows, or even death, if bleeding can’t be stopped in vital organs like the brain.

Hemophilia treatment is super expensive. As a baseline, average health care costs are approximately $155,000 per year. For hemophilia treatment with complications like inhibitors (antibodies that prevent clotting factor treatments from being able to stop bleeding), costs can rise to $697,000 to $1 million per year. Approximately 25% to 30% of those with Hemophilia A and 1% to 5% of those with Hemophilia B develop inhibitors. Plus, hemophilia currently requires lifelong treatment.

That’s where gene therapy could come in. Strong results were reported in two important studies published in 2017. In the first study, seven men with Hemophilia A were given a single high dose gene therapy treatment. The study found that these participants produced normal or near normal levels of clotting factors on their own after treatment. Annual bleeding events went from 16 to one after treatment. The second study of ten men with severe Hemophilia B found that all participants generated missing clotting factors after gene therapy treatment. Nine out of ten participants had no bleeding events after treatment, down from an average of 11 bleeding events per year. Despite these positive results, it’s important to remember these short term studies have not determined long term treatment outcomes. In addition, more and larger studies will be needed to evaluate the general safety of gene therapy treatments.

Though gene therapy for hemophilia is not yet FDA approved and on the market, analysts predict the price could be steep. If the results are shown to be of lasting benefit, analysts think the price tag could be 2.5 times the current annual cost of treatment. This could be in the range of $1 million to $2 million per patient.


Gene therapy is exciting because it potentially offers the chance not just to treat symptoms but actually cure disease. However, you may be wondering how this will impact your bottom line. It’s important to remember hemophilia is very rare – affecting only about 20,000 males in the US. The chance that any given employer may be impacted is very low. However, there are ways to be prepared for health conditions with extremely high price tags:

  • Stay informed. Gene therapy is a very new but quickly developing technology. Stay on top of what’s going on. Your health plan or broker can help.

  • Understand your employees’ health conditions. Look at the health condition make up of your organization. Determine those that could require high cost treatments. Assess whether your organization’s annual costs of treatment may be higher or lower than anticipated/expected.

  • Learn more about what your carrier can do. Talk with your health plan carrier about strategies to proactively manage gene therapy and other high cost treatments. It’s also important to discuss coverage strategies that can help your organization be prepared should extremely high cost claims occur.

  • Manage health conditions holistically. Integrated medical and pharmacy benefits may offer cost management and improved health outcomes. This is especially important for costly, high touch conditions like bleeding disorders.

Check out our new infographic to learn more about hemophilia and its impact on employers.

Hemophilia treatment