$3.5M Gene Therapy Treatment Gives Benefits Industry a Glimpse Into the Future

Top 3 Things You’ll Learn

  • How does gene therapy work?
  • Will gene therapy make a meaningful difference for hemophilia patients?
  • New treatments bring experimental coverage models

The first FDA approved gene therapy treatment for hemophilia B debuted as the most expensive drug in the world. Hemgenix drew a lot of attention with its $3.5 million per dose price tag, but it’s not the only gene therapy treatment in the works for people with hemophilia. Currently, a person with severe hemophilia may incur an annual drug cost exceeding $250K. The development of inhibitors, which can prevent treatment from working, can increase that to upwards of $1M a year. Gene therapy treatments, like Hemgenix, could combat these yearly costs with a one-time treatment that forever alters the genetics of the individual receiving the treatment. Benefit advisors preparing clients for the future need to learn more about gene therapy, hemophilia, and how eliminating millions in clotting factor cost could impact plan sponsors and patients in the long run.

Gene therapy – experimental techniques to treat or prevent disease

Even if you’re not a pharmacy expert, you might know that our genes are responsible for forming proteins in our bodies. Gene therapy is a newer technique that uses genes to treat or prevent disease. Mutated genes are replaced with healthy ones, inactivating mutated genes that function improperly or introducing new genes. Once inside a cell, the healthy gene can replace the defective one.

The FDA has approved five gene therapies, and more than 30 are in the mid-to-late development stages. This new sector of possibility has produced promising treatment options for several diseases, but there are risks, including unwanted immune system reactions, infection, and impact on the wrong cells.

New treatments for hemophilia A and B

People with hemophilia experience excessive bleeding, which may be fatal if it occurs in the brain. A defective gene that fails to create enough of a crucial factor in blood clotting, factor VIII (hemophilia A) or factor IX (hemophilia B), causes the disease, and patients can suffer from bleeding in the joints — causing severe disfigurement and disability.

Historically, hemophilia has been treated with an infusion of the missing factor, but gene therapies like Hemgenix and others in development allow the body to produce much more of the missing clotting factor on its own.

Roctavian, which treats hemophilia A, is likely to be launched in the U.S. at the beginning of 2023. In the EU, the treatment has gained conditional approval with an orphan-drug designation. It would require only one dose, with a price tag likely to approach $3M.

Out of the 54 participants in the trial that gained Hemgenix FDA approval, 94% of patients ended their previous continuous therapy. Unfortunately, gene therapy has not been proven to be an all-out cure. Sometimes, the response is incomplete or temporary; some patients still have abnormal bleeding and/or require factor infusions. Still, even a partial and/or temporary response is likely to make a meaningful difference.

The one-time cost for Hemgenix and Roctavian could mitigate against a lifetime of infusions for a hemophilia patient, not only mitigating cost but bringing relief to a patient previously thought to have no other option.

How will these new techniques be covered?

With high success rates of these one-time treatments, gene therapy may prove to be a bargain in the long run by eliminating the need for consistent blood clotting factor cost of $1M to $2M yearly for a member’s entire life.

Gene therapies are mostly currently covered under the medical benefit. But they could be split between pharmacy and medical and may impact pharmacy spend by reducing the need for consistent drug treatment. Although it may be cost-effective in the long run, coverage of gene therapies is problematic because under current payment models, the payer that covers the treatment cost wouldn’t be likely to retain the member long enough to recoup the cost, and some payers may not have funding to pay such a large sum at one time.

Experts are still experimenting with coverage models that will resolve these issues. Among the approaches being considered for gene therapies are models where money is refunded if treatment is ineffective, dividing total cost into smaller payments, adding reinsurance or stop-loss insurance to reduce risk, or spreading payments out over years.

Fortunately, many measures designed over the years to mitigate member and payer pain from hemophilia treatments are as valid for gene therapy as for factor treatments. These include:

  • Investigating hemophilia-specific foundation support programs
  • Implementing evidence-based utilization and care management programs offered by your benefit provider
  • Participating in a Center of Excellence or similar program that includes Hemophilia Treatment Centers
  • Exploring any relevant high-cost therapy benefit protection insurance offerings
  • Considering product discounts, rebates, and reimbursement rates available under the pharmacy vs. the medical benefit before deciding where to direct claims.

Finding the right path to relieve the financial pressure of these treatments may well be the key to success for all parties involved, but since these treatments impact smaller populations, it may take time before you see a client impacted by these new options. For now, the focus should be on the impact these drugs can have on patients and the amazing scientific research that makes it possible to reprogram our bodies to fight back.


First published on: November 9, 2022 as 11 Possible Gene Therapies for Hemophilia Are On The Horizon | Updated on December 19, 2023



Inhibitors and Hemophilia | CDC

Hemophilia Treatment Centers | Gateway Hemophilia Association 

Hemophilia– Update on Treatment Management. Bay Harbor Islands, FL: IPD Analytics.

Impact of the 340B Pharmacy Program on Services and Supports for Persons Served by Hemophilia Treatment Centers in the United States | Springer Link

National Hemophilia Foundation NHF Policy Statement: Hemophilia Treatment Center Participation in the 340B Drug Discount Program | National Hemophilia Foundation

Approval, check. Now, can BioMarin make a commercial success of its hemophilia A gene therapy in Europe? | Fierce Pharma



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