Top 3 Things You’ll Learn
- How does gene therapy work?
- Will gene therapy make a meaningful difference for hemophilia patients?
- Experimental coverage models for new treatments
Thanks to recent advances in gene therapy, the chance to cure diseases and relieve pain and suffering for patients previously without hope grows. For people living with hemophilia, the impact of gene therapy could be profound. Currently, a person with severe hemophilia may incur an annual drug cost exceeding $250K. For those who develop inhibitors, which prevent treatment from working, costs can climb upwards of $1M a year. The FDA approved the first gene therapy for hemophilia B in November, and at $3.5 million per treatment, it is now the most expensive drug in the world.
With more treatments on the horizon, you will need to learn more about gene therapy and hemophilia to understand the potential impact on your plan and members.
Gene Therapy: From Theory to Reality
You might know that our genes are responsible for the formation of proteins in our bodies. Gene therapy is a new technique that uses genes to treat or prevent a specific disease. This new sector has produced promising treatment options for several diseases, but that doesn’t come without risk, which may include unwanted immune system reactions, infection, and impact on the wrong cells. The research is focused on diseases that have no cures, and many of the treatments are expensive, with some reaching more than $1M.
The FDA has so far approved five gene therapies, and more than 30 are currently in the mid-to-late stages of development.
Hemophilia new treatments and how they may be paid for
Hemophilia A is a rare, chronic, inherited disease impacting about 20K to 33K men in the U.S. People with hemophilia experience excessive bleeding caused by a defective gene that fails to create enough of a crucial factor in blood clotting, factor VIII (hemophilia A) or factor IX (hemophlia B). Patients can suffer from bleeding in the brain, which may be fatal, and the joints — causing severe disfigurement and disability.
Hemophilia now has one approved gene therapy and six gene therapies that need FDA approval. In gene therapy for hemophilia, the replacement gene allows the body to produce much more of the missing clotting factor on its own. Hemgenix, for hemophilia B (which is much more rare than hemophilia A) was approved in November of 2022; Roctavian is likely to be the first gene therapy for hemophilia A launched in the U.S., possibly at the beginning of 2023. These products require only one dose, with a price tag of $2 to 3.5 million. The one-time cost could mitigate a lifetime of drug costs for a hemophilia patient.
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.
How will these new techniques be covered?
Most gene therapies are 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, gene therapy coverage is problematic under current payment models, because the payer covering the treatment wouldn’t be likely to retain the member long enough to recoup the cost, and some payers may simply not have the funding.
Experts are still experimenting with coverage models that will resolve these issues, including refunding ineffective treatments, dividing total cost into smaller payments, adding reinsurance or stop-loss insurance to reduce risk, or spreading payments out over years.
Fortunately, measures designed over the years to mitigate member and payer pain from hemophilia treatments are valid for gene therapy. 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 that would be available under the pharmacy vs. the medical benefit before deciding where to direct claims.
Finding the right path to relieve the financial pressure that comes with these treatments may well be the key to success for all parties involved.
- Inhibitors and Hemophilia | CDC
- Hemophilia Treatment Centers | Gateway Hemophila
- 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 | SpringerLink
- 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