March 21, 2023
Independence Blue Cross has launched a new network under which it will cover gene-based therapies for members at Penn Medicine and Children's Hospital of Philadelphia. These types of therapy involve modifying a person's genes to treat rare genetic disorders.
The Advanced Network for Gene-Based Therapeutics focuses on gene-replacement therapies, in which patients' cells are given missing genes or new copies of malfunctioning ones.
"These are potentially life-changing gene-based therapies for patients who may have few options," said Dr. Rodrigo Cerdá, Independence's senior vice president of health services and chief medical officer.
Each of the five gene-based therapies approved by the U.S. Food and Drug Administration will be available through the network.
Three of the therapies will be available at Penn Medicine and CHOP, which were involved in developing one called Luxturna. It's used to treat adults and pediatric patients with a rare form of childhood blindness. The other two are Zynteglo, which is used to treat adult and pediatric patients with a blood disorder known as beta thalassemia, and Hemgenix, which is used to treat adult patients with hemophilia B.
The remaining two therapies will be available exclusively at CHOP. They are Zolgensma, which is used to treat children with spinal muscular atrophy; and Skysona, which is used to slow the progression of neurologic dysfunction in children with early, active cerebral adrenoleukodystrophy.
The therapies are administered in one-time treatments. Their cost ranges from $850,000 for Luxturna to $3.5 million for Hemgenix, Cerdá said.
"The pipeline for these therapies continues to grow each year and each new therapy is priced higher than the last," he said. "Although there are only five gene-based therapies on the market now, we anticipate that eight to 10 new products could be approved by the end of 2024."
Those could include a gene therapy for hemophilia B that was originally developed at CHOP and is in the final stages of clinical testing, according to Dr. Joseph W. St. Geme, physician-in-chief and chairman of the Department of Pediatrics at CHOP.
To help reduce how much Independence and its members will pay for new gene-based therapies, the insurer is working on outcome-based agreements with their manufacturers.
"We agree to a set price and measurable expected outcomes with a manufacturer for a specific therapy," said Susan Larkin, Independence's senior vice president for health markets. "If the therapy delivers on its intended outcomes, we pay the set price. If it doesn't, the manufacturer refunds a portion of the cost."
Gene therapies offer so much promise because they don't just treat a condition's symptoms; they fix the defective genes that cause the condition, St. Geme said.
"They offer the potential for near curative therapies for numerous genetic diseases," he said. "In many cases, the only current treatments available for these conditions treat the symptoms of the disease but not the fundamental cause, sometimes with a significant impact on the patient's quality of life. For some conditions, like a rare inherited form of blindness called Leber congenital amaurosis, there are no conventional treatments at all."
Gene therapies are administered in two ways:
• In vivogene therapies are infused directly into the patient, typically near the site most affected by the defective gene (for example, the liver or the eye).
• In ex vivo therapies, a patient's cells are modified outside the body and then infused into the patient by what is effectively a bone marrow transplant. That's the method used to treat such blood disorders as beta thalassemia, which is one of the diseases the network provides treatment for.
Both Penn Medicine and CHOP have a long history of working with gene-based therapies.
Penn Medicine has been doing direct gene therapy since the late 1990s, said Dr. David Porter, the director of cell therapy and transplantation at Penn Medicine's Abramson Cancer Center.
"Our teams have specialized experience developing these types of therapies in laboratory settings, conducting clinical trials to test the safety and efficacy of these new treatments, and in developing the processes and clinical care needed to scale up operations needed to treat larger numbers of patients once the therapies are approved," Porter said.
CHOP sponsored the first gene therapy clinical trial for an eye disorder called Leber congenital amaurosis in 2007, St. Geme said.
"We are hopeful that in the future, technological advances will allow these therapies to be produced more efficiently and less expensively, so that all children with genetic diseases have access to potentially life-saving treatments," he said.
Independence members approved to receive the therapies will receive extra support from registered nurse health coaches employed by the insurer. The health coaches work in partnership with the care teams at Penn Medicine and CHOP and can help members through every step of the treatment process.
Independence members who live more than 40 miles from the main facilities of Penn Medicine and CHOP may be able to receive treatment at a different facility.
"We take members' age, physical location, and plan benefits into consideration during the precertification review process and will help determine where members can get treatment," Cerdá said.