Gene therapies offer promise where none previously existed to treat complex conditions, and many more are in the pipeline to target additional disease states over the next several years. Yet these therapies come at ultra-high prices that can cost unprotected plan sponsors millions of dollars to treat one patient.
Gene therapies take the form of highly targeted medications that introduce genetic material into a person’s DNA to “edit” and replace faulty or missing genetic material that leads to disease. There are two types of gene therapies: Gene modification, which edits the patient’s DNA at the site of the gene defect by introducing new genetic code to fill a gap or fix a flaw, and cellular therapies, which involves removing cells from the patient’s body (or from a live donor), modifying the genes of those cells in a laboratory, and then introducing the modified cells back into the body.
“Families managing a rare disease are excited by the opportunity of gene therapies, but they also are concerned about treatment safety, cost, and durability,” said Dr. Christine Gilroy, chief medical officer at Express Scripts, the pharmacy benefits management arm of Evernorth. “They need to focus their energy on the patient, but all too often they are consumed by financial concerns and scrambling to crowd-source a million dollars or more.”
A robust pipeline of gene therapies is coming to market
Over the previous five years, the U.S. Food and Drug Administration (FDA) approved 12 gene therapies. Eight more are in the pipeline for approval during 2023, and 15 are expected to enter the market in 2024 and beyond. While the prices of these treatments are unknown, they are expected to cost millions of dollars. A recently approved treatment for hemophilia topped $3.5 million per treatment — making it the most expensive drug ever created.
Together, these therapies will enable us to treat a wide range of serious conditions, including spinal muscular atrophy (SMA), the top genetic cause of infant deaths, and hemophilia, a bleeding disorder that affects more than 30,000 people in the U.S.
“Evernorth is constantly monitoring the gene therapy pipeline, anticipating which drugs are nearing FDA approval,” Dr. Gilroy said. “We created Embarc Benefit Protection to help our clients navigate this complex environment, particularly those scenarios where a potentially life-saving treatment may only work in 40% of treated patients, or a patient may not receive the full expected benefit.”
Embarc shields payers from high costs while their patients receive personalized, expert care and have no out-of-pocket costs beyond their normal deductibles. In addition, enrolled clients are eligible for reimbursement based on overall utilization and program performance.
The $2 million baby
One of the first approved gene therapies is Zolgensma, which treats young children diagnosed with a severe type of SMA. Many of these babies who are not treated die before their second birthday.
Recently, a newborn was diagnosed with this disorder. Fortunately, the plan sponsor had joined the Embarc program a few months before the baby’s birth. The baby received Zolgensma within two months, quickly putting the child on the path to good health. The family paid nothing for the treatment, which was successful, and the plan sponsor was protected from the cost of the therapy, thanks to Embarc.
“This client saved more than $2 million and was able to provide this family with the help they needed during an extraordinarily difficult time. As gene therapies become more available, more and more plan sponsors will face similar scenarios,” Dr. Gilroy said.
“Since we launched Embarc in July 2020, we’ve provided nearly $110 million in the form of gene therapy claims paid out and program surplus dollars returned to clients. With every claim, and every dollar returned, we’re pleased to be able to help the plans and the members we serve.”