The inclusion of cell and gene therapies in employer health plans remains rare but is becoming more common, creating a growing financial risk for employers and plan sponsors, according to a new study from the Employee Benefit Research Institute.
Cell and gene therapies are among the most promising and expensive treatments in modern medicine. Rather than managing symptoms, they modify a patient's genes or cells to address the root cause of disease. They are currently used to treat certain cancers and rare genetic disorders such as sickle cell disease and hemophilia, and are being studied for a broader range of inherited and autoimmune conditions.
As of 2025, the U.S. Food and Drug Administration has approved 48 cell and gene therapies, with dozens more in development. Increasingly, these therapies are targeting conditions that are more common than traditional rare diseases, raising the likelihood that individuals covered under employer-sponsored plans may qualify for treatment in the coming years.
Utilization remains limited but is gradually rising. In 2018, 7.9 per 100,000 enrollees received a cell or gene therapy. By 2022, that figure increased to 9.2 per 100,000. Although users represent fewer than 0.1% of enrollees, they account for roughly 0.5% of total health care spending, including the cost of the therapies and related medical services, the report found.
Among enrollees in the top 1% of total health care spenders, just 0.58% used these therapies. Yet they accounted for 1.6% of spending within that high-cost group, underscoring how even a small number of cases can influence overall cost trends, EBRI said.
"Even though use of cell and gene therapy remains rare, the financial impact of a single case can be significant, particularly for smaller self-insured employers," said Paul Fronstin, director of health benefits research at EBRI. "Employers should be evaluating coverage and financing strategies now — not because this is a widespread budget driver today, but because the trajectory of approvals and innovation suggests it could become more consequential over time."
Traditional cost-sharing tools such as deductibles and out-of-pocket maximums offer limited protection against these claims. For therapies that can exceed $1 million per treatment, standard cost-sharing mechanisms have little impact on utilization, particularly among the highest-cost patients, said EBRI.
Employer size shapes financial exposure. Larger self-insured employers can spread risk across broader populations. Smaller employers are more vulnerable to catastrophic claims and often rely on stop-loss coverage, which may include exclusions for known high-cost individuals. For some smaller plans, a single seven-figure claim could represent a double-digit share of annual health spending.
To manage this risk, employers and insurers are exploring alternative strategies, including enhanced stop-loss coverage, carve-out programs, gene therapy reinsurance pools, amortization models that spread costs over multiple years, broader risk-sharing arrangements and performance-based payment tied to clinical outcomes. While promising, many of these approaches remain largely untested at scale, making ongoing evaluation critical as the therapy pipeline expands, the report said.
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