Cell and Gene Therapies in Employment-Based Health Insurance: Financing the High-Cost, High-Impact Future

Summary

Cell and gene therapies (CGTs) are a relatively new frontier in medicine, offering the potential to treat or potentially cure rare diseases by targeting their root causes at the cellular or genetic level. These therapies can dramatically improve a patient’s quality of life, but they come with extremely high costs. While only a limited number of CGTs are approved, their use is growing, and more therapies are in the pipeline. As a result, the financial implications for employers and employment-based health plans are likely to increase over time.

In this Issue Brief, we use claims data to examine the evolving landscape of CGTs within employment-based health plans. We analyze the prevalence of conditions targeted by CGTs, patterns in therapy utilization, and the broader spending implications for enrollees who use these therapies. Our analysis further highlights the disproportionate concentration of CGT-related costs among a small subset of high-cost claimants and explores emerging strategies that employers and insurers may consider to manage the associated financial risks.

Key Findings:

• Rising Number of Approvals: As of 2025, the U.S. Food and Drug Administration (FDA) has approved 48 CGTs, with more in development. A growing number of these therapies are targeting conditions that are more common than traditional rare diseases.
• Modest Growth in Use: Use of CGTs remains limited but has shown a gradual increase. In 2018, 7.9 per 100,000 enrollees received a CGT; by 2022, the rate rose to 9.2 per 100,000.
• Small Share of Spending: CGT users represent fewer than one-tenth of one percent of the enrollee population but account for approximately one-half of one percent of total spending. This includes both the cost of CGTs and spending on other health services. These proportions have remained relatively stable between 2018 and 2022.
• Impact on High-Cost Claimants: Among enrollees in the top 1 percent of total health care spenders, just 0.58 percent used CGTs. However, these individuals accounted for 1.6 percent of spending within that top-spending group. While the share is small, it signals that CGTs can meaningfully impact spending patterns among high-cost claimants.

As CGTs evolve from rare to more common disease applications, their financial footprint within employment-based health plans is likely to grow. Employers are beginning to explore innovative solutions to manage these costs, including stop-loss insurance, carve-out programs, gene therapy reinsurance, value-based payment arrangements, and performance-based contracts.

Understanding the trajectory and implications of CGTs is critical for employers, policymakers, and health plan sponsors. While current utilization and spending remain limited, the pipeline of therapies and their high costs warrant close attention to ensure sustainable access and benefit design in the years ahead.