Key takeaways:
Cell and gene therapies (CGTs) are becoming a benefits-planning priority for employers. Rising utilization and an expanding pipeline mean self-insured plans may face greater exposure to high-cost therapies in the years ahead.
Current CGT spend may look modest, but the financial risk can be significant. Even a small number of eligible members can create meaningful budget impact when therapies carry high upfront costs.
Data can help benefits leaders move from reactive cost management to proactive planning. Prevalence modeling, pricing assumptions, and pipeline monitoring can help employers estimate exposure before costs occur.
CGT strategy must account for more than the therapy claim. Employers should also plan for care coordination, member support, vendor alignment, and long-term outcomes tracking.
Cell and gene therapies (CGTs) are changing what is possible in modern medicine. For people living with certain cancers, rare genetic conditions, blood disorders, retinal diseases, and other serious conditions, these therapies may offer outcomes that were difficult to imagine just a decade ago.
But for employers, health plans, and benefits advisors, CGTs are also changing the way healthcare risk needs to be understood and managed. These therapies can involve complex care journeys, high upfront costs, ongoing follow-up, and coordination across medical, pharmacy, behavioral health, travel, caregiver, and leave benefits. As more products move through the pipeline, employers may need to shift from reacting to rare events toward proactively assessing risk across their covered populations.
Truven experts recently led a webinar on how organizations can use data, analytics, and planning frameworks to anticipate risk, model potential financial impact, and make informed decisions about coverage and plan design.
Today, CGT utilization may still appear small in broad population reporting. From 2022 through 2025, CGT utilization increased more than 30%, reaching 4.2 patients per 100,000, while allowed spend more than doubled to $13 per member per year. Those numbers may look modest next to larger medication cost drivers, but the trajectory matters.
CGTs are today where some other major drug trends were just a few years before they became boardroom-level cost concerns. The difference is that many CGTs can carry six- or seven-figure price tags, often concentrated in a single plan year. Even when there are only small increases in the number of eligible members, the financial impact can be substantial—especially for self-insured employers.
There are nearly 50 approved CGT products today, with hundreds more in clinical trials.
Oncology remains one of the biggest drivers, especially for cell and gene-modified cell therapies such as CAR-T products. Hematologic cancers, including multiple myeloma, lymphoma, and leukemia, are heavily represented, while therapies for lung, colon, prostate, and breast cancer are also emerging. For employers and plans, this reinforces the importance of preventive care, screening strategies, and closing screening gaps.
Other pipeline areas may have different implications. Neurology includes gene therapies for conditions such as Duchenne muscular dystrophy, ALS, Huntington’s disease, Parkinson’s disease, and Alzheimer’s disease—many of which may be expensive and require specialized follow-up. Immunology could become especially important due to higher condition prevalence; conditions with products in the pipeline include lupus, multiple sclerosis, rheumatoid arthritis, Crohn’s disease, ulcerative colitis, and psoriasis. Metabolic disorders also warrant attention, with emerging therapies for Type 1 diabetes, Type 2 diabetes, obesity, MASH, and metabolic syndrome.
A strong CGT strategy starts with understanding the covered population. Employers and health plans can use claims and clinical history to:
Estimate the prevalence of indicated conditions
Identify populations that may be more likely to qualify for therapy
Benchmark their exposure against broader market trends
From there, organizations can layer in pricing assumptions to model potential financial impact. Not every member with an indicated condition will qualify for or elect therapy, but prevalence and eligibility modeling can help benefits leaders communicate potential exposure before treatment begins.
For example, Type 1 diabetes has a cell therapy in the pipeline. Looking at historic claims data, prevalence and allowed spend were relatively stable from 2022 through 2025, with 2025 prevalence at 5.5 patients per 1,000 and spend just over $85 PMPY. But when projected for a 50,000-member plan, even conservative uptake among higher-severity eligible patients could increase costs for that condition by 26% to 71% in the following year, depending on eligibility criteria and utilization assumptions.
Because the CGT landscape changes quickly, static reporting is not enough. Products may move forward or be delayed based on clinical trial results, public interest, competitive dynamics, pricing, or FDA timelines. At the same time, the population itself changes as members enter or leave the plan, receive new diagnoses, or progress in disease severity.
Benefits leaders should consider several reporting principles:
Use enough clinical history. Rare conditions may not produce continuous treatment patterns, so a short lookback period can miss potential exposure.
Go beyond diagnosis codes when appropriate. Diagnostic information alone may not identify likely eligibility. Supportive care needs, admissions, severity markers, and medical policy criteria may provide additional context.
Avoid relying on genetic testing data. Instead, use ICD-10 codes and published mutation-rate studies where relevant to estimate potential eligibility.
Refresh assumptions frequently. Pipeline timing, eligibility criteria, pricing, uptake, and covered population characteristics can all change.
CGTs also create operational and member-experience challenges. A member’s journey may involve multiple care settings, lab and radiology services, prescription and infusion therapies, travel benefits, time away from work, mental health support, caregiver coordination, and long-term outcomes tracking. Even one-time therapies often require ongoing follow-up care, monitoring, and support.
That means employers should evaluate more than the therapy price alone. They may need to ask whether stop-loss coverage is adequate, whether vendor partners can coordinate across benefits, how medical and pharmacy policies align, how members will be guided through a complex care journey, and how outcomes will be tracked over time.
Questions benefits leaders should be asking now
Which approved and pipeline CGTs are most relevant to our covered population?
Do we have enough claims and clinical history to identify rare or emerging-risk cohorts?
How would one or more high-cost CGT claims affect annual budgets, stop-loss coverage, and renewal discussions?
What eligibility, severity, and uptake assumptions should be used in our financial models?
How are our carriers, PBMs, consultants, and vendor partners coordinating member support?
How will we monitor long-term outcomes, follow-up care, and total cost beyond the initial therapy claim?
CGTs represent both remarkable medical innovation and a new level of benefits complexity. For employers and health plans, the goal is not to restrict access to transformative therapies; it is to prepare for them with better data, clearer assumptions, stronger partner alignment, and more informed decision-making.
By monitoring the pipeline, modeling exposure across covered populations, and refreshing projections as clinical and pricing information evolves, benefits leaders can move from reactive cost management to proactive CGT planning. That preparation can help organizations support members through complex care journeys while protecting the long-term sustainability of their health benefits strategy.
Connect with Truven today to see how we can help with using your own data to assess CGT risk.
Watch the full webinar here.