Healthcare costs continue to rise, leaving employers scrambling to manage benefits expenses. The Business Group on Health 2025 Employer Health Care Strategy Survey found that 76% of employers are “very concerned” about overall pharmacy costs. While GLP-1 drugs have dominated recent conversations, a new pharmaceutical cost challenge is emerging for benefits leaders: gene therapies.
These cutting-edge medications carry price tags reaching hundreds of thousands to millions of dollars per treatment. Although their per-member-per-year (PMPY) impact has remained relatively low due to the rare nature of the conditions they treat, that’s changing quickly. After reviewing trends in a health data set covering 300 million people in the U.S., we see that utilization rates for gene therapies have grown 42%, and PMPY spend doubled from 2022 to 2024. And we’re expecting this expansion to accelerate even further.
Employers are entering a formative period for gene therapy management – while product counts remain small, momentum is undeniable. Assuming regulatory success for the current late-stage pipeline, which the FDA just made easier with its new fast track framework, this class of medications may triple within 36 months, pressuring the still-experimental financing architecture to mature rapidly.
Understanding gene therapies and their costs
Gene therapies represent a subcategory of precision medicine — also known as personalized medicine. They differ from other precision medicine approaches, such as cell therapies, cell-based gene therapies, immunotherapies, and targeted therapies. Below are examples of each subcategory:
- Gene therapy
- Definition: Treatments insert genetic material into a patient’s cells to correct or replace a faulty gene, resulting in preventing or fighting disease.
- Example: Zolgensma for Spinal Muscular Atrophy
- Cell therapy
- Definition: Living cells are used to repair or replace damaged cells. These cells could either come from the patient themselves or from a donor.
- Examples: Stem cell transplants
- Cell-based gene therapy
- Definition: This is a combination of cell and gene therapy where a patient’s cells are genetically modified outside of the body, then to repair or replace damaged cells.
- Examples: CAR-T treatments such as Kymriah and Yescarta for cancer
- Immunotherapies
- Definition: These medications boost the patient’s immune system to fight disease.
- Examples: Keytruda, Opdivo, and Yervoy for cancer
- Targeted therapies
- Definition: Medications target specific proteins or genes to interfere with cellular processes.
- Examples: Adcentris. Gleevec, and Herceptin for cancer, Humira for Rheumatoid Arthritis
Currently, there are more than 40 gene therapy products on the market, treating conditions like hemophilia, cancer, and various rare diseases. But this field is expected to grow significantly over the next five years.
As expensive as the medications are, the total cost of gene therapy treatment goes well beyond the drugs’ price tag. Often, these mediations require rigorous testing, wraparound supportive care, ancillary medications, inpatient stays, and sometimes, even patient travel.
Gene therapy medications are often administered in hospital settings, either inpatient or outpatient, so they’re typically covered through medical benefits. While some medications require only infusion with additional monitoring and supportive care, other medications, especially ex vivo therapies, may involve multiple visits and inpatient stays at highly specialized hospitals. As payers consider coverage strategies for gene therapies, it is important to recognize and consider how benefits and care coordination work for these wraparound services, too.
The cell and gene therapy pipeline is growing
The cell and gene therapy (CGT) pipeline continues to expand rapidly, and oncology is among the fastest-growing areas. According to the 2025 Cell and Gene Therapy Report: Advancing the Future of Medicine, 178 oncology-focused drug candidates entered the late-stage pipeline in the past year alone, signaling continued momentum in cancer research.
Beyond oncology, neurological disorders are also emerging with promising results, including medications for conditions like Parkinson’s and Alzheimer’s. For metabolic disorders, numerous gene and cell therapies are being developed for Type 1 diabetes, with an expansion into Type 2 diabetes likely. These advances reflect CGT’s growing potential to benefit broader patient populations.
Managing CGTs in benefit programs
The 2025 Cell and Gene Therapy Report: Advancing the Future of Medicine interviewed payers to gather their perspectives on CGTs. The report found that while 80% of respondents believe CGTs are safe and effective, they remain skeptical about high upfront costs and limited long-term data. Sixty percent of payers believe payment models could reduce risk; however, they emphasize that model effectiveness depends on the availability of clinically relevant endpoints that are easy to measure.
This skepticism is understandable. We’re dealing with medications that can fundamentally change how we treat disease, but the financial models supporting them are still evolving. Leaders who prepare flexible benefit designs, capture real-world data, and develop performance-based contracts will be best positioned to absorb the coming wave while preserving both innovation incentives and fiscal sustainability.
As gene therapies become more prevalent, benefits professionals should consider three essential strategies to manage rising costs while ensuring members have access to life-changing treatments.
1. Review integrated data about your employees
Precise medication requires precise data. Integrated data reveals patterns in how gene therapies are being used within your population. What medical policies or prior authorizations are in place to make sure the right medications are selected? Are these medications prescribed at centers of excellence? Are outcomes tracked? This information is critical for making informed decisions about benefit design and vendor partnerships.
Employers who understand their population’s health needs can also anticipate future gene therapy utilization and plan accordingly. Using claims data, predictive modeling, and demographic information, we can identify members who may benefit from these treatments and anticipate future plan costs. This proactive approach helps organizations budget more accurately and avoid financial surprises.
2. Enforce rigorous clinical management
Not every patient who could receive gene therapy should receive one. Clinical management ensures these expensive treatments go to patients most likely to benefit.
Work with specialty pharmacies and medical management teams to establish clear coverage criteria. This includes verifying that patients meet FDA-approved indications, have tried appropriate alternatives when applicable, and are receiving care at qualified facilities.
Rigorous clinical management also means tracking outcomes. If a gene therapy doesn’t deliver the promised results, performance-based contracts can help mitigate financial risk. These arrangements tie reimbursement to measurable health improvements, aligning incentives between payers and drug manufacturers.
3. Prepare for the financial impact
Traditional benefit structures weren’t designed for million-dollar therapies. Some employers are exploring outcome-based payment arrangements, risk-sharing agreements, and collective carve-outs where costs are spread over time and tied to treatment success.
Collaboration with vendors and consultants who specialize in gene therapy management can also provide valuable support. These partners bring expertise in negotiating with manufacturers, identifying cost-saving opportunities, and implementing innovative financing models.
Building sustainable benefit structures
The rise of metabolic gene therapies represents a critical juncture for benefits management. These treatments offer genuine hope for patients with serious conditions, but they also pose significant financial challenges.
By leveraging integrated data, enforcing rigorous clinical management, and preparing for the financial impact of precision medicine, health plans and employers can build sustainable benefit structures that support member health while managing escalating costs. Those who act now to build the right infrastructure, partnerships, and policies will be best positioned to navigate this new frontier successfully.
Photo: doyata, Getty Images
Katherine Shanahan is Senior Pharmacy Analytic Advisor at Truven by Merative, where she helps commercial and government payers, providers, and researchers to develop and deliver actionable pharmacy-related analytics. She supports customer efforts to identify cost-saving opportunities and healthcare quality improvement strategies through improved medication management and prescribing.
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