By Long Tran, PharmD; Beckie Fenrick, PharmD; and Jason Peterson, RPh
A single cell or gene therapy can cost several million dollars, creating significant financial exposure for self-funded employer health plans. These treatments offer the potential for long-term and, in some cases, curative benefits for patients with genetic disorders, rare diseases and certain cancers.
As the treatment pipeline expands, plan sponsors must navigate high upfront costs, specialized care requirements and uncertainty about long-term outcomes. Understanding the available therapies, their clinical risks and the financing and stop-loss options is becoming an important part of benefits planning.
What Are Cell and Gene Therapies?
Cell and gene therapies (CAGTs) are advanced treatments designed to restore, replace or modify biological functions to treat or prevent disease.
While often grouped together, the two approaches work differently:
- Gene therapy introduces or modifies genetic material to treat, modify or prevent disease.
- Cell therapy uses specific cell types to restore, replace or enhance biological function to treat or prevent disease.
The category has grown considerably since 2017, when the FDA approved Kymriah for patients up to age 25 with B-cell precursor acute lymphoblastic leukemia. Subsequent approvals have expanded treatment options beyond conventional regimens, including more individualized, one-time interventions. For example, CAR-T therapies genetically engineer a patient’s T cells to attack cancer cells and may cure some patients with hematologic cancers.
How Much Do Cell and Gene Therapies Cost?
Several approved therapies carry published list prices exceeding $2 million, with some costing more than $4 million. The following tables summarize notable therapies, their indications and published costs or market status.
Gene Therapies
| Brand (Generic Name) | Indication | Published Cost (WAC) |
| Hemgenix (etranacogene dezaparvovec) | Moderate-to-severe hemophilia B | $3,500,000 |
| Roctavian (valoctocogene roxaparvovec) | Moderate-to-severe hemophilia A | Removed from market by manufacturers |
| Elevidys (delandistrogene moxeparvovec) | Duchenne muscular dystrophy | $3,200,000 |
| Beqvez (fidanacogene elaparvovec-dzkt) | Moderate-to-severe hemophilia B | Removed from market by manufacturers |
| Waskyra (etuvetidigene autotemcel) | Wiskott-Aldrich syndrome (WAS) | $3,250,000 |
| Skysona (elivaldogene autotemcel) | Cerebral adrenoleukodystrophy | $3,000,000 |
| Zevaskyn (prademagene zamikeracel) | Dystrophic epidermolysis bullosa | $3,147,000 |
| Lenmeldy (atidarsagene autotemcel) | Metachromatic leukodystrophy | $4,250,000 |
| Casgevy (exagamglogene autotemcel) | Sickle cell disease; transfusion-dependent beta-thalassemia | $2,365,000 |
| Zynteglo (betibeglogene autotemcel) | Beta-thalassemia | $2,800,000 |
| Lyfgenia (lovotibeglogene autotemcel) | Sickle cell disease | $3,100,000 |
| Luxturna (voretigene neparvovec-rzyl) | Inherited retinal dystrophy (blindness) | $913,750 |
| Zolgensma (onasemnogene abeparvovec-xioi) | Spinal muscular atrophy | $2,715,961 |
| Vyjuvek (beremagene geperpavec-svdt) | Dystrophic epidermolysis bullosa | $1,352,000 |
| Encelto (revakinagene taroretcel-lwey) | Macular telangiectasia type 2 | $250,000 |
| Papzimeos (zopapogene imadenovec-drba) | Recurrent respiratory papillomatosis (RRP) | $460,000 |
| Itvisma (onasemnogene abeparvovec-brve) | Spinal muscular atrophy | $2,664,229 |
| Kebilidi (eladocagene exuparvovec-tneq) | Aromatic L-amino acid decarboxylase deficiency | $3,950,000 |
| Kresladi (marnetegragene autotemcel) | Leukocyte adhesion deficiency type 1 | Price pending launch, expected in Q4 2026 |
| Otarmeni (lunsotogene parvec-cwha) | Sensorineural hearing loss associated with confirmed biallelic OTOF variants | Provided at no cost to clinically eligible U.S. residents; other out-of-pocket costs may apply. |
| Adstiladrin (nadofaragene firadenovec-vncg) | High-grade, BCG-unresponsive non-muscle-invasive bladder cancer | $247,254 |
| Tecelra (afamitresgene autoleucel) | Metastatic synovial sarcoma | $781,525 |
| Imlygic (talimogene laherparepvec) | Malignant melanoma | $762,848 |
| Genglycos (pariglasgene brecaparvovec-opnr) | Glycogen storage disease type Ia | $2,700,000 |
| Tudriqev (vusolimogene oderparepvec-wtpg) | Unresectable advanced cutaneous melanoma | $450,000 |
Cell Therapies
| Brand (Generic Name) | Indication | Published Cost (WAC) |
| Ryoncil (remestemcel-L-rknd) | Steroid-refractory acute graft-versus-host disease (GVHD) | $1,552,000 |
| Rethymic (allogeneic processed thymus tissue–agdc) | Congenital athymia | $2,923,840 |
| Amtagvi (lifileucel) | Malignant melanoma | $515,000 |
| Lantidra (donislecel) | Adult type 1 diabetes with recurrent and severe hypoglycemia | $300,000 |
| Omisirge (omidubicel-onlv) | Hematopoietic stem cell transplant; severe aplastic anemia | $693,900 |
| MACI (matrix-induced autologous chondrocyte implantation) | Repair of cartilage defects of the knee | $77,164 per implant |
| Yescarta (axicabtagene ciloleucel) | Large B-cell lymphoma, follicular lymphoma, non-Hodgkin lymphoma | $548,900 |
| Kymriah (tisagenlecleucel) | Acute lymphoblastic leukemia, large B-cell lymphoma, follicular lymphoma, non-Hodgkin lymphoma | $523,152 |
| Provenge (sipuleucel-T) | Hormone-refractory prostate cancer | $187,808 |
| Tecartus (brexucabtagene autoleucel) | Acute lymphoblastic leukemia, mantle cell lymphoma, non-Hodgkin lymphoma | $503,580 |
| Breyanzi (lisocabtagene maraleucel) | Large B-cell lymphoma, follicular lymphoma, non-Hodgkin lymphoma | $557,918 |
| Abecma (idecabtagene vicleucel) | Relapsed or refractory multiple myeloma | $544,162 |
| Carvykti (ciltacabtagene autoleucel) | Relapsed or refractory multiple myeloma | $571,970 |
| Aucatzyl (obecabtagene autoleucel) | Acute lymphoblastic leukemia | $572,250 |
| Tregzi (allogeneic regulatory T cell immunotherapy with HSPC and T cell-vldq) | Improve chronic graft-versus-host disease (cGVHD)-free survival in patients with blood cancers undergoing allogeneic hematopoietic stem cell transplantation | $428,000 |
*WAC represents the manufacturer’s published list price, not necessarily what a health plan ultimately pays. Actual costs may reflect negotiated discounts, rebates, administration and facility charges, and other treatment-related expenses. Patient weight, dose and treatment duration may also affect total cost. Manufacturers may change prices over time.
Cell and Gene Therapy Safety Risks and Specialized Care
Cell and gene therapies often require specialized care and close monitoring. Strict eligibility criteria limit who can receive treatment, and many therapies are administered at centers of excellence that coordinate multidisciplinary care, pharmacy and infusion services, and long-term patient monitoring.
These treatments can also carry serious safety risks. CAR-T therapies may cause severe adverse events, including cytokine release syndrome (CRS) and neurologic toxicities that require prompt intervention. Other CAGTs can cause serious liver injury, prolonged cytopenias, infections, or complications from conditioning chemotherapy. Elevidys, a gene therapy for Duchenne muscular dystrophy, carries a boxed warning for serious liver injury and acute liver failure, including fatal outcomes. Safety concerns have also led to temporary pauses in certain investigational CAR-T therapies.
How Can Employers Manage Cell and Gene Therapy Costs?
As development expands into cardiometabolic, hematologic and neurological conditions affecting broader populations, including working-age adults, employers may face greater exposure to high-cost treatments.
The impact can be especially significant for smaller employers, while larger groups can generally spread high-cost claims across more covered members. Uncertainty about long-term treatment durability and safety further complicates financial planning.
Several financing and reimbursement approaches have emerged to help employers manage this exposure:
- Value-linked pricing agreements: Tie payment to a therapy’s clinical value and outcomes achieved.
- Outcomes-based contracts: Make reimbursement dependent on predefined clinical or durability outcomes.
- Manufacturer warranties: Provide financial protection if a therapy doesn’t achieve expected results.
- Subscription or risk-pooling models: Spread financial risk across payers or larger covered populations.
- Installment payment programs: Distribute treatment costs over multiple years rather than requiring the full amount upfront.
- Federal carve-out benefit approaches: A proposed federal coverage model that would shift responsibility for funding certain gene therapies to a centralized government or dedicated funding program.
These approaches may help manage financial risk and improve patient access, but each presents challenges involving implementation, administration, outcomes tracking, and long-term sustainability.
Stop-loss insurance is another important consideration. While it can protect self-funded employers against catastrophic claims, policies may exclude certain conditions that qualify for gene therapy, charge higher premiums or impose condition-specific or member-specific deductibles, known as lasering.
Traditional stop-loss contracts also generally operate on annual claim periods, which can complicate multi-year or outcomes-based payment arrangements. Employers should review exclusions, reimbursement provisions, and renewal conditions to understand their potential financial responsibility.
Preparing Plan Sponsors for the Next Generation of Cell and Gene Therapies
Cell and gene therapies are creating new possibilities for patients, but they also require employers to think differently about the financial risks associated with advanced treatments. Reviewing benefit design, clinical management programs, financing arrangements, and stop-loss coverage can help employers identify potential gaps before a high-cost treatment claim occurs.
As the pipeline grows, plans that evaluate these considerations early will be better positioned to manage financial exposure while supporting appropriate access to potentially life-changing care.
