New Gene Therapies Spark Transformative Healthcare Changes

While gene therapy shows significant promise, outcomes can vary by the condition and the person. These treatments may also carry serious risks and may not eliminate the need for ongoing care and support. For employers and health plans, this makes careful clinical review, appropriate utilization management, and long-term cost planning essential.

It’s estimated that genetic and rare diseases affect about 1 in 10 people in the United States. While these treatments signal a new era of healthcare innovation, they also come with significant financial implications. By 2032, the cost of gene therapies is expected to reach up to $36.6B in the U.S. Four to eight new gene and cell therapies are expected to receive FDA approval each year over the next decade. Given this escalating cost of care, health insurance companies and employers are adopting strategies to help manage the expense of gene therapies.

The Value Of Treating Whole Health

Employers are increasingly recognizing the value of addressing a person’s whole health, not just a specific diagnosis. By working with a trusted partner like Anthem, employers can offer health plans that integrate medical management, healthcare data, specialty pharmacy expertise, and care support for rare genetic conditions.

Anthem Stop Loss products can help protect self-insured employers from unexpected financial risk while supporting care management for employees and family members with rare or complex conditions. Stop Loss policies generally align with the employer’s benefit plan, including eligible medical and pharmacy costs, to help provide protection for gene therapy claims.

Anthem also uses clinical and contractual strategies to help employers manage cost of care. These include utilization management (UM) criteria, strategic provider contracting, and CarelonRx contracting approaches designed to help secure value for clients based on condition and therapy type.

A Growing Gene Therapy Pipeline

Anthem standard medical benefits currently cover 14 approved gene therapies that could potentially impact about 160,000 members in the U.S. population, and three additional therapy approvals are expected by the end of 2026.

The list below includes the gene therapy name, the condition it treats, the current estimated treatment cost, and the estimated number of people living with the condition in the U.S.*

• Luxturna, Hereditary retinal dystrophies, $914,000, < 3,000
• Zolgensma, Spinal muscular atrophy (SMA) < 2 years old, $2.6M, < 1,000
• Zynteglo, Beta-thalassemia, $2.8M, ~ 1,500
• Skysona, Cerebral adrenoleukodystrophy (CALD), $3M, < 1,000 males
• Hemgenix, Hemophilia B, $3.5M, ~ 4,200 males
• Elevidys, Duchenne muscular dystrophy (DMD), $3.2M, < 20,000 males
• Roctavian, Hemophilia A, ~ $2.9M, Product discontinued
• Lyfgenia, Sickle cell anemia, $3.1M, ~ 100,000
• Casgevy, Sickle cell anemia, $2.2M, ~ 100,000
• Casgevy, Beta thalassemia, $2.2M, ~ 1,500
• Lenmeldy, Metachromatic leukodystrophy, $4.25M, ~ 2,500
• Beqvez, Hemophilia B, $3.5M, Product discontinued
• Kebilidi, Aromatic L-amino acid decarboxylase (AADC) deficiency, $3.95M, < 1,000
• Itvisma, Spinal muscular atrophy (SMA) ≥ 2 years old, $2.6M, ~ 5,000 
• Waskyra, Wiskott-Aldrich Syndrome (WAS), Not yet listed, ~ 500
• Kresladi, Leukocyte adhesion deficiency-I, Not yet listed, < 5,000
• Otarmeni, Congenital hearing loss, No cost to “clinically eligible individuals" per Regeneron, < 15,000

Connecting Employees To Transformative Care

As more gene therapies become available, Anthem will continue to monitor the drug and biologic pipeline, evaluate the potential efficacy of new treatments and ensure they are medically necessary in accordance with medical policy. 

As gene therapy advances, it’s vital for employers to consider health coverage and cost-management strategies that connect employees to appropriate, potentially life-changing treatments. By integrating medical and pharmacy benefits and working with a trusted health partner, employers can support healthcare innovation, improve employee well-being, and help protect their financial interests.

* All gene therapies listed above have been approved by the FDA. Prevalence is noted unless otherwise stated. Several factors not represented here may affect how many people living with a medical condition are candidates for therapy. Prices are estimates and may change over time. 

All therapy brand names or products in this article are trademarks or registered trademarks of their respective owners.