Unlocking The Future: How Gene Therapy Is Transforming Treatment For Rare Disorders
Aug 05,2025
Read Time 2 Minutes
Gene therapy is a radical and groundbreaking shift in the treatment of people with rare disorders. Its approach involves targeting the origin of a genetic disease by replacing a deficient or nonexistent gene with a new, operational "copy" — and by doing so, restoring its function. This healthcare innovation is one that holds immense promise in improving the outlook for those affected by genetic conditions.
A Growing Gene Therapy Pipeline
Right now, there are 12 FDA-approved gene therapies to treat 143,000 potential candidates in the United States.* But by the end of 2026, it’s estimated that there will be 26 additional FDA-approved gene therapies to treat more than 6.1 million people in the United States.*
As more gene therapies become available, Anthem’s pharmacy benefits manager, CarelonRx, will continue to monitor the drug and biologic pipeline, evaluating the potential efficacy of new treatments and making certain they are medically necessary according to medical policy.
Managing The Costs Of Gene Therapies
Experts are recognizing the value of treating the whole person, not just the disease. This is why insurers like Anthem are taking a holistic approach to treating rare genetic conditions by bringing together health services, medical management, healthcare data, and specialty pharmacy expertise.
Anthem’s Gene Therapy Solution works as an endorsement of its stop loss products to protect self-insured employers from unknown financial risk, while also providing robust care management for employees and their family members with rare and complex conditions. In addition to covering both treatments and medical costs, there are no exclusions or lasers planned on future gene therapies that are covered by the Anthem Gene Therapy Solution.
Anthem also uses a comprehensive blend of clinical and contractual strategies to manage costs. This includes utilization management criteria to help guide essential treatments and reduce unnecessary costs, strategic provider contracting for drug and service costs, and agreements with pharmaceutical companies for rebates or payments for unsuccessful treatments.
Connecting Employees To Transformative Care
As gene therapy advances, it's vital for employers to consider health coverage plans and cost management strategies that connect employees to these life-changing holistic treatments. Employers can foster healthcare innovation and enhance their employees' well-being, while also protecting their financial interests, by integrating medical and pharmacy benefits and collaborating with a dependable health partner dedicated to improving lives.
* Internal analysis, CarelonRx, March 2025.