Gene therapies are a type of medicine that uses genetic material to treat or prevent disease. To achieve the best possible results, these groundbreaking and potentially life-changing treatments must be prescribed and administered in a thoughtful, evidence-based process. In addition, they are extremely costly, can be grueling for the patient, and require a sophisticated, specialized team of medical professionals and other caregivers for optimal success.
Before gene therapies are prescribed and administered, these questions are among those that should be taken into consideration:
- Does the patient understand the risks and benefits of all therapy options?
- Is gene therapy the best treatment? Would an existing therapy be equally or more effective?
- How can patients receive high-touch, ultra-personalized care that keeps them on track and maximizes therapy success?
The promise and costs of gene therapy
Biotechnology companies typically price these therapies from approximately $500,000 to more than $3.5 million per course of treatment. Theoretically, the high price of these treatments is offset by avoiding the potential lifetime costs of comprehensive disease management.
Researchers have linked about 10,000 diseases to genetic errors, and many of those diseases currently lack effective treatment. More than 1,000 gene and cellular therapies are in development, and more than 1,500 gene modification and cellular therapy trials (clinical studies) are in process. As therapies emerge to target relatively common conditions, potential patient populations are expected to grow exponentially.
Identifying the right patients for gene therapy
Gene therapies are not right for every patient. Genomic testing is often required to determine suitability; the disease stage, the patient’s overall health, their stability on any current (non-gene) therapies, and many other factors also may need to be considered.
Given the short history of gene therapies, the efficacy of a treatment outside of a trial environment may differ from expectations. In addition, longer-term efficacy and risks are still unknown. These factors add to the complexity of determining if gene therapy is the right choice for a patient.
Specialized expertise, infrastructure, and patient-focused care are vital
The complexities and therapeutic nuances specific to gene therapies require a customized, case-by-case approach. Each step of the process must be coordinated carefully, and many therapies require complex shipping and handling procedures, time-sensitive delivery, and ultra-cold environments.
Connectivity and collaboration among the provider team – clinicians, pharmacists, nurses, dietitians, social workers, patient care advocates, and others – help ensure patients receive the services they need to maximize the effectiveness of their gene therapy.
Gene therapy and long-term value
Treatment with a gene therapy can potentially deliver cost savings over the course of a patient’s life by reducing or eliminating the need for ongoing treatment, such as specialty or other medications, long-term disease management by specialists and other clinicians, emergency department visits, hospitalizations, and other health services. For example, the lifetime cost of hemophilia B can exceed $20 million – far more than the $3.5 million list price of the gene therapy Hemgenix. However, durability – how long gene therapy might be effective – remains a question. That uncertainty needs to be factored into total savings estimates until more long-term study results are available.
By supporting and encouraging evidence-based access to gene therapy, payers and other segments of U.S. society will be positioned to benefit from future advancements and biopharmaceutical breakthroughs that drive systemic change – away from a lifetime of chronic disease management and toward disease intervention and prevention.
Download Evernorth's new white paper exploring the current and future states of gene therapies.
This article was created with the assistance of AI tools. It was reviewed, edited, and fact-checked by Evernorth's editorial team.
