Medicine Section - Gene Therapy

IMPORTANT REMINDER

This Medical Policy has been developed through consideration of medical necessity, generally accepted standards of medical practice, and review of medical literature and government approval status.

Benefit determinations should be based in all cases on the applicable contract language. To the extent there are any conflicts between these guidelines and the contract language, the contract language will control.

The purpose of medical policy is to provide a guide to coverage. Medical Policy is not intended to dictate to providers how to practice medicine. Providers are expected to exercise their medical judgment in providing the most appropriate care.

DESCRIPTION

Gene therapy is a technique that uses genes to treat or prevent disease. (1) The different techniques currently being investigated in clinical trials include:  

• Inserting a normal gene to replace an abnormal gene;
• Swapping an abnormal gene for a normal one;
• Repairing an abnormal gene; and
• Altering the degree to which a gene is turned on or off.

Initially, gene therapy was targeted towards genetic disorders, e.g., cystic fibrosis, Gaucher's disease, hyperlipidemia. At this time, the majority of human gene therapy protocols in clinic trials are for cancer therapy. Outside of genetic diseases, gene therapy is also being investigated in the treatment of inflammatory, vascular and immune-mediated disorders.

The FDA's Center for Biologics Evaluation and Research (CBER) regulates human gene therapies. At this time, the FDA has not yet granted final approval for any human gene therapy product. (2)

POLICY/CRITERIA

Gene therapy is considered investigational.

POSITION SUMMARY

Although there is a great deal of ongoing research, it is not yet established that gene therapy is able to either prevent or cure disease.  In addition, as noted in the policy description, the FDA has not yet granted final approval for any gene therapy product. (2)

References

1. Genes and Gene Therapy, NIH website:  http://www.nlm.nih.gov/medlineplus/genesandgenetherapy.html (Verified 05/11/09)
2. Human Gene Therapy and The Role of the Food and Drug Administration. Updated March 24, 2003 www.fda.gov/cber/infosheets/genezn.htm  (Verified 05/11/09)

Cross References

None

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