What does "off label" use mean? Is it safe?

"Off label" means that a drug or biologic product is being used for a purpose or at a dose other than that specified on its label. All drug indications, which are approved by the FDA, must be clearly listed on the label.

An "off label" use is often supported by a number of credible studies that validate its safety and efficacy for a particular disease or condition. Although, it has not been approved by the FDA, the scientific basis may be sufficient for it be considered medically acceptable and, in some cases, even a standard of care, for the disease.

How often is IVIG used off label?

No precise statistics exist, but most experts now believe that > 60% of all IVIG dispensed in the USA is for off label indications. In fact, some experts claim that this number may even be higher. All agree that off label use is increasing faster than use for any of the label indications.

Aren't the terms "off lable" and "experimental" really the same?

In terms of IVIG therapy, what is the difference? Off label use refers to treatment of any diagnosis that has not been approved by the FDA. Over the years, many off label uses have been validated by research. For IVIG, well recognized off label uses include Guillian-Barre' Syndrome, CIDP and NAIT.

However, an experimental use is either not supported by a body of evidence or the published studies are few, small and/or lack scientific rigor. Experimental uses of IVIG have included Alzheimer's Disease, Autism, Antiphospholipid Syndrome and many others.

Remember: All experimental use of IVIG is off label. But not all off label use is experimental.

If off label use of IVIG for some diseases is now considered medically acceptable, why aren't these diseases included on the label? Why hasn't the FDA approved them?

The costs of conducting the research necessary to apply for a new drug indication from the FDA are extremely high. At the same time, the number of patients with these specific conditions may be very low.

Both the practical aspects of conducting the required studies, as well as the potiential return on such an investment for manfacturers, are major obstacles in pursing new label indications. However, manufacturers continue to evaluate this issue closely and at least one new indication for IVIG is expected sometime in 2007.

Are off label uses of IVIG reimbursed by insurance? Is this off label therapy "covered?"

Today, a health plan will most likely cover the costs of IVIG if 1) all other treatment options have been unsuccessful 2) use of IVIG for the disease is medically necessary and acceptable, according to current standards of care and 3) supporting documentation, often including lab or other diagnostic tests, justify IVIG treatment.

Health plans vary in their coverage of IVIG. Many require prior authorization of IVIG before the therapy can be given. It is always essential that that physician, patient and the IVIG provider be aware of the reimbursement issues whenever IVIG is being considered and/or when long term use is expected.