A term used to describe the prescribing of a drug for a condition for which it was not specifically approved by the Food and Drug Administration (FDA). Increasingly, medical researchers are discovering that many drugs have multiple effects throughout the body and can have beneficial effects on completely unrelated medical conditions. Because the FDA does not regulate the practice of medicine, doctors may legally prescribe drugs for off-label uses at their own discretion.
As an example of off-label prescribing, some ophthalmologists are using a drug called bevacizumab (brand name Avastin), which was approved by the FDA in 2004 for treating metastatic colorectal cancer (cancer that has spread to other areas of the body), in people with macular edema to see whether it can prevent or correct vision loss. In macular edema, the blood vessels may become leaky in the center of the retina, an area known as the macula, causing the macular tissue to swell and blur central vision. Bevacizumab fights metastatic colorectal cancer by blocking the action of a protein called vascular endothelial growth factor (VEGF), an action thought to inhibit the growth of new blood vessels to the tumor and effectively cut off the tumorís blood supply. In people with macular edema, the drug may keep blood vessels in the retina from leaking and causing swelling of the macula.
Another example of off-label drug use includes prescribing tricyclic antidepressants such as amitriptyline to treat painful diabetic neuropathy. Additionally, ACE inhibitors, which were originally approved for treating high blood pressure, are now also used to prevent diabetic kidney disease, independent of their effects on blood pressure.