Before a pharmaceutical company can market a new medication to the public, the drug and its uses and dosages must be approved by the FDA. In the interest of protecting consumers, the regulatory agency maintains a strict set of clinical trials, evaluations, and performance standards that new substances and treatments must satisfy before being approved for public sales.
Part of this approval process includes creating the “drug label” that specifies the exact uses the pharmaceutical compound may be prescribed for, including the patient populations and dosages. Although many substances may have potential alternative applications, approval is only given for those treatments that meet the most stringent testing. Using a medication to treat an illness in a way other than what the FDA has approved is known as off-label use.
Physicians, however, may sometimes prescribe drugs for off-label use in the patient’s best interest. Some medicines can be, in fact, effective to treat some conditions that affect a small number of selected patients. For example, more than half of all cancer-treating drugs are prescribed for off-label usage, particularly for those types of cancer that are too rare to conduct a full clinical trial.