Indications for Drugs (uses), Approved vs. Non-approved

Pharmacy Author: Omudhome Ogbru, Pharm.D.
Medical Editor: Jay Marks, M.D.

In medical terminology, an "indication" for a drug refers to the use of that drug for treating a particular disease. For example, diabetes is an indication for insulin. Another way of stating this relationship is that insulin is indicated for the treatment of diabetes. Drugs often have more than one indication, which means that there is more than one disease for which it is used. The Food and Drug Administration (FDA) classifies indications for drugs in the United States. Indications for drugs can be classified in two categories:

  1. FDA-approved, also called labeled indications, and
  2. Non FDA-approved, also called off-label indications.

We will discuss how indications for drugs are established and the differences between these two categories of indications.

Early in the process of developing a drug, the drug's manufacturer decides what disease(s) the drug might treat effectively based on the drug's known effects on the body. The manufacturer then conducts studies using people with the disease(s) to determine if the drug is, in fact, effective. These studies are designed and conducted under strict FDA guidelines and under the watchful eye of the FDA. If the studies show efficacy (benefit to patients with the disease) and no serious side effects for the drug, the manufacturer applies to the FDA for approval of the drug for that indication (disease).

  1. Labeled indications

    If the FDA determines that there is enough evidence to approve the drug for the indication (treatment of the disease), the indication becomes a labeled indication for the drug. The approval by the FDA means that the company can include the information in their package insert (product label) regarding the use of the drug for that indication. More importantly, the manufacturer can claim that the drug is effective for the approved indication and use this information to market their drug to patients and physicians.