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:
- FDA-approved, also called labeled indications, and
- 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
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).
- 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.
Manufacturers are not allowed to market their drugs for indications that have
not been approved by the FDA. Since marketing is very important for selling
drugs, FDA approval of indications is critical to the financial success of a
drug. The decision by the manufacturer to apply for approval for a particular
indication is primarily economic. Manufacturers seek approval for indications
that allow the broadest use of the drug in order to maximize the drug's use and
the financial return on their investment in developing the drug. Once a drug is
marketed for one FDA-approved indication, the manufacturer can add indications
for the drug by meeting FDA requirements for the additional indications. Thus,
approved use of the drug can be expanded, and the drug can be marketed to a
- Off-label indications
Once a drug has been approved by the FDA for an indication and then marketed
for that indication, physicians are allowed to prescribe the drug for any other
indication (treatment for other diseases or conditions) if there is reasonable
scientific evidence that the drug is effective for that indication. These uses
that have not been approved by the FDA are the off-label indications. This means
that the drug manufacturer has not gone through the process of applying to the
FDA for approval of the indication. Off-label indications typically are
supported by less extensive studies than labeled indications; however, this does
not necessarily mean that the drugs are less effective when prescribed for
off-label indications than for FDA-approved indications.
Last Editorial Review: 10/26/2009
There are several reasons why manufacturers do not apply to the FDA for
certain indications. Most often, it is because the number of people with the
disease for which the drug might be indicated is small, and sales will not
generate enough revenue to justify the cost of the studies required by the FDA.
Sometimes, there is a similar drug on the market that already has the disease as
a labeled indication, and the manufacturer assumes that its own drug also will
be used as a treatment for the disease because the two drugs have similar
effects. In still other instances, off-label use of the drug may be so common
that obtaining FDA approval for the indication would not make much of a
difference in the use of the drug and the revenue that is generated.
The difference between labeled and off-label indications are important to the
drug manufacturer but not as important to the patient. Because drugs have many
uses, most medical references include only labeled indications and the most
common off-label indications. Regardless of whether drugs are prescribed for
labeled or off-label indications, what is important is that the drug is
effective and safe for the condition for which it is prescribed.