Is it Safe to Use Medicines for Indications for Which They Are Not Approved?

Indications for a drug are diseases for the treatmentparticularly with regard to the indication for which it is
of which it can be used effectively. There are twobeing approved, dosage, storage, and route of
types of indications:administration.However, there is no binding on doctors
to prescribe a particular drug only for its
1. Labeled indications: These are indications approvedFDA-approved indication. It is entirely legal for doctors
for treatment by a specific drug. In the Unitedto prescribe drugs for diseases for which they (the
States, the authority granting the approval is thedrug) are not approved. The doctor's judgment
Food and Drug Administration (FDA). Labeledcomes into play here, and law respects it.
indications are mentioned, along with information onManufacturers however are not allowed to market
dosage, etc. on the drug's label.their drugs for off-label indications, howsoever
2. Off-label indications: These are indications that theeffective they may be in the treatment of those
FDA has not approved for treatment by a specificindications. (Note: The only exceptions are certain
drug and are not mentioned on the drug's label. An'controlled substances' such as opiates, which must
apparent anomaly: Some drugs are used more oftenbe used only for approved purposes).Why
for off-label indications that labeled indications. Formanufacturers don't seek approval for
example, a research by the US General Accountingmulti-indications: FDA approval costs money.
Office in 1991 showed that a third of all drugs usedManufacturers have to invest in trials to satisfy the
for treatment of cancer are off-label. Though this isFDA on an indication-by-indication basis. Thus, more
old research data, the overall picture has notindications they seek to obtain approval for, the
changed. A more recent example would be that of,more the manufacturers have to spend money.
say, Lexapro, the anti-depressant introduced in theManufacturers don't consider this extra expenditure
market in 2002. Lexapro's labeled indication is unipolarand effort as commercially justifiable because of one
depression, but doctors often prescribe it for bipolaror more of the following reasons:
depression to, and with good results. Another- There are already drugs in the market that are
example is the tricyclic anti-depressant group, whoseestablished for the treatment of those additional
approved indication is depression, but is used moreindications.
for treatment of neuropathic pain.The fact that so- The market for those indications is small.
many drugs are widely prescribed for off-label- The manufacturer wants to have a specific
indications surely suggests that such uses are notpositioning for the new drug. So, is off-label safe?: As
only not dangerous, but may also be more effectivecan be understood from the above, the answer is a
than approved drugs. How does one explain thisresounding 'yes'. The difference between labeled and
apparent anomaly?The FDA approval process: Tooff-label indications is important for the manufacturer,
understand the above question, it is necessary tobut not the patient or the doctor who are only
understand the FDA approval process. The FDAconcerned with the effect. Therefore, if your doctor
requires manufacturers of new drugs to conductprescribes you a medicine on whose label (or insert)
many clinical trials to demonstrate the safety andyour specific ailment is not named, don't think that
efficacy of the new product. If satisfied, the FDAthe doctor has erred. He or she is just following a
and the manufacturer decide on the wording towidespread and trusted practice.
appear on the drug's label and package insert,