What Is Off Label Use of Drugs?



Off-label drug use

One in 5 prescriptions in the US is for off-label drug use.

One in 5 prescriptions in the US is for off-label drug use or simply put, the medicines are prescribed for a use that has not been approved by Food and Drug Administration (FDA). It should be borne in mind that the drug as such is FDA approved but is being used to treat an ailment that is not approved by FDA.

So, what exactly is off-label use of drugs?

When a physician prescribes a drug for an unapproved use, it is called as off-label use of drugs. The term is derived from the fact that all drugs are labeled with descriptions pertaining to use and these are mostly based on results of research and studies conducted and approved by FDA.

Use of drugs for unapproved use is legal and is a common practice. The legality stems from the views that once a drug has been approved and is made available in the market, doctors and physicians know the best way to use them.

Drug Approvals by FDA


Off-label drug use.

FDA approval is mandatory for prescription drugs.

All prescription drugs must have FDA approval. A drug manufacturer conducts clinical trials to test a medicine and submits results to the FDA for approval. The studies should prove efficacy and safety of drug in question. The FDA follows certain prescribed rules and regulations during approval process and this more-or-less encourage focus of drug for one main single use. Hence, even if the drug has multiple uses, drug company has to make a choice and conduct studies in support to get the drug approved. A classic example is drugs used for treating cancer. Most of these have FDA approval for use against only one particular type of cancer while these are often cited as having multiple uses.

Types of Off-Label Drug Use

Based on how drugs are used, off-label use of drugs are classified into two types:

  1. Type 1 – An FDA approved drug for one particular disease is used for treating a very different ailment. For example, drugs approved for treating seizures are used for treating nerve pain.
  2. Type 2 – An approved drug is used for treating a condition it is approved for but not always in keeping with the intended use. Use of Viagra and other drugs for erectile dysfunction like Levitra and Cialis are good examples of off-label drug use of this type. Even though these drugs are approved for erectile dysfunction (ED), it is often prescribed to men for enhancing sexual performance.

Growing Concern of Off-Label Drug Use

The increase in off-label use of drugs is a cause for concern since patients are unaware that they are being prescribed drugs for off-label use. The risk is high and if prescription is not appropriate, it could turn out to be a waste of money and health.

Having said all this, there are quite a few successful off-label use of drugs and one of them was the off label use of aspirin to prevent a second heart attack in heart patients until the FDA approved it in 1998.

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