Distinguishing Between Intended and Off-label Drug Use

It's so common today for medications, especially prescription, to be associated with off-label uses. The practice of using medicine for intentions other than what it was created for can be beneficial or dangerous.

Focusing only on the United States, when a drug is submitted to the FDA for approval, the pharmaceutical company who produces it has already conducted several clinical trials that support its use that the FDA reviews. This labeled use(s) is referred to as FDA-approved indications. If the drug is found to be safe, effective, and beneficial the FDA will approve it for distribution. Pharmaceutical companies establish a patent on new medications well before seeking FDA approval, and when it expires generic forms of the drug can be produced by any other pharmaceutical company. This is why generic drugs come in many shapes, color, and sizes. Each company makes its own version of the drug so it can be recognized as being created by their company. Once a drug becomes generic it still is only approved for the original indication.

So who promotes off-label uses? Not the pharmaceutical companies. They are legally only allowed to promote a drugs FDA-approved use. If they have a desire to add a new indication to a drug, they must follow the same process used for original approval. Sometimes the new use is similar to the original and is easily approved by the FDA. A good percentage of the cases are completely different than the original use, and trends show it is harder to get FDA approval. If a pharmaceutical company is found to be promoting off-label uses for their drugs, they are charged in criminal court for violating FDA regulations. A sneaky way pharmaceutical companies may try to get around breaking any laws is by continuing research on a medication after FDA approval by outside research firms to discover off-label uses, and have the results (if they're supportive) published in medical journals and other medias. Sometimes drug research firms don't have any dealing with the pharmaceutical company and perform their own research to find off-label uses.

Doctors and other professionals who have the power to dispense prescriptions are the only people who can promote off-label uses. There is no law against this practice and it is used every day by thousands of doctors. Off-label uses are easy to find by just typing in a medication + off-label use in an internet search engine. One benefit of having off-label uses with doctors is that they are not obligated to write prescriptions for the off-label uses. A lot of doctors will completely disagree with an off-label use and will choose not to dispense the medicine for that purpose. Because the FDA does not require strict clinical trials on off-label uses there is a chance an off-label use was completely fabricated out of no research. Doctors will usually conduct research of their own on an off-label use they've never heard of before writing a prescription.

There is a naive attitude most patients have to never question or research a medicine that has been prescribed to them. But, those that do research and discover that the logic behind a new prescription isn't immediately found, let alone found at all can become very panicked. Patients should never assume their doctor has unquestionable proof about an off-label use. We should always "double check" with our doctors about off-label uses.

But, the same can be said for patients who discover off-label uses of drugs and run to their doctor for a prescription. Checking many resources and finding patient testimonials should be done before accepting a drugs off-label use. Sometimes doctors will have negative biased or concrete beliefs about an off-label use and will refuse to write the prescription on those terms only. Drugs with intended use should usually be a first option before off-label drug usage because the evidence supporting an intended use is precise and has been tested through time.