“Off-label” is the term used when a drug is prescribed to treat a condition for which it is not intended. Such drugs have not been approved for use by the FDA because there is no evidence shown to the FDA that it works for that condition. It may be alarming, but almost 20% of all drugs are prescribed are “off label."

The FDA approves drugs for specific uses only after the drug has been subjected to clinical trials which demonstrate both the effectiveness of the drug and its safety. When a doctor chooses to prescribe a drug “off-label,” he or she is giving patients a treatment for a condition that has not been rigorously tested. It may or may not work; it may or may not be safe for the patient to whom it has been given; and it may or may not have serious adverse effects for the patient.

In effect, an off-label prescription makes a human guinea pig of the unwitting patient.

Why are prescriptions written “off-label”?

When pharmaceutical companies want to market a drug in the United States, they are first required by law to prove to the Food and Drug Administration that the drug is an effective treatment for the condition for which it is to be marketed. Proving a drug’s effectiveness can be a lengthy and expensive procedure, requiring drug studies and clinical trials intended to test a drug for both effectiveness and safety.

When a drug is approved by the FDA for use in the United States, that approval is specific to the diagnosis or treatment of the specific symptoms, conditions, and diseases for which it has been tested. However, although pharmaceutical companies are not permitted to promote additional, untested uses, doctors are free to prescribe any approved drug for any purpose, even a use that the FDA has not approved.

And doctors do write those prescriptions, often based on anecdotal or even falsified proof that the drug will be useful. This most often is provided by drug company representatives against the law, and done so by word of mouth so there is no documented proof it occurred. This is often done by physicians hired by the drug company. These physicians promote to well-intentioned doctors who trust these physicians, unaware of the fact that they were paid by the drug company to shill these drugs.

In short, pharmaceutical companies have found that once a drug has been approved for a single use, expensive clinical testing for other uses can be dispensed with. All that is really needed is a substantial marketing budget, an able public relations team, and a few unscrupulous pharmaceutical reps, doctors, and researchers.

This has been repeatedly shown to occur in criminal prosecutions of drug companies where billions of dollars in fines for fraudulent, off-label promotional activities have been paid. Now, they are seeking aggressively to extend their ability to promote off-label uses to doctors free of regulation. Now, more than ever, consumers need to stay informed, and they need to demand that their physicians learn everything that they can about the efficacy and safety of a drug before they prescribe it.

Ask your doctor

Off-label prescribing is not restricted by law, and may in some cases actually be beneficial. Your doctor can prescribe any drug he or she thinks is most appropriate for your treatment. But while some drugs have solid scientific evidence to support their off-label use, the majority of them do not. Your doctor may not volunteer information to you about your prescription or may not even know it is off label use, but you still need that information in order to make an informed choice.

Any time your doctor writes you a prescription, or offers you a “free sample” of a new medication, you should ask him these questions:

1. Has this medication been approved by the FDA to treat my condition?
   If the answer is no, then ask
2. Why do you think that this drug will be better treatment for my condition than one that has been approved by the FDA?
3. What evidence supports the use of this drug in my case?
4. Is there an alternative treatment for my condition?

Sources:

• Consumer Reports, “Off-label drug prescribing: What does it mean for you?” May, 2009
• Companies that have been fined for fraudulent off-label promotion include Pfizer, Eli Lilly, Astra Zeneca, GastroSmithKline, Schering Plough, Cell Therapeutics, Purdue-Pharma, and Cephalon. Most have pled guilty to charges, and paid multi-billion dollar fees to the government.
• See, e.g., Los Angeles Times, “FDA’s off-label rule under attack,” November, 2011

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