Glenn Ellis

*Millions of Americans each year are prescribed drugs that are not approved for their specific medical condition, a practice that is legal and logical in many cases. It is common for doctors to prescribe drugs for conditions they aren’t specifically approved for, but in many cases there is not enough evidence to justify the practice.

In a recent national survey, a substantial minority of physicians falsely believed that certain off-label uses of prescription drugs were approved by the Food and Drug Administration. This mistaken belief could encourage them to prescribe these drugs, despite the lack of scientific evidence supporting such use.

Let’s start with an informal and short definition of so-called “off-label” prescribing: A drug is being prescribed “off-label” when it has not been approved for treatment of the patient’s medical condition and, instead, is being used experimentally.

While most people assume that the medicines prescribed by doctors in the United States have the FDA’s stamp of approval, that’s only partially true. The FDA regulates the marketing of prescription drugs, not prescribing. The agency approves drugs for marketing with an official “label” that stipulates an indication, dose, intended population, duration of use, and other specifications. However, physicians and other licensed prescribers are free to prescribe any approved drug for any indication, whether or not the indication is included on the drug’s FDA-approved label. As long as the FDA has approved a drug for one condition, physicians are free to prescribe it for anything.

In fact, according to the FDA website, the agency says, “Good medical practice and the best interests of the patient require that physicians use legally available drugs, biologics and devices according to their best knowledge and judgment. If physicians use a product for an indication not in the approved labeling, they have the responsibility to be well informed about the product, to base its use on firm scientific rationale and on sound medical evidence, and to maintain records of the product’s use and effects.”

To get a drug approved by the FDA, a pharmaceutical company must complete three rounds ( or phases) of testing in human subjects to demonstrate its safety and effectiveness in treating a specific condition. Once a drug is approved and on the market, though, physicians may choose to prescribe it for any condition. But this carries unknown risks because often the drug hasn’t been rigorously tested on patients with that condition.

However, drug companies have only been allowed to advertise and market drugs that have been FDA-approved for specific uses or disorders. That means that generally an antidepressant drug can only be marketed and advertised for depression. If a company wants to market and advertise its drug for other uses, it has to go back to the U.S. Food and Drug Administration (FDA) and file additional applications, with a wealth of supporting research to support those uses. Doctors, on the other hand, have always been able to prescribe whatever drug they want, for whatever they want.

A 2006 examination of off-label prescribing of 160 common drugs, found that off-label use accounted for 21 percent of all prescriptions and 73 percent of these uses had little or no scientific support A fifth of all drugs are already being prescribed off-label, and amongst psychiatric drugs, one out of three psychiatric drugs are being prescribed for a use not approved by the FDA.

The real problem with off-label use is that it has less evidence, and lower quality evidence, to support prescribing the drug. No more than 30% of the off-label practices we observed were supported by strong scientific evidence.

That means in 70% of the data they analyzed — the vast majority — doctors were writing prescriptions for off-label use of drugs with minimal empirical evidence to support that prescription.

It is illegal for pharmaceutical companies to actively market their drugs for uses that they are not specifically approved for, but sales reps are allowed to tell doctors about published research that supports these off-label uses.

There are, however, clearly examples of the benefit of off-label prescribing.

Originally approved by the FDA to be used for treating pulmonary hypertension, doctors began noticing a common side effect of men who were taking the drug Sildenafil. They reported that their sex life had improved due to noticeable improvement in their ability to have an erection.

The world of erection impairment changed dramatically on March 27, 1998—the day the Food and Drug Administration approved the little blue pill. Viagra is used for treatment of erection impairment, now medically called erectile dysfunction (ED). Viagra took the U.S.—and the world—by storm. During its first month of availability, American doctors wrote more than 300,000 prescriptions, making Viagra the fastest-selling new drug in history. Within six months, repeat prescriptions were being written at the rate of 100,000 per month. Today, Viagra is one of the most successful drugs ever marketed. More than 10 million men have taken more than 125 million tablets. A class of drugs called beta-blockers, for example, was initially approved in the 1980s to treat high blood pressure. But researchers and doctors quickly theorized — and evidence mounted from subsequent studies — that beta-blockers (such as propranolol and metoprolol) would be effective against angina and heart attack.

Doctors began to prescribe beta-blockers “off-label”. They were right and it worked. The labeling of all beta-blockers now reflects their significant benefit for heart disease generally.

So what should you do when it seems that your doctor is suggesting, or prescribing, a drug for an “indication”, or treatment use, other than what that drug was approved for by the FDA? Talk with your doctor if you have concerns about any medicine or treatment, particularly if it may be off label. Here are several questions to ask:

  • Is this the approved use of the medicine? You may not know if the use is off label. This question can help you start the conversation with your doctor about your medicines.
  • Is the off-label use of this drug likely to be more effective than one approved to treat my illness? This is important because the off-label drug may not be as well tested for your condition.
  • What evidence shows that this off-label drug can treat my condition?
  • What are the risks and benefits of off-label treatment with this drug? Will my health insurance cover off-label treatment with this drug?

Critics say that off-label drug prescribing can amount to an uncontrolled experiment, and that not only do most patients not know when a drug is being prescribed for a condition for which it has not been approved, neither do most doctors.

Off-label drug use is sometimes necessary – in cases of last resort where all other approved drugs have failed.

Whatever you decide, it is important that you make an informed decision. That’s why I do what I do.

Remember, I’m not a doctor. I just sound like one.

Take good care of yourself and live the best life possible.


This column is written for informational use only. It should not take the place of advice from your doctor or healthcare provider.

Glenn Ellis is a Health Advocacy Communications Specialist and author of, Which Doctor? He writes a national column, provides media commentary, and lectures on relevant health issues.

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