Off-Label Use of Medications

MYTH: 

Medications must have full FDA approval for use in the treatment of bipolar disorders.  Off-label use is experimental medicine and is frowned upon.

FACT: 

FDA approval means only that the FDA has approved the drug company to label or advertise its drug for a particular use.  Nothing about the labeling system is meant to control, limit or guide doctors and their prescriptive practice.  In fact, a great deal of medicine is practiced off-label.  Those who don’t understand this do not understand the practice of medicine.

As stated by Henry Crabbe, M.D., Ph.D., M.B.A. in a text he authored for clinicians practicing in Connecticut, “Considerable confusion exists regarding the prescription of psychotropic medication with unlabeled indications.  Information included in the Physicians’ Desk Reference (PDR) may not necessarily reflect current and standard medical practice and is used by drug companies for marketing and advertising purposes.”

The Food and Drug Act of 1938/1962 provides first that a drug is safe and effective for the use in the U.S. and, second, that the claims made by the drug manufacturer are true and accurate.  Thus, low-dose aspirin is used by doctors to prevent first heart attack in people at risk – though aspirin is not FDA approved for that indication.  The label on the bottles of 81mg aspirin even states you must take eight pills for the “treatment of headache” - yet both doctors and the manufacturers know that the 81mg dose is only of use in heart attack and stroke prevention.  However, because of  the high cost to the manufacturer of the FDA process to add the indication “to prevent first heart attack”, and because of the availability of generic forms of aspirin, no manufacturer would recover, in increased sales, its’ cost of going thorough the FDA process.

Other examples of off-label uses of medication include stimulant medication to treat adults with Attention Deficit Hyperactivity Disorder, Klonopin™ or Xanax™ to treat panic disorder, Elavil™ to treat chronic pain and almost any antidepressant or mood stabilizer to treat children or adolescents.

The only medication currently FDA approved for the prophylaxis (prevention of relapse) is lithium.  Other drugs are approved only for acute treatment.  Nevertheless, good practice demands that patients be able to benefit from a host of off-label uses of medications.

Robert Post, M.D., Chief, Biological Psychiatry Branch of the National Institute of Mental Health and Director of the Stanley Foundation Bipolar Network has stated, “To the extent one continues to utilize only the FDA approved mode of therapy in patients who are unresponsive to these modalities, it constitutes inferior medical treatment, if not outright malpractice.”