Broadly speaking, “off-label” drug use occurs when a doctor prescribes a medication for a condition or ailment that is not specifically approved by the FDA. When the FDA approves a drug, the manufacturer is required to submit studies showing that a drug works for a specific condition and that it is safe. Sometimes, however, a drug is shown to have beneficial effects on a condition, but the FDA will not approve the drug for that specific use. For instance, I take a drug for migraine headaches that has only officially been approved for treatment of clinical depression. This is considered “off-label” use.
No law prevents a physician from writing a prescription for an off-label use, and private insurance companies (my own included) frequently pay for drugs even when used for an “off-label” purpose. Nevertheless, “off-label” drug use presents the potential for fraud. Drug companies are barred by the FDA from marketing a drug for off-label use – for obvious safety reasons.
This rule, however, has not stopped the deep-pocketed pharmaceutical companies from trying to find back door methods to promote their drugs for off-label uses, in order to increase their bottom line. A few years ago, GlaxoSmithKline paid over $1 billion to settle False Claims Act allegations regarding its illicit marketing of drugs, including Paxil and Wellbutrin, for off-label uses. Among other problems, the government found that GlaxoSmithKline had been paying kickbacks to doctors in exchange for prescribing the drugs for off-label purposes. (The total settlement was over $3 billion, but only about one-third was related to off-label marketing.)