When receiving a doctor’s prescription, you can assume that the drug has been approved for use by a federal regulatory agency (e.g., Food and Drug Administration). This approval process includes providing evidence that the drug is safe and effective for specified medical uses. But can you assume that your doctor is using this medicine for one of the approved uses?
OLD DRUGS, NEW USES. Once approved for any medical indication, a drug can be prescribed by licensed doctors in the United States and the United Kingdom for any purpose they professionally believe to be safe and effective. Off-label prescribing is the use of a drug for a non-approved use, and one in five of all prescribed drugs is off-label. This figure is far higher in pediatrics and for psychiatric, antiseizure, and cancer medications.
Advocates for off-label drug use argue that the approval process can take a decade, during which time patients do not benefit from a drug that has the potential for saving lives. The cost of bringing a new drug to market is said to approach $1 billion, and approval of a new use for an already approved drug can cost hundreds of millions of dollars. Why would a drug sponsor (pharmaceutical manufacturer) expend such funds to convince the FDA that an older generic drug is effective for a new condition when it’s being already prescribed? Why invest in orphan drugs that are intended for conditions in a very small fraction of the population, when the costs of testing might never be recouped by sales?
Although off-label prescribing is legal and recognized by the FDA, it is illegal for a pharmaceutical manufacturer to actively promote a drug for an off-label use. Companies found to have done so have been fined many hundreds of millions of dollars—usually far less than the profits made from such sales.
Off-label prescribing raises a number of ethical questions: Should doctors inform patients that their prescribed drug is not FDA-approved for that use? In the absence of evidence that the drug is safe and effective for a condition, is the prescriber experimenting on patients? Should patients be offered the opportunity to consent to an off-label use?
SEE ALSO Clinical Testing of Drugs (1753), Food and Drug Administration (1906), Federal Food, Drug, and Cosmetic Act (1938), Kefauver-Harris Amendment (1962).
This doctor may be prescribing an FDA-approved medicine for an off-label use—that is, for a medical condition for which the drug has not been evaluated to be safe or effective by the FDA.