Off-Label Promotion, On-Target Sales

Adriane Fugh-Berman; Douglas Melnick


PLoS Med. 2008;5(10) 

In This Article


For prescription drugs, demonstrated benefits define the parameters of acceptable risks. For example, liver toxicity may be acceptable in a drug approved for cancer, but unacceptable in a drug approved for acne. Government regulatory bodies review laboratory, animal, and human data to confirm that a drug has the claimed efficacy and safety prior to approving its release in the market for specific approved ("labeled") uses.

Once a drug is approved for at least one indication, it may be prescribed off-label for a different condition, a different population, or in a different dose than what the drug is approved for. However, off-label uses have not been subject to the testing and review that is a precondition for marketing approval. The scientific review of evidence of effectiveness and safety that regulators weigh prior to approval for a labeled indication protects the patient. With off-label use, this protection often does not exist.

Off-label prescription of a drug is generally legal, but promotion of off-label uses by a drug manufacturer is usually illegal. This paper addresses public health issues associated with off-label use, and describes techniques by which pharmaceutical companies covertly promote off-label use even where such promotion is illegal.