Risks and Benefits for Patients
Off-label use is sometimes unavoidable; three-quarters of marketed prescription drugs have no labeling indications for children, a population only recently included in clinical trials.[1] Pregnant women are also routinely excluded from studies, so most drug treatment during pregnancy is off-label.
Some off-label use is demonstrably beneficial. For example, in the United States, misoprostol, a prostaglandin, is approved only to prevent ulcers. However, misoprostol is widely used off-label for ripening the cervix, inducing abortion, and other indications, and more than 200 studies involving more than 16,000 pregnant women support the use of misoprostol in obstetrics.[2]
Pregnant women and children, however, do not account for most off-label use. In 2001, 150 million off-label prescriptions were written -- 21% of all prescriptions written for 160 common medications in the US. About three-quarters (73%) of off-label prescriptions were written for conditions for which there was little or no scientific support for efficacy.[3] Up to 75% of drug use in cancer care, and about 90% of drug use in rare diseases, is off-label.[1]
Off-label use of drugs has been associated with serious adverse effects. For example, Duract (bromfenac), an analgesic, was approved only for treating acute pain, and only for short-term use (less than ten days). However, some physicians prescribed Duract off-label for longer durations. Duract caused liver failure, and was withdrawn from the market less than a year after approval.[4] The appetite suppressant Pondimin (fenfluramine), approved for short-term use, was widely prescribed with phentermine and used long-term. The off-label combination "fen-phen" caused valvular heart disease.[4,5] In children, off-label use of drugs is associated with an increased number and severity of adverse effects.[6]
PLoS Med. 2008;5(10) © 2008 Public Library of Science
This is an open-access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. See original citations on the articles and examples below from contract.
Cite this: Off-Label Promotion, On-Target Sales - Medscape - Oct 01, 2008.
