Off-Label Prescribing: A Call for Heightened Professional and Government Oversight

TLDR

Off‑label prescribing is a common and often necessary practice in modern medicine, providing therapeutic options when approved indications are lacking, yet it can pose risks, particularly when lacking robust evidence, and gaps remain in understanding its safety and efficacy, especially in pediatric populations. A 2006 analysis of 169 frequently prescribed drugs revealed that off‑label use was widespread and frequently unsupported by scientific evidence.

Abstract

Off-label prescribing is an integral part of contemporary medicine. Many patients benefit when they receive drugs or devices under circumstances not specified on the label approved by the Food and Drug Administration (FDA). An off-label use may provide the best available intervention for a patient, as well as the standard of care for a particular health problem. In oncology, pediatrics, geriatrics, obstetrics, and other practice areas, patient care could not proceed without off-label prescribing. When scientific and medical evidence justify off-label uses, physicians promote patients’ interests by prescribing products off label. Off-label prescribing can also harm patients, however. The potential for harm is greatest when an offlabel use lacks a solid evidentiary basis. A 2006 study examining prescribing practices for 169 commonly prescribed drugs found high rates of off-label use with little or no scientific support. Researchers examining off-label use in U.S. children’s hospitals concluded, "[W]e still have incomplete knowledge about the safety and efficacy of many medications commonly used to treat children across a range of drug classes and clinical diagnoses.”