Off-Label Use of Drugs and Medical Devices: A Review of Policy Implications

“Off‐label use” occurs when the use of a medication or device deviates from what is mentioned in its US Food and Drug Administration (FDA) product label. Off‐label use is common, legal, and an important source of innovation; however, it can be costly, and strong evidence of the efficacy and safety of such use may be lacking.1 Given the contradictory and unresolved expectations of major stakeholders, off‐label use remains problematic. It requires a new policy paradigm that can successfully balance the need for innovation against the imperatives of evidence‐based practice and finite health‐care resources. Clinical Pharmacology & Therapeutics (2012); 91 5, 920–925. doi:10.1038/clpt.2012.22