Approval from the Food and Drug Administration (FDA) does not make any and all uses of that drug approved under federal law. The regulatory approval process requires drug manufacturers to objectively validate the claims submitted. Although a drug manufacturer may not advertise or promote an approved drug for a non-approved use, physicians are not prohibited from making a clinical judgment to prescribe the drug for a so-called "off-label" use by a particular patient. That does not mean that the decision is without risk. The further away the use is from scientific evidence and similar use of the drug by other clinicians, the more likely the use may be viewed as experimental. An informed consent process and documentation of a dialogue regarding the rationale, risks, benefits, and alternatives to the off-label option should be part of the clinician's and the patient's decision making.
An emerging risk in this area could stem from the FDA's "expedited development and approval" and "accelerated approval" processes. Both of these programs involve a shortened and potentially less stringent approval process by the FDA. These may increase risk exposure in off-label use of a drug which had undergone an accelerated review that may or may not have demonstrated all potentially significant adverse drug effects.
