On the other hand, evidence of overall safety of off-label prescription is “limited,” with many “individual reports of harm.”4 For example, off-label prescribing resulting in adverse outcomes includes use of antipsychotics to manage behavioral issues in elderly patients with dementia, certain anticonvulsants to treat bipolar disorder, and fluoxetine hydrochloride for pain. Off-label use does not only refer to prescription of a medication for an indication other than the one for which it was FDA-approved; another off-label category might include long-term prescription of an agent approved only for short-term use.
Off-label prescribing is an “unknown arena,” since the agent has not been subjected to the rigorous review of safety and efficacy that supports FDA approval for a given indication. Many clinicians do not have time or motivation to become familiar with evidence that might affect the off-label indication for which they are prescribing the drug.4 And although the term “off-label drug use” is mentioned extensively in the medical literature, continuing medical education activities, and the media, many healthcare professionals underappreciate its definition, prevalence, and implications.1
Egual et al called upon physicians and physician organizations to “recognize the enormity of the problem and be active participants in the promotion of cautious prescribing of drugs for off-label use lacking strong scientific evidence.”3 They encouraged use of “appropriately configured decision support systems” to “fill the knowledge gap in off-label prescribing by supplying clinicians with information on drug approval status and the degree of scientific evidence at the point of care.” Including in the electronic prescription the indication for which a drug is prescribed would “facilitate communication among physicians, pharmacists, and patients” and “may decrease medication errors.”