Using a drug for purposes not approved by regulatory authorities
Off-label use refers to the prescription or administration of a pharmaceutical product for a purpose, population, dosage, or route of administration not approved by the regulatory authority (the FDA in the United States). While physicians can legally prescribe drugs off-label based on their clinical judgment, pharmaceutical companies are generally prohibited from marketing drugs for unapproved uses.
The prohibition on off-label marketing exists because FDA approval requires rigorous testing demonstrating safety and efficacy for specific indications. Off-label use bypasses this safety framework — patients may be taking drugs whose risks and benefits for their specific condition have never been systematically studied.
Despite the legal prohibition, pharmaceutical companies have repeatedly been caught promoting off-label uses through various channels: funding studies designed to generate data supporting unapproved uses, paying physicians as "key opinion leaders" to give lectures about off-label applications, and using sales representatives to directly promote unapproved uses to prescribers. GlaxoSmithKline paid $3 billion in 2012 to settle charges including off-label promotion of antidepressants for children. Pfizer paid $2.3 billion in 2009 for off-label promotion of multiple drugs. These fines, while large in absolute terms, often represent a fraction of the revenues generated by the off-label sales.
