The Food and Drug Administration approves, or labels, medications to treat specific conditions based on rigorous research.
Many drugs treat multiple ailments, and doctors may prescribe – and health insurers frequently cover – them for "off-label" purposes, sometimes when no medication has been approved for the condition.
But off-label uses have not undergone the extensive – and, for drug companies, extremely expensive – large clinical trials required for FDA approval, and manufacturers are limited by law in how they may promote them off-label. Marketing for uses that are specifically contraindicated on the label is forbidden.
Companies have paid billions of dollars to settle state and federal charges of off-label promotion, and the regulatory environment may be changing. A federal judge ruled last year that the First Amendment right to free speech allowed a small company to market its drug for unapproved uses as long as its language was truthful; the FDA then made an exception in that case.
"Off-label communication is not necessarily bad. It is frequently beneficial," said Rick Collier, an adjunct professor of drug and medical device law at Temple.
The FDA has scheduled a two-day meeting about off-label communication in November.