Making Decisions on When to Use Off-Label Drugs

But talking to patients about a term—“off-label”—that sounds more jarring to them than to physicians can sometimes unnecessarily worry patients.

Dr. Sataloff said he puts patients at ease, in part, by never using a scripted message. “I tailor my discussions to the individual patient. Sometimes that happens with extremely bright, well-educated people. Sometimes that happens with people with no medical knowledge at all, who just went onto the Internet,” he added. “Depending on who they are and what their concerns are, we explain to them what we’re doing, why we’re doing it, what the alternatives are—which, in many cases, are no treatment—and what the risks are associated with no treatment.”

Usually, that does the job.

“There are always people who get it in their head that something is not right, and, no matter what you tell them, you can’t change their mind,” Dr. Sataloff said. “But they are a small minority.”

Dr. Gillespie suggested explaining to patients that the term off label is really what he calls a “bureaucratic decision.”

“That really doesn’t say much about the state of the science, because the FDA will often accept things on label with lower levels of evidence than Level 1 evidence,” he said. “It’s really just that someone’s taken the time to go through the government application process to get the government’s stamp of approval on a particular treatment.

Dr. Gillespie noted that if a patient trusts their otolaryngologist, then they shouldn’t be put off by the lack of a randomized, controlled trial backing that physicians’ recommendation. “There’s never been a randomized, controlled trial on whether a parachute works,” he said. “But we know that if you jump out of a plane without a parachute, things will not go well. And there’s things as equally evident that we do … that almost make a randomized, controlled trial unnecessary.”—RQ