Pediatricians can’t always follow FDA medication labels

The Pediatric Insider

© 2014 Roy Benaroch, MD

You know those little packets of paper, folded over eight times, written in type that can’t possibly be seen? You’ve seen them glommed onto the side of stock bottles of medications, or shoved into the paper box alongside a tube of prescription ointment. That unreadable thing is the official “product information”, often called the “label”, required for every FDA-approved medication. It contains all sorts of information, including lists of side effects (Nausea! Anal leakage! Growing a tail!) and what a medicine is “supposed” to be used for.

But often, that information isn’t the best information. It’s just what the FDA approved. Science marches on, and new published information is not routinely included in the product insert. Doctors who pay attention know that there’s usually a whole lot of more-reliable, more-specific, and more-accurate information about here than what’s on the label.

The discrepancy is especially stark with babies and children. According to a new AAP report, “Off Label Use of Drugs in Children,” less than 50% of medications have information about pediatric usage in the FDA-approved product labeling. If you have a child who has been prescribed medication, it’s almost certain that at least some of them have been used “off-label,” in a way that is not specifically approved by the FDA.

As the AAP report outlines, using drugs in this way is not incorrect, unethical, or inappropriate:

The absence of labeling for a specific age group or for a specific disorder does not necessarily mean that the drug’s use is improper for that age or disorder. Rather, it only means that the evidence required by law to allow inclusion in the label has not been approved by the FDA.

Those sound like weasel legal words, I know—but it worth understanding what the FDA’s role here is. The FDA is empowered to regulate the manufacturing, labeling, advertisement, and safety of medications. For a drug to be “FDA approved” for sale in the US, a sponsor has to provide information usually including two large, randomized trials to prove that medicine is safe and effective. Safe in the population studied, and effective for the disease studied. The FDA “approves” the drug for sale using specific parameters from these studies, and does not allow the sponsoring company to promote the use of the medication for any other purpose.

But once a drug is approved, a licensed physician can legally prescribe it for any use. The FDA does not regulate the practice of medicine or how doctors use medication (that can be regulated by state medical boards, if a doctor does stupid or unethical things with a prescription pad.) It is up to licensed prescribers to use their own judgment, based on the totality of the evidence, to make prescribing recommendations. Limiting our knowledge to what’s in the product information sheet—which is seldom modified or updated after a drug is approved—would be unethical and foolish.

In most circumstances, using a drug off-label (for an indication or age not explicitly approved in the FDA label) is not “experimental” or “research.” As long as it’s in a patient’s best interests, using a medication this way does not require any specific information be shared with the patient, beyond what we should be talking about with any medication (risks, benefits, side effects, etc.) However, it’s usually best for doctors to talk about the off-label use in cases where off-label prescribing is unusual or cutting-edge. In pediatrics, that’s seldom the case. We write off-label meds every day, in ways that have sometimes been used for decades, and I would bet that most pediatricians don’t even know when they’re doing it.

That’s because the best therapeutic decision making is not based on the label. It’s based on all of the evidence out there: published studies, experience, judgment, and the best interests of the patient.

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