Copyright ©2008 NPR. For personal, noncommercial use only. See Terms of Use. For other uses, prior permission required.


Testing drugs takes a lot of time and money, and it's even harder with children than with grownups. That means fewer medications approved for kids and a real dilemma for parents and pediatricians. When is it OK to use a drug off-label, meaning for a purpose or a group that's not formally approved by the FDA? Dr. Sydney Spiesel reviews medical literature for us, and he writes for the online magazine Slate. He's a professor at Yale Medical School, and he has his own practice. Syd, welcome back to the show. There's been a new study on this. What do you find there?

Dr. SYDNEY SPIESEL (Pediatrics, Yale Medical School): The study, in fact, looks at the use of off-label medications in general. Mostly it's focused on psychiatric medications. You know, when a drug company comes up with a new medication, the FDA approves it, usually, for a specific application and for a specific population. But once a medication has been approved for anything, doctors can use it for other applications, and that's what off-label treatment is. And unfortunately, it's very expensive to test medications, and so, very often, once a pharmaceutical company can sell a medication directed at adults, they don't bother to test it on kids. In fact, somewhere between 50 percent and 75 percent of all medications used by pediatricians are, in fact, used off-label.

One of the great serious things that are confronting all of us is the problem of increasing awareness of mental health problems. And in the old days, mental health problems were addressed largely by psychotherapy. And then it turned out that there were very helpful medications that could sometimes be used. And in general, it's cheaper to use medications for mental health problems.

CHADWICK: And how do these off-label drugs generally perform there?

Dr. SPIESEL: Some of them have performed pretty well. The SSRIs, the antidepressants, which are very commonly used - drugs like Prozac are very commonly used - and for some kids with depressive disorders and other kinds of disorders, the drugs are sometimes helpful.

CHADWICK: So, in the world of psychiatric treatments, how far have we come in developing medications specifically for children, and where are some of the places you think we need to go?

Dr. SPIESEL: It's hard to think of any medications that have been developed specifically for children. We're using, as I say, the antidepressants. We're using the new anti-psychotic drugs for serious mental illness. One place which has gotten a tremendous amount of pediatric uses are the stimulant drugs for attention deficit syndromes. But there are things that are tremendously missing, things like addiction disorders and anxiety disorders and oppositional defiant disorders. Most of these we think would be best treated with psychotherapy, but it would be really nice to know, do we have medications that would help psychotherapy and perhaps medications alone would be helpful for some of these?

CHADWICK: Does the paper maybe explain how a doctor can use off-label drugs in a way that's going to be ethically sound so the doctor doesn't feel that they're maybe abusing their authority to prescribe medications?

Dr. SPIESEL: If I could only prescribe things on label, I would not be doing my patients a favor. Kids would be horribly sick, would be hospitalized. The paper does address that. For example, when we know that there's a risk, based really often on adult stuff, adult experiences, if a doctor knows that there's some risk of certain specific medical problems associated with drugs, it suggests, and I think appropriately, we should do appropriate laboratory testing, based on known side effects.

Another thing which is really important to do, and I don't think we do it enough, is to actually be looking seriously at effectiveness when we start treating kids with these medications. For example, are there changes in school performance? Is there improvement in social development? One of the things that we don't look at enough is, is there good compliance? In general, when kids feel they are being helped, and adults, too, feel they're being helped by medication, they're more likely to continue to take it. If the kids are kind of blowing it off, that suggests the medication may not be very helpful.

CHADWICK: Dr. Sydney Spiesel. He's a practicing pediatrician and a professor at the Yale Medical School. You can read his "Medical Examiner" column with our friends at Syd, thank you again.

Dr. SPIESEL: Thank you, Alex.

CHADWICK: Stay with us. More to come on Day to Day.

Copyright © 2008 NPR. All rights reserved. No quotes from the materials contained herein may be used in any media without attribution to NPR. This transcript is provided for personal, noncommercial use only, pursuant to our Terms of Use. Any other use requires NPR's prior permission. Visit our permissions page for further information.

NPR transcripts are created on a rush deadline by a contractor for NPR, and accuracy and availability may vary. This text may not be in its final form and may be updated or revised in the future. Please be aware that the authoritative record of NPR's programming is the audio.



Please keep your community civil. All comments must follow the Community rules and terms of use, and will be moderated prior to posting. NPR reserves the right to use the comments we receive, in whole or in part, and to use the commenter's name and location, in any medium. See also the Terms of Use, Privacy Policy and Community FAQ.