Off-Label Prescribing of Drugs for Children

Albert      Wertheimer

Abstract

Under normal circumstances, children are excluded from clinical trials of new drug candidates. Yet, when the drug product is approved for marketing by the authorities, it is only a matter of time before physicians use that drug on pediatric patients. We do know that the practice of prescribing drugs for off-label conditions is found in approximately 50% of all physician prescribing. We should not look at off-label pediatric drug use as always a bad or negative behavior. It may be that a manufacturer has conducted a successful clinical trial for a new pediatric condition and has submitted the documentation to the FDA staff to support that new indication, but the regulatory agency has not completed its review and formally awarded the new indication to the sponsor. Or it might be the case that over a dozen years of clinician experience informs all clinicians that an offlabel use of a drug works most effectively and yet the manufacturer is reluctant to spend the money required for a supplemental investigation when it loses patent protection in under a year. As with many other situations where not all of the facts may be known, it seems that the wisest path is to approach the use of drugs for off-label conditions with great caution and with as much knowledge as may be gained before the prescribing decision is arrived at.

Keywords: Off-label prescribing, off-label drug use, children, pediatrics, FDA, anticonvulsants, NDA, FDAMA, antibiotics, antidepressants, SSRIs, drug therapies, USP-DI