Less than half of medications include specific labeling for
children, which means pediatricians often must decide what is appropriate to
prescribe based on their clinical judgment. In a revised policy statement, the
American Academy of Pediatrics (AAP) offers guidance to physicians using drugs
The policy statement, “Off-Label Use of Drugs in Children,”
published in the March 2014 Pediatrics (released online Feb. 24), updates a
statement that was published in 2002 and reaffirmed in 2005.
“Pediatricians must prescribe drugs off-label, simply
because an overwhelming number of critical drugs still have no information on
the label for use in children,” said Kathleen Neville, MD, FAAP, lead author of
the policy statement and chair of the AAP Committee on Drugs. “This is an even
larger issue for special populations of children, including preterm infants and
newborns, and in children with chronic or rare diseases.”
According to the AAP, a drug’s off-label status does not
imply an improper or experimental use. Health care practitioners caring for
children can make therapeutic decisions to use drugs off-label based on expert
opinion or on evidence for the medication’s use in a different population.
The passage of the Best Pharmaceuticals for Children Act and
the Pediatric Research Equity Act has resulted in more than 500 labeling
changes, including expanded labeling that includes pediatric information.
According to the AAP, the two laws are an “essential first step” in expanding
the evidence on use of medications in children, but more work remains to ensure
the best possible outcomes for children.
The AAP encourages pediatricians to advocate for research of
drugs for children, and supports the publication of drug trials, including
negative studies, in academic journals. The AAP also advises that health
insurance companies should not use labeling status as the sole criterion to
determine whether a medication is eligible to be reimbursed for use in
children. Less expensive medications that are considered appropriate for adults
should not be automatically considered as first-line treatment in children.
The American Academy of Pediatrics is an organization of 62,000 primary
care pediatricians, pediatric medical subspecialists and pediatric surgical
specialists dedicated to the health, safety and well-being of infants, children,
adolescents and young adults. For more information, visit www.aap.org.