NEW ORLEANS – “Off label”
drugs are medications that have not been tested for safety or efficacy for a
specific patient age or condition. New research presented Oct. 21 at the
American Academy of Pediatrics (AAP) National Conference and Exhibition in New
Orleans found that off-label treatments were ordered for 96 percent of all
pediatric patients, and 100 percent of patients ages 13-17, in the intensive care unit of an urban
In “Off-Label Drug Use in the
Pediatric Intensive Care Unit,” researchers collected data on all patients
admitted to the 32-bed pediatric intensive care unit (PICU) at Primary
Children’s Medical Center (PCMC) in Salt Lake City from October 2002 to February
2003, including age, diagnosis, medications ordered and indication for each
medication ordered. Each drug was assessed for whether it was used in an
on-label or off-label manner.
Off-label use was declared
when a drug was prescribed for a patient whose age was not listed on the
package label, no pharmacokinetic (PK) data was listed in the package insert,
and/or if the drug was used for a non-FDA
A total of 335 drugs were
prescribed in 492 pediatric patients from birth to age 17. Ninety-six percent of
patients received at least one off-label drug. Off-label prescribing occurs in
almost every patient in the PICU.
“Treatment with off-label medications is the rule rather than the exception in
the PICU,” said study author Susan Sorenson, a doctor of pharmacy.
“Numerous problems emanate
from the lack of drug data in children, including uncertainty about whether a
drug is effective in children for a particular disease, questions about the
side effect profile, and lack of dosing information,” said Dr. Sorenson.
“It is very difficult when
you stand at the bedside and want to treat a sick child with a drug and you
don’t know if the dose or drug you have chosen or recommended will harm the
child or help the child. Everyone does the best they can to find
suggested doses and do the right thing, however, it is better medicine to dose
or recommend doses based on evidence.
“This study is attempting to determine the drugs that are
used most frequently in our sickest patient population that do not carry dosing
information, with the intent that studies of dosing, safety, and efficacy will
be carried out on those drugs,” said Dr. Sorenson. “More studies need to be
conducted so that prescribing in our youngest and sickest patients can be done
based on evidence.”
The American Academy of Pediatrics is an organization of 60,000 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.