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Guidance on off-label drug use in pediatric population updated


The American Academy of Pediatrics (AAP) has issued a policy statement updating its recommendations for off-label drug use in the pediatric population.

The American Academy of Pediatrics (AAP) has issued a policy statement updating its recommendations for off-label drug use in the pediatric population.

“Off-label use of drugs in children” updates the Academy’s recommendations from a decade ago, which resulted in more than 500 labeling changes.

Less than half of all medications include specific labeling for children, according to Children’s Mercy Hospital in Kansas City, Mo., which is the practicing home of Kathleen Neville, MD, who led the AAP’s Committee on Drugs in drafting the updated guidelines. Thus, the new statement attempts to further define and discuss the status of off-label use of medications, particularly for special pediatric populations such as preterm infants and children with chronic or rare diseases.



The new recommendations emphasize that the prescribing practitioner is ultimately responsible for deciding which drug and dosing regimen is appropriate and for what purpose, and that the decision should be based on information contained in the drug’s labeling or other available data, such as sound scientific evidence, expert medical judgment, or published literature. As long as use is based on one of these, off-label use is neither incorrect nor investigational, according to the AAP.

The AAP encourages: 1) pediatricians to continue to advocate for necessary incentives and requirements to promote the study of drugs in children; 2) researchers to continue to study the use of drugs in children with well-designed pediatric drug studies and national consortium studies; and 3) journals to publish the results of those investigations.

Lastly, the Committee on Drugs instructs institutions and payers to not use labeling status as the sole criterion for determining reimbursement, and to not automatically consider less-expensive therapeutic alternatives for adults as appropriate first-line treatment for children. 

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