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AAP updates off-label drug-use policy
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The number of drugs approved by the FDA with pediatric indications or expanded labeling has increased in recent years, but many pharmaceuticals used in children still have no labeling for pediatrics, according to the AAP.
The Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act resulted in more than 500 labeling changes, but off-label drug use in pediatrics remains an important public health issue, according to the AAP Committee on Drugs, which drafted the most recent off-label use of drugs in children policy statement published in Pediatrics.
The AAP states that physicians prescribing drugs are responsible for deciding the drug and dosing regimen. The AAP recommends that information contained in the drug’s labeling and other information available to the prescriber should be used when making the decision. Scientific evidence, expert medical judgment or published literature should be the basis for the choice of drug, which makes off-label drug use neither incorrect nor investigational.
The statements also recommends:
- Advocating necessary incentives and requirements to promote the study of drugs in children should be continued.
- The rational and critical study of drugs in children should be continued by researchers.
- Results of well-designed investigations, including negative studies, should be published by journals.
- And labeling status should not be used as the only criterion by institutions and payers determining the availability on formulary or reimbursement status for medication in children.
“Off-label drug use remains an important public health issues, especially for infants, young children, and children with rare diseases,” the researchers wrote. “Evidence, not label indication, remains the gold standard from which practitioners should draw when making therapeutic decisions for their patients. The Pediatric Research Equity Act and Best Pharmaceuticals for Children Act have been extremely successful and represent an essential first step in expanding this evidence as a means of achieving the ultimate goal that any and all drugs used to treat children will have age-appropriate evidence sufficient to provide information for labeling. However, labeling with pediatric information still exists in less than 50% of products, such that much work remains to be done to ensure the best possible practice for therapeutic decision-making in pediatrics.”
Disclosure: The researchers report no relevant financial disclosures.
Perspective
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Edward A. Bell, PharmD, BCPS
This statement by the Committee on Drugs updates a previous policy statement from 2002 and highlights that much has occurred to the better in assessing and labeling drug products for use in the pediatric population. Two significant regulatory acts, the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act, have resulted in more than 500 pediatric labeling changes. While this is certainly an improvement, greater than 50% of currently available commercial drug products still do not contain pediatric labeling. Thus, we have come a long way, but we still have quite a long way to go. This short statement is an important read for pediatric prescribers, as it summarizes how pediatric drug labeling may affect prescribing, including therapeutic, legal and ethical manifestations. This statement additionally summarizes “where we are” with respect to off-label drug use in pediatrics. Nearly every pediatric health care provider has likely prescribed a drug product off-label to a patient. As discussed in this statement, evidence, and not label indication, remains the driver for sound therapeutic prescribing habits when caring for our patients. It is, therefore, helpful for prescribers to keep abreast of the medical literature for this evidence. Representatives of pharmaceutical manufacturers are not good sources for off-label use of drug products, as law prohibits them from doing so. The published literature remains the best information source.
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