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Off-label Drug Prescriptions

In February 2014, the American Academy of Pediatrics (AAP) released a statement on the off-label use of drugs in pediatric patients. As the AAP pointed out, changes in the federal regulations and incentives for pediatric drug research over the last decade have led the Food and Drug Administration (FDA) and pharmaceutical companies to add pediatric guidance to the prescribing labels for more than 500 medications.

Yet, the topic of off-label use remains highly important in pediatrics and public health, because many medications prescribed for children in the U.S. today have never been studied in children.

"While the rate can vary greatly depending on the specialty and environment, in general, less than 50 percent of medication labels have any guidance for providers on a drug's use in children," says Florence Bourgeois, MD, MPH, an emergency physician at Boston Children's Hospital who studies pediatric drug development practices. "At an academic medical center, that rate might even be as high as 60 to 80 percent for the medications used among hospitalized patients."

However, lack of child-specific prescribing guidance does not mean that the FDA prohibits a given drug's use in children. Nor does it mean that the drug is experimental or requires special consent from the patient's family or guardian.

Rather, it means that there are no data or "substantial evidence" from "adequate and well-controlled investigations" upon which the FDA can make a determination either way regarding that drug's safety and effectiveness in children.

"'Off-label' use has negative connotations, but a lack of pediatric guidance is not the same as if a drug were to have a pediatric contraindication," Bourgeois explains. "Rather, it points to a paucity in the evidence about how a given drug should be used."

Looking beyond the label for prescription guidance

When deciding whether to prescribe a drug that has no pediatric labeling, both the AAP and Bourgeois agree that providers must rely on their best judgment and the best evidence available. As both point out, there are several sources providers can turn to for evidence regarding a given medication's pediatric use and safety, such as*:

  • Peer-reviewed literature
  • Consensus statements
  • AAP policies and practice guidelines
  • Information from organizations such as the Cochrane Collaboration and UpToDate
  • The FDA's MedWatch program

"Providers should also rely on their own and their colleagues' expertise," Bourgeois adds, "as well as on any expert opinions and trial data they can access.

"A lack of formal evidence does not mean that there is no evidence to guide prescribing," she continues. "However, finding, compiling and assessing that evidence may be challenging and take additional effort on the provider's part."

Providers can play an important role in helping to build the pediatric evidence base for a given drug, especially for newborns or for children with rare conditions where there may be little to no data available. "The AAP's statement encourages providers to become part of the process," Bourgeois says. "If you routinely prescribe a drug off-label, consider publishing or presenting your experiences."

Cautions and concerns

She notes that providers are sometimes reluctant to prescribe medications to children off-label for fear of causing harm. And while that could prevent children from receiving a potentially beneficial treatment, some hesitation is understandable. For instance, there can be greater medical and legal risks when writing an off-label prescription. Also, some insurance carriers will not cover the cost of drugs prescribed off-label or will discourage patients from filling the prescription.

"Ultimately, every provider is responsible for her own decisions, and if you prescribe a drug and there's harm, there may be increased risk for legal liability," she says.

She adds that providers should consider providing more information to patients and families than they normally would with an approved drug and engage them more closely in the decision.

"Inform the family about the evidence behind your recommendation to prescribe the drug, and be sure to document that evidence before writing the prescription," she advises. "And above all, use your best judgment. Only prescribe a drug off-label if you think there is sufficient evidence that the treatment will benefit the child."

*This list is provided for informational purposes only, and should not be construed as an endorsement by Boston Children's Hospital of any of the included sources.