Off-label Drugs Given to Pediatric Patients: Risky Medicine?


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Albuterol helps a child breathe, even though it is considered off-label. Photo by: Intropin.

Risky Drugs? Parent Concerns

Parents are scared enough when their child is in the hospital, especially the PICU.

Do they need to be concerned about doctors using untested drugs on their kids?

According to Dr. Sorenson, although the drugs are used off-label, the doctors are not prescribing untested drugs without careful forethought.

She said, “When drugs are used in this manner, literature searches are done and resources are checked to find any information on that drug in the condition for what it is being used.”

What’s Next? More Testing Needed

Now that we know that doctors are prescribing drugs off-label to children in critical-care situations, I asked Dr. Sorenson about the next steps.

What will ensure the safety of our kids, and are there any plans to forbid off-label drug use in kids?

Dr. Sorenson said, “You can’t stop using drugs just because they are used off-label, like I stated before, if a child needs therapy, literature is checked to see if there is any data and then best judgment is used to treat the child with the drug.”

According to Dr. Sorenson, the answer is better testing in advance of treatment. “The study found that 75% of the drugs used in the PICU were used off- label at least once.   I reported the top 40 drugs that were used most frequently in an off-label manner, 19 still lack pediatric safety and efficacy information.  They are listed and the list can be used to pick drugs to study in children.”

Intensive Care Treatments

It is important to remember that the children in the Intensive Care Unit are very sick with life-threatening illnesses. If there is a drug that may help, a doctor is going to research it thoroughly, and use his or her best judgment to decide if the drug, although not intended for that purpose or tested with that age group, is necessary to save the child’s life. Some of the drugs that were studied included Albuterol, used to help a child breathe, and Epinephrine, used to treat life-threatening allergic reactions. These medications are off-label, but can provide potentially life-saving treatment – the question that remains is, why haven’t these critical-care medicines been tested for proper use and dosage in children?


American Academy of Pediatrics National Conference and Exhibition. Off-label Drug Use in the Pediatric Intensive Care Unit. (2012). Accessed November 2, 2012.

American Academy of Pediatrics. Uses of Drugs Not Described in the Package Insert (Off label uses). (2002). Accessed November 2, 2012.

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