Posted On: November 2, 2012 by Patrick A. Malone

Off-Label Drugs Commonly Given to Intensive Care Patients

At the American Academy of Pediatrics conference last month, a researcher presented a paper that should give pause to anyone receiving or delivering treatment in a pediatric intensive care unit (PICU).

Susan Sorenson, who holds a doctorate in pharmacy, studied the use of 335 different drugs used in a PICU over a five-month period and found that 3 out of 4 were prescribed “off-label” at least once. “Off-label” is the practice of prescribing a drug for a condition or demographic other than the one for which it received FDA approval. Doctors have the right to so prescribe, and it’s often appropriate to do so, but the drug manufacturer may not promote a drug for any use other than what the FDA granted.

But as described in a report on MedPage Today, what appears to be a routine practice exposes patients to medications that may not have been adequately studied in children. Even if some compounds are suitable for pediatric use, if they haven’t been studied for that population it can be difficult to determine the proper dose and know the possible side effects and risks.

The study has not been published in a peer-reviewed journal, so it should be considered preliminary. The study group was limited to a single, 32-bed facility in Salt Lake where nearly all the patients (96 percent) were given a drug off-label at least once.

Drugs were considered to have been used off-label when prescribed for patients whose ages were not listed on the label, when no pharmacokinetic data (how drugs behave in the body over a period of time) were listed for the patient's age or when they were used for a disorder not approved by the FDA. The study involved 490 patients with ages ranging from 4 days to 17 years.

The most common medications prescribed off-label were morphine, ondansetron (Zofran, for the nausea and vomiting caused by chemotherapy and radiation treatments ), metoclopramide (Reglan, for heartburn and esophageal problems associated with gastroesophageal reflux disease), dopamine (for a wide variety of problems including shock, low blood pressure/heart attack, trauma and infections) and fentanyl (for breakthrough pain from cancer; that is, sudden pain that occurs despite other, ongoing analgesic treatment).

Recent legislation, including the the FDA Modernization Act (FDAMA) and the Best Pharmaceuticals for Children Act (BPCA), has addressed the issue of improved labeling for pediatric drugs. But, Sorenson told MedPage Today, “There’s still some work to do.”

We’ve written about the difficulty in researching and labeling drugs for pediatric applications and the fact that not even half of drug labels carry child-specific information.

Since passage of the BPCA in 2002, only 14 of 40 drugs with the highest number of off-label medication orders during the study have had labeling changes; 19 still lack safety and efficacy information for a pediatric population.

That seems like a roadmap of where to go next, but as Sorenson said, most of the drugs used in the study are off-patent, so it will be difficult to find funding to perform such expensive studies. If manufacturers cannot monetize new or additional research, they’re not motivated to conduct it.

There is some legislative relief: The BPCA extends patent protection for six months if a drug manufacturer agrees to perform pediatric studies. That can be added to the six months of market exclusivity granted by the FDAMA for performing studies in children.

If your child is in the PICU, closely monitor whatever drugs the doctors prescribe. Ask what they are, why they are being given, what is their intended effect and if they have been tested specifically for children or if they are being prescribed off-label. If the latter, ask if there are alternative therapies. And always ask what are the potential side effects and harms.

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