It is a conundrum that has frustrated pediatricians for decades: children get sick and need drugs, yet few medications have been approved for their use. A recent study and a government report published in February concluded that, most of the time, doctors are forced to prescribe drugs to young patients without adequate data, putting kids at risk for overdoses, side effects and long-term health problems. In late June Congress was poised to strengthen existing laws that encourage pharmaceutical companies to test medicines in kids, but that won't solve the safety problems associated with pediatric drugs.

Drugmakers resist testing their products in children because doing so is risky, expensive and difficult—and it is rarely worthwhile from a business perspective because children make up a small percentage of the world's drug consumers. Yet children metabolize drugs differently from adults. “The adage that a child is not just a small adult is true: you can't simply scale down a dose of a drug from adults and expect it to behave identically in a small child,” says Peter Adamson, a pediatrician and pharmacologist at Children's Hospital of Philadelphia. A 2000 study revealed, for instance, that the seizure drug gabapentin (Neurontin) requires higher than expected doses for children younger than five and that it can make them hostile and hyperactive. Pain relievers, antibiotics and asthma medications are among other drugs often prescribed off-label.

Adamson was a co-author of a February Institute of Medicine report highlighting other ongoing issues in pediatric drug safety: some studies have never been made public, others have been too small to yield clinically useful data, and few studies have investigated the long-term effects of drugs in youngsters. The June legislation would grant more power to the FDA to ensure that drugmakers follow through with trials, test drugs in newborns and make past studies public. But it won't address the vital need for data on how drugs taken in youth might affect long-term health.

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