Safeguarding prescription medications for children

Published: October, 2003

Here's a disturbing fact: Just one-third of medications used to treat children have actually been studied for safety and effectiveness in children. Information on the proper use is lacking for the other two-thirds of medications.

What is the cause of this problem? The reasons for this situation are multi-faceted. Drug companies have little financial incentive to test drugs in children, as they represent a small part of the market. The issue also poses an ethical dilemma because minors can't make informed decisions for themselves. Parents who enroll their child in a study often gain a financial reward regardless of whether or not the study benefits the child. In addition, childhood represents a wide spectrum of developmental stages, each of which may require study.

So what do doctors do? If physicians were to prescribe only those drugs specifically approved and labeled for children they would be left with little to choose from. So most physicians don't limit themselves. Instead, they write "off-label" prescriptions, meaning they use their best clinical judgment to determine how to prescribe a medication that has not been tested in children. This method of prescribing can work out fine. But in some cases, the drug may not work because of under-dosing or it may cause serious side effects related to how the drug works in a child's body.

What's being done to change this? The Food and Drug Administration (FDA) is trying to encourage pharmaceutical companies to study drugs in children. In 1997, Congress passed the FDA Modernization Act (FDAMA), which provides companies that test the safety and effectiveness of their product in children with extended patent protection. A recent investigation set out to determine just what the change in policy has done. Researchers examined the results of studies of 33 drugs in children that were requested by the FDA as part of the Modernization Act. The results of 36% of the studies led to improved understanding of adverse side effects and the drug's action in a child's body. In a few cases, studies revealed that the drug was ineffective. Information gathered from 21% of the studies led to major dosing changes. An added benefit was the development of new formulations specifically designed for children in 15% of the drugs tested.

What can you do to protect your children and ensure their health? Until more drug companies test their products in children, doctors have to make decisions based on their clinical judgment. When a medication is prescribed for your child, talk to the physician about drug choices. Ask why the specific medication was chosen and what is known about this drug and its use in children. Be sure you know about possible side effects, and pay close attention to how your child's body responds to the medication.

October 2003 Update

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