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Should Your Child Take Part in A Clinical Trial?

You may have heard of clinical trials, and your doctor may even have suggested your child enroll in one. Here, from the experts at the federal Food and Drug Administration (FDA), are some of the things to consider:

Clinical trials are voluntary research studies conducted in people and designed to answer specific questions about the safety and/or effectiveness of drugs, vaccines, other therapies, or new ways of using existing treatments. If your child were to enroll in one, he or she could be offered a new treatment that may (or may not) be better than those already being used.

By law, giving a study drug to children must provide a potential for clinical benefit that justifies the risk of taking the drug, and the children who participate must have the disease or condition being studied. Under limited circumstances, children may be exposed to the risks of a drug (or other intervention) that does not offer any clinical benefit, provided that the risks are sufficiently low and the information to be gained is important to understanding or improving the disease or condition.

In addition, clinical trials yield important information on a drug’s safety, dosing, and/or effectiveness. Together, this information forms the basis for FDA approval; in addition, it guides whatever information goes in the product label, including about the right dose to use for which patients.

Only about 50% of drugs approved by FDA have been labeled for use in children to date. So, by necessity, doctors routinely treat children with a drug that has been tested in adults and approved for adult use, but may be untested in children or tested in a different group of children. (For example, the drug may have been tested in adolescents, but not in neonates.)

Children’s responses to drugs can’t always be predicted from data collected in studies of adults. A child grows, and the metabolism changes as he or she gets older. So how a drug affects a newborn may be quite different from how it affects an adolescent.

“These changes mean the child may have a different susceptibility to side effects over time, even if the dose remains the same. Or we may need to use a different dose to have the same treatment effect. We may think we can predict some of these differences, but we really can’t without studying them,” says Robert (Skip) Nelson, M.D., Ph.D., Deputy Director and Senior Ethicist of the Office of Pediatric Therapeutics at the FDA.

What Can You Expect to Happen?

Depending on the type of trial and the product being tested, a child may get an experimental drug, a treatment known to be effective, or an inactive pill (placebo).

In a clinical trial, a child may not be deprived of a treatment that is necessary for the child’s health. For example, if the child has seizures and is taking a particular drug which helps control them, he or she would continue to take that drug—but also take the experimental drug being studied in the trial.