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Should Your Child Be in a Clinical Trial?

Why Clinical Trials in Children are Important

Clinical trials yield important information on a medical product's safety, dosing, and effectiveness, which is the basis for FDA approval and product labeling. Health care providers use labeling information to prescribe the right product for their patients and to monitor them for potential side effects. This includes prescribing the right drug at the right dose.

Historically, only 20-30% of drugs approved by FDA have been labeled for use in children. So, by necessity, doctors routinely give drugs to children off label." We need to do clinical trials in children so that a child will not be an experiment of one every time a doctor prescribes a drug," says Murphy.

Children's responses to drugs can't always be predicted from data collected in adult studies, says FDA's Pediatric Bioethicist Robert Nelson, M.D., M.Div., Ph.D. A child grows and the metabolism changes as he or she gets older, he says. "These changes mean the child has a different susceptibility to side effects over time. We may think we can predict some of these differences, but we really can't without studying them."

Potential Benefit

Clinical trials in children, by law, must provide the potential for at least a minimal benefit, and the child must have the disease or condition being studied or be at risk for it. For example, many children have had otitis media (middle ear infections), says William Rodriguez, M.D., FDA's Science Director for Pediatrics, and they have the potential to benefit from studies of a treatment for otitis media. But a child could not be in a study to test a diabetes drug or a glucose monitor unless the child has diabetes or is at risk for it.

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

If a placebo is used in a trial, it is because either the period of time the child is on the placebo is short and doesn't pose risks (for example, a couple of weeks without a therapy for a mild condition of high blood pressure) or because the therapy being tested is being used to treat discomfort or symptoms of a disease but not the underlying disease (for example, a runny nose).

A child should not be deprived of a treatment in a clinical trial that is necessary for the child's health. For example, if the child has seizures, an experimental drug may be added on to a medication the child is already taking to help control seizures, or added onto another treatment that is known to work.

People often think that a clinical trial that tests an experimental drug is riskier than being treated in your doctor's office with an already approved drug, says Nelson. "Some research is riskier, but some is safer than being prescribed a drug in an off-label environment where you don't know the implications. Monitoring for adverse events would be much more intensive in a research study than in an off-label practice."