By: FDA Consumer Health Information
You may have heard of clinical trials, and your doctor may even have suggested your child enroll in one.
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 U.S. Food and Drug Administration (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.
“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 that has not been tested in children,” says Nelson. “Some research may be riskier, but some may be safer than being prescribed a drug that has not been studied in children. Further, monitoring for adverse events would be much more intensive in a research study than when a drug is prescribed by a doctor outside of a clinical trial.”
Making A Decision
Nelson suggests parents use a child’s pediatrician as an advisor before enrolling in, and during, a clinical trial. “A pediatrician can help a parent evaluate the risks and potential benefits, assess the qualifications of the research team, clarify a child’s understanding of the research, and support the parent and child throughout the research study,” he says.
Some suggestions for possible discussion with the investigator include:
- Write down a list of questions and take it with you when you meet with the investigator. Some examples are: What is the evidence that the investigational drug might help my child? How closely will my child be monitored if there are problems with the drug? Who do I call if I’m concerned that there is a problem?
- Take notes during the discussion. Read the notes back to the investigator to make sure you heard everything correctly. Ask, “Can I tell you what I think this is about and you tell me if I’m right or not?”
- Ask for contact information if you would like to talk to other parents whose child is participating in the study. The study team can provide this information if they get permission from other parents to have you contact them.
- Think about taking a little time before signing the informed consent document. Ask if you can instead go home and think about it, talk it over with your family, your child’s pediatrician, and other trusted people before signing.
Listen to your “gut.” If you’re not satisfied with the information given to you and the answers to your questions, don’t enroll your child.
“Parents need to understand that they can take their child out of a clinical trial at any time,” says Nelson. “However, there may need to be some follow-up to make sure a child does not have a serious side effect from stopping the drug being tested.”