Clinical Trials Can Help Improve Medical Treatments
Clinical trials are voluntary research studies conducted in people and designed to answer specific questions about the safety and/or effectiveness of new drugs, vaccines, and medical devices.
Interventional studies may also seek to answer questions about new ways of using existing products that are approved, cleared or licensed by the U.S. Food and Drug Administration for certain uses.
If your child enrolls in a pediatric clinical trial, they could be offered an experimental product that may (or may not) be better than those already being used. Generally speaking, by law, giving an experimental product to children must provide a potential clinical benefit that justifies the risk of using the experimental product. Also, the children who participate in the clinical trial generally must have the disease or condition being studied.
Under limited circumstances, children may be exposed to the risks of an experimental medical product that does not offer any potential clinical benefit. In that case, the risks must be sufficiently low and the information to be gained must be important to understanding or treating the children’s disease or condition.
Clinical studies give us important information about the safety and effectiveness of an experimental medical product. This information informs the FDA’s review of medical products. In addition, it guides whatever information goes in the approved, cleared, or licensed product labeling, including, for example, about the right dose of a drug to use.
The Importance of Clinical Trials in Children
Not all drugs or devices approved, licensed, cleared or authorized for marketing by the FDA have been labeled for use in children. So, by necessity, doctors may routinely treat children with a drug or device that has been studied in adults and approved for adult use but may not have been studied in children, or may have been studied in a different group of children. For example, a drug may have been evaluated in adolescents but not in newborns.
Children’s responses to medical products can’t always be predicted from data collected in studies of adults. As children grow, their bodies change. Those changes can mean children of different ages may experience differences in the effectiveness or side effects of a medical product and, for example, may need a different dosage.
For devices, clinical trials may also be needed to verify design modifications for children and to establish age-appropriate use of the device. Designing a medical device for children can be challenging because children are often smaller and more active than adults.
Medical devices may be diagnostic or therapeutic. Some devices may require different settings and algorithms to ensure they are safe and effective for children. Others may be implanted and may need to be adjusted as a child grows. Adult data may not always predict the potential risks in children.
How Clinical Trials Are Conducted
Safeguards are in place to ensure the rights and well-being of children in clinical trials. Depending on the type of clinical trial and the product being evaluated, a child may get an investigational product, an approved, cleared, or licensed product known to be effective, or an inactive substance or device (placebo).
In a clinical trial, children may not be deprived of a treatment necessary for their health. So, in some cases the investigational product a child receives in a clinical trial may be added to the treatment they are already receiving for their condition, unless the risks of stopping the established treatment are low. For example, if the child has seizures and is taking a particular drug that helps control the seizures, they would likely continue to take that drug and also take the investigational product (or placebo) being studied in the clinical trial to treat seizures.
A clinical trial that evaluates an investigational product might be no riskier than your child being treated with an FDA-approved, cleared, licensed or authorized product that has not been evaluated in children. For one, monitoring for possible side effects (also known as adverse events) generally would be much more intensive in a research study than when a drug is prescribed, or a device used, by a doctor outside of a clinical trial.
What You Should Know Before Enrolling Your Child in a Clinical Trial
Talk with your child’s pediatrician before enrolling in, and during, a clinical trial. Your health care professional can serve as an adviser to your family and help you evaluate the potential benefits and risks of the study.
They can help you think about important questions to ask the investigator in charge of the clinical trial and decide if it is right for your child. The people doing the research study may include doctors, nurses, research coordinators, social workers and other health care professionals.
Enrolling your child a clinical trial is an important decision. Learn as much as you can beforehand.
Ask questions. Write down a list of questions and take it with you when you meet with the investigator in charge of the clinical trial. Some examples include:
What is the evidence that the investigational product might help my child?
How will you check to see if the investigational product is working?
Are there any concerns that the investigational product could be unsafe?
How closely will my child be monitored if there are problems?
Who do I call if I’m concerned that there is a problem?
