Clinical trials help provide reliable evidence needed when studying medical products to diagnose, prevent, treat, & sometimes even cure, childhood diseases. Children who participate in these research studies may also potentially benefit from the medical products under investigation.

Before deciding whether your child should participate in a clinical trial, there’s a lot to know, including the risks & benefits. Let’s take a look.

Clinical Trials Can Help Improve Medical Treatments

Clinical trials are voluntary research studies conducted in people & designed to answer specific questions about the safety &/or effectiveness of new drugs, vaccines, & 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 & 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 & 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 & 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 & 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 &, for example, may need a different dosage.

For devices, clinical trials may also be needed to verify design modifications for children & to establish age-appropriate use of the device. Designing a medical device for children can be challenging because children are often smaller & more active than adults.

Medical devices may be diagnostic or therapeutic. Some devices may require different settings & algorithms to ensure they are safe & effective for children. Others may be implanted & 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 & well-being of children in clinical trials. Depending on the type of clinical trial & 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 & is taking a particular drug that helps control the seizures, they would likely continue to take that drug & 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, & during, a clinical trial. Your health care professional can serve as an adviser to your family & help you evaluate the potential benefits & risks of the study.

They can help you think about important questions to ask the investigator in charge of the clinical trial & decide if it is right for your child. The people doing the research study may include doctors, nurses, research coordinators, social workers & 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 & 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?

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, & you tell me if I’m right or not?”

Ask for contact information if you would like to talk to other parents whose children are participating in the clinical trial. The study team can provide this information if they get permission from other parents to have you contact them.

Take your time. Before signing the informed consent document, think about it. Talk it over with your child, your family, your child’s health care professional & other trusted people before signing.

Follow your instincts. If you’re not satisfied with the information given to you & the answers to your questions, don’t enroll your child.

Clinical trials are voluntary. After you enroll in a study, you can take your child out of the clinical trial at any time. It’s important to talk to the study team if you want to stop participating in the trial. You may need to follow up even after stopping to make sure your child does not have any side effects from the investigational product.

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