About clinical trials

We conduct pediatric clinical trials because children need treatments that were studied specifically in their age groups. Sometimes medicines are developed and approved specifically for children, but often they are developed for adults first and then later for children. Since children’s bodies are still developing, medicines used to treat conditions in adults may not affect children in the same way. Pediatric clinical trials are important to help us understand and address the differences.

A pediatric clinical trial could help us develop the correct dose of a medicine for a child, or it could focus on development of a liquid formulation that is easier to swallow. Participation from children in clinical trials helps us develop medicines that are safe and effective for children.

In all clinical trials, the researchers must follow guidelines and requirements to protect the participants’ rights, safety, and well-being. Pediatric clinical trials have additional safety guidelines. As with clinical trials with adult participants, the guidelines are governed by regulatory agencies such as the U.S. FDA, and oversight is provided by institutional review boards or independent ethics committees. In addition, the regulatory agencies require pediatric-specific drug development plans that are reviewed and agreed to by the agencies’ pediatric experts.

Pediatric clinical studies are also designed with special considerations for children. For example, needle pokes are minimized when possible. The amount of blood taken during the study may be limited based on the age of the child.

As part of a clinical trial, your child’s study-related medical care is provided by the study team at the location where he or she is participating. This includes the principal investigator (or study doctor) — the healthcare professional who conducts and takes responsibility for the clinical trial at that location — as well as other study team members.

During the clinical trial, your child may also continue to see his or her regular medical doctors for any care that is not related to the study. It is important for you to tell your study team about other conditions your child might have, and to let the team know which other doctors are caring for him or her. This helps the study team coordinate your child’s overall care.

If your child participates, the clinical trial will provide study-related medical care, and your child’s regular medical doctors will address the other aspects of your child’s health. Your child’s regular medical doctors can also work with the study team to understand the details of the study and any impact it may have on their regular medical care.

Legally, children are not able to provide informed consent until they reach adulthood. However, children who are old enough and able to do so provide something called assent. Assent is when the child understands and agrees to take part in the clinical trial. Assent forms are age-appropriate and provide information on clinical trials in a format that children can easily understand.

Once a child gives assent to participate, the parents or legal guardians are the ones who then provide consent for their child to participate in the study.