Is a Clinical Trial Right for My Child?

Every year, hundreds of millions of prescriptions are given to children for the treatment of a wide range of conditions. However, the U.S. Food and Drug Administration (FDA) reported in 2016 that only about 20% of prescribed medications were actually approved for use in children. In Europe, more than 70% of marketed drugs have been not been licensed for use in children.

Due to the lack of pediatric research data, doctors use their medical judgment when prescribing medications to children and alter doses based on adult study findings. Although there are valid reasons for doing this, it highlights the importance of involving children in clinical trials.

Why Clinical Trials for Children Matter

It is often said that “children are not small adults”, yet they are frequently prescribed medicines that have only been tested in adults. Children’s bodies are constantly changing, and their reactions to medications differ from those of adults.

Clinical trials, whether for adults or children, help researchers discover treatments that are safe and work well. Where appropriate, clinical trials specifically designed for children can help develop and test medicines that are safe and effective for a child’s developing body – without these insights, doctors, and developers need to rely on adult-tested medicines.

Clinical Research in Children

To ensure the safety of participants, all clinical trials adhere to a set of rules and regulations and are closely monitored by institutional review boards, or research ethics committees. Although children are a unique group, the process for clinical trials in children is the same as adults.

What is involved

Generally, research in children involves:

• Testing drugs, treatments, or procedures that may or may not have already been tested in adults to verify whether or not they work for children
• Finding drugs, treatments, or procedures that are safe and work well to treat diseases or conditions in children according to their specific health needs
• Investigating diseases or conditions that are mostly or only found in children

Possible benefits and risks

Each clinical trial has its own set of benefits and risks, which vary depending on the type of trial and what it’s aiming to discover. In pediatric trials, the most common benefit for parents is that their child may have access to a new drug or treatment. Another potential benefit is that your child’s health will be closely monitored by medical specialists and doctors throughout the trial.

On the other hand, the treatment being tested may not work well or at all for your child. There’s also a risk of potential side effects that may or may not be known at the time of the trial. Children may also need to see different doctors, go to the doctor more frequently, and have more tests and procedures than they are used to.

Your child joining a clinical trial may be a step toward a better health outcome. Therefore, the most important factor to consider is whether the benefits outweigh the risks.

Making a decision

Before joining a trial, you and your child should both have the opportunity to ask any questions you have. Obtaining as much information as possible about the trial and speaking to your child’s regular doctor will assist you in making an informed decision about whether or not to enroll your child in a clinical trial. For example, you want to ask:

• What are the possible benefits to my child?
• What are the possible risks or side effects?
• What kind of tests and procedures are involved?
• How will we know if the trial medicine is working?
• Who can answer questions before, during, and after the trial?

When you decide to enroll your child in a trial, you must provide informed consent for them to participate. In some cases, depending on their age, children may also need to give their own permission (also known as assent) to participate.

Prepare your child for clinical trial participation

Children may face different challenges than adults when participating in a clinical trial. Before joining a trial, think about potential concerns for your child and find ways to help your child overcome them. Ask yourself the following:

• What are my child’s fears (e.g. needles, having to go to an unfamiliar doctor’s office, having to take more medication)?
• Will my child feel comfortable and be able to complete the tests and procedures that will be performed on him or her?
• How will my child feel about having more or longer doctor visits?
• Will my child understand what is happening during the trial? How will I explain what a trial is and why participation may be a good idea?

How will I explain the possible side effects of the trial treatment and the impact it might have on my child’s illness? How will I explain if the trial treatment does not help my child?

Think of other things that may affect your child during a trial. Encourage your child to ask questions, if appropriate. Depending on their age, children can understand basic medical information if presented at the appropriate developmental level. Work closely with the research team to help you answer any questions your child may have.

Keep in mind that participating in a trial is completely voluntary. Your child can leave a trial at any time and for any reason. To determine what is best for your child, always consult with your child’s regular doctor first.

Find a Clinical Trial

If you and your child are interested in a clinical trial or want to learn more, search for trials on ClinicalTrials.gov.