Clinical studies for children are important for many reasons. The goal of such studies is to find out whether treatment is safe, well-tolerated and beneficial for children. Researchers doing these studies may test vaccines, new drugs, procedures, devices, and different ways to use current treatments.

Children are not just small adults and medical studies help us understand how medicines affect their bodies and brains as they grow. They result in treatments for problems that occur only in children, like prematurity and for diseases or conditions that act differently in children and adults, like heart disease or arthritis.

Studies can result in treatments for new and existing diseases that improve the health of children in the future. They also uncover the best doses of medicines to prevent harmful side effects and chewable, liquids or tablets can be developed that are easier for children to take.

Stages of growth

Children grow consistently and at each stage of growth, they may need different types of therapy, different size devices, or different doses of medicine. Medicines work differently within a child as the liver or kidneys develop and studies are needed to find out about the long-term effects on these organs.

Benefits of clinical studies

Helping future generations

Clinical research in children often looks a lot like standard medical care but standard care focuses on the needs of an individual child whereas research is done to find out whether a drug or treatment will work for a large group of children in the future. 

Children today are safer from death and disability from many childhood diseases, like measles and polio, thanks to parents in the past who allowed a child to be part of a study to test vaccines. Many procedures and medicines need to be tested to see if they are effective and safe before they are accepted for use.

Access to new treatments or drugs

Researchers test new treatments and drugs because they believe they may work better than what’s currently available. Children who participate in studies have access to what’s not yet available and if successful, they are the first to benefit. 

Access to more information

Enrolling in a clinical study may also provide an opportunity to see more doctors and find out more about a condition. It can also put parents in touch with families going through similar experiences and the study team may offer information about groups, websites and organizations dealing with the condition of the child.

 Closer monitoring

Another benefit of enrolling a child in a clinical study is closer monitoring and additional testing which may not be part of standard care. Sometimes parents are requested to keep a diary and bring a child in for weekly visits. A child must be carefully monitored when taking part in the clinical study to understand whether a treatment is working and to identify any side effects.

Possible risks

Clinical studies are designed to test if a drug or procedure works and is safe. There may be benefits to a particular child but there is also the chance that treatment is ineffective. A child may find the treatment unpleasant or experience unwelcome side effects. Treatment may involve visits to the study site, more blood tests, more hospital stays, etc.

Protocols to minimize risk

Clinical studies follow a protocol (detailed plan) carefully designed to minimize any risks and increase the chance of getting useful results. Informed consent is required from parents to give permission for a child to join a research study. They must fully understand what they’re signing up for and all the risks and benefits involved.