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Clinical Trials and Children

Introductory image: Dr. Margolis with Young Patient - High Five

Until recently, children were rarely included in studies of medical treatments. As a result, much is still unknown about how children respond to drugs, some biologics (such as gene therapy) and medical devices. According to the U.S. Food and Drug Administration (FDA), only 20-30% of approved drugs have actually been labeled for use in children.

Doctors and nurses may give medicines, with doses adjusted for their weight, to a child even though the treatment may not have been studied and approved by the FDA for use in children. This is known as "off-label" use. Most of the time, this works well but, because children are not merely little adults, there is a chance that the dose used could be ineffective or even harmful.

Without research studies on children, the healthcare team may not have the level of information needed to understand the way medicines and other treatments work in a child’s body over time, or how those therapies impact a child’s growth and development. The only way to examine the scientific value of a new therapy for children is by conducting a pediatric clinical trial.

Clinical research in children helps to more appropriately treat children, rather than treating them as if they were little adults. The studies are geared to:

  • Finding the best dose of medicines to prevent harmful effects or under-treatment
  • Making medicines that are easier for children to take, yet still safe
  • Finding treatments for problems that occur only (or more often) in children
  • Finding treatments for diseases or conditions that occur in both children and adults but which can act differently in children and adults
  • Understanding how medicines affect children's brains and bodies as they grow and develop

Clinical trials in children, by law, must provide the potential for at least a minimal benefit, and the child must have the disease or condition being studied or be at risk for it. When considering enrolling your child in a clinical trial, make sure you understand the difference between the regular care your child gets at his or her doctor’s office and what's involved in research. Parents who are considering enrolling a child will be invited to the research site to talk with the study investigator(s) or other scientist or health care professional on the research team.  Experts from the FDA offer some suggestions for this discussion:

  • Write down a list of questions at home and take it with you when you meet with the investigator. Make sure you get all of your questions answered.
  • Take notes during the discussion. Read the notes back to the investigator to make sure you heard everything correctly. Ask the investigator, "Can I tell you what I think this is about and you tell me if I'm right or not?"
  • If you do not understand any part of the trial, ask more questions. You should feel that the study team is receptive to your questions and concerns.
  • Ask what information is already known about the medical product that your child may be given. Studies may be in progress elsewhere and some data may be available.
  • Ask for contact information if you would like to talk to another parent whose child is participating in the study. The study team can provide this information if they get permission from other parents to have you contact them.
  • After being presented with information and an informed consent document, you do not have to sign it right away. Go home and think about it, talk it over with your family, your child's pediatrician and other trusted people.
  • Listen to your "gut." If you're not satisfied with the information given to you and the answers to your questions, don't enroll your child.

How is this different from the standard care my child would receive?

  • Will my child see different doctors and nurses for the clinical trial?
  • Will my child go to a different hospital or clinic for the trial?
  • Will the doctors and nurses ask me a lot more questions about my child's condition?
  • Will there be more paperwork or additional tests when we are in the clinical trial?
  • Will there be more rules and deadlines in the clinical trial?

Learn about the clinical trial:

  • Why is the trial being done?
  • Why do researchers think the approach may be effective?
  • Who is paying for the clinical trial?
  • Who has reviewed and approved the clinical trial?
  • How are the trial results and the safety of the participants being monitored and checked?
  • How long will the clinical trial last?
  • What will our responsibilities be while our child participates in the clinical trial?

Researchers test new drugs and treatments because they have reason to believe they might work better or be safer than the standard care. In a study, your child may have access to something that is not available yet. If the drug or treatment is found to be helpful, your child may be among the first to benefit. In addition, sometimes enrolling in a clinical study can give your child a chance to see extra doctors or find out more facts about your child's condition. A clinical study may be able to put you in touch with families going through what you are going through.

A clinical study may offer closer monitoring or additional testing for your child, which may not be part of regular care. Sometimes a study asks parents to keep a diary or to bring a child in to be seen more often, such as weekly visits. Children in a clinical study will be watched closely for side effects and to understand how the treatment is working. Whatever the reason, remember that clinical studies are designed to test if a drug or procedure works and is safe. There may be benefits for your child, but there may not be.

Parents say that one reason they join a pediatric study is to help other families in similar situations. Today our children have more protection from death, disability and discomfort from many childhood diseases like polio or measles because parents in the past made a decision to allow their child to be in a study to test vaccines. But still, there are many medicines and procedures that have not been tested in kids. And before they can be accepted for use, they must be tested to see if they are safe and effective.

While research can have risks, there is a lot that goes on "behind the scenes" to ensure safety before families are invited to be in a study.

  • Many people review a study in detail before a single person is enrolled. These people include:
    • the scientists who focus on identifying the right treatment
    • the statisticians who help to design the right study with the right number of participants to get good results
    • the medical investigators (nurses, doctors, pharmacists, psychologists and technicians) who will make sure that the study is done in the right way with the right participants.
  • Informed consent documents are developed to describe why a study is being done, what will happen and risks and benefits.
  • Institutional Review Boards (IRB) are independent committees that review research plans and consent forms to make sure that people in a study are informed and protected during studies. They review studies both before they start and throughout the study. They are also known as Research Ethics Boards (REB) or Ethics Committees (EC).
  • The U.S. Department of Health and Human Services has special protections in place for children as research subjects.

As a parent, you must be comfortable with what your child will be doing in a study:

  • Even with efforts to make your child safe, remember there may still be risks. Make sure you understand the possible risks and benefits and understand how they apply to your child.
  • Get familiar with the study team, whose job it is to protect your child.
  • Find out what resources are available to help you understand your rights. The National Heart, Lung, and Blood Institute (NHLBI) of the National Institutes of Health reminds families of their rights in clinical research studies.  Keep in mind you can say “no” at any time. That is one of your rights.
  • What are the potential benefits?
  • What are the short-term risks, such as side effects?
  • What are the possible long-term risks?
  • What other options do children with similar conditions have?
  • How do the possible risks and benefits of this trial compare with standard care for my child’s bone marrow failure disease?

Clinical Studies don't just "happen". Before your child is even asked to participate, the procedure or medication being researched has likely been through a series of steps and phases.  To learn more about children and clinical studies, visit the National Heart, Lung and Blood Institute or the FDA. Help your children understand more about clinical trials with interactive games and activity pages from the NIH “The Kids Files” program.