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Medicines for Children

The Importance of Children in Clinical Trials

Dr. Daniel Benjamin and Dr. Michael Cohen-Wolkowiez

Dr. Daniel Benjamin (right) and colleague Dr. Michael Cohen-Wolkowiez discuss research on safe medications for children at the Duke Clinical Research Institute in Durham, North Carolina.
Photo Duke University Health System

An interview with Daniel K. Benjamin, Jr., MD, PhD, professor of pediatrics at the Duke Clinical Research Institute and Director of the BPCA Pediatric Trials Network (PTN).

What do you say to a parent who asks you why children’s clinical trials are important?

Clinical research is critically important to your child’s health. It was only 100 years ago that nearly one out of every six children born in the United States died prior to their fifth birthday. Infant and early childhood mortality in the United States is now uncommon. It is from prior research studies conducted in children that we have reduced early childhood mortality to a relatively rare event.

Can you point to some of the successes with children from clinical trials research in the past?

Yes, here are several important examples:

  • Clinical trials showed that surfactant—a substance that keeps air sacs in the lungs inflated—helps premature infants breathe. As a result, the lives of thousands of babies who would otherwise die of respiratory failure are saved each year.
  • Clinical trials showed that giving antiretroviral therapy (AZT) to pregnant women with HIV-AIDS prevents HIV transmission. We have saved the lives of thousands of children born in this country every year thanks to this research.
  • As a result of repeated clinical trials in children with cancer, most children who develop leukemia survive. Only 50 years ago, acute leukemia was almost universally fatal in young children.
  • Prior to the development of the polio vaccine, 15,000 American children developed paralytic polio each year. In the last five years, there have been no cases of paralytic polio in the United States. This advance was only possible through clinical trials in children.

Can we just do clinical studies in adults?

No. Investigators at Duke University and elsewhere have shown that if we rely on adult data to guide how to give medicines to children, we will be wrong almost half the time.

Can we predict which drugs work differently in adults and children?

No. The differences in how drugs are metabolized relate to the age of the child, the organ that breaks down the drug (liver or kidney), and how much the brain has developed. No one can predict how the developing child’s body will respond to medicines.

Can’t we just do clinical studies in juvenile animals?

No. Animal research is an important part of clinical research but is not definitive.

Will my child benefit from this research?

This is the most difficult of the frequently asked questions. In general terms, there are some data to suggest that people who participate in clinical research have better outcomes compared to similar patients with the same disease.

The reason why research participants tend to fare better is unclear. Improved outcomes may relate to the close observation often demanded by clinical trials, or closer follow-up after care that is often dictated by the research. Children may benefit from participating in clinical research of new medicines because these medicines are usually tested and proven to be safe and effective in adults before they are studied in clinical trials in children.

These short-term benefits are small in comparison to the benefits that will certainly be recognized by future children.

Two of my four children, Jack (now 10 years old) and Eli (nine years old), have benefited from medicines that were initially studied in children over a decade ago; and the children of our parents’ and grandparents’ generations participated in clinical trials that resulted in life-saving therapies for diseases that would otherwise have claimed thousands of lives of children who are young adults today. This long-term societal benefit is the best reason that clinical research is important in both adults and children.

Are You Interested In a Children’s Clinical Trial?

People volunteer to take part in clinical trials for many reasons. So do parents considering whether their own children would be a good fit for a clinical trial. They often hope that by participating, their health will improve in some way.

Researchers compare different treatments to determine which is better—for example, more effective or less risky. Sometimes the newer treatment is better, but sometimes the standard is.

Clinical trials can entail risks. These must be disclosed to potential participants as part of the informed consent process prior to enrollment. Always discuss a clinical trial with your health professional before participating.

Many times, people volunteer simply because they want to contribute to a medical research effort that may help others in the future.


How to Participate In a Clinical Trial

More information about having your child participate in a clinical trial is available at government Web sites, such as:

What Is “Informed Consent”?

Before you decide whether or not to participate in a clinical trial, you should learn the key facts about it. To help you decide, the health professionals involved in the study explain its details. You also receive a document describing the study, including its purpose, duration, required procedures, risks, potential benefits, and key contacts.

If you agree to participate, you give your informed consent. Informed consent is not a contract. You may withdraw from the trial at any time and for any reason.


Read More "Medicines for Children" Articles

Developing Safe and Effective Medicines for Children / The Importance of Children in Clinical Trials / Cokie Roberts Cares about Kids!

Winter 2012 Issue: Volume 6 Number 4 Page 6-7