Today’s research benefits the next generation

While seeing my patients in our outpatient clinic I often have the opportunity to offer them participation in a clinical trial where a new medicine or vaccine may be evaluated before being licensed.

Frequently the trials are with new vaccines or drug therapies but many include questionnaires about lifestyle and how families are coping with today’s stresses.

Dr. Daniel Benjamin, a specialist in clinical trial research at Duke, explains below why we should strongly consider having our children participate in clinical trial research and answers frequently asked questions about the topic.

-- Dennis Clements MD, PhD, MPH

Daniel Benjamin Jr., MD, MPH, PhDClinical 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.

Here is a sample of some of the successes of the 20th century:

• Clinical trials showed that surfactant 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.

Frequently Asked Questions

Can we just do experiments 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, that we will be wrong almost half of the time.

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

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

Can’t we just do experiments in juvenile animals?

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

Is the hospital making large sums of money from clinical research in children?

No. Duke University Hospital actually loses money on research.

Don’t we know enough already?

No. Approximately 75 percent of medicines prescribed to children today have insufficient safety, dosing, and efficacy data. This is because prior to 1998, pharmaceutical companies did not have to study medicines in children prior to getting the drugs approved.

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 individuals 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 that study new medicines because these products are usually studied and proven to work in adults before they are tested 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 seven years old) and Eli (six years old), have benefited from medicines that were initially studied in children over a decade ago; and the children of our parent’s and our grandparent’s generations participated in clinical trials that resulted in life-saving therapy 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.

-- Daniel Benjamin Jr., MD, MPH, PhD, specializes in infectious diseases in Duke's Department of Pediatrics.

-- Dennis Clements, MD, PhD, MPH, is the chief of primary care pediatrics at Duke Children's Hospital.