Children remain underrepresented in clinical trials

SAN FRANCISCO — To receive medical care that is tailored to their unique needs, children’s participation in clinical trials is essential, according to a speaker here at the 2010 American Academy of Pediatrics National Conference and Exhibition.

Gail Pearson, MD, of the Children’s National Medical Center in Washington, D.C., said that research in the pediatric population is needed because this group of patients has special needs that require more than just “hand-me-down” information derived from studies conducted in adults.

“One of the major reasons we should do research in kids is because they have diseases that adults don’t have,” Pearson said. She pointed out that the Salk vaccine trial, which involved 1.8 million children, was integral to developing a successful polio vaccine.

Research in the pediatric population is also important now because children are now presenting with diseases typically seen in adults. “We’re a fast food nation, so we’re all seeing more high blood pressure, type 1 and type 2 diabetes and dyslipidemias, and we really don’t know what to do about it,” Pearson said.

Pediatrics recently published two articles highlighting this problem, she noted, with one paper recommending universal cholesterol screening because data indicated that family history was an insufficient predictor for severe dyslipidemia in children.

Developmental differences

Unlike adults, children also have developmental stages that affect therapeutic interventions. The Best Pharmaceuticals for Children Act addresses these stages and much valuable information has been derived from this act, such as increased awareness and understanding of dosing-related efficacy issues and how certain medications affect children’s growth. Additionally, more than 170 drugs have been patented since the law’s enactment, she noted.

“Another reason to do research in children is because expert opinion is fallible,” Pearson said. She pointed out that new data can debunk popular beliefs and practices and conducting quality studies in children will allow clinicians to base their decisions on the best information possible.

Pearson also said improved outcomes in pediatric patients reflect the benefits of research in children. Increased survival rates for children with acute lymphoblastic leukemia or reductions in overall infant mortality, for example, demonstrate how practical application of data from studies in children significantly benefit the pediatric population as a whole.

Barriers to research

Many factors hinder researchers’ willingness to conduct clinical trials in children.

“They don’t represent a lucrative market, and they don’t vote,” Pearson said.

She also noted that researchers have more to consider, such as wide variations in height and weight or developmental stages, in children than in adults. Additionally, because children are a vulnerable population, more health care personnel are required to monitor them during clinical trials.

Parents are also often concerned about the safety of enrolling their children in these studies. “Among adults and children, there’s no shared public understanding of the importance of biomedical research,” Pearson said.

In response to this problem, Pearson said the National Heart, Lung and Blood Institute, in conjunction with other partners, have put together a resource for children and their families who are approached about doing research. The Web site not only provides information but also has a documentary video with children explaining why they believe pediatric research is critical.

“Our premise is that kids are not just little adults,” said Pearson. “There should be no more hand-me-down research.” – by Melissa Foster

For more information:

Pearson G. P3069. Presented at: 2010 AAP National Conference and Exhibition; Oct. 2-5, 2010; San Francisco.

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