When it comes to research, ailments in children take a back seat
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The U.S. has made great biomedical strides in recent decades that have produced drug therapies and medical procedures benefiting the world, particularly when it comes to prolonging the life expectancy of adults.

But from asthma to diabetes, cancer to the common cold, studying diseases in children has taken a back seat to investigating diseases in adult populations. The scientific knowledge base of how a particular ailment behaves in children -- and discovery of the therapies that might be used to counter it -- pale in comparison.

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While the U.S. Food and Drug Administration has taken strong actions in recent years to increase research into pediatric therapies, the findings of an Institute of Medicine report from 15 years ago still hold true. Pediatricians and scientists, the study found, “have argued that infants, children and adolescents have not shared equally with adults in advances in biomedicine. In particular, many drugs with potential pediatric uses have not been tested in studies that include children.”

Children are approximately 27 percent of the world’s population, yet research into pediatric ailments accounts for only 16.7 percent of clinical trial research, according to the British Journal of Clinical Pharmacology. A robust effort to address ailments in children should be much closer to their population percentage.

A recent article in the journal Pediatrics underscores the problem. “Optimal treatment decisions in children require sufficient evidence on the safety and efficacy of pharmaceuticals in pediatric patients,” it noted. “However, there is concern that not enough trials are conducted in children and that pediatric trials differ from those performed in adults.”

In practical terms, what this means is that children are often the last to receive new therapies. And among new treatments used to address conditions in children, many are what are known as “off label” therapies. These are drugs that have been studied in adults and approved by the FDA for adult use, which are later found to have benefit for children. In other words, children wait at the end of the line because therapies are not sufficiently studied at the front end of the research process with both children and adults.

Why does pediatric research take a back seat?

One reason is trepidation among scientists. A recent study in Pediatrics found that researchers said “they were perceived by some pediatricians, the community and politicians to be ‘using’ children and were described as ‘vampires after your blood.’”

Another reason is that research using children as study participants presents ethical hurdles regarding informed consent, since they are minors. Recruitment of children is more difficult because parents understandably are exceedingly protective. Where parents might willingly participate themselves in a research trial, the threshold is raised for their child. This is exacerbated by a lack of training among pediatric researchers: They are not effectively trained to persuade parents about the need for and value of pediatric research.

Still another reason is lack of interest from the pharmaceutical industry. The journal Pediatrics found that among a selection of diseases in which children are the most affected, very few clinical trials were conducted aimed at addressing the condition in children. And among those pediatric clinical trials, 56 percent were conducted without industry funding, compared with 35 percent of adult trials.

The institute that I lead intends to help fill the pediatric research void. The Gale and Ira Drukier Institute for Children’s Health is bringing together the best physician-scientists with a laser focus on childhood diseases. It is developing advanced methods of studying diseases in children, using big data and computational biology. Initially, our research will be on diseases that are mediated by the immune system, such as diabetes, allergies, asthma and lupus. Our goal is straightforward: We will delve deep into how these diseases affect children, and we will develop new therapies for them. 

Our efforts, and those of other pediatric centers, can only succeed if more studies include children. But that will only occur if the scientific and pharmaceutical communities make a concerted effort in addition to thoughtfully, compassionately reaching out to parents and persuading them that pediatric research saves lives and alleviates suffering in one of the world’s most vulnerable populations.

Dr. Pascual, a pediatric rheumatologist, is director of the Gale and Ira Drukier Institute for Children’s Health at Weill Cornell Medicine in New York.