Medical Studies Involving Children Often Go Unpublished

Aug 4, 2016
Originally published on August 5, 2016 4:13 pm

Many medical studies involving children never end up being put to use because scientists frequently don't publish the results of their work, according to an analysis published online Thursday.

The findings raise both scientific and ethical issues regarding research on this vulnerable population.

Previous studies have documented that about a third of all clinical trials conducted in the United States end up as largely wasted effort, because the scientists doing that work don't take the effort to publish and share their results with the scientific community.

Pediatricians Florence Bourgeois and Natalie Pica at Harvard Medical School and Boston Children's Hospital wondered whether researchers focusing on children took this obligation to publish more seriously. As they report in the journal Pediatrics, the experience with childhood research is just about as bad.

The report, reviewing clinical trials started in 2008-10, finds that 19 percent of the studies that recruited children didn't run to completion. That was often because researchers weren't able to recruit as many volunteers as they needed to run the experiments. And of the 455 trials that were completed, the results from 30 percent weren't published.

"That means all the participants who are enrolled in these studies aren't able to contribute in a meaningful way to our clinical information and knowledge," Bourgeois said. One reason may be that scientists didn't get the results they were hoping for. It's less rewarding to publish results that report a failed trial, but Bourgeois says it's just as important for science to do so.

"The harm is we end up with scientific literature that only shows all the things that do work," she said. "It may falsely appear that certain interventions do work. So our literature may become biased and may not be representative of the true efficacy or safety of an intervention."

One result of that is that other scientists may try to run the same failed experiment, and end up down the same blind alley as scientists who had tried it before. "That leads to a lot of inefficiency and waste," she said.

Parents volunteer their children for these studies with an understanding that their efforts are contributing to the advancement of medical science.

Vincent del Gaizo, the parent of a child with a hard-to-treat form of juvenile arthritis, is passionate about that commitment. He's taken a very active role in managing his son's disease. When his son was about 9, he suffered from a flare-up so acute that he couldn't bend his right elbow at all. Del Gaizo learned of a clinical trial of an experimental drug near his home, in Hackensack, N.J., and decided to take the risk that the drug would help.

Del Gaizo paid close attention to how the drug affected his son. But he also cared about how the work benefited the community more broadly. "It is hugely important to me as a patient-family," for scientists to publish their results and explain them clearly, he said. It's important "not only for the patient-families but also for the clinicians," del Gaizo said. Doctors need to know what works — and what doesn't work — to guide their treatment of children who aren't participating in studies.

The results of the clinical trial his son participated in were eventually published: The drug worked better than an injection of plain salt water but hasn't ended up on the market to treat this condition. But del Gaizo says he talks to many other families, as a patient advocate, and finds they frequently don't learn the results of the experiments involving their children. "Just having information available to you ... would make it so patient-families like us can sleep at night with the decisions we have to make on a daily basis."

The shortcomings of pediatric research come as no surprise to Dr. Joseph Ross at the Yale School of Public Health. He arrived at similar results when he analyzed clinical trials that include adults. Scientists have many explanations for why they don't publish their results. "Maybe the results don't show what the investigator wants and they move on," Ross said. "But more often people are busy and people don't focus enough time and attention on getting those results out."

Ross considers this an ethical lapse. "When you do a clinical study and you're asking patients to participate and subject themselves to a risk, in order to inform science and generate knowledge, you have an ethical obligation to disseminate those results to the wider scientific community," he said.

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Tens of thousands of American parents enroll their children in medical research studies every year. Parents make those sometimes risky decisions in hopes the studies will help advance medical treatment. But an analysis out today finds that many of those results are never published. NPR's Richard Harris reports.

RICHARD HARRIS, BYLINE: Central New Jersey businessman Vincent del Gaizo has a son with a hard-to-treat form of juvenile arthritis. When the boy was about 9 years old, he had a particularly difficult flare-up. Del Gaizo learned that a nearby medical facility was running a clinical trial of an experimental drug.

VINCENT DEL GAIZO: When he enrolled in this study, he went through the previous three weeks of life without being able to straighten his elbow, like, literally one degree. It was locked at a 90 degree position.

HARRIS: The experimental drug helped the boy, and the scientists ultimately published their overall results in the medical literature. Del Gaizo said that last step was hugely important in his mind, part of the implicit deal he made with the researchers when he agreed to put his son at risk for unknown side effects in the study.

DEL GAIZO: The patients need to know. They need to know the results of the study. They need this information to help make these treatment decisions.

HARRIS: Have you encountered patients who have gone through clinical trials and then heard absolutely nothing about the results of the trials?

DEL GAIZO: Yeah, I mean that's commonplace. There's so much research that was conducted and a lot of it finished and maybe some of it not finished. But still, the results are never disseminated.

HARRIS: For the benefit of other patients. Del Gaizo has become an advocate over this issue, and now scientists at the Harvard Medical School and Children's Hospital in Boston have put some hard numbers on the problem based on studies that were conducted between 2008 and 2010.

They found that about 20 percent of pediatric studies weren't published because they were never completed. And among pediatric studies that were completed, about 30 percent still didn't publish their results in the scientific literature. Dr. Florence Bourgeois co-authored that analysis in the journal Pediatrics.

FLORENCE BOURGEOIS: We found that tens of thousands of children had been enrolled in these trials, and the results are then never published - means that they can never be incorporated into our clinical decision making and will never benefit the care we provide.

HARRIS: Sometimes scientists don't bother to publish their results because they didn't find what they'd hoped to find. But even those disappointing results can be important.

BOURGEOIS: Well, the harm is - is that we end up with scientific literature that only shows all the things that do work.

HARRIS: It's just as important to know what doesn't work. For one thing, when those results aren't published, Bourgeois says other scientists may waste their time trying to do the same experiment and hitting the same dead end.

BOURGEOIS: It creates a lot of inefficiencies and waste.

HARRIS: That's not just a problem for pediatric studies. Dr. Joseph Ross at the Yale School of Public Health is among those who have documented similar failures in medical research involving adults as well. He points to the ethical issues here which are intensified with research involving vulnerable groups such as children.

JOSEPH ROSS: When you do a clinical study and you're asking patients to participate and subject themselves to a risk, in order to inform science and generate knowledge - that you have an ethical obligation to disseminate those findings to the wider scientific community.

ROSS: But the new study underscores that even in the sensitive world of Pediatrics, medical researchers don't always take that obligation to heart. Richard Harris, NPR News. Transcript provided by NPR, Copyright NPR.