Developing Better Clinical Trials in Pediatric Nephrology

Columbus, OH — November 2017

Nearly half of pediatric clinical trials are unfinished or unpublished. Some don’t enroll a single patient. It’s a problem across pediatrics, but small pediatric subspecialties such as nephrology – with relatively few clinicians and patients – have an especially difficult time bringing new therapies to market.

Among the problems is that drug companies, clinicians, regulators and others don’t often speak to each other about the best ways to develop new therapies, says William Smoyer, MD, pediatric nephrologist and director of the Center for Clinical and Translational Research at Nationwide Children’s Hospital. So Dr. Smoyer and his colleagues are trying to change that.

Dr. Smoyer is a founding member and co-chair of the Therapeutics Development Committee of the American Society of Pediatric Nephrology. It brings together pediatric nephrologists, representatives from the drug industry, the U.S. Food and Drug Administration, the National Institute of Diabetes and Digestive and Kidney Diseases, the Eunice Kennedy Shriver National Institute of Child Health and Human Development, the Patient-Centered Outcomes Research Institute and patient advocacy groups.

The committee’s first publication, highlighting the differences between the industry and academic approaches to clinical trials and how they can be bridged, appeared recently in Pediatric Research.

“The current way of doing this has not worked well for anyone, especially for children,” says Dr. Smoyer, senior author of the publication. “Many trials are not well-designed, but that’s not clear until a year down the road when money has already been invested.”

“We all want to design trials that would be effective, that doctors want to participate in, that parents will enroll their children in. The idea is that if we develop relationships between all of the stakeholders we can have more of these trials.”

Just by getting the participants talking to each other, the committee feels that it already has made progress, says William Schnaper, MD, a pediatric nephrologist at Lurie Children’s Hospital in Chicago, committee co-chair and lead author of the publication.

“By having e-mail exchanges and face-to-face meetings, the committee members have learned from each other and gained new perspectives on conducting clinical trials,” he says. “The next step is sharing our insight with our colleagues in pediatrics and industry.”

In some ways, the committee reverses the traditional, “transactional” way of approaching drug development, says Dr. Smoyer, who is also a professor of Pediatrics at The Ohio State University College of Medicine. That model involves stakeholders only when it becomes necessary – a pharmaceutical company may contact a nephrologist only when looking for patients, for example. The committee focuses on communication between all stakeholders before a drug is even considered, with the idea that transactions follow from mutual trust and respect.

The committee deals specifically with nephrology because it was born out of a meeting of pediatric nephrologists in 2012. But its strategy may apply across medical fields.

“We have patients but we don’t make drugs,” says Dr. Smoyer. “Companies make drugs but don’t have patients. Drug trials are already complicated. We can help by better understanding each other.”


Schnaper HW, Flynn JT, Gross C, Cropp AB, Dehmel B, Patel LB, Greenbaum LA, Houtsmuller E, Kaskel F, Moxey-Mims M, Nowak K, Silverstein D, Thompson A, Yao L, Darsey E, Smoyer WE. Enhancing clinical trial development for pediatric kidney diseases. Pediatric Research. 2017 Nov; 82(5):727-732.

Pica N, Bourgeois F. Discontinuation and nonpublication of randomized clinical trials conducted in children. Pediatrics. 2016 Sep ;138(3).