October 2, 2019 – Research guidance recently issued by the FDA provides clarity regarding clinical trial study design, drug dosing, and analysis in studies of newborns.
Years of research undertaken by the DCRI Pediatrics group recently contributed to the U.S. FDA’s publication of draft guidance on neonatal studies.
Pediatric researchers at the DCRI have been working on dosing studies in babies for over 10 years, in conjunction with the Pediatric Trials Network (PTN), a research network coordinated by the DCRI that studies drug safety and efficacy in pediatric populations. DCRI investigators shared new findings with the FDA as their research progressed, and this collaboration eventually developed into the publication of FDA guidance. The DCRI’s Kanecia Zimmerman, MD, MPH, who oversees the daily operations of the PTN, and Michael Cohen-Wolkowiez, MD, PhD, who serves as a PTN investigator, recently wrote about their work and the importance of the new FDA guidance in STAT News.
Research from the DCRI and the PTN has shown that medications function differently in newborns than in other children, both because of their smaller size and because their organs are not fully developed. In response, researchers from both groups have developed methods to make studying infants easier. These methods include techniques to take smaller and fewer samples from infants, as well as innovative mathematical modeling that enables better predictions so that fewer babies can be enrolled in each study. All of these findings and more are included in the new FDA guidance for neonates, which the DCRI group began to draft in 2015.
“We knew from studies at the DCRI and within the PTN that accurate dosing is critical for safety and efficacy of medication,” said Danny Benjamin, MD, PhD, director of DCRI Pediatrics. “Just as the PTN was formed because we didn’t want to dose children like small adults, we also knew we had to drill down further and develop specific guidelines for studies in newborns.”
The guidance outlines clinical pharmacology considerations that should be taken into account when conducting neonatal studies for drugs and biological products, and it provides clarity regarding clinical trial study design, drug dosing, and analysis. One of the recommendations calls for input from multidisciplinary teams during the study design period, including parents.
“We are pleased that our work will now help to improve medication safety for newborns, one of our most vulnerable populations, across the clinical research enterprise,” Cohen-Wolkowiez said. “Along with the FDA, we expect that all organizations working on studies in newborns, from industry sponsors to the NIH, will rely on this guidance.”
The FDA also anticipates that clearer guidelines could help spur development of new therapies for newborns.
“The FDA is committed to gaining more information on the safety and efficacy of existing therapies given to our youngest patients,” the FDA’s Susan McCune, MD, director of the Office of Pediatric Therapeutics, said in a statement issued by the FDA. “We hope the recommendations will encourage drug development for newborns, and ultimately help families make more informed decisions about the care of their babies.”