Pediatrics

Pediatrics

Our commitment to advance the scientific investigation of medical therapies in children begins at the bedside. We know firsthand the limits and risks of providing treatments based on evidence generated through studies of adults. Through innovative design methods and data monitoring, the DCRI's Pediatrics team is leading the world in expanding the impact of pediatric research.

Protecting Our Most Vulnerable Population

The safety and well-being of children must be at the center of all pediatric clinical research. Our Pediatrics faculty ensures this at every step. They—along with our statistical faculty, site and data management experts, and project leaders—work closely with sponsors at all stages of a study to ensure accurate and efficient execution.

Unparalleled Pediatric Expertise

Our pediatric research experience ranges from pharmacokinetic studies in neonates to large multicenter trials in adolescent populations. We focus on:

  • Cardiology
  • Central nervous system disorders
  • Infectious disease/HIV
  • Primary care
  • Rheumatology
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Highlights

Baby in incubator

Helping to Shape FDA Guidance

DCRI's Pediatrics team contributed to the FDA's draft guidance on research in newborns, which provided clarity on study design, data analysis, and drug dosing. Kanecia Zimmerman, MD, MPH, and Michael Cohen-Wolkowiez, MD, PhD, recently wrote about their work and the importance of the new FDA guidance in STAT News.

Developing Pain Measures for Children

DCRI investigators, in collaboration with Duke's Department of Population Health Sciences, are developing a set of endpoints and measures that will be used in clinical trials and spur development of new pain medications for young children.

Jeff Guptill

DCRI Research Contributes to FDA Approval of First Drug for Rare Disease in Pediatric Patients

DCRI Pediatrics and the DCRI Pharmacometrics Center team used pharmacokinetic modeling to assess how the drug would behave in children, leading to FDA approval without conducting a pediatric clinical trial.

The Pediatric Trials Network (PTN)

Most of our pediatric clinical trials are conducted through the Pediatric Trials Network (PTN), founded in 2010 with a $95 million contract from the National Institutes of Health (NIH) and renewed in 2018. The PTN is a groundbreaking collaboration between the DCRI and several of the country’s preeminent pediatric-medicine experts, hospitals, and medical centers. Through the PTN we study the formulation, dosing, efficacy, and safety of drugs—as well as the development of medical devices—used in pediatric patients.

Every PTN study is closely examined to determine how to obtain the most valuable and generalizable results while minimizing the risk to participants. Over the last 10 years, we have enrolled more than 7,000 children in 38 studies, including 26 clinical trials. Data collected from PTN trials has already helped regulators to revise 10 drug labels for safer and more effective use in infants and children. PTN has submitted pediatric data to the FDA for more than 20 products.

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Watch the video above to learn why PTN research matters.

The Global Pediatric Clinical Trials Network (G-PCTN)

In October 2017, the DCRI and its strategic partners were awarded a grant from the U.S. Food and Drug Administration to establish a coordinating center for a Global Pediatric Clinical Trials Network (G-PCTN). The G-PCTN will support efficient pediatric clinical trials worldwide by developing scientific and operational infrastructure, fostering collaborative networks, sharing knowledge, and engaging stakeholders.

DCRI Releases White Paper Providing Guidance on Pediatric Research

DCRI pediatric researchers, in collaboration with members of the DCRI’s communications team, authored “Plan and Design With the Child in Mind: Global Pediatric Clinical Trials Network Recommendations and Insights for Sponsors of Pediatric Research.” To gain these insights, the team conducted extensive research with pharmaceutical companies via an online focus group, in-depth interviews, and in-person educational workshops during which DCRI researchers worked with companies to identify and address their specific pediatric research challenges.

Leveraging Real-World Data to Deliver Insights into Pediatric Migraine

The real-world data collected from pediatric patients diagnosed with migraine via a registry co-led by Christoph Hornik, MD, PhD, MPH, could open the door for future drug development in the space. DCRI’s Research Together program contributed to the registry to ensure it collects data that is important to patients and their families.

Pediatrics Leadership at the DCRI

Danny Benjamin, MD, MPH, PhD, Director

Danny Benjamin completed his undergraduate degree, medical degree, and pediatric residency at the University of Virginia. Board-certified in pediatrics and pediatric infectious disease, he completed pediatric infectious disease subspecialty training at Duke University and received a PhD in epidemiology from the University of North Carolina. His clinical and translational research interests are in neonatal infectious disease and neonatal pharmacology. In his role as a mentor for the Clinical Research Fellowship Program, he works to recruit, develop, and retain physicians committed to the scientific investigation of medical therapies. During his tenure as an advisor to the Food and Drug Administration on improved pediatric clinical trial design, Benjamin held a joint appointment as a medical officer and special pediatric consultant providing programmatic advice on pediatric research regulation.