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

Our Pediatrics faculty ensure that the safety and well-being of children is at the center of every step of pediatric clinical research. Along with our statisticians, site and data management experts, and project leaders, our faculty work closely with sponsors to ensure accurate and efficient study delivery.

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
contextual photo

The Pediatric Trials Network (PTN)

The Pediatric Trials Network (PTN) is a research network born from a groundbreaking collaboration between the DCRI and several of the country’s preeminent pediatric-medicine experts, hospitals, and medical centers. Many of the DCRI's pediatric clinical trials are conducted through the PTN, which was founded in 2010 with a $95 million contract from the National Institutes of Health (NIH) and renewed in 2018. Through the PTN, our Pediatrics faculty and their colleagues across the nation 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. The PTN has enrolled more than 11,000 patients in studies and trials that focus on 18 different therapeutic areas. Data collected from PTN trials has already helped regulators to revise 15 drug labels for safer and more effective use in infants and children. PTN has submitted pediatric data to the FDA for 26 products.

Read the articles below to learn how:

PTN logo

Watch the video above to learn why PTN research matters.

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DCRI's Pediatrics team contributed to the FDA's draft guidance on research in newborns, which provided clarity on study design, data analysis, and drug dosing.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

Uncovering Treatments for Rare Diseases

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 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 authored a white paper outlining recommendations and insights into planning and designing patient-centered pediatric clinical research. The paper is the culmination of extensive research and direct work with pharmaceutical companies to identify and address challenges specific to pediatric research.

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

Michael (Micky) Cohen-Wolkowiez, MD, PhD, Director

Michael Cohen-Wolkowiez, MD, PhD, is a Professor of Pediatrics at Duke University and head of Pediatrics Research at the Duke Clinical Research Institute. His research focuses on computational and patient-centric methods to advance and accelerate pediatric drug development. Cohen-Wolkowiez serves as the Principal Investigator for a diversified portfolio of research that is funded via a peer-review process, including grants from the NIH, FDA, BARDA, various foundations, and industry sponsors. In order to amplify the impact of this research, Cohen-Wolkowiez worked for two years as a Scientific Advisor to the FDA, Office of Clinical Pharmacology and Office of Pediatric Therapeutics. He has served as protocol chair, principal investigator, or held an investigational new drug (IND) application for 15 trials in children resulting in enrollment of more than 3,500 children. He has performed or led more than 50 PK/PD analyses across multiple therapeutic areas that were used to support data submission to the FDA. He is the author of more than 140 peer-reviewed publications and has mentored numerous trainees in clinical pharmacology.

Cohen-Wolkowiez completed his medical degree at the Universidad Central de Venezuela and his residency in Pediatrics at the Nicklaus Children’s Hospital in Miami, Florida. Board-certified in pediatrics and pediatric infectious disease, he completed infectious disease subspecialty training at Duke University and received a PhD in Pharmaceutical Sciences from the University of North Carolina at Chapel Hill School of Pharmacy.