DCRI Releases White Paper Providing Guidance on Pediatric Research

October 30, 2019 – Through its work with the Global Pediatric Clinical Trials Research Network, the DCRI worked with sponsors to help navigate challenges associated with pediatric clinical research.

The DCRI has released a new white paper that provides guidance for sponsors on how to more effectively conduct pediatric research.

Christoph Hornik, MD, PhD, MPHDCRI 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 challenges.

“The half-day educational workshops we conducted with sponsor companies really helped us better understand some of the specific challenges companies face in real time,” said the DCRI’s Christoph Hornik, MPD, PhD, MPH, (pictured left), who co-led the workshops with the DCRI’s Michael Cohen-Wolkowiez, MD, PhD (pictured right). “While we gave customized advice to each company, we noticed the same themes kept popping up—themes which also were found in our other avenues involved in this research.”

Sponsor challenges with pediatric research vary significantly from sometimes small patient populations, which inhibits enrollment, to the special needs of pediatric populations, which makes trial Michael Cohen-Wolkowiez, MD, PhDdesign more challenging. However, the Global Pediatric Clinical Trial Network found that the majority of issues could be better addressed after considering three high-level solutions:

  • Collaborating early and often with stakeholders, including patients and their caregivers;
  • Capitalizing on novel data sources, such as real-world data and registries; and
  • Engaging early and often with regulatory bodies to ensure alignment at the beginning of the development process.

“We hope that the guidance put forth in our white paper will speed up pediatric research timelines and potentially even open the door for new pediatric drug development,” Cohen-Wolkowiez said.