Investigators will develop a core set of endpoints and measures to be used in clinical trials and spur development of new pain medications for young children.
The DCRI was recently awarded a Phase 1 grant from the FDA to develop endpoint models and measures for acute pain in children ages 0 to 2 years.
The grant, which is expected to total $4.1 million over 5 years, will support development of the endpoint measures throughout the initial phase of funding, and the evaluation and validation of the measures during the second phase.
The project, called Clinical Outcome Assessments for Acute Pain Therapeutics in Infants and young Children (COA-APTIC), is a response to a request for applications from the FDA, which asked for studies on clinical outcome assessments for a pilot program focused on patient-centered outcomes. This program is part of the FDA’s Patient Focused Drug Development (PFDD) efforts to support the use of meaningful and valid outcomes in regulatory trials.
DCRI’s Kanecia Zimmerman, MD, MPH (pictured top), who oversees the daily operations of the Pediatric Trials Network (PTN), had already been working with DCRI’s Bryce Reeve, PhD (pictured bottom), who serves as the director for the Center for Health Measurement, which is based in Duke’s Department of Population Health Sciences. Through their collaboration on a PTN study on pain and sedation, they created a novel sedation measure for use in children undergoing procedures. To further build on this work, they decided to apply for the FDA grant together as co-principal investigators—and recently received notice that they were one of three awardees.
“Few pain medications are approved for children 2 years and younger because no one knows how to measure pain in that population,” Zimmerman said. “Our job is to identify outcomes that matter in a rigorous way.”
Outcome assessment development will begin with interviews to identify what outcomes are important for key stakeholders, including patients and their parents. The Center for Health Measurement will provide its expertise and experiences in the development and evaluation of measures to be included in this study. The team will leverage their experience working with the FDA to ensure successful project delivery.
Once these outcome assessments are developed, they will be evaluated and validated in a clinical trial setting within the PTN. The ultimate goal, Zimmerman said, is to develop measures that could be used in clinical trials in the future, which would help open the door for new pain medications to be developed and approved for this age range.
Fellow contributors to this study include the DCRI’s Christoph Hornik, MD, PhD, MPH; Christy Zigler, PhD, assistant professor in Population Health Sciences; and Katie Gustafson, professor in Psychiatry and Behavioral Sciences.