A major barrier to the identification, development, and regulatory approval of therapeutics for treatment of acute pain in infants and young children is the lack of standardized, high-quality measures and endpoints for use in clinical trials. To address this need, Drs. Zimmerman and Reeve will leverage existing infrastructure of the Pediatric Trials Network and the Duke Center for Health Measurement. This partnership capitalizes on strong expertise in pediatric drug development, design and successful execution of pediatric clinical trials, evaluation of analgesic safety and efficacy in children, and design and evaluation of high-quality clinical outcome assessments (COAs). Drs. Zimmerman and Reeve will lead a team of uniquely-qualified investigators in the identification of high quality COAs and endpoints for use in the development of acute pain therapeutics in infants and young children, with a primary focus on those 0-2 years. Specifically, the research team will accomplish the following goals: 1) conduct formative research to identify important outcomes for acute pain drug trials that matter most to patients, caregivers, and clinicians; 2) identify existing COAs for each outcome and determine each COA?s fit-for-purpose for acute pain trials or the need for new COAs; and 3) gather further evidence of the psychometric properties of clinician-, observer-, and patient-reported COAs in a prospective trial among infants and young children. With input from a diverse and well-rounded external technical advisory committee, completion of these goals will help facilitate patient-centered acute pain drug trials and ease the burden of investment in pediatric drug development.
Development of safe and effective drugs to treat pain in infants and young children may help alleviate suffering and improve quality of life. However, there is a limited understanding of the optimal measures and endpoints for use in acute pain clinical trials that involve this vulnerable population. This proposal will use rigorous qualitative and quantitative methods to identify concepts important to patients, caregivers, and providers and will provide a standardized set of high-quality clinical outcome assessments and endpoints for use in trials of acute pain therapeutics in infants and young children.