Pediatric assessments are now required as part of every New Drug Application and Biologics Licensing Application in the US and every Marketing Authorization Application in Europe (unless a waiver has been granted). An important consideration for this pediatric use is to determine how to support its safety. Unlike the highly defined study designs of the standard core of toxicity studies conducted to support registration for adult indications, the need for and design of juvenile toxicity studies are driven on a case-by-case basis taking into consideration whether or not adequate, relevant data can be obtained from other nonclinical toxicity studies in adult animals or from prior clinical experience. Studies in juvenile animals can provide valuable safety data to support pediatric clinical trials and for use in drug labeling, for example, long-term effects and effects on developing systems that cannot be assessed in clinical trials (e.g., effects on reproductive potential).

The first presentation will provide a regulatory perspective on these studies, including possible study designs, endpoints to be assessed, and tests used to assess these endpoints. Examples of juvenile animal studies that have been conducted will be presented, as well as how the results from these studies were used in regulatory decision-making and labeling.

The second presentation will discuss the challenges in the industry to develop a nonclinical strategy to support global pediatric drug development that is both technically feasible and scientifically justified while providing meaningful information for the patient, parent and healthcare professional. This talk will address considerations, challenges, and experiences when designing and conducting juvenile toxicology studies from the perspective of the sponsor. There are many design elements that we could include but the real question is what should we include.