Preclinical safety evaluations supporting pediatric drug development with biopharmaceuticals: strategy, challenges, current practices


  • The authors are part of the Biotechnology Industry Organization's (BIO) juvenile toxicity studies for biotherapeutics taskforce. This taskforce is a committee within the BioSafe Preclinical Safety Committee, comprised of BIO members working to serve as a resource for BIO members and BIO staff by identifying and responding to key scientific and regulatory issues related to the preclinical safety evaluation of biopharmaceuticals products.


Evaluation of pharmaceutical agents in children is now conducted earlier in the drug development process. An important consideration for this pediatric use is how to assess and support its safety. This article is a collaborative effort of industry toxicologists to review strategies, challenges, and current practice regarding preclinical safety evaluations supporting pediatric drug development with biopharmaceuticals. Biopharmaceuticals include a diverse group of molecular, cell-based or gene therapeutics derived from biological sources or complex biotechnological processes. The principles of preclinical support of pediatric drug development for biopharmaceuticals are similar to those for small molecule pharmaceuticals and in general follow the same regulatory guidances outlined by the Food and Drug Administration and European Medicines Agency. However, many biopharmaceuticals are also inherently different, with limited species specificity or immunogenic potential which may impact the approach taken. This article discusses several key areas to aid in the support of pediatric clinical use, study design considerations for juvenile toxicity studies when they are needed, and current practices to support pediatric drug development based on surveys specifically targeting biopharmaceutical development. Birth Defects Res (Part B) 92:359–380, 2011. © 2011 Wiley-Liss, Inc.