An FDA Oncology Perspective of Juvenile Toxicity Studies to Support Pediatric Drug Development

Abstract

FDA Oncology previously published a perspective on the utility of juvenile animal studies (JAS) to support clinical development for drugs intended for the treatment of cancer in pediatric patients. FDA Oncology concluded that, consistent with the position described in the ICH S9 Guidance, given how clinical development in these populations proceeds, JAS did not generally provide critical data informing on the starting dose or clinical monitoring and that lack of JAS should not generally be the basis for delaying pediatric clinical development of oncology drugs. FDA Oncology has now reexamined this conclusion with consideration of both JAS submitted over the last 5 years and new publications. FDA Oncology determined that a drug’s pharmacology may be the most important driver of whether a drug should be studied in pediatric patients. This paper will discuss recently submitted JAS, with specific emphasis on anti-GD2 antibodies and TRK inhibitors. Juvenile animal studies were conducted for these products, some at the request of FDA to answer specific questions that could not be addressed through clinical studies. Other drugs with JAS were also examined, but are not discussed since there was little impact on clinical development.