Last September the International Council for Harmonization (ICH) released its draft guideline “Nonclinical Safety Testing in Support of Development of Paediatric Medicines – S11” to provide direction on safety studies required to support the development of pediatric medicines. The FDA is an active member in the ICH and published a copy of the ICH guidance for public comment on January 22, 2019. The guideline focuses on a Weight of Evidence approach to determine the number and type of juvenile animal safety studies appropriate to enable pediatric clinical trials. A number of illustrative case studies are presented as well as tables of data comparing the different stages of organ development in species commonly used for preclinical studies. The comments are extensive, appropriate, and interesting. For example, PhRMA commented that this guideline should also apply to studies supporting a marketing application or registration for pediatric indications. Other comments came from the International Consortium for Innovation and Quality in Pharmaceutical Development (requesting several points of clarification), the International Plasma and Fractionation Association (noting the preclinical models are not relevant for plasma derived therapeutic proteins as these are highly immunogenic in nonhuman models), International Council on Animal Protection in Pharmaceutical Programs (expressing concern that the guideline appeared to advocate a “tick the box” approach to juvenile animal studies), and the Biotechnology Innovation Organization (calling for additional clarification on several points). 

Text Copyright © 2019 Katrina Rogers