Abstract
In the 1960ies children were described as therapeutic orphans, but without consequence until the 1990ies. Today pharmaceutical industry is expected to consider potential use of new drugs in children. Pediatric assessments are mandatory in US and a comparable EU legislation is discussed. In 2002, implementation of pediatric aspects into drug development was analyzed in Novartis by a cross-functional team. A pediatric strategy was decided. An internal pediatric advisory group was established spring 2003. It coordinates implementation of pediatric aspects into standard development plans and has built up an internal training program in pediatric drug development. The EU commission wants pediatric data at submission. We analyzed this in the content of ICHE11 (guideline on drug development in children), which lists serious diseases in children only, serious diseases affecting both adults & children, and other diseases. The first two scenarios justify early development in children. Exposure of children to new experimental drugs is ethical only for life threatening conditions without acceptable therapeutic alternative. In this case, major development blocks are shifted to earlier stages, e.g. preclinical safety & toxicology and pediatric formulation. Higher investments at early project stages increase overall drug development costs. Project teams have to carefully balance potential therapeutic value, ethical concerns, risks and business considerations. Scenario (3) with deferral at registration point and a commitment to pediatric development later will mostly be the routine. In the complex modern drug development the introduction of pediatric thinking is a major challenge. It requires internal training and organized exchange of knowledge & experience. The decision for early pediatric development will probably be the exception reserved for therapeutic brake-throughs, but a careful analysis of pediatric aspects at several development decisions points is becoming integral part of drug development.
Article PDF
Author information
Authors and Affiliations
Rights and permissions
About this article
Cite this article
Rose, K. 308 Implementation of Pediatric Aspects into the Global Drug Development Process. Pediatr Res 58, 407 (2005). https://doi.org/10.1203/00006450-200508000-00337
Issue Date:
DOI: https://doi.org/10.1203/00006450-200508000-00337