title: Drug Development for Pediatric Populations: Regulatory Aspects.
abstract: Pediatric aspects are nowadays integrated early in the development process of a new drug. The stronger enforcement to obtain pediatric information by the regulatory agencies in recent years resulted in an increased number of trials in children. Specific guidelines and requirements from, in particular, the European Medicines Agency (EMA) and the Food and Drug Administration (FDA) form the regulatory framework. This review summarizes the regulatory requirements and strategies for pediatric drug development from an industry perspective. It covers pediatric study planning and conduct, considerations for first dose in children, appropriate sampling strategies, and different methods for data generation and analysis to generate knowledge about the pharmacokinetics (PK) and pharmacodynamics (PD) of a drug in children. The role of Modeling and Simulation (M&S) in pediatrics is highlighted-including the regulatory basis-and examples of the use of M&S are illustrated to support pediatric drug development.
authors.affiliation: Actelion Pharmaceuticals Ltd, Clinical Pharmacology, Gewerbestrasse 16, CH-4123 Allschwil, Switzerland. jochen.zisowsky@actelion.com.
authors.forename: Jochen
authors.initials: J
authors.lastname: Zisowsky
authors.affiliation: Actelion Pharmaceuticals Ltd, Clinical Pharmacology, Gewerbestrasse 16, CH-4123 Allschwil, Switzerland.
authors.forename: Andreas
authors.initials: A
authors.lastname: Krause
authors.affiliation: Actelion Pharmaceuticals Ltd, Clinical Pharmacology, Gewerbestrasse 16, CH-4123 Allschwil, Switzerland.
authors.forename: Jasper
authors.initials: J
authors.lastname: Dingemanse
keywords: Modeling and Simulation; PIP; PK/PD  modeling; children; clinical studies; health authorities; pediatric drug development; pediatric legislation; pediatrics; regulatory guidelines