Recent public awareness and scientific advances have launched a reversal in the historical trend to virtually ignore the importance of obtaining reliable data from pediatric patients during the process of drug development. The need for well-designed clinical trials in children is now clearly recognized, and a few regulatory bodies have addressed the requirements for the conduct of these trials. Global consensus on the unique issues related to pediatric trials, however, has been lacking. The International Conference on Harmonization recently released a draft of its guideline, “Clinical Investigation of Medicinal Products in the Pediatric Population.” The objective of this paper is to discuss critical issues addressed by the guideline. It begins with a brief overview on the underlying need for the guideline and factors that have contributed to shifting attitudes toward the conduct of pediatric clinical trials. The five major considerations outlined in the guideline are then summarized. The discussion for each of the key areas is supplemented with background information, representative cases, and points for further consideration.
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