Clinical Trial Design and Alterations in Drug Disposition with Age *

Abstract

Most drugs lack information on their safe and effective use in children. This dilemma limits the number of potentially useful medications in children or places children at increased risk for therapeutic misadventures. A number of problems have hampered the study of drugs in children: childhood is a period of rapid physiologic change, children represent a small market share, drug development is costly, and there are ethical concerns of using children as research subjects. Enactment of the Food and Drug Administration (FDA) Modernization Act (FDAMA) and issuance of the Pediatric Studies regulations will have a dramatic impact on increasing the number of drugs approved for use in children. Important issues must be addressed regarding the design of clinical trials in children, including delineation of disease, use of suitable controls, when investigational drugs should be studied, and which pediatric age groups need to be studied. There is real optimism that the increased interest in obtaining pediatric labeling will lead to a greater number of drugs being approved for use in children.