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Review
. 2016 Nov;82(5):1308-1314.
doi: 10.1111/bcp.13058. Epub 2016 Aug 8.

Design and conduct of early phase drug studies in children: challenges and opportunities

Michael Rieder  1   2 , Daniel Hawcutt  3   4
Affiliations
Review

Design and conduct of early phase drug studies in children: challenges and opportunities

Michael Rieder et al. Br J Clin Pharmacol. 2016 Nov.

Abstract

It has historically been very difficult to conduct early phase drug studies in children for a number of reasons related to ethics, acceptability, rarity, standardization, end points, safety, dosing and feasibility. Over the past decade there have been a number of developments including novel clinical trial design, in silico pharmacology and microdosing that have significantly enhanced the ability of investigators to conduct early phase drug studies in children. While the evolution of drug therapy is creating a series of new challenges, there has never been a better time for conducting drug studies in children.

Keywords: children; clinical trials; drug development; early phase drug studies.

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Figures

Figure 1
Figure 1
General overview of the drug development process. Modified from 6
Figure 2
Figure 2
Considerations in planning the design of an early phase drug trial in children. A key aspect in planning relates to early decisions as to the need for a flexible design. Modified from 6
See this image and copyright information in PMC

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