On optimal designs for clinical trials: an updated review. (English) Zbl 1437.62620

Summary: Optimization of clinical trial designs can help investigators achieve higher quality results for the given resource constraints. The present paper gives an overview of optimal designs for various important problems that arise in different stages of clinical drug development, including phase I dose-toxicity studies; phase I/II studies that consider early efficacy and toxicity outcomes simultaneously; phase II dose-response studies driven by multiple comparisons (MCP), modeling techniques (Mod), or their combination (MCP-Mod); phase III randomized controlled multi-arm multi-objective clinical trials to test difference among several treatment groups; and population pharmacokinetics-pharmacodynamics experiments. We find that modern literature is very rich with optimal design methodologies that can be utilized by clinical researchers to improve efficiency of drug development.


62P10 Applications of statistics to biology and medical sciences; meta analysis
62K05 Optimal statistical designs
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