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Experimental design and sample size determination

1 Experimental designand sample size determinationKarl W BromanDepartment of BiostatisticsJohns Hopkins ~kbromanNote This is a shortened version of a lecture which is part of a web-based course on Enhancing Humane Science/ImprovingAnimal Research (organized by Alan Goldberg, Johns HopkinsCenter for Alternatives to Animal Testing) Few details mostly design3 Basic question/goal in (aka blocking) experiments43 ExampleQuestion:Does salted drinking water affect blood pressure (BP) in mice? a mouse with water containing 1% 14 experiments are comparative. Compare BP in mice fed salt water to BP in micefed plain water. Compare BP in strain A mice fed salt water to BPin strain B mice fed salt , the Experimental group is compared toconcurrent controls (rather than to historical controls).64 Replication7 Why replicate? Reduce the effect of uncontrolled variation( , increase precision). Quantify related point:An estimate is of no value without some statement of the uncertainty in the subjects ( units ) should be assigned totreatment groups at random does not mean needs to explicitly randomize using A computer, or Coins, dice or randomize?

•Decrease the variability in the measurements. –Make subjects more homogeneous. –Use stratification. –Control for other variables (e.g., weight). –Average multiple measurements on each subject. 35 Final conclusions •Experiments should be designed. •Good design and good analysis can lead to reduced sample sizes.

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