Sample size requirements for evaluating intervention effects in three-level cluster randomized clinical trials

Moonseong Heo, Mimi Kim

Research output: Chapter in Book/Report/Conference proceedingChapter

Abstract

Experimental clinical trial settings are now often extended to community entities beyond academic research centers. In such settings, a cluster randomized clinical trial (cluster-RCT) design can be useful to rigorously test the effectiveness of a new intervention. Investigators are most commonly interested in assessing the following three types of intervention effects: overall intervention effect, change in intervention effect over time, and local intervention effect at the end of the study. At the design stage of the cluster-RCT, it is essential to estimate a sample size sufficient for adequate statistical power to evaluate the different intervention effects. However, the sample size estimation must account for the multilevel data structure that is necessitated by the nature of the cluster-RCT design. In this review, we consider a three-level data structure and summarize sample size approaches for testing intervention effects within a unified framework of mixedeffects linear models which offer flexibility in the analysis of multilevel data and hypotheses testing in a cluster-RCT. The sample size methods are presented in closed form and have been validated by simulation studies. Important features of sample size determination for each primary hypothesis are also discussed.

Original languageEnglish (US)
Title of host publicationBiometrics: Theory, Applications, and Issues
PublisherNova Science Publishers, Inc.
Pages1-28
Number of pages28
ISBN (Print)9781617287657
StatePublished - 2011

Fingerprint

Sample Size
Randomized Controlled Trials
Data structures
Size determination
Testing
Multilevel Analysis
Linear Models
Research Personnel
Clinical Trials
Research

ASJC Scopus subject areas

  • Biochemistry, Genetics and Molecular Biology(all)

Cite this

Heo, M., & Kim, M. (2011). Sample size requirements for evaluating intervention effects in three-level cluster randomized clinical trials. In Biometrics: Theory, Applications, and Issues (pp. 1-28). Nova Science Publishers, Inc..

Sample size requirements for evaluating intervention effects in three-level cluster randomized clinical trials. / Heo, Moonseong; Kim, Mimi.

Biometrics: Theory, Applications, and Issues. Nova Science Publishers, Inc., 2011. p. 1-28.

Research output: Chapter in Book/Report/Conference proceedingChapter

Heo, M & Kim, M 2011, Sample size requirements for evaluating intervention effects in three-level cluster randomized clinical trials. in Biometrics: Theory, Applications, and Issues. Nova Science Publishers, Inc., pp. 1-28.
Heo M, Kim M. Sample size requirements for evaluating intervention effects in three-level cluster randomized clinical trials. In Biometrics: Theory, Applications, and Issues. Nova Science Publishers, Inc. 2011. p. 1-28
Heo, Moonseong ; Kim, Mimi. / Sample size requirements for evaluating intervention effects in three-level cluster randomized clinical trials. Biometrics: Theory, Applications, and Issues. Nova Science Publishers, Inc., 2011. pp. 1-28
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