The design and conduct of clinical trials to limit missing data

R. J. Little, M. L. Cohen, K. Dickersin, S. S. Emerson, J. T. Farrar, J. D. Neaton, W. Shih, J. P. Siegel, H. Stern

Research output: Contribution to journalArticle

Abstract

This article summarizes recommendations on the design and conduct of clinical trials of a National Research Council study on missing data in clinical trials. Key findings of the study are that (a) substantial missing data is a serious problem that undermines the scientific credibility of causal conclusions from clinical trials; (b) the assumption that analysis methods can compensate for substantial missing data is not justified; hence (c) clinical trial design, including the choice of key causal estimands, the target population, and the length of the study, should include limiting missing data as one of its goals; (d) missing-data procedures should be discussed explicitly in the clinical trial protocol; (e) clinical trial conduct should take steps to limit the extent of missing data; (f) there is no universal method for handling missing data in the analysis of clinical trials - methods should be justified on the plausibility of the underlying scientific assumptions; and (g) when alternative assumptions are plausible, sensitivity analysis should be conducted to assess robustness of findings to these alternatives. This article focuses on the panel's recommendations on the design and conduct of clinical trials to limit missing data. A companion paper addresses the panel's findings on analysis methods.

Original languageEnglish (US)
Pages (from-to)3433-3443
Number of pages11
JournalStatistics in Medicine
Volume31
Issue number28
DOIs
StatePublished - Dec 10 2012

Keywords

  • Clinical trial ethics
  • Dropouts
  • Incomplete data
  • Randomized withdrawal
  • Run-in period

ASJC Scopus subject areas

  • Epidemiology
  • Statistics and Probability

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  • Cite this

    Little, R. J., Cohen, M. L., Dickersin, K., Emerson, S. S., Farrar, J. T., Neaton, J. D., Shih, W., Siegel, J. P., & Stern, H. (2012). The design and conduct of clinical trials to limit missing data. Statistics in Medicine, 31(28), 3433-3443. https://doi.org/10.1002/sim.5519