Assurance for clinical trial design with normally distributed outcomes: Eliciting uncertainty about variances.

Pharmaceutical Statistics
Ziyad A Alhussain, Jeremy E Oakley

Abstract

The assurance method is growing in popularity in clinical trial planning. The method involves eliciting a prior distribution for the treatment effect, and then calculating the probability that a proposed trial will produce a "successful" outcome. For normally distributed observations, uncertainty about the variance of the normal distribution also needs to be accounted for, but there is little guidance in the literature on how to elicit a distribution for a variance parameter. We present a simple elicitation method, and illustrate how the elicited distribution is incorporated within an assurance calculation. We also consider multi-stage trials, where a decision to proceed with a larger trial will follow from the outcome of a smaller trial; we illustrate the role of the elicited distribution in assessing the information provided by a proposed smaller trial. Free software is available for implementing our methods.

References

Jan 1, 1986·Statistics in Medicine·D J Spiegelhalter, L S Freedman
Apr 3, 2009·Pharmaceutical Statistics·Richard M NixonAlan Brennan
Jul 19, 2013·Statistics in Medicine·Shijie Ren, Jeremy E Oakley
Apr 1, 2018·Pharmaceutical Statistics·Nigel DallowTimothy H Montague

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Citations

Mar 7, 2021·International Journal of Environmental Research and Public Health·Danila AzzolinaIleana Baldi
Dec 16, 2021·Pharmaceutical Statistics·Lisa V HampsonSteffen Ballerstedt

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