2020/11/23 by Geoffrey S Johnson, Johnson, Geoffrey S
Mathematics · #Applications (stat.AP) #FOS: Computer and information sciences #Machine Learning (stat.ML) #Methodology (stat.ME) #stat.AP #stat.ME #stat.ML
paper · pdf · doi:10.48550/arxiv.2011.11583
Clinical Trial Recruitment, Time on Treatment, Probability of Success, Prediction Interval, Tolerance Interval
arxiv created 2022/01/17 · arxiv updated 2022/01/19
A prediction interval covers a future observation from a random process in repeated sampling, and is typically constructed by identifying a pivotal quantity that is also an ancillary statistic. Analogously, a tolerance interval covers a population percentile in repeated sampling and is often based on a pivotal quantity. One approach we consider in non-normal models leverages a link function resulting in a pivotal quantity that is approximately normally distributed. In settings where this normal approximation does not hold we consider a second approach for tolerance and prediction based on a confidence interval for the mean. These methods are intuitive, simple to implement, have proper operating characteristics, and are computationally efficient compared to Bayesian, re-sampling, and machine learning methods. This is demonstrated in the context of multi-site clinical trial recruitment with staggered site initiation, real-world time on treatment, and end-of-study success for a clinical endpoint.