Abstract
This chapter will give examples of particular clinical trial designs that are fundamentally flawed. Each example will illustrate a fairly common practice. The first example is a futility rule that aims to stop accrual to a single-arm trial early if the interim data show that it is unlikely the experimental treatment provides at least a specified level of anti-disease activity. The rule is given in terms of progression-free survival time. An alternative, much more sound, and reliable futility monitoring rule that accounts for each patient’s complete time-to-event follow-up data will be presented. The second example will show how the routine practice of defining patient evaluability can lead one astray when estimating treatment effects, by misrepresenting the actual patient outcomes. The next two examples pertain to the problems of incompletely or vaguely specified safety monitoring rules. The final example shows what can go wrong when one ignores fundamental experimental design issues, including bias and confounding, when evaluating and comparing multiple treatments. As an alternative approach, the family of randomized select-and-test designs will be presented.
Two things are infinite: the universe and human stupidity; and I’m not sure about the universe.
Albert Einstein
Access this chapter
Tax calculation will be finalised at checkout
Purchases are for personal use only
Author information
Authors and Affiliations
Corresponding author
Rights and permissions
Copyright information
© 2020 Springer Nature Switzerland AG
About this chapter
Cite this chapter
Thall, P.F. (2020). Just Plain Wrong. In: Statistical Remedies for Medical Researchers. Springer Series in Pharmaceutical Statistics. Springer, Cham. https://doi.org/10.1007/978-3-030-43714-5_10
Download citation
DOI: https://doi.org/10.1007/978-3-030-43714-5_10
Published:
Publisher Name: Springer, Cham
Print ISBN: 978-3-030-43713-8
Online ISBN: 978-3-030-43714-5
eBook Packages: Mathematics and StatisticsMathematics and Statistics (R0)