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An application of a multi‐stage strategy involving confidence intervals to evaluate whether a response rate is favourable or not
Author(s) -
Murray Sharon C.,
Otterness Mary F.,
Forster John K.,
Catellier Diane J.,
Koch Gary G.
Publication year - 2004
Publication title -
pharmaceutical statistics
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.421
H-Index - 38
eISSN - 1539-1612
pISSN - 1539-1604
DOI - 10.1002/pst.89
Subject(s) - confidence interval , stage (stratigraphy) , computer science , identification (biology) , statistics , type i and type ii errors , binomial distribution , binomial (polynomial) , decision rule , mathematics , artificial intelligence , paleontology , botany , biology
In early clinical development of new medicines, a single‐arm study with a limited number of patients is often used to provide a preliminary assessment of a response rate. A multi‐stage design may be indicated, especially when the first stage should only include very few patients so as to enable rapid identification of an ineffective drug. We used decision rules based on several types of nominal confidence intervals to evaluate a three‐stage design for a study that includes at most 30 patients. For each decision rule, we used exact binomial calculations to determine the probability of continuing to further stages as well as to evaluate Type I and Type II error rates. Examples are provided to illustrate the methods for evaluating alternative decision rules and to provide guidance on how to extend the methods to situations with modifications to the number of stages or number of patients per stage in the study design. Copyright © 2004 John Wiley & Sons, Ltd.