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Activity Number:
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215
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Type:
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Contributed
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Date/Time:
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Tuesday, August 13, 2002 : 12:00 PM to 1:50 PM
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Sponsor:
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Biopharmaceutical Section*
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| Abstract - #300723 |
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Title:
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An Application of a Multi-Stage Strategy Involving Confidence Intervals to Evaluate Whether a Response Rate is Favorable or Not
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Author(s):
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Sharon Murray*+ and Mary Otterness and John Forster and Diane Catellier and Gary Koch
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Affiliation(s):
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GlaxoSmithKline, Inc. and Medtronic, Inc. and GlaxoSmithKline, Inc. and University of North Carolina and University of North Carolina
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Address:
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PO Box 13398, RTP, North Carolina, 27709-3398, USA
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Keywords:
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Binomial distribution ; Confidence intervals ; Sequential testing ; Multi-stage designs ; Sample size
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Abstract:
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In early clinical development of new medicines, a single-arm study with a limited number of subjects 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 subjects 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, thirty 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.
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