| Autor: |
Mary F. Otterness, Gary G. Koch, Diane J. Catellier, John K. Forster, Sharon C. Murray |
| Rok vydání: |
2004 |
| Předmět: |
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| Zdroj: |
Pharmaceutical Statistics. 3:25-37 |
| ISSN: |
1539-1604 |
| Popis: |
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. |
| Databáze: |
OpenAIRE |
| Externí odkaz: |
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