Abstract #301859

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JSM 2003 Abstract #301859
Activity Number: 212
Type: Contributed
Date/Time: Tuesday, August 5, 2003 : 8:30 AM to 10:20 AM
Sponsor: Biopharmaceutical Section
Abstract - #301859
Title: Analyses of Pilot and Early Phase Studies with Small Sample Sizes
Author(s): Yong Lin*+ and Pamela A. Ohman Strickland and Weichung J. Shih
Companies: The Cancer Institute of New Jersey and UMDNJ and University of Medicine and Dentistry of New Jersey
Address: 28 Francis Dr., Bridgewater, NJ, 08807-5714,
Keywords: multiple test adjustment ; normal mixtures ; pilot study ; small n study ; proof of concept
Abstract:

The need for statistical methodologies for analyzing a small size study, such as a pilot or so-called "proof of concept"study, has not been paid much attention in the past. Recently the Institute of Medicine (IOM) formed a committee and held a workshop to discuss methodologies for conducting clinical trials with small number participants. We argue that the hypothesis of treatment effect in a small pilot study should be set up to test whether any individual subject has an effect rather than whether the group mean or median has shifted as often done for large, confirmatory clinical trials. Based on this paradigm, we propose to use multiple test procedures when individuals have enough observations, and a mixture-distribution approach when individuals have only one or two observations and present the likelihood ratio test in the latter situation. Examples are given to demonstrate the methods.

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