Statistical design considerations for pilot studies transitioning therapies from the bench to the bedside

被引：30

作者：

Carter R.E. ^{[1
]}

Woolson R.F. ^{[1
]}

机构：

[1] Dept. Biostatist. Bioinfo./Epidemiol, Medical University of South Carolina, Charleston, SC 29425

来源：

Journal of Translational Medicine | / 2卷 / 1期

关键词：

Hearing Loss; Translational Research; Transition Therapy; Augmentative Therapy; Initial Sample Size;

D O I：

10.1186/1479-5876-2-37

中图分类号：

学科分类号：

摘要：

Pilot studies are often used to transition therapies developed using animal models to a clinical setting. Frequently, the focus of such trials is on estimating the safety in terms of the occurrence of certain adverse events. With relatively small sample sizes, the probability of observing even relatively common events is low; however, inference on the true underlying event rate is still necessary even when no events of interest are observed. The exact upper limit to the event rate is derived and illustrated graphically. In addition, the simple algebraic expression for the confidence bound is seen to be useful in the context of planning studies. © 2004 Carter and Woolson; licensee BioMed Central Ltd.

引用

页数：3

共 11 条

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