Statistical design considerations for pilot studies transitioning therapies from the bench to the bedsideReport as inadecuate




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Journal of Translational Medicine

, 2:37

First Online: 28 October 2004Received: 07 October 2004Accepted: 28 October 2004DOI: 10.1186-1479-5876-2-37

Cite this article as: Carter, R.E. & Woolson, R.F. J Transl Med 2004 2: 37. doi:10.1186-1479-5876-2-37

Abstract

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.

Electronic supplementary materialThe online version of this article doi:10.1186-1479-5876-2-37 contains supplementary material, which is available to authorized users.





Author: Rickey E Carter - Robert F Woolson

Source: https://link.springer.com/







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