Skip to Main content Skip to Navigation
Journal articles

Decision-theoretic designs for small trials and pilot studies: A review

Abstract : Pilot studies and other small clinical trials are often conducted but serve a variety of purposes and there is little consensus on their design. One paradigm that has been suggested for the design of such studies is Bayesian decision theory. In this article, we review the literature with the aim of summarizing current methodological developments in this area. We find that decision-theoretic methods have been applied to the design of small clinical trials in a number of areas. We divide our discussion of published methods into those for trials conducted in a single stage, those for multi-stage trials in which decisions are made through the course of the trial at a number of interim analyses, and those that attempt to design a series of clinical trials or a drug development programme. In all three cases, a number of methods have been proposed, depending on the decision maker’s perspective being considered and the details of utility functions that are used to construct the optimal design.
Complete list of metadata

Cited literature [101 references]  Display  Hide  Download
Contributor : Sarah Zohar Connect in order to contact the contributor
Submitted on : Monday, January 27, 2020 - 3:10:04 PM
Last modification on : Thursday, February 24, 2022 - 1:38:04 PM


Publication funded by an institution



Siew Wan Hee, Thomas Hamborg, Simon Day, Jason Madan, Frank Miller, et al.. Decision-theoretic designs for small trials and pilot studies: A review. Statistical Methods in Medical Research, SAGE Publications, 2016, 25 (3), pp.1022-1038. ⟨10.1177/0962280215588245⟩. ⟨inserm-02456662⟩



Record views


Files downloads