The Treatment Versus Experimentation Dilemma in Dose-finding Studies

David Azriel, Micha Mandel () and Yosef Rinott

Discussion Paper Series from The Federmann Center for the Study of Rationality, the Hebrew University, Jerusalem

Abstract: Phase I clinical trials are conducted in order to find the maximum tolerated dose (MTD) of a given drug from a finite set of doses. For ethical reasons, these studies are usually sequential, treating patients or group of patients with the best available dose according to the current knowledge. However, it is proved here that such designs, and, more generally, designs that concentrate on one dose from some time on, cannot provide consistent estimators for the MTD unless very strong parametric assumptions hold. We describe a family of sequential designs that treat individuals with one of the two closest doses to the estimated MTD, and prove that such designs, under general conditions, concentrate eventually on the two closest doses to the MTD and estimate the MTD consistently. It is shown that this family contains randomized designs that assign the MTD with probability that approaches 1 as the size of the experiment goes to infinity. We compare several designs by simulations, studying their performances in terms of correct estimation of the MTD and the proportion of individuals treated with the MTD.

Pages: 17 pages
Date: 2010-09
New Economics Papers: this item is included in nep-ecm
References: View complete reference list from CitEc
Citations: View citations in EconPapers (4)

Published in Journal of Statistical Planning and Inference 141, 2759--2768. (2011).

Downloads: (external link)
http://ratio.huji.ac.il/sites/default/files/publications/dp559.pdf (application/pdf)
Our link check indicates that this URL is bad, the error code is: 404 Not Found (http://ratio.huji.ac.il/sites/default/files/publications/dp559.pdf [302 Moved Temporarily]--> https://ratio.huji.ac.il/sites/default/files/publications/dp559.pdf)

Related works:
This item may be available elsewhere in EconPapers: Search for items with the same title.

Export reference: BibTeX RIS (EndNote, ProCite, RefMan) HTML/Text

Persistent link: https://EconPapers.repec.org/RePEc:huj:dispap:dp559

Access Statistics for this paper

More papers in Discussion Paper Series from The Federmann Center for the Study of Rationality, the Hebrew University, Jerusalem Contact information at EDIRC.
Bibliographic data for series maintained by Michael Simkin ().