IDEAS home Printed from https://ideas.repec.org/p/huj/dispap/dp559.html
   My bibliography  Save this paper

The Treatment Versus Experimentation Dilemma in Dose-finding Studies

Author

Listed:
  • David Azriel
  • Micha Mandel

    ()

  • Yosef Rinott

    ()

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.

Suggested Citation

  • David Azriel & Micha Mandel & Yosef Rinott, 2010. "The Treatment Versus Experimentation Dilemma in Dose-finding Studies," Discussion Paper Series dp559, The Federmann Center for the Study of Rationality, the Hebrew University, Jerusalem.
  • Handle: RePEc:huj:dispap:dp559
    as

    Download full text from publisher

    File URL: http://ratio.huji.ac.il/sites/default/files/publications/dp559.pdf
    Download Restriction: no

    Citations

    Citations are extracted by the CitEc Project, subscribe to its RSS feed for this item.
    as


    Cited by:

    1. Azriel, David, 2012. "A note on the robustness of the continual reassessment method," Statistics & Probability Letters, Elsevier, vol. 82(5), pages 902-906.

    More about this item

    NEP fields

    This paper has been announced in the following NEP Reports:

    Statistics

    Access and download statistics

    Corrections

    All material on this site has been provided by the respective publishers and authors. You can help correct errors and omissions. When requesting a correction, please mention this item's handle: RePEc:huj:dispap:dp559. See general information about how to correct material in RePEc.

    For technical questions regarding this item, or to correct its authors, title, abstract, bibliographic or download information, contact: (Michael Simkin). General contact details of provider: http://edirc.repec.org/data/crihuil.html .

    If you have authored this item and are not yet registered with RePEc, we encourage you to do it here. This allows to link your profile to this item. It also allows you to accept potential citations to this item that we are uncertain about.

    We have no references for this item. You can help adding them by using this form .

    If you know of missing items citing this one, you can help us creating those links by adding the relevant references in the same way as above, for each refering item. If you are a registered author of this item, you may also want to check the "citations" tab in your RePEc Author Service profile, as there may be some citations waiting for confirmation.

    Please note that corrections may take a couple of weeks to filter through the various RePEc services.

    IDEAS is a RePEc service hosted by the Research Division of the Federal Reserve Bank of St. Louis . RePEc uses bibliographic data supplied by the respective publishers.