| Published Version Download ( PDF | 365kB) | License: Creative Commons Attribution 4.0 |
A benchmark for dose-finding studies with unknown ordering
Mozgunov, Pavel, Paoletti, Xavier and Jaki, Thomas
(2021)
A benchmark for dose-finding studies with unknown ordering.
Biostatistics 23 (3), pp. 721-737.
Date of publication of this fulltext: 30 Sep 2025 06:01
Article
DOI to cite this document: 10.5283/epub.77859
Abstract
An important tool to evaluate the performance of a dose-finding design is the nonparametric optimal benchmark that provides an upper bound on the performance of a design under a given scenario. A fundamental assumption of the benchmark is that the investigator can arrange doses in a monotonically increasing toxicity order. While the benchmark can be still applied to combination studies in which ...
An important tool to evaluate the performance of a dose-finding design is the nonparametric optimal benchmark that provides an upper bound on the performance of a design under a given scenario. A fundamental assumption of the benchmark is that the investigator can arrange doses in a monotonically increasing toxicity order. While the benchmark can be still applied to combination studies in which not all dose combinations can be ordered, it does not account for the uncertainty in the ordering. In this article, we propose a generalization of the benchmark that accounts for this uncertainty and, as a result, provides a sharper upper bound on the performance. The benchmark assesses how probable the occurrence of each ordering is, given the complete information about each patient. The proposed approach can be applied to trials with an arbitrary number of endpoints with discrete or continuous distributions. We illustrate the utility of the benchmark using recently proposed dose-finding designs for Phase I combination trials with a binary toxicity endpoint and Phase I/II combination trials with binary toxicity and continuous efficacy.
Alternative links to fulltext
Involved Institutions
Details
| Item type | Article | ||||
| Journal or Publication Title | Biostatistics | ||||
| Publisher: | Oxford Academic, Oxford University Press | ||||
|---|---|---|---|---|---|
| Volume: | 23 | ||||
| Number of Issue or Book Chapter: | 3 | ||||
| Page Range: | pp. 721-737 | ||||
| Date | 4 January 2021 | ||||
| Institutions | Informatics and Data Science > Department Machine Learning & Data Science > Lehrstuhl für Computational Statistics (Prof. Dr. Thomas Jaki) | ||||
| Identification Number |
| ||||
| Keywords | Benchmark; Combination trial; Dose finding; Partial ordering; Power likelihood | ||||
| Dewey Decimal Classification | 000 Computer science, information & general works > 004 Computer science | ||||
| Status | Published | ||||
| Refereed | Yes, this version has been refereed | ||||
| Created at the University of Regensburg | No | ||||
| URN of the UB Regensburg | urn:nbn:de:bvb:355-epub-778596 | ||||
| Item ID | 77859 |
Download Statistics
Download Statistics