Direkt zum Inhalt

Mozgunov, Pavel ; Paoletti, Xavier ; Jaki, Thomas

A benchmark for dose-finding studies with unknown ordering

Mozgunov, Pavel, Paoletti, Xavier und Jaki, Thomas (2021) A benchmark for dose-finding studies with unknown ordering. Biostatistics 23 (3), S. 721-737.

Veröffentlichungsdatum dieses Volltextes: 30 Sep 2025 06:01
Artikel
DOI zum Zitieren dieses Dokuments: 10.5283/epub.77859


Zusammenfassung

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.



Beteiligte Einrichtungen


Details

DokumentenartArtikel
Titel eines Journals oder einer ZeitschriftBiostatistics
Verlag:Oxford Academic, Oxford University Press
Band:23
Nummer des Zeitschriftenheftes oder des Kapitels:3
Seitenbereich:S. 721-737
Datum4 Januar 2021
InstitutionenInformatik und Data Science > Fachbereich Maschinelles Lernen und Data Science > Chair for Computational Statistics (Prof. Dr. Thomas Jaki)
Identifikationsnummer
WertTyp
10.1093/biostatistics/kxaa054DOI
Stichwörter / KeywordsBenchmark; Combination trial; Dose finding; Partial ordering; Power likelihood
Dewey-Dezimal-Klassifikation000 Informatik, Informationswissenschaft, allgemeine Werke > 004 Informatik
StatusVeröffentlicht
BegutachtetJa, diese Version wurde begutachtet
An der Universität Regensburg entstandenNein
URN der UB Regensburgurn:nbn:de:bvb:355-epub-778596
Dokumenten-ID77859

Bibliographische Daten exportieren

Nur für Besitzer und Autoren: Kontrollseite des Eintrags

nach oben