Direkt zum Inhalt

Greenstreet, Peter ; Jaki, Thomas ; Bedding, Alun ; Harbron, Chris ; Mozgunov, Pavel

A multi‐arm multi‐stage platform design that allows preplanned addition of arms while still controlling the family‐wise error

Greenstreet, Peter, Jaki, Thomas , Bedding, Alun, Harbron, Chris und Mozgunov, Pavel (2024) A multi‐arm multi‐stage platform design that allows preplanned addition of arms while still controlling the family‐wise error. Statistics in Medicine 43 (19), S. 3613-3632.

Veröffentlichungsdatum dieses Volltextes: 22 Sep 2025 07:13
Artikel
DOI zum Zitieren dieses Dokuments: 10.5283/epub.77772


Zusammenfassung

There is growing interest in platform trials that allow for adding of new treatment arms as the trial progresses as well as being able to stop treatments part way through the trial for either lack of benefit/futility or for superiority. In some situations, platform trials need to guarantee that error rates are controlled. This paper presents a multi-stage design, that allows additional arms to be ...

There is growing interest in platform trials that allow for adding of new treatment arms as the trial progresses as well as being able to stop treatments part way through the trial for either lack of benefit/futility or for superiority. In some situations, platform trials need to guarantee that error rates are controlled. This paper presents a multi-stage design, that allows additional arms to be added in a platform trial in a preplanned fashion, while still controlling the family-wise error rate, under the assumption of known number and timing of treatments to be added, and no time trends. A method is given to compute the sample size required to achieve a desired level of power and we show how the distribution of the sample size and the expected sample size can be found. We focus on power under the least favorable configuration which is the power of finding the treatment with a clinically relevant effect out of a set of treatments while the rest have an uninteresting treatment effect. A motivating trial is presented which focuses on two settings, with the first being a set number of stages per active treatment arm and the second being a set total number of stages, with treatments that are added later getting fewer stages. Compared to Bonferroni, the savings in the total maximum sample size are modest in a trial with three arms, <1% of the total sample size. However, the savings are more substantial in trials with more arms.



Beteiligte Einrichtungen


Details

DokumentenartArtikel
Titel eines Journals oder einer ZeitschriftStatistics in Medicine
Verlag:Wiley
Band:43
Nummer des Zeitschriftenheftes oder des Kapitels:19
Seitenbereich:S. 3613-3632
Datum16 Juni 2024
InstitutionenInformatik und Data Science > Fachbereich Maschinelles Lernen und Data Science > Chair for Computational Statistics (Prof. Dr. Thomas Jaki)
Identifikationsnummer
WertTyp
10.1002/sim.10135DOI
Stichwörter / KeywordsMAMS, multi-arm, multi-stage, platform trials, strong control of FWER
Dewey-Dezimal-Klassifikation000 Informatik, Informationswissenschaft, allgemeine Werke > 004 Informatik
StatusVeröffentlicht
BegutachtetJa, diese Version wurde begutachtet
An der Universität Regensburg entstandenZum Teil
URN der UB Regensburgurn:nbn:de:bvb:355-epub-777725
Dokumenten-ID77772

Bibliographische Daten exportieren

Nur für Besitzer und Autoren: Kontrollseite des Eintrags

nach oben