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Burnett, Thomas ; König, Franz ; Jaki, Thomas

Adding experimental treatment arms to multi‐arm multi‐stage platform trials in progress

Burnett, Thomas, König, Franz und Jaki, Thomas (2024) Adding experimental treatment arms to multi‐arm multi‐stage platform trials in progress. Statistics in Medicine 43 (18), S. 3447-3462.

Veröffentlichungsdatum dieses Volltextes: 22 Sep 2025 07:29
Artikel
DOI zum Zitieren dieses Dokuments: 10.5283/epub.77774


Zusammenfassung

Multi-arm multi-stage (MAMS) platform trials efficiently compare several treatments with a common control arm. Crucially MAMS designs allow for adjustment for multiplicity if required. If for example, the active treatment arms in a clinical trial relate to different dose levels or different routes of administration of a drug, the strict control of the family-wise error rate (FWER) is paramount. ...

Multi-arm multi-stage (MAMS) platform trials efficiently compare several treatments with a common control arm. Crucially MAMS designs allow for adjustment for multiplicity if required. If for example, the active treatment arms in a clinical trial relate to different dose levels or different routes of administration of a drug, the strict control of the family-wise error rate (FWER) is paramount. Suppose a further treatment becomes available, it is desirable to add this to the trial already in progress; to access both the practical and statistical benefits of the MAMS design. In any setting where control of the error rate is required, we must add corresponding hypotheses without compromising the validity of the testing procedure.To strongly control the FWER, MAMS designs use pre-planned decision rules that determine the recruitment of the next stage of the trial based on the available data. The addition of a treatment arm presents an unplanned change to the design that we must account for in the testing procedure. We demonstrate the use of the conditional error approach to add hypotheses to any testing procedure that strongly controls the FWER. We use this framework to add treatments to a MAMS trial in progress. Simulations illustrate the possible characteristics of such procedures.



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Details

DokumentenartArtikel
Titel eines Journals oder einer ZeitschriftStatistics in Medicine
Verlag:Wiley
Band:43
Nummer des Zeitschriftenheftes oder des Kapitels:18
Seitenbereich:S. 3447-3462
Datum9 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.10090DOI
Stichwörter / Keywordsadaptive designs, conditional error, design modification, multi-arm multi-stage
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-777749
Dokumenten-ID77774

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