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Yarahmadi, Amin ; Dodd, Lori E. ; Jaki, Thomas ; Horby, Peter ; Stallard, Nigel

Group sequential designs for clinical trials when the maximum sample size is uncertain

Yarahmadi, Amin, Dodd, Lori E., Jaki, Thomas , Horby, Peter und Stallard, Nigel (2024) Group sequential designs for clinical trials when the maximum sample size is uncertain. Statistics in Medicine 43 (24), S. 4667-4683.

Veröffentlichungsdatum dieses Volltextes: 22 Sep 2025 06:51
Artikel
DOI zum Zitieren dieses Dokuments: 10.5283/epub.77769


Zusammenfassung

Motivated by the experience of COVID-19 trials, we consider clinical trials in the setting of an emerging disease in which the uncertainty of natural disease course and potential treatment effects makes advance specification of a sample size challenging. One approach to such a challenge is to use a group sequential design to allow the trial to stop on the basis of interim analysis results as soon ...

Motivated by the experience of COVID-19 trials, we consider clinical trials in the setting of an emerging disease in which the uncertainty of natural disease course and potential treatment effects makes advance specification of a sample size challenging. One approach to such a challenge is to use a group sequential design to allow the trial to stop on the basis of interim analysis results as soon as a conclusion regarding the effectiveness of the treatment under investigation can be reached. As such a trial may be halted before a formal stopping boundary is reached, we consider the final analysis under such a scenario, proposing alternative methods for when the decision to halt the trial is made with or without knowledge of interim analysis results. We address the problems of ensuring that the type I error rate neither exceeds nor falls unnecessarily far below the nominal level. We also propose methods in which there is no maximum sample size, the trial continuing either until the stopping boundary is reached or it is decided to halt the trial.



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Details

DokumentenartArtikel
Titel eines Journals oder einer ZeitschriftStatistics in Medicine
Verlag:Wiley
Band:43
Nummer des Zeitschriftenheftes oder des Kapitels:24
Seitenbereich:S. 4667-4683
Datum21 August 2024
InstitutionenInformatik und Data Science > Fachbereich Maschinelles Lernen und Data Science > Chair for Computational Statistics (Prof. Dr. Thomas Jaki)
Identifikationsnummer
WertTyp
10.1002/sim.10203DOI
Stichwörter / Keywordsconditional error group-sequential stopping boundary sequential probability ratio test spending function underrunning analysis
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-777697
Dokumenten-ID77769

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