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Robertson, David S. ; Burnett, Thomas ; Choodari‐Oskooei, Babak ; Dimairo, Munya ; Grayling, Michael ; Pallmann, Philip ; Jaki, Thomas

Confidence Intervals for Adaptive Trial Designs II: Case Study and Practical Guidance

Robertson, David S., Burnett, Thomas, Choodari‐Oskooei, Babak, Dimairo, Munya, Grayling, Michael, Pallmann, Philip und Jaki, Thomas (2025) Confidence Intervals for Adaptive Trial Designs II: Case Study and Practical Guidance. Statistics in Medicine 44 (18-19), e70202.

Veröffentlichungsdatum dieses Volltextes: 22 Sep 2025 04:47
Artikel
DOI zum Zitieren dieses Dokuments: 10.5283/epub.77709


Zusammenfassung

In adaptive clinical trials, the conventional confidence interval (CI) for a treatment effect is prone to undesirable properties such as undercoverage and potential inconsistency with the final hypothesis testing decision. Accordingly, as is stated in recent regulatory guidance on adaptive designs, there is the need for caution in the interpretation of CIs constructed during and after an adaptive ...

In adaptive clinical trials, the conventional confidence interval (CI) for a treatment effect is prone to undesirable properties such as undercoverage and potential inconsistency with the final hypothesis testing decision. Accordingly, as is stated in recent regulatory guidance on adaptive designs, there is the need for caution in the interpretation of CIs constructed during and after an adaptive clinical trial. However, it may be unclear which of the available CIs in the literature are preferable. This paper is the second in a two-part series that explores CIs for adaptive trials. Part I provided a methodological review of approaches to construct CIs for adaptive designs. In this paper (Part II), we present an extended case study based around a two-stage group sequential trial, including a comprehensive simulation study of the proposed CIs for this setting. This facilitates an expanded description of considerations around what makes for an effective CI procedure following an adaptive trial. We show that the CIs can have notably different properties. Finally, we propose a set of guidelines for researchers around the choice of CIs and the reporting of CIs following an adaptive design.



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Details

DokumentenartArtikel
Titel eines Journals oder einer ZeitschriftStatistics in Medicine
Verlag:Wiley
Band:44
Nummer des Zeitschriftenheftes oder des Kapitels:18-19
Seitenbereich:e70202
Datum8 August 2025
InstitutionenInformatik und Data Science > Fachbereich Maschinelles Lernen und Data Science > Chair for Computational Statistics (Prof. Dr. Thomas Jaki)
Identifikationsnummer
WertTyp
10.1002/sim.70202DOI
Stichwörter / Keywordsadaptive design bootstrap conditional inference coverage estimation group sequential interim analyses
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-777093
Dokumenten-ID77709

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