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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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| Dokumentenart | Artikel | ||||
| Titel eines Journals oder einer Zeitschrift | Statistics in Medicine | ||||
| Verlag: | Wiley | ||||
|---|---|---|---|---|---|
| Band: | 44 | ||||
| Nummer des Zeitschriftenheftes oder des Kapitels: | 18-19 | ||||
| Seitenbereich: | e70202 | ||||
| Datum | 8 August 2025 | ||||
| Institutionen | Informatik und Data Science > Fachbereich Maschinelles Lernen und Data Science > Chair for Computational Statistics (Prof. Dr. Thomas Jaki) | ||||
| Identifikationsnummer |
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| Stichwörter / Keywords | adaptive design bootstrap conditional inference coverage estimation group sequential interim analyses | ||||
| Dewey-Dezimal-Klassifikation | 000 Informatik, Informationswissenschaft, allgemeine Werke > 004 Informatik | ||||
| Status | Veröffentlicht | ||||
| Begutachtet | Ja, diese Version wurde begutachtet | ||||
| An der Universität Regensburg entstanden | Zum Teil | ||||
| URN der UB Regensburg | urn:nbn:de:bvb:355-epub-777093 | ||||
| Dokumenten-ID | 77709 |
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