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Statistics in Medicine
Article . 2023 . Peer-reviewed
License: CC BY NC ND
Data sources: Crossref
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zbMATH Open
Article . 2024
Data sources: zbMATH Open
https://dx.doi.org/10.48550/ar...
Article . 2023
License: CC BY NC ND
Data sources: Datacite
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Group sequential two‐stage preference designs

Group sequential two-stage preference designs
Authors: Ruyi Liu; Fan Li; Denise Esserman; Mary M. Ryan;

Group sequential two‐stage preference designs

Abstract

The two‐stage preference design (TSPD) enables inference for treatment efficacy while allowing for incorporation of patient preference to treatment. It can provide unbiased estimates for selection and preference effects, where a selection effect occurs when patients who prefer one treatment respond differently than those who prefer another, and a preference effect is the difference in response caused by an interaction between the patient's preference and the actual treatment they receive. One potential barrier to adopting TSPD in practice, however, is the relatively large sample size required to estimate selection and preference effects with sufficient power. To address this concern, we propose a group sequential two‐stage preference design (GS‐TSPD), which combines TSPD with sequential monitoring for early stopping. In the GS‐TSPD, pre‐planned sequential monitoring allows investigators to conduct repeated hypothesis tests on accumulated data prior to full enrollment to assess study eligibility for early trial termination without inflating type I error rates. Thus, the procedure allows investigators to terminate the study when there is sufficient evidence of treatment, selection, or preference effects during an interim analysis, thereby reducing the design resource in expectation. To formalize such a procedure, we verify the independent increments assumption for testing the selection and preference effects and apply group sequential stopping boundaries from the approximate sequential density functions. Simulations are then conducted to investigate the operating characteristics of our proposed GS‐TSPD compared to the traditional TSPD. We demonstrate the applicability of the design using a study of Hepatitis C treatment modality.

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Keywords

FOS: Computer and information sciences, Other Statistics (stat.OT), preference trial, two-stage randomized clinical trial, Patient Preference, Article, Applications of statistics to biology and medical sciences; meta analysis, group sequential monitoring, Methodology (stat.ME), Statistics - Other Statistics, independent increments, Treatment Outcome, Research Design, Sample Size, Humans, preference effect, Statistics - Methodology

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selected citations
These citations are derived from selected sources.
This is an alternative to the "Influence" indicator, which also reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).
BIP!Citations provided by BIP!
popularity
This indicator reflects the "current" impact/attention (the "hype") of an article in the research community at large, based on the underlying citation network.
BIP!Popularity provided by BIP!
influence
This indicator reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).
BIP!Influence provided by BIP!
impulse
This indicator reflects the initial momentum of an article directly after its publication, based on the underlying citation network.
BIP!Impulse provided by BIP!
0
Average
Average
Average
Green
hybrid