Optimization of the two-stage group sequential three-arm gold-standard design for non-inferiority trials.
gold-standard design
group sequential
hierarchical testing
non-inferiority
optimal design
Journal
Statistics in medicine
ISSN: 1097-0258
Titre abrégé: Stat Med
Pays: England
ID NLM: 8215016
Informations de publication
Date de publication:
20 02 2023
20 02 2023
Historique:
revised:
06
09
2022
received:
03
11
2021
accepted:
08
12
2022
pubmed:
29
12
2022
medline:
24
1
2023
entrez:
28
12
2022
Statut:
ppublish
Résumé
If design parameters are chosen appropriately, group sequential trial designs are known to be able to reduce the expected sample size under the alternative hypothesis compared to single-stage designs. The same holds true for the so-called 'gold-standard' design for non-inferiority trials, a design involving an experimental group, an active control group, and a placebo group. However, choosing design parameters that maximize the advantages of a two-stage approach for the three-arm gold-standard design for non-inferiority trials is not a straightforward task. In particular, optimal choices of futility boundaries for this design have not been thoroughly discussed in existing literature. We present a variation of the hierarchical testing procedure, which allows for the incorporation of binding futility boundaries at interim analyses. We show that this procedure maintains strong control of the family-wise type I error rate. Within this framework, we consider the futility and efficacy boundaries as well as the sample size allocation ratios as optimization parameters. This allows the investigation of the efficiency gain from including the option to stop for futility in addition to the ability to stop for efficacy. To analyze the extended designs, optimality criteria that include the design's performance under the alternative as well as the null hypothesis are introduced. On top of this, we discuss methods to limit the allocation of placebo patients in the trial while maintaining relatively good operating characteristics. The results of our numerical optimization procedure are discussed and a comparison of different approaches to designing a three-arm gold-standard non-inferiority trial is provided.
Types de publication
Journal Article
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
536-558Informations de copyright
© 2022 The Authors. Statistics in Medicine published by John Wiley & Sons Ltd.
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