Adaptive enrichment designs in clinical trials

PF Thall - Annual review of statistics and its application, 2021 - annualreviews.org
Adaptive enrichment designs for clinical trials may include rules that use interim data to
identify treatment-sensitive patient subgroups, select or compare treatments, or change entry …

Adaptive enrichment designs with a continuous biomarker

N Stallard - Biometrics, 2023 - Wiley Online Library
A popular design for clinical trials assessing targeted therapies is the two‐stage adaptive
enrichment design with recruitment in stage 2 limited to a biomarker‐defined subgroup …

Bayesian cancer clinical trial designs with subgroup-specific decisions

PF Thall - Contemporary Clinical Trials, 2020 - Elsevier
Two illustrative applications are presented of Bayesian clinical trial designs that make
adaptive subgroup-specific decisions based on elicited utilities of patient outcomes to …

Isotonic design for single-arm biomarker stratified trials

L Li, A Ivanova - Statistical Methods in Medical Research, 2024 - journals.sagepub.com
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12pt]?> In single-arm trials with a predefined subgroup based on baseline biomarkers, it is …

Blinded sample size recalculation in adaptive enrichment designs

M Placzek, T Friede - Biometrical Journal, 2023 - Wiley Online Library
In the precision medicine era,(prespecified) subgroup analyses are an integral part of
clinical trials. Incorporating multiple populations and hypotheses in the design and analysis …

A unified framework for weighted parametric group sequential design

KM Anderson, Z Guo, J Zhao, LZ Sun - Biometrical Journal, 2022 - Wiley Online Library
Group sequential design (GSD) is widely used in clinical trials in which correlated tests of
multiple hypotheses are used. Multiple primary objectives resulting in tests with known …

Adaptive identification of populations with treatment benefit in clinical trials: machine learning challenges and solutions

A Curth, A Hüyük… - … Conference on Machine …, 2023 - proceedings.mlr.press
We study the problem of adaptively identifying patient subpopulations that benefit from a
given treatment during a confirmatory clinical trial. This type of adaptive clinical trial has …

Group sequential two‐stage preference designs

R Liu, F Li, D Esserman, MM Ryan - Statistics in Medicine, 2024 - Wiley Online Library
The two‐stage preference design (TSPD) enables inference for treatment efficacy while
allowing for incorporation of patient preference to treatment. It can provide unbiased …

Bags: A Bayesian adaptive group sequential trial design with subgroup-specific survival comparisons

R Lin, PF Thall, Y Yuan - Journal of the American Statistical …, 2021 - Taylor & Francis
A Bayesian group sequential design is proposed that performs survival comparisons within
patient subgroups in randomized trials where treatment–subgroup interactions may be …

A conditional error function approach for adaptive enrichment designs with continuous endpoints

M Placzek, T Friede - Statistics in Medicine, 2019 - Wiley Online Library
Adaptive enrichment designs offer an efficient and flexible way to demonstrate the efficacy of
a treatment in a clinically defined full population or in, eg, biomarker‐defined subpopulations …