Optimizing randomized trial designs to distinguish which subpopulations benefit from treatment

DOI10.1093/BIOMET/ASR055MaRDI QIDQ3107976zbMATH OpenOpenAlexWikidataFDO

Authors Michael Rosenblum, Mark J. Van der Laan

Publication date 28 December 2011

Published in Biometrika (Search for Journal in Brave)

Full work available at URL http://europepmc.org/articles/pmc3413180

zbMATH Keywords

optimizationgroup sequential designadaptive designenrichment designpatient-oriented research

Mathematics Subject Classification ID

Applications of statistics to biology and medical sciences; meta analysis (62P10) Medical applications (general) (92C50) Sequential statistical design (62L05)

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Cited in

(13)

• Analysis of ``learn-as-you-go (LAGO) studies
• Adaptive patient enrichment designs in therapeutic trials
• Adaptive randomized trial designs that cannot be dominated by any standard design at the same total sample size
• Recursive partitioning for heterogeneous causal effects
• Special study designs: early escape, enrichment, studies in non-responders
• Random norming AIDS analysis of non-linear regression models with sequential informative dose selection
• New insights into adaptive enrichment designs
• Uniformly most powerful tests for simultaneously detecting a treatment effect in the overall population and at least one subpopulation
• Inference for a two-stage enrichment design
• A predictive enrichment procedure to identify potential responders to a new therapy for randomized, comparative controlled clinical studies
• Confidence intervals for the selected population in randomized trials that adapt the population enrolled
• Optimal tests of treatment effects for the overall population and two subpopulations in randomized trials, using sparse linear programming
• Optimal treatments in cost-effectiveness analysis in the presence of covariates: improving patient subgroup definition

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