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9 papers · 1 filter
Multivariate Adjustments for Average Equivalence Testing
Younes Boulaguiem, Luca Insolia, Maria-Pia Victoria-Feser +2
Multivariate (average) equivalence testing is widely used to assess whether the means of two conditions of interest are `equivalent' for different outcomes simultaneously. The mult…
Randomization-based Inference for MCP-Mod
Lukas Pin, Oleksandr Sverdlov, Frank Bretz +1
Dose selection is critical in pharmaceutical drug development, as it directly impacts therapeutic efficacy and patient safety of a drug. The Generalized Multiple Comparison Procedu…
Implementing Response-Adaptive Randomisation in Stratified Rare-disease Trials: Design Challenges and Practical Solutions
Rajenki Das, Nina Deliu, Mark Toshner +1
Although response-adaptive randomisation (RAR) has gained substantial attention in the literature, it still has limited use in clinical trials. Amongst other reasons, the implement…
Partial Ordering Bayesian Logistic Regression Model for Phase I Combination Trials and Computationally Efficient Approach to Operational Prior Specification
Weishi Chen, Pavel Mozgunov
Recent years have seen increased interest in combining drug agents and/or schedules. Several methods for Phase I combination-escalation trials are proposed, among which, the partia…
Dynamic factor analysis for sparse and irregular longitudinal data: an application to metabolite measurements in a COVID-19 study
Jiachen Cai, Robert J. B. Goudie, Brian D. M. Tom
It is of scientific interest to identify essential biomarkers in biological processes underlying diseases to facilitate precision medicine. Factor analysis (FA) has long been used…
Inference procedures in sequential trial emulation with survival outcomes: comparing confidence intervals based on the sandwich variance estimator, bootstrap and jackknife
Juliette M. Limozin, Shaun R. Seaman, Li Su
Sequential trial emulation (STE) is an approach to estimating causal treatment effects by emulating a sequence of target trials from observational data. In STE, inverse probability…