|Title||A model-based conditional power assessment for decision making in randomized controlled trial studies.|
|Publication Type||Journal Article|
|Year of Publication||2017|
|Authors||Zou, Baiming, Jianwen Cai, Gary G. Koch, Haibo Zhou, and Fei Zou|
|Date Published||2017 Dec 30|
|Keywords||Clinical Decision-Making, Computer Simulation, Diabetes Mellitus, Type 1, Humans, Likelihood Functions, Models, Statistical, Monte Carlo Method, Multivariate Analysis, Nonlinear Dynamics, Normal Distribution, Randomized Controlled Trials as Topic, Regression Analysis, Sample Size|
Conditional power based on summary statistic by comparing outcomes (such as the sample mean) directly between 2 groups is a convenient tool for decision making in randomized controlled trial studies. In this paper, we extend the traditional summary statistic-based conditional power with a general model-based assessment strategy, where the test statistic is based on a regression model. Asymptotic relationships between parameter estimates based on the observed interim data and final unobserved data are established, from which we develop an analytic model-based conditional power assessment for both Gaussian and non-Gaussian data. The model-based strategy is not only flexible in handling baseline covariates and more powerful in detecting the treatment effects compared with the conventional method but also more robust in controlling the overall type I error under certain missing data mechanisms. The performance of the proposed method is evaluated by extensive simulation studies and illustrated with an application to a clinical study.
|Alternate Journal||Stat Med|
|Original Publication||A model-based conditional power assessment for decision making in randomized controlled trial studies.|
|PubMed Central ID||PMC5995155|
|Grant List||P01 CA142538 / CA / NCI NIH HHS / United States |
T32 ES007018 / ES / NIEHS NIH HHS / United States
UL1 TR000064 / TR / NCATS NIH HHS / United States
UL1 TR001427 / TR / NCATS NIH HHS / United States
A model-based conditional power assessment for decision making in randomized controlled trial studies.