Randomized Trials, Statistics, and Clinical Inference

Statistical inference of adaptive randomized clinical trials for personalized medicine

Feifang Hu*,1, Yanqing Hu2, Wei Ma3, Lixin Zhang4 & Hongjian Zhu5 Department of Statistics, George Washington University, Washington, DC 20052, USA Department of Statistics, West Virginia University, Morgantown, WV 26506, USA Biogen Idec, 14 Cambridge Center, Cambridge, MA 02142, USA Department of Mathematics, Zhejiang University, Hangzhou 310006, PR China Department of Biostatistics, The Unive...

Statistics and Interpretation Of Clinical Trials

Clinical Trials Advantages and disadvantages. Retrospective and prospective studies. Controlled and uncontrolled trials. Historical and concurrent controls. Blind and double-blind studies. Phase I, II and III trials. Ethics (Helsinki declaration). Planning a Trial Establishing objectives, short-term and long-term. Determining the appropriate criteria. Establishing grounds for inclusion and excl...

Causal Inference in Randomized Trials with Noncompliance

Randomized Experiments and Observational Studies: Causal Inference in Statistics

This talk describes the theory of causal inference in randomized experiments and nonrandomized observational studies, using two simple theoretical/actual examples for illustration. Key ideas: causal effects, randomized experiments, adjustments for observed covariates, sensitivity analysis for unobserved covariates, reducing sensitivity to hidden bias using design strategies. 1. Seven Key Contri...

Randomized Clinical Trials in HFpEF

Heart Failure With Preserved Ejection Fraction Heart failure with preserved ejection fraction (HFpEF) or diastolic heart failure (HF) accounts for approximately half of HF cases in the community, and the portion of HF with preserved ejection fraction (EF) is increasing. Patients with HFpEF have limited functional capacity and poor prognosis. With ongoing shifts in the age distribution of the po...