Statistical thinking in clinical trials / Michael A. Proschan, Julian Barreiro-Gomez.

Format:

Book

Author/Creator:

Proschan, Michael A., author.

Barreiro-Gomez, Julian, author.

Contributor:

Taylor & Francis eBooks.

Dr. Adolph E. Schecter Memorial Fund.

Series:

Chapman & Hall/CRC biostatistics series

Language:

English

Subjects (All):

Clinical trials--Statistical methods.

Clinical trials.

Physical Description:

1 online resource (270 pages).

polychrome

Edition:

First edition.

Place of Publication:

Boca Raton : CRC Press, Taylor & Francis Group, 2022.

System Details:

text file

Biography/History:

Michael Proschan is a mathematical statistician and Fellow of the American Statistical Association with 32 years of clinical trial experience in cardiovascular and infectious diseases, including HIV/AIDS, Ebola virus disease, and COVID-19. He has expertise in statistical monitoring of clinical trials, having taught short courses and co-authored the book Statistical Monitoring of Clinical Trials: A Unified Approach with Gordon Lan and Janet Wittes. He co-authored, with Sally Hunsberger, one of the first papers on adaptive clinical trial methods using the observed treatment effect at an interim analysis. More recently, Dr. Proschan has written about the vital role re-randomization tests play in adaptive methods before breaking the treatment blind. He has recently been an adjunct faculty member at George Washington University and Johns Hopkins University Advanced Academic Programs.

Contents:

1. Evidence and Inference Terminology and Paradigm of Inference Classical Inference Hypothesis Tests and P-Values Confidence Intervals Criticisms of Classical Methods The Bayesian Approach Large Sample Inference Robust Methods Are Preferred in Clinical Trials Summary 2. 2 2 Tables Measures of Treatment Effect Exact Tests and Confidence Intervals Fisher Exact Test Exact Confidence Interval for Odds Ratio Oddities of FisherExact Test and Confidence Interval Unconditional Tests As Alternatives to FisherExact Test Appendix: P(X = x S = s) in Table Summary 3. Introduction to Clinical Trials Summary 4. Design of Clinical Trials Different Phases of Trials Blinding Baseline Variables Controls Regression to the Mean Appropriate Control Choice of Primary Endpoint Reducing Variability Replication and Averaging Differencing Stratification Regression Different Types of Trials Superiority Versus Noninferiority Parallel Arm Trials Crossover Trials Cluster-Randomized Trials Multi-Arm Trials Appendix: The Geometry of Stratification Summary 5. Randomization/Allocation Sanctity and Placement of Randomization Simple Randomization Permuted Block Randomization Biased Coin Randomization Stratified Randomization Minimization and Covariate-Adaptive Randomization Response-Adaptive Randomization Adaptive Randomization And Temporal TrendsSummary 6. Randomization-Based Inference Introduction Paired Data An Example Control of Conditional Type Error Rate Asymptotic Equivalence to a T-test The Null Hypothesis and Generalizing Does A Re-randomization Test Assume Independence? Unpaired Data: Traditional Randomization Introduction Control of Conditional Type Error Rate The Null Hypothesis and Generalizing Does a Re-randomization Test Require Independence? Asymptotic Equivalence to a t-Test Protection Against Temporal Trends FisherExact Test As a Re-Randomization Test Unpaired Data: Covariate-Adaptive Randomization Introduction Control of Conditional Type Error Rate Protection Against Temporal Trends A More Rigorous Null Hypothesis Unpaired Data: Response-Adaptive Randomization Introduction Re-randomization Tests & Strength of Randomized Evidence Confidence Intervals A Philosophical Criticism of Re-randomization Tests Appendix: The Permutation Variance of YC YT Summary 7. Survival Analysis Introduction to Survival Methods Comparing Survival Across Arms Comparing Survival At A Specific Time The Logrank Test The Hazard Rate and Cox Model Competing Risk Analysis Parametric Approaches Conditional Binomial Procedure Appendix: Partial Likelihood Summary 8. Sample Size/Power Introduction The EZ Principle Illustrated through the -Sample t-Test Important Takeaways from the EZ Principle EZ Principle Applied More Generally -Sample t-test Test of Proportions Logrank Test Cluster-Randomized Trials In a Nutshell Nonzero Nulls Practical Aspects of Sample Size Calculations Test of Means Test of Proportions Specification of Treatment Effect Exact Power t-Tests Exact Power for FisherExact Test Adjusting for Noncompliance and Other Factors Appendix: Other Sample Size Formulas for Two Proportions Summary 9. Monitoring Introduction Efficacy Monitoring A Brief History of Efficacy Boundaries Z-scores, B-Values, and Information Revisiting O Brien-Fleming Alpha Spending Functions The Effect of Monitoring on Power Small Sample Sizes Futility Monitoring What is Futility? Conditional Power Beta Spending Functions Practical Aspects of Monitoring Inference after A Monitored Trial Statistical Contrast between Unmonitored and Monitored Trials Defining A P-Value after a Monitored Trial Defining A Confidence Interval after A Monitored Trial Correcting Bias after A Monitored Trial Bayesian Monitoring Summary 10. M&Ms: Multiplicity & Missing Data Introduction Multiple Comparisons The Debate Control of Familywise Error Rate (FWER) Showing Strong Control by Enumeration Intuition Behind Multiple Comparison Procedures Independent Comparisons Closure Principle The Dunnett Procedure And A Conditioning Technique Missing Data Definitions And An Example Methods for Data That Are MAR Sensitivity Analyses Summary 11. Adaptive Methods Introduction Adaptive Sample Size Based on Nuisance Parameters Continuous Outcomes Binary Outcomes Adaptive Sample Size Based on Treatment Effect Introduction and Notation Non-adaptive Two-Stage Setting Adaptation Principle Bauer-Kohne () Proschan and Hunsberger, () Criticisms of Adaptive Methods Based on The Treatment Effect Unplanned Changes before Breaking the Blind Summary Index

Notes:

Includes bibliographical references and index.

"A Chapman & Hall book."

Electronic reproduction. London Available via World Wide Web.

Online resource; title from PDF title page (Taylor & Francis, viewed November 29, 2021).

Local Notes:

Acquired for the Penn Libraries with assistance from the Dr. Adolph E. Schecter Memorial Fund.

ISBN:

9781315164090

1315164094

9781351673105

1351673106

9781351673112

1351673114

9781351673099

1351673092

Publisher Number:

99989607755

Access Restriction:

Restricted for use by site license.