Rothmann / Wiens / Chan Design and Analysis of Non-Inferiority Trials

Erscheinungsjahr 2016
ISBN: 978-1-58488-805-5
Verlag: Taylor & Francis
Format: PDF
Kopierschutz: Adobe DRM (»Systemvoraussetzungen)

E-Book, Englisch, 454 Seiten

Reihe: Chapman & Hall/CRC Biostatistics Series

The increased use of non-inferiority analysis has been accompanied by a proliferation of research on the design and analysis of non-inferiority studies. Using examples from real clinical trials, Design and Analysis of Non-Inferiority Trials brings together this body of research and confronts the issues involved in the design of a non-inferiority trial. Each chapter begins with a non-technical introduction, making the text easily understood by those without prior knowledge of this type of trial.

Topics covered include:

- A variety of issues of non-inferiority trials, including multiple comparisons, missing data, analysis population, the use of safety margins, the internal consistency of non-inferiority inference, the use of surrogate endpoints, trial monitoring, and equivalence trials

- Specific issues and analysis methods when the data are binary, continuous, and time-to-event

- The history of non-inferiority trials and the design and conduct considerations for a non-inferiority trial

- The strength of evidence of an efficacy finding and how to evaluate the effect size of an active control therapy

A comprehensive discussion on the purpose and issues involved with non-inferiority trials, Design and Analysis of Non-inferiority Trials will assist current and future scientists and statisticians on the optimal design of non-inferiority trials and in assessing the quality of non-inferiority comparisons done in practice.

Zielgruppe

Researchers and practitioners from statistics.

Autoren/Hrsg.

Rothmann, Mark D.

Wiens, Brian L.

Chan, Ivan S.F.

Fachgebiete

• Medizin | Veterinärmedizin Medizin | Public Health | Pharmazie | Zahnmedizin Medizin, Gesundheitswesen Epidemiologie, Medizinische Statistik
• Mathematik | Informatik Mathematik Numerik und Wissenschaftliches Rechnen Angewandte Mathematik, Mathematische Modelle
• Sozialwissenschaften Soziologie | Soziale Arbeit Soziologie Allgemein Empirische Sozialforschung, Statistik
• Mathematik | Informatik Mathematik Stochastik Mathematische Statistik
• Interdisziplinäres Wissenschaften Wissenschaften: Forschung und Information Datenanalyse, Datenverarbeitung

Inhaltsverzeichnis

What Is a Non-Inferiority Trial?
Definition of Non-Inferiority
Reasons for Non-Inferiority Trials
Different Types of Comparisons
A History of Non-Inferiority Trials
References
Non-Inferiority Trial Considerations
Introduction
External Validity and Assay Sensitivity
Critical Steps and Issues
Sizing a Study
Example of Anti-Infectives
References
Strength of Evidence and Reproducibility
Introduction
Strength of Evidence
Reproducibility
References
Evaluating the Active Control Effect
Introduction
Active Control Effect
Meta-Analysis Methods
Bayesian Meta-Analyses
References
Across-Trials Analysis Methods
Introduction
Two Confidence Interval Approaches
Synthesis Methods
Comparing Analysis Methods and Type I Error Rates
A Case in Oncology
References
Three-Arm Non-Inferiority Trials
Introduction
Comparisons to Concurrent Controls
Bayesian Analyses
References
Multiple Comparisons
Introduction
Comparing Multiple Groups to an Active Control
Non-Inferiority on Multiple End Points
Testing for Both Superiority and Non-Inferiority
References
Missing Data and Analysis Sets
Introduction
Missing Data
Analysis Sets
References
Safety Studies
Introduction
Considerations for Safety Study
Cardiovascular Risk in Antidiabetic Therapy
References
Additional Topics
Introduction
Interaction Tests
Surrogate End Points
Adaptive Designs
Equivalence Comparisons
References
Inference on Proportions
Introduction
Fixed Thresholds on Differences
Fixed Thresholds on Ratios
Fixed Thresholds on Odds Ratios
Bayesian Methods
Stratified and Adjusted Analyses
Variable Margins
Matched-Pair Designs
References
Inferences on Means and Medians
Introduction
Fixed Thresholds on Differences of Means
Fixed Thresholds on Ratios of Means
Analyses Involving Medians
Ordinal Data
References
Inference on Time-to-Event End Points
Introduction
Censoring
Exponential Distributions
Nonparametric Inference Based on a Hazard Ratio
Analyses Based on Landmarks and Medians
Comparisons Over Preset Intervals
References
Appendix: Statistical Concepts
Frequentist Methods
Bayesian Methods
Comparison of Methods
Stratified and Adjusted Analyses
References
Index

Über Autor(innen)

Dr. Mark Rothmann earned his Ph. D. in Statistics at the University of Iowa. He taught several years as a professor and has worked at the U. S. Food and Drug Administration. He has done research in many areas involving the design and analysis of clinical trials.

Dr. Brian L. Wiens, received his MS in statistics from Colorado State University and his Ph.D. in statistics from Temple University. He has worked at several pharmaceutical, biotechnology and medical device companies since 1991. He has published research in several areas of design and analysis of clinical trials. Dr. Wiens is a Fellow of the American Statistical Association.

Dr. Ivan S.F. Chan received his M.S. in Statistics from The Chinese University of Hong Kong and his Ph.D. in Biostatistics from University of Minnesota. He has worked at Merck Research Laboratories since 1995 and is currently Senior Director and Franchise Head for vaccines, leading the statistical support for all vaccine clinical research programs at Merck. Dr. Chan has published research in many areas of statistics including exact inference, analysis of non-inferiority trials, and methodologies for clinical trials.