Atkinson / Biswas Randomised Response-Adaptive Designs in Clinical Trials

Erscheinungsjahr 2013
ISBN: 978-1-58488-694-5
Verlag: Taylor & Francis
Format: PDF
Kopierschutz: Adobe DRM (»Systemvoraussetzungen)

E-Book, Englisch, 339 Seiten

Reihe: Chapman & Hall/CRC Monographs on Statistics & Applied Probability

Randomised Response-Adaptive Designs in Clinical Trials presents methods for the randomised allocation of treatments to patients in sequential clinical trials. Emphasizing the practical application of clinical trial designs, the book is designed for medical and applied statisticians, clinicians, and statisticians in training.

After introducing clinical trials in drug development, the authors assess a simple adaptive design for binary responses without covariates. They discuss randomisation and covariate balance in normally distributed responses and cover many important response-adaptive designs for binary responses. The book then develops response-adaptive designs for continuous and longitudinal responses, optimum designs with covariates, and response-adaptive designs with covariates. It also covers response-adaptive designs that are derived by optimising an objective function subject to constraints on the variance of estimated parametric functions. The concluding chapter explores future directions in the development of adaptive designs.

Zielgruppe

Researchers and practitioners in statistics and statisticians in the pharmaceutical industry.

Autoren/Hrsg.

Atkinson, Anthony C

Biswas, Atanu

Fachgebiete

• Medizin | Veterinärmedizin Medizin | Public Health | Pharmazie | Zahnmedizin Medizinische Fachgebiete Pharmakologie, Toxikologie
• Mathematik | Informatik Mathematik Stochastik

Inhaltsverzeichnis

Introduction: Stories and Data
Scope and Limits
Two-Treatment Trials with a Binary Response
Equal Randomisation
Adaptive Allocation
Urn Model
Some Motivating Clinical Trials

Adaptive Design: Controversies and Progress
Why Adaptive?
How Adaptive?
Criticism
What Next?

Randomised Balanced Sequential Treatment Allocation
Introduction
Balance with Two Treatments
Designs with Three or More Treatments
Designs with Covariates
The Distribution of Loss and of Bias
Heteroscedastic Models
More about Biased-Coin Designs
Further Reading

Response-Adaptive Designs for Binary Responses
Introduction
Urn Designs
Play-the-Winner Rule
Randomised Play-the-Winner Rule
Generalised Pólya Urn
Success Driven Design (SDD)
Failure-Driven Design (FDD)
Birth and Death Urn (BDU)
Birth and Death Urn with Immigration
Drop-the-Loser Rule
Odds Ratio-Based Adaptive Designs
Delayed Response in the RPW Rule
Prognostic Factors in Urn Designs
Targeting an Allocation Proportion
Adaptive Designs for Categorical Responses
Comparisons and Recommendations

Response-Adaptive Designs for Continuous Responses
Motivation
Some Trials with Continuous Responses
Doubly Adaptive Biased-Coin Designs (DBCD)
Nonparametric Designs
Adaptive Designs for Survival Data
Link Function-Based Adaptive Design (BB)
Multi-Treatment Multivariate Design
DL Rule for Continuous Responses (CDL)

Response-Adaptive Designs for Longitudinal Responses
Repeated Responses
Binary Longitudinal Responses (SLPW)
Design and Analysis for the PEMF Data
Longitudinal Categorical Responses
Longitudinal Multivariate Ordinal Responses
Models with Covariates
Continuous Longitudinal Responses
Random Number of Responses
Numerical Illustrations

Optimum Biased-Coin Designs with Covariates
Modelling and Design
Biased-Coin DA-Optimum Designs
Numerical Comparisons for Two Treatments
Designs for Three Treatments
Distribution of Loss
Skewed Allocations
Skewed Allocation – Numerical
Heteroscedastic Normal Models
Allocation Rules for Heteroscedastic Models
Generalized Linear Models
Binary Data
Allocation Rules for Binomial Models
Gamma Data
Loss, Power, Variability
Further Reading: Skewed Designs

Optimum Response-Adaptive Designs with Covariates
Introduction
Link-Function-Based Adaptive Design
Adaptive Designs Maximising Utility
Power Comparisons for Four Rules
Redesigning a Trial: Fluoxetine Hydrochloride
Extensions
Further Reading

Optimal Response-Adaptive Designs with Constraints
Optimal Designs Subject to Constraints
Design of Jennison and Turnbull
RSIHR Design
Maximising Power: Neyman Allocation
Other Designs
BM Design
ZR Design
A General Framework: BBZ Design
Two Normal Populations with Unknown Variances
Two-Sample Nonparametric Design
BM Design for More Than Two Treatments
Optimal Designs with More than One Constraint
Designs for Survival Times
Covariates
Implementation
Adaptive Constraints
Back to Chapter 7

Adaptive Design: Further Important Issues
Bayesian Adaptive Designs
Two-Stage Adaptive Design
Group Sequential Adaptive Design
Optimal Design for Binary Longitudinal Responses
Inverse Sampling
Robustness in Adaptive Designs
Missing Data in Response-Adaptive Designs
Asymptotic Results for CARA Designs
How to Bridge Theory and Practice

Appendix: Optimum Design

Bibliography

Index

Über Autor(innen)

Anthony C. Atkinson is an Emeritus Professor of Statistics at the London School of Economics and Political Science.

Atanu Biswas is a professor in the Applied Statistics Unit at the Indian Statistical Institute, Kolkata.