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.