Dmitrienko / Pulkstenis Clinical Trial Optimization Using R

1. Auflage 2017
ISBN: 978-1-4987-3508-7
Verlag: CRC Press
Format: PDF
Kopierschutz: Adobe DRM (»Systemvoraussetzungen)

E-Book, Englisch, 337 Seiten

Reihe: Chapman & Hall/CRC Biostatistics Series

The main goal of this book is to define a unified framework for clinical trial optimization based on a comprehensive quantitative evaluation of relevant clinical scenarios (using the clinical scenario evaluation approach) and introduce best practices for simulationbased optimization. The book will be aimed at a broad audience and will emphasize a hands-on approach with a detailed discussion of practical issues arising in clinical trial optimization and R software implementation (relevant statistical methodology will be moved to the appendix).

Autoren/Hrsg.

Dmitrienko, Alex

Pulkstenis, Erik

Fachgebiete

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

Inhaltsverzeichnis

Clinical Scenario Evaluation and Clinical Trial Optimization

Alex Dmitrienko and Gautier Paux

Introduction

Clinical scenario evaluation

Components of Clinical Scenario Evaluation

Software implementation

Case study 1.1: Clinical trial with a normally distributed endpoint

Case study 1.2: Clinical trial with two time-to-event endpoints

Clinical trial optimization

Optimization strategies

Optimization algorithm

Sensitivity assessments

Direct optimization

Case study 1.3: Clinical trial with two patient populations
Qualitative sensitivity assessment

Quantitative sensitivity assessment

Optimal selection of the target parameter

Tradeoff-based optimization

Case study 1.4: Clinical trial with an adaptive design

Optimal selection of the target parameter

Clinical Trials with Multiple Objectives

Alex Dmitrienko and Gautier Paux

Introduction

Clinical Scenario Evaluation framework
Case study 2.1: Optimal selection of a multiplicity adjustment
Qualitative sensitivity assessment
Quantitative sensitivity assessment

Software implementation

Conclusions and extensions

Case study 2.2: Direct selection of optimal procedure parameters
Case study 2.3: Tradeoff-based selection of optimal procedure parameters

Clinical trial

Subgroup Analysis in Clinical Trials

Alex Dmitrienko and Gautier Paux

Introduction

Clinical Scenario Evaluation in confirmatory subgroup analysis

Case study 3.1: Optimal selection of a multiplicity adjustment

Case study 3.2: Optimal selection of decision rules to support two potential claims

Case study 3.3: Optimal selection of decision rules to support three potential claims

Decision Making in Clinical Development

Kaushik Patra, Ming-Dauh Wang, Jianliang Zhang, Aaron Dane, Paul Metcalfe, Paul Frewer, and Erik Pulkstenis

Introduction

Clinical Scenario Evaluation in Go/No-Go decision making and determination of probability of success

Case study 4.1: Bayesian Go/No-Go decision criteria

Case study 4.2: Bayesian Go/No-Go evaluation using an alternative decision criterion
Case study 4.3: Bayesian Go/No-Go evaluation in a trial with an interim analysis

Case study 4.4: Decision criteria in Phase II trials based on Probability of Success

Case study 4.5: Updating POS using interim or external information

Bibliography

Über Autor(innen)

Alex Dmitrienko, Ph.D., Vice President, Center for Statistics in Drug Development, Quintiles Innovation, has over 15 years of pharmaceutical experience and has been actively involved in biostatistical research with emphasis on multiple testing procedures, subgroup analysis and adaptive design in clinical trials. He has authored/edited two SAS Press books (Analysis of Clinical Trials Using SAS and Pharmaceutical Statistics Using SAS) and a Chapman and Hall/CRC Press book (Multiple Testing Problems in Pharmaceutical Statistics). Dr. Dmitrienko is an Associate Editor for Statistics in Medicine and a Fellow of the American Statistical Association.