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Handbook of Methods for Designing, Monitoring, and Analyzing Dose-Finding Trials

Handbooks of Modern Statistical Methods

Edited by: John O’Quigley , Alexia Iasonos , Björn Bornkamp

Print publication date:  April  2017
Online publication date:  April  2017

Print ISBN: 9781498746106
eBook ISBN: 9781315151984
Adobe ISBN:

10.1201/9781315151984
 Cite  Marc Record

Book description

Handbook of Methods for Designing, Monitoring, and Analyzing Dose-Finding Trials gives a thorough presentation of state-of-the-art methods for early phase clinical trials. The methodology of clinical trials has advanced greatly over the last 20 years and, arguably, nowhere greater than that of early phase studies. The need to accelerate drug development in a rapidly evolving context of targeted therapies, immunotherapy, combination treatments and complex group structures has provided the stimulus to these advances. Typically, we deal with very small samples, sequential methods that need to be efficient, while, at the same time adhering to ethical principles due to the involvement of human subjects.

Statistical inference is difficult since the standard techniques of maximum likelihood do not usually apply as a result of model misspecification and parameter estimates lying on the boundary of the parameter space. Bayesian methods play an important part in overcoming these difficulties, but nonetheless, require special consideration in this particular context. The purpose of this handbook is to provide an expanded summary of the field as it stands and also, through discussion, provide insights into the thinking of leaders in the field as to the potential developments of the years ahead. With this goal in mind we present:

  • An introduction to the field for graduate students and novices
  • A basis for more established researchers from which to build
  • A collection of material for an advanced course in early phase clinical trials
  • A comprehensive guide to available methodology for practicing statisticians on the design and analysis of dose-finding experiments
  • An extensive guide for the multiple comparison and modeling (MCP-Mod) dose-finding approach, adaptive two-stage designs for dose finding, as well as dose?time?response models and multiple testing in the context of confirmatory dose-finding studies.?

John O?Quigley is a professor of mathematics and research director at the French National Institute for Health and Medical Research based at the Faculty of Mathematics, University Pierre and Marie Curie in Paris, France. He is author of Proportional Hazards Regression and has published extensively in the field of dose finding.

Alexia Iasonos is an associate attending biostatistician at the Memorial Sloan Kettering Cancer Center in New York. She has over one hundred publications in the leading statistical and clinical journals on the methodology and design of early phase clinical trials. Dr. Iasonos has wide experience in the actual implementation of model based early phase trials and has given courses in scientific meetings internationally.

Björn Bornkamp is a statistical methodologist at Novartis in Basel, Switzerland, researching and implementing dose-finding designs in Phase II clinical trials. He is one of the co-developers of the MCP-Mod methodology for dose finding and main author of the DoseFinding R package. He has published numerous papers on dose finding, nonlinear models and Bayesian statistics, and in 2013 won the Royal Statistical Society award for statistical excellence in the pharmaceutical industry.

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Table of contents

Prelims Download PDF
Chapter  1:  Overview of Phase I Designs Download PDF
Chapter  2:  Model-Based Designs for Safety Download PDF
Chapter  3:  Dose-Finding Methods for Nonbinary Outcomes Download PDF
Chapter  4:  Dose-Finding Trials in Pediatric Oncology Download PDF
Chapter  5:  Phase I/II Dose-Finding Designs with Efficacy and Safety Endpoints Download PDF
Chapter  6:  Designing Early-Phase Drug Combination Trials Download PDF
Chapter  7:  Dose–Schedule Finding in Early-Phase Clinical Trials Download PDF
Chapter  8:  Patient Heterogeneity in Dose-Finding Trials Download PDF
Chapter  9:  Nonparametric Optimal Design in Adaptive Dose-Finding Studies Download PDF
Chapter  10:  Practical Implementation: Protocol Development Download PDF
Chapter  III:  Phase II Dose-Finding Trials Download PDF
Chapter  11:  Dose-Finding Studies in Phase II: Introduction and Overview Download PDF
Chapter  12:  The MCP-Mod Methodology: Practical Considerations and the DoseFinding R Package Download PDF
Chapter  13:  Designing Phase II Dose-Finding Studies: Sample Size, Doses, and Dose Allocation Weights Download PDF
Chapter  14:  Two-Stage Designs in Dose Finding Download PDF
Chapter  15:  Longitudinal Dose–Response Models Download PDF
Chapter  16:  Multiple Test Strategies for Comparing Several Doses with a Control in Confirmatory Trials Download PDF
Chapter  17:  A Regulatory View on Dose-Finding Studies and on the Value of Dose–Exposure–Response Analysis Download PDF
Index Download PDF
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