Introduction to Statistics in Pharmaceutical Clinical Trials / Edition 1

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Overview

All students of pharmaceutical sciences and clinical research need a solid knowledge and understanding of the nature, methods, application, and importance of statistics.

Introduction to Statistics in Pharmaceutical Clinical Trials is an ideal introduction to statistics presented in the context of clinical trials conducted during pharmaceutical drug development. This novel approach both teaches the computational steps needed to conduct analyses and provides a conceptual understanding of how these analyses provide information that forms the rational basis for decision making throughout the drug development process.

This textbook conveys: why and how data are collected in clinical studies, how these data are summarized and analyzed, what the results mean in the context of the clinical research question, how the results are communicated to regulatory agencies and to the scientific and medical communities. Introduction to Statistics in Pharmaceutical Clinical Trials is designed primarily for students of pharmacy, clinical research, and allied health professionals. It will also be invaluable to professionals entering the pharmaceutical, biotechnology, and contract research organization industries who wish to gain a broader understanding of study design, research methodology, and statistical analysis and interpretation in clinical trials.

About the Author:
Todd A Durham is Senior Director of Biostatistics and Data Management, Inspire Pharmaceuticals, Durham, North Carolina, USA

About the Author:
J Rick Turner is Chairman, Department of Clinical Research, Campbell University School of Pharmacy, North Carolina, USA

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Editorial Reviews

Doody's Review Service
Reviewer: Michael R Jacobs, BS, PharmD (Temple University School of Pharmacy)
Description: This book covers the essential components of statistics and research design related to new drug development.
Purpose: It is intended to serve as an introductory text for students of pharmacy and other health-related disciplines as well as students who might be interested in clinical research and the drug development process. It fills the gap that exists between books that focus on research design and those that address statistical methods. The authors have clearly met this worthy objective.
Audience: Although written for students in the health sciences, the book may also be of value to residents and practitioners who have little experience in clinical research and would like to acquire a basic understanding of this material. Both authors have credentials in clinical research and education.
Features: The book covers the essential topics in study design and statistical analysis as they relate to the drug development process. It uses the unique approach of interweaving research methodology with relevant statistical applications. The chapters follow a logical progression from a short discussion of statistical terminology, through the development of a research hypothesis, to the demonstration of drug efficacy and safety. Illustrations are used when appropriate to deliver a point. Some of the chapters have exercises that can be used for practice problems. Excluding the appendixes, the book is under 200 pages; consequently, although it meets the objective of providing an introduction, some topics may not be covered to the depth some readers desire.
Assessment: The book fills a unique position and uses an innovative method. Although not stated by the authors, it is quite possible that additional users of the book will be the statisticians and researchers who are asked to teach an introductory course in clinical research. The research process is now so sophisticated that some find it difficult to simplify. This book addresses this need.
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Product Details

  • ISBN-13: 9780853697145
  • Publisher: Pharmaceutical Press
  • Publication date: 4/1/2008
  • Edition description: New Edition
  • Edition number: 1
  • Pages: 226
  • Sales rank: 1,261,635
  • Product dimensions: 7.50 (w) x 9.70 (h) x 0.50 (d)

Table of Contents

Foreword     x
Preface     xii
Dedications     xiv
The discipline of Statistics: Introduction and terminology     1
Introduction     1
The discipline of Statistics     2
The term "statistic" and the plural form "statistics"     3
The term "statistical analysis"     3
Association versus causation     3
Variation and systematic variation     4
Compelling evidence     4
The terms "datum" and "data"     5
Results from statistical analyses as the basis for decision-making     5
Blood pressure and blood pressure medication     5
Organization of the book     6
Some context before reading Chapters 2-11     7
Review     8
References     8
The role of clinical trials in new drug development     9
Introduction     9
Drug discovery     9
Regulatory guidance and governance     10
Pharmaceutical manufacturing     13
Nonclinical research     14
Clinical trials     15
Postmarketing surveillance     18
Ethical conduct during clinical trials     19
Review     20
References     20
Research questions and research hypotheses     23
Introduction     23
The concept of scientific research questions     23
Useful research questions     23
Useful information     24
Moving from the research question to the research hypotheses     24
The placebo effect     24
The drug treatment group and the placebo treatment group     25
Characteristics of a useful research question     25
The reason why there are two research hypotheses     26
Other forms of the null and alternate hypotheses     27
Deciding between the null and alternate hypothesis     28
An operational statistical definition of "more"     29
The concept of statistically significant differences     30
Putting these thoughts into more precise language     30
Hypothesis testing     31
The relationship between hypothesis testing and ethics in clinical trials     31
The relationship between research questions and study design     32
Review     33
References     33
Study design and experimental methodology     35
Introduction     35
Basic principles of study design     36
A common design in therapeutic exploratory and confirmatory trials     38
Experimental methodology     40
Why are we interested in blood pressure?     41
Uniformity of blood pressure measurement     43
Measuring change in blood pressure over time     43
The clinical study protocol     44
Review     45
References     45
Data, central tendency, and variation     47
Introduction     47
Populations and samples     47
Measurement scales     48
Random variables     49
Displaying the frequency of values of a random variable     49
Central tendency     52
Dispersion     53
Tabular displays of summary statistics of central tendency and dispersion     55
Review     56
References     56
Probability, hypothesis testing, and estimation     57
Introduction     57
Probability     57
Probability distributions     60
Binomial distribution     61
Normal distribution     62
Classical probability and relative frequency probability     67
The law of large numbers     68
Sample statistics and population parameters     69
Sampling variation     69
Estimation: General considerations     70
Hypothesis testing: General considerations     74
Hypothesis test of a single population mean     78
The p value     80
Relationship between confidence intervals and hypothesis tests     81
Brief review of estimation and hypothesis testing     82
Review     83
References     83
Early phase clinical trials     85
Introduction     85
A quick recap of early phase studies     85
General comments on study designs in early phase clinical studies     86
Goals of early phase clinical trials     86
Research questions in early phase clinical studies     87
Pharmacokinetic characteristics of interest     87
Analysis of pharmacokinetic and pharmacodynamic data     89
Dose-finding trials     91
Bioavailability trials     92
Other data acquired in early phase clinical studies     93
Limitations of early phase trials     94
Review     95
References     95
Confirmatory clinical trials: Safety data I     97
Introduction     97
The rationale for safety assessments in clinical trials     97
A regulatory view on safety assessment     98
Adverse events     99
Reporting adverse events     99
Using all reported AEs for all participants     100
Absolute and relative risks of participants reporting specific AEs     101
Analyzing serious AEs     102
Concerns with potential multiplicity issues     102
Accounting for sampling variation     103
A confidence interval for a sample proportion     103
Confidence intervals for the difference between two proportions     105
Time-to-event analysis     107
Kaplan-Meier estimation of the survival function     109
Review     114
References     115
Confirmatory clinical trials: Safety data II     117
Introduction     117
Analyses of clinical laboratory data     117
Vital signs     123
QT interval prolongation and torsades de pointes liability     124
Concluding comments on safety assessments in clinical trials     125
Review     126
References     126
Confirmatory clinical trials: Analysis of categorical efficacy data     127
Introduction: Regulatory views of substantial evidence     127
Objectives of therapeutic confirmatory trials     130
Moving from research questions to research objectives: Identification of endpoints     131
A brief review of hypothesis testing     132
Hypothesis tests for two or more proportions     133
Concluding comments on hypothesis tests for categorical data     145
Review     145
References     146
Confirmatory clinical trials: Analysis of continuous efficacy data     147
Introduction     147
Hypothesis test of two means: Two-sample t test or independent groups t test     147
Hypothesis test of the location of two distributions: Wilcoxon rank sum test     150
Hypothesis tests of more than two means: Analysis of variance     152
A worked example with a small dataset     155
A statistical methodology for conducting multiple comparisons     159
Bonferroni's test     160
Employing Bonferroni's test in our example     161
Tukey's honestly significant difference test     163
Implications of the methodology chosen for multiple comparisons     164
Additional considerations about ANOVA      166
Nonparametric analyses of continuous data     167
The Kruskal-Wallis test     167
Hypothesis test of the equality of survival distributions: Logrank test     169
Review     171
References     172
Additional statistical considerations in clinical trials     173
Introduction     173
Sample size estimation     173
Multicenter studies     181
Analysis populations     182
Dealing with missing data     184
Primary and secondary objectives and endpoints     185
Evaluating baseline characteristics     186
Equivalence and noninferiority study designs     187
Additional study designs     189
Review     190
References     190
Concluding comments     191
Reference     191
Appendices     193
Standard normal distribution areas     195
Percentiles of t distributions     205
Percentiles of X[superscript 2] distributions     207
Percentiles of F distributions ([alpha] = 0.05)     209
Values of q for Tukey's HSD test ([alpha] = 0.05)     211
Review exercise solutions by chapter      215
Index     219

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