# Statistical Design and Analysis of Clinical Trials

Developed from the authors' courses taught to public health and medical students, residents, and fellows during the past 15 years, the text shows how biostatistics in clinical trials is an integration of many fu

Author: Weichung Joe Shih

Publisher: CRC Press

ISBN: 9781482250503

Category: Mathematics

Page: 244

View: 694

Statistical Design and Analysis of Clinical Trials: Principles and Methods concentrates on the biostatistics component of clinical trials. Developed from the authors’ courses taught to public health and medical students, residents, and fellows during the past 15 years, the text shows how biostatistics in clinical trials is an integration of many fundamental scientific principles and statistical methods. Teach Your Students How to Design, Monitor, and Analyze Clinical Trials The book begins with ethical and safety principles, core trial design concepts, the principles and methods of sample size and power calculation, and analysis of covariance and stratified analysis. It then focuses on sequential designs and methods for two-stage Phase II cancer trials to Phase III group sequential trials, covering monitoring safety, futility, and efficacy. The authors also discuss the development of sample size reestimation and adaptive group sequential procedures, explain the concept of different missing data processes, and describe how to analyze incomplete data by proper multiple imputations. Turn Your Students into Better Clinical Trial Investigators This text reflects the academic research, commercial development, and public health aspects of clinical trials. It gives students a multidisciplinary understanding of the concepts and techniques involved in designing and analyzing various types of trials. The book’s balanced set of homework assignments and in-class exercises are appropriate for students in (bio)statistics, epidemiology, medicine, pharmacy, and public health.
Categories: Mathematics

# Statistical Issues in the Design and Analysis of Clinical Trials

Chapters 1-5 concern statistical methods in designing and analyzing data for survival clinical trials, and predicting trial duration.

Author: Yanning Liu

Publisher:

ISBN: 365984554X

Category:

Page: 248

View: 333

Categories:

# Design and Analysis of Clinical Trials

Praise for the First Edition of Design and Analysis of Clinical Trials "An excellent book, providing a discussion of the clinical trial process from designing the study through analyzing the data, and to regulatory requirement . . . could ...

Author: Shein-Chung Chow

Publisher: John Wiley & Sons

ISBN: 9780471473299

Category: Mathematics

Page: 752

View: 961

Praise for the First Edition of Design and Analysis of Clinical Trials "An excellent book, providing a discussion of the clinical trial process from designing the study through analyzing the data, and to regulatory requirement . . . could easily be used as a classroom text to understand the process in the new drug development area." –Statistical Methods in Medicine A complete and balanced presentation now revised, updated, and expanded As the field of research possibilities expands, the need for a working understanding of how to carry out clinical trials only increases. New developments in the theory and practice of clinical research include a growing body of literature on the subject, new technologies and methodologies, and new guidelines from the International Conference on Harmonization (ICH). Design and Analysis of Clinical Trials, Second Edition provides both a comprehensive, unified presentation of principles and methodologies for various clinical trials, and a well-balanced summary of current regulatory requirements. This unique resource bridges the gap between clinical and statistical disciplines, covering both fields in a lucid and accessible manner. Thoroughly updated from its first edition, the Second Edition of Design and Analysis of Clinical Trials features new topics such as: Clinical trials and regulations, especially those of the ICH Clinical significance, reproducibility, and generalizability Goals of clinical trials and target population New study designs and trial types Sample size determination on equivalence and noninferiority trials, as well as comparing variabilities Also, three entirely new chapters cover: Designs for cancer clinical trials Preparation and implementation of a clinical protocol Data management of a clinical trial Written with the practitioner in mind, the presentation assumes only a minimal mathematical and statistical background for its reader. Instead, the writing emphasizes real-life examples and illustrations from clinical case studies, as well as numerous references-280 of them new to the Second Edition-to the literature. Design and Analysis of Clinical Trials, Second Edition will benefit academic, pharmaceutical, medical, and regulatory scientists/researchers, statisticians, and graduate-level students in these areas by serving as a useful, thorough reference source for clinical research.
Categories: Mathematics

# Statistical Aspects of the Design and Analysis of Clinical Trials

Fully updated, this revised edition describes the statistical aspects of both the design and analysis of trials, with particular emphasis on the more recent methods of analysis.About 8000 clinical trials are undertaken annually in all areas ...

Author: Brian Everitt

Publisher: World Scientific

ISBN: 9781860944413

Category: Medical

Page: 323

View: 360

Fully updated, this revised edition describes the statistical aspects of both the design and analysis of trials, with particular emphasis on the more recent methods of analysis.About 8000 clinical trials are undertaken annually in all areas of medicine, from the treatment of acne to the prevention of cancer. Correct interpretation of the data from such trials depends largely on adequate design and on performing the appropriate statistical analyses. This book provides a useful guide to medical statisticians and others faced with the often difficult problems of designing and analysing clinical trials.
Categories: Medical

# Design and Analysis of Clinical Trials for Predictive Medicine

Design and Analysis of Clinical Trials for Predictive Medicine provides statistical guidance on conducting clinical trials for predictive medicine.

Author: Shigeyuki Matsui

Publisher: CRC Press

ISBN: 9781466558168

Category: Mathematics

Page: 400

View: 653

Design and Analysis of Clinical Trials for Predictive Medicine provides statistical guidance on conducting clinical trials for predictive medicine. It covers statistical topics relevant to the main clinical research phases for developing molecular diagnostics and therapeutics—from identifying molecular biomarkers using DNA microarrays to confirming their clinical utility in randomized clinical trials. The foundation of modern clinical trials was laid many years before modern developments in biotechnology and genomics. Drug development in many diseases is now shifting to molecularly targeted treatment. Confronted with such a major break in the evolution toward personalized or predictive medicine, the methodologies for design and analysis of clinical trials is now evolving. This book is one of the first attempts to contribute to this evolution by laying a foundation for the use of appropriate statistical designs and methods in future clinical trials for predictive medicine. It is a useful resource for clinical biostatisticians, researchers focusing on predictive medicine, clinical investigators, translational scientists, and graduate biostatistics students.
Categories: Mathematics

# Clinical Trials

This book explains statistics specifically for a medically literate audience. Readers gain not only an understanding of the basics of medical statistics, but also a critical insight into how to review and evaluate clinical trial evidence.

Author: Duolao Wang

Publisher: Remedica

ISBN: 9781901346725

Category: Medical

Page: 480

View: 650

This book aims to demystify clinical trials. It is divided into five sections: fundamentals of trial design, alternative trial designs, basics of statistical analysis, special trial issues in data analysis, and reporting of trials. Using simple language the book explains with illustrations of numerous trial examples, the conceptual and methodological issues that occur at all stages of clinical trial covering trial design, conduct, analysis and reporting. The book is an educational and approachable reference in a difficult area of medicine where clinicians often feel uncertain and this material helps them review, appraise and publish trials and clinical evidence.
Categories: Medical

# The Design and Analysis of Sequential Clinical Trials

This book details all aspects of sequential clinical trials from preliminary planning, through the monitoring of the trial, to the final analysis of the results.

Publisher: John Wiley & Sons

ISBN: 0471975508

Category: Science

Page: 328

View: 327

This book details all aspects of sequential clinical trials from preliminary planning, through the monitoring of the trial, to the final analysis of the results. Emphasis is placed on the triangular test and other procedures based on straight line stopping boundaries. These methods allow for frequent or occasional interim analyses and permit the analysis of a wide variety of patient responses. Alternative procedures are also covered in detail, and these include -spending function methods, repeated confidence intervals and Bayesian approaches to sequential clinical trials.
Categories: Science

# Textbook of Clinical Trials in Oncology

This book offers the requisite background and expert guidance for the innovative statistical design and analysis of clinical trials in oncology.

Author: Susan Halabi

Publisher: CRC Press

ISBN: 9781351620970

Category: Medical

Page: 626

View: 415

There is an increasing need for educational resources for statisticians and investigators. Reflecting this, the goal of this book is to provide readers with a sound foundation in the statistical design, conduct, and analysis of clinical trials. Furthermore, it is intended as a guide for statisticians and investigators with minimal clinical trial experience who are interested in pursuing a career in this area. The advancement in genetic and molecular technologies have revolutionized drug development. In recent years, clinical trials have become increasingly sophisticated as they incorporate genomic studies, and efficient designs (such as basket and umbrella trials) have permeated the field. This book offers the requisite background and expert guidance for the innovative statistical design and analysis of clinical trials in oncology. Key Features: Cutting-edge topics with appropriate technical background Built around case studies which give the work a "hands-on" approach Real examples of flaws in previously reported clinical trials and how to avoid them Access to statistical code on the book’s website Chapters written by internationally recognized statisticians from academia and pharmaceutical companies Carefully edited to ensure consistency in style, level, and approach Topics covered include innovating phase I and II designs, trials in immune-oncology and rare diseases, among many others
Categories: Medical

# Recent Advances in Clinical Trial Design and Analysis

Recent Advances in Clinical Trial Design and Analysis brings together biostatisticians doing cutting-edge research and explains some of the more recent developments in biostatistics to clinicians and scientists who work in clinical trials.

Author: Peter F. Thall

Publisher: Springer Science & Business Media

ISBN: 9781461520092

Category: Medical

Page: 258

View: 887

Clinical trials have two purposes -- to treat the patients in the trial, and to obtain information which increases our understanding of the disease and especially how patients respond to treatment. Statistical design provides a means to achieve both these aims, while statistical data analysis provides methods for extracting useful information from the trial data. Recent advances in statistical computing have enabled statisticians to implement very rapidly a broad array of methods which previously were either impractical or impossible. Biostatisticians are now able to provide much greater support to medical researchers working in both clinical and laboratory settings. As our collective toolkit of techniques for analyzing data has grown, it has become increasingly difficult for biostatisticians to keep up with all the developments in our own field. Recent Advances in Clinical Trial Design and Analysis brings together biostatisticians doing cutting-edge research and explains some of the more recent developments in biostatistics to clinicians and scientists who work in clinical trials.
Categories: Medical

# Design and Analysis of Clinical Trials with Time to Event Endpoints

Design and Analysis of Animal Studies in Pharmaceutical Development, Shein-
Chung Chow and Jen-pei Liu 2. Basic Statistics and Pharmaceutical Statistical
Applications, James E. De Muth 3. Design and Analysis of Bioavailability and ...

Author: Karl E. Peace

Publisher: CRC Press

ISBN: 9781420066401

Category: Mathematics

Page: 616

View: 958

Using time-to-event analysis methodology requires careful definition of the event, censored observation, provision of adequate follow-up, number of events, and independence or "noninformativeness" of the censoring mechanisms relative to the event. Design and Analysis of Clinical Trials with Time-to-Event Endpoints provides a thorough presentation of the design, monitoring, analysis, and interpretation of clinical trials in which time-to-event is of critical interest. After reviewing time-to-event endpoint methodology, clinical trial issues, and the design and monitoring of clinical trials, the book focuses on inferential analysis methods, including parametric, semiparametric, categorical, and Bayesian methods; an alternative to the Cox model for small samples; and estimation and testing for change in hazard. It then presents descriptive and graphical methods useful in the analysis of time-to-event endpoints. The next several chapters explore a variety of clinical trials, from analgesic, antibiotic, and antiviral trials to cardiovascular and cancer prevention, prostate cancer, astrocytoma brain tumor, and chronic myelogonous leukemia trials. The book then covers areas of drug development, medical practice, and safety assessment. It concludes with the design and analysis of clinical trials of animals required by the FDA for new drug applications. Drawing on the expert contributors’ experiences working in biomedical research and clinical drug development, this comprehensive resource covers an array of time-to-event methods and explores an assortment of real-world applications.
Categories: Mathematics

# Design Analysis of Clinical Trials for Economic Evaluation Reimbursement

Published Titles Noninferiority Testing in Clinical Trials: Issues and Challenges
Tie-Hua Ng Optimal Design for ... Yin Bun Cheung Statistical Design and
Analysis of Clinical Trials: Principles and Methods Weichung Joe Shih and
Joseph ...

Author: Iftekhar Khan

Publisher: CRC Press

ISBN: 9781466505483

Category: Mathematics

Page: 339

View: 395

Economic evaluation has become an essential component of clinical trial design to show that new treatments and technologies offer value to payers in various healthcare systems. Although many books exist that address the theoretical or practical aspects of cost-effectiveness analysis, this book differentiates itself from the competition by detailing
Categories: Mathematics

# Design and Analysis of Non Inferiority Trials

Using examples from real clinical trials, Design and Analysis of Non-Inferiority Trials brings together this body of research and confronts the issues involved in the design of a non-inferiority tr

Author: Mark D. Rothmann

Publisher: CRC Press

ISBN: 9781584888055

Category: Mathematics

Page: 454

View: 245

The increased use of non-inferiority analysis has been accompanied by a proliferation of research on the design and analysis of non-inferiority studies. Using examples from real clinical trials, Design and Analysis of Non-Inferiority Trials brings together this body of research and confronts the issues involved in the design of a non-inferiority trial. Each chapter begins with a non-technical introduction, making the text easily understood by those without prior knowledge of this type of trial. Topics covered include: A variety of issues of non-inferiority trials, including multiple comparisons, missing data, analysis population, the use of safety margins, the internal consistency of non-inferiority inference, the use of surrogate endpoints, trial monitoring, and equivalence trials Specific issues and analysis methods when the data are binary, continuous, and time-to-event The history of non-inferiority trials and the design and conduct considerations for a non-inferiority trial The strength of evidence of an efficacy finding and how to evaluate the effect size of an active control therapy A comprehensive discussion on the purpose and issues involved with non-inferiority trials, Design and Analysis of Non-inferiority Trials will assist current and future scientists and statisticians on the optimal design of non-inferiority trials and in assessing the quality of non-inferiority comparisons done in practice.
Categories: Mathematics

# Design and Analysis of Clinical Experiments

Currently available in the Series: T.W. Anderson The Statistical Analysis of Time Series T.S. Arthanari & Yadolah Dodge Mathematical Programming in Statistics Emil Artin Geometric Algebra Norman T. J. Bailey The Elements of Stochastic ...

Author: Joseph L. Fleiss

Publisher: John Wiley & Sons

ISBN: 9781118031179

Category: Mathematics

Page: 448

View: 300

First published in 1986, this unique reference to clinical experimentation remains just as relevant today. Focusing on the principles of design and analysis of studies on human subjects, this book utilizes and integrates both modern and classical designs. Coverage is limited to experimental comparisons of treatments, or in other words, clinical studies in which treatments are assigned to subjects at random.
Categories: Mathematics

# Analysis of Clinical Trials Using SAS

This book also features recommendations from clinical trial experts and a discussion of relevant regulatory guidelines.

Author: Alex Dmitrienko

Publisher: SAS Institute

ISBN: 9781635261448

Category: Computers

Page: 410

View: 599

Analysis of Clinical Trials Using SAS®: A Practical Guide, Second Edition bridges the gap between modern statistical methodology and real-world clinical trial applications. Tutorial material and step-by-step instructions illustrated with examples from actual trials serve to define relevant statistical approaches, describe their clinical trial applications, and implement the approaches rapidly and efficiently using the power of SAS. Topics reflect the International Conference on Harmonization (ICH) guidelines for the pharmaceutical industry and address important statistical problems encountered in clinical trials. Commonly used methods are covered, including dose-escalation and dose-finding methods that are applied in Phase I and Phase II clinical trials, as well as important trial designs and analysis strategies that are employed in Phase II and Phase III clinical trials, such as multiplicity adjustment, data monitoring, and methods for handling incomplete data. This book also features recommendations from clinical trial experts and a discussion of relevant regulatory guidelines. This new edition includes more examples and case studies, new approaches for addressing statistical problems, and the following new technological updates: SAS procedures used in group sequential trials (PROC SEQDESIGN and PROC SEQTEST) SAS procedures used in repeated measures analysis (PROC GLIMMIX and PROC GEE) macros for implementing a broad range of randomization-based methods in clinical trials, performing complex multiplicity adjustments, and investigating the design and analysis of early phase trials (Phase I dose-escalation trials and Phase II dose-finding trials) Clinical statisticians, research scientists, and graduate students in biostatistics will greatly benefit from the decades of clinical research experience and the ready-to-use SAS macros compiled in this book.
Categories: Computers

# Statistical Design and Analysis of Stability Studies

Design and Analysis of Animal Studies in Pharmaceutical Development, Shein-
Chung Chow and Jen-pei Liu . ... Statistical Design for the Researcher, Daryl S.
Paulson Advances in Clinical Trial Biostatistics, Nancy L. Geller Statistics in the ...

Author: Shein-Chung Chow

Publisher: CRC Press

ISBN: 1584889063

Category: Mathematics

Page: 352

View: 458

The US Food and Drug Administration's Report to the Nation in 2004 and 2005 indicated that one of the top reasons for drug recall was that stability data did not support existing expiration dates. Pharmaceutical companies conduct stability studies to characterize the degradation of drug products and to estimate drug shelf life. Illustrating how stability studies play an important role in drug safety and quality assurance, Statistical Design and Analysis of Stability Studies presents the principles and methodologies in the design and analysis of stability studies. After introducing the basic concepts of stability testing, the book focuses on short-term stability studies and reviews several methods for estimating drug expiration dating periods. It then compares some commonly employed study designs and discusses both fixed and random batch statistical analyses. Following a chapter on the statistical methods for stability analysis under a linear mixed effects model, the book examines stability analyses with discrete responses, multiple components, and frozen drug products. In addition, the author provides statistical methods for dissolution testing and explores current issues and recent developments in stability studies. To ensure the safety of consumers, professionals in the field must carry out stability studies to determine the reliability of drug products during their expiration period. This book provides the material necessary for you to perform stability designs and analyses in pharmaceutical research and development.
Categories: Mathematics

# Biomarker Analysis in Clinical Trials with R

The world is awash in data. This volume of data will continue to increase. In the pharmaceutical industry, much of this data explosion has happened around biomarker data.

Author: Nusrat Rabbee

Publisher: CRC Press

ISBN: 9780429766800

Category: Mathematics

Page: 204

View: 371

The world is awash in data. This volume of data will continue to increase. In the pharmaceutical industry, much of this data explosion has happened around biomarker data. Great statisticians are needed to derive understanding from these data. This book will guide you as you begin the journey into communicating, understanding and synthesizing biomarker data. -From the Foreword, Jared Christensen, Vice President, Biostatistics Early Clinical Development, Pfizer, Inc. Biomarker Analysis in Clinical Trials with R offers practical guidance to statisticians in the pharmaceutical industry on how to incorporate biomarker data analysis in clinical trial studies. The book discusses the appropriate statistical methods for evaluating pharmacodynamic, predictive and surrogate biomarkers for delivering increased value in the drug development process. The topic of combining multiple biomarkers to predict drug response using machine learning is covered. Featuring copious reproducible code and examples in R, the book helps students, researchers and biostatisticians get started in tackling the hard problems of designing and analyzing trials with biomarkers. Features: Analysis of pharmacodynamic biomarkers for lending evidence target modulation. Design and analysis of trials with a predictive biomarker. Framework for analyzing surrogate biomarkers. Methods for combining multiple biomarkers to predict treatment response. Offers a biomarker statistical analysis plan. R code, data and models are given for each part: including regression models for survival and longitudinal data, as well as statistical learning models, such as graphical models and penalized regression models.
Categories: Mathematics

# Statistical Methods for Clinical Trials

"Summarizes graphical analysis, analysis of variance, meta-analysis, and design of comparable treatment groups.

Author: Mark X. Norleans

Publisher: CRC Press

ISBN: 0824704673

Category: Mathematics

Page: 276

View: 942

"Summarizes graphical analysis, analysis of variance, meta-analysis, and design of comparable treatment groups. Streamlines the analytical techniques for continuous, categorical, longitudinal, and survival data-focusing on generalized linear models, GEEs, and mixed linear models, -ahd highlihgts p-value, and more."
Categories: Mathematics

# Developments in Statistical Evaluation of Clinical Trials

This book describes various ways of approaching and interpreting the data produced by clinical trial studies, with a special emphasis on the essential role that biostatistics plays in clinical trials.

Author: Kees van Montfort

Publisher: Springer

ISBN: 9783642553455

Category: Mathematics

Page: 361

View: 643

This book describes various ways of approaching and interpreting the data produced by clinical trial studies, with a special emphasis on the essential role that biostatistics plays in clinical trials. Over the past few decades the role of statistics in the evaluation and interpretation of clinical data has become of paramount importance. As a result the standards of clinical study design, conduct and interpretation have undergone substantial improvement. The book includes 18 carefully reviewed chapters on recent developments in clinical trials and their statistical evaluation, with each chapter providing one or more examples involving typical data sets, enabling readers to apply the proposed procedures. The chapters employ a uniform style to enhance comparability between the approaches.
Categories: Mathematics

# Clinical Trials

CRM – Implements the continual reassessment methods for dose finding clinical trials. OPTIMAL – Calculates two-stage optimal phase II designs using the Simon method. POWER – This is a power and sample size program for clinical trials.

Publisher: John Wiley & Sons

ISBN: 9781118959206

Category: Mathematics

Page: 920

View: 161

Categories: Mathematics

# Clinical Trial Biostatistics and Biopharmaceutical Applications

This book clarifies important issues when designing and analyzing clinical trials, including several misunderstood and unresolved challenges. It will help readers choose the right method for their biostatistical application.

Author: Walter R. Young

Publisher: CRC Press

ISBN: 9781482212181

Category: Mathematics

Page: 580

View: 581

Since 1945, "The Annual Deming Conference on Applied Statistics" has been an important event in the statistics profession. In Clinical Trial Biostatistics and Biopharmaceutical Applications, prominent speakers from past Deming conferences present novel biostatistical methodologies in clinical trials as well as up-to-date biostatistical applications from the pharmaceutical industry. Divided into five sections, the book begins with emerging issues in clinical trial design and analysis, including the roles of modeling and simulation, the pros and cons of randomization procedures, the design of Phase II dose-ranging trials, thorough QT/QTc clinical trials, and assay sensitivity and the constancy assumption in noninferiority trials. The second section examines adaptive designs in drug development, discusses the consequences of group-sequential and adaptive designs, and illustrates group sequential design in R. The third section focuses on oncology clinical trials, covering competing risks, escalation with overdose control (EWOC) dose finding, and interval-censored time-to-event data. In the fourth section, the book describes multiple test problems with applications to adaptive designs, graphical approaches to multiple testing, the estimation of simultaneous confidence intervals for multiple comparisons, and weighted parametric multiple testing methods. The final section discusses the statistical analysis of biomarkers from omics technologies, biomarker strategies applicable to clinical development, and the statistical evaluation of surrogate endpoints. This book clarifies important issues when designing and analyzing clinical trials, including several misunderstood and unresolved challenges. It will help readers choose the right method for their biostatistical application. Each chapter is self-contained with references.
Categories: Mathematics