Planning And Analyzing Clinical Trials With Composite Endpoints

DOWNLOAD

Download Planning And Analyzing Clinical Trials With Composite Endpoints PDF/ePub or read online books in Mobi eBooks. Click Download or Read Online button to get Planning And Analyzing Clinical Trials With Composite Endpoints book now. This website allows unlimited access to, at the time of writing, more than 1.5 million titles, including hundreds of thousands of titles in various foreign languages. If the content not found or just blank you must refresh this page

Planning And Analyzing Clinical Trials With Composite Endpoints

DOWNLOAD
Author : Geraldine Rauch
language : en
Publisher: Springer
Release Date : 2018-05-22

Planning And Analyzing Clinical Trials With Composite Endpoints written by Geraldine Rauch and has been published by Springer this book supported file pdf, txt, epub, kindle and other format this book has been release on 2018-05-22 with Medical categories.

This book addresses the most important aspects of how to plan and evaluate clinical trials with a composite primary endpoint to guarantee a clinically meaningful and valid interpretation of the results. Composite endpoints are often used as primary efficacy variables for clinical trials, particularly in the fields of oncology and cardiology. These endpoints combine several variables of interest within a single composite measure, and as a result, all variables that are of major clinical relevance can be considered in the primary analysis without the need to adjust for multiplicity. Moreover, composite endpoints are intended to increase the size of the expected effects thus making clinical trials more powerful. The book offers practical advice for statisticians and medical experts involved in the planning and analysis of clinical trials. For readers who are mainly interested in the application of the methods, all the approaches are illustrated with real-world clinical trial examples, and the software codes required for fast and easy implementation are provided. The book also discusses all the methods in the context of relevant guidelines related to the topic. To benefit most from the book, readers should be familiar with the principles of clinical trials and basic statistical methods.

Methods For Planning And Analyzing Clinical Trials With Composite Endpoints

DOWNLOAD
Author : Svenja Schüler
language : en
Publisher:
Release Date : 2020

Methods For Planning And Analyzing Clinical Trials With Composite Endpoints written by Svenja Schüler and has been published by this book supported file pdf, txt, epub, kindle and other format this book has been release on 2020 with categories.

Multiple Testing Problems In Pharmaceutical Statistics

DOWNLOAD
Author : Alex Dmitrienko
language : en
Publisher: CRC Press
Release Date : 2009-12-08

Multiple Testing Problems In Pharmaceutical Statistics written by Alex Dmitrienko and has been published by CRC Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2009-12-08 with Mathematics categories.

Useful Statistical Approaches for Addressing Multiplicity Issues Includes practical examples from recent trials Bringing together leading statisticians, scientists, and clinicians from the pharmaceutical industry, academia, and regulatory agencies, Multiple Testing Problems in Pharmaceutical Statistics explores the rapidly growing area of multiple comparison research with an emphasis on pharmaceutical applications. In each chapter, the expert contributors describe important multiplicity problems encountered in pre-clinical and clinical trial settings. The book begins with a broad introduction from a regulatory perspective to different types of multiplicity problems that commonly arise in confirmatory controlled clinical trials, before giving an overview of the concepts, principles, and procedures of multiple testing. It then presents statistical methods for analyzing clinical dose response studies that compare several dose levels with a control as well as statistical methods for analyzing multiple endpoints in clinical trials. After covering gatekeeping procedures for testing hierarchically ordered hypotheses, the book discusses statistical approaches for the design and analysis of adaptive designs and related confirmatory hypothesis testing problems. The final chapter focuses on the design of pharmacogenomic studies based on established statistical principles. It also describes the analysis of data collected in these studies, taking into account the numerous multiplicity issues that occur. This volume explains how to solve critical issues in multiple testing encountered in pre-clinical and clinical trial applications. It presents the necessary statistical methodology, along with examples and software code to show how to use the methods in practice.

Sharing Clinical Trial Data

DOWNLOAD
Author : Institute of Medicine
language : en
Publisher: National Academies Press
Release Date : 2015-04-20

Sharing Clinical Trial Data written by Institute of Medicine and has been published by National Academies Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2015-04-20 with Medical categories.

Data sharing can accelerate new discoveries by avoiding duplicative trials, stimulating new ideas for research, and enabling the maximal scientific knowledge and benefits to be gained from the efforts of clinical trial participants and investigators. At the same time, sharing clinical trial data presents risks, burdens, and challenges. These include the need to protect the privacy and honor the consent of clinical trial participants; safeguard the legitimate economic interests of sponsors; and guard against invalid secondary analyses, which could undermine trust in clinical trials or otherwise harm public health. Sharing Clinical Trial Data presents activities and strategies for the responsible sharing of clinical trial data. With the goal of increasing scientific knowledge to lead to better therapies for patients, this book identifies guiding principles and makes recommendations to maximize the benefits and minimize risks. This report offers guidance on the types of clinical trial data available at different points in the process, the points in the process at which each type of data should be shared, methods for sharing data, what groups should have access to data, and future knowledge and infrastructure needs. Responsible sharing of clinical trial data will allow other investigators to replicate published findings and carry out additional analyses, strengthen the evidence base for regulatory and clinical decisions, and increase the scientific knowledge gained from investments by the funders of clinical trials. The recommendations of Sharing Clinical Trial Data will be useful both now and well into the future as improved sharing of data leads to a stronger evidence base for treatment. This book will be of interest to stakeholders across the spectrum of research-from funders, to researchers, to journals, to physicians, and ultimately, to patients.

Behavioral Clinical Trials For Chronic Diseases

DOWNLOAD
Author : Lynda H. Powell
language : en
Publisher: Springer Nature
Release Date : 2021-10-13

Behavioral Clinical Trials For Chronic Diseases written by Lynda H. Powell and has been published by Springer Nature this book supported file pdf, txt, epub, kindle and other format this book has been release on 2021-10-13 with Psychology categories.

This is the first comprehensive guide to the design of behavioral randomized clinical trials (RCT) for chronic diseases. It includes the scientific foundations for behavioral trial methods, problems that have been encountered in past behavioral trials, advances in design that have evolved, and promising trends and opportunities for the future. The value of this book lies in its potential to foster an ability to “speak the language of medicine” through the conduct of high-quality behavioral clinical trials that match the rigor commonly seen in double-blind drug trials. It is relevant for testing any treatment aimed at improving a behavioral, social, psychosocial, environmental, or policy-level risk factor for a chronic disease including, for example, obesity, sedentary behavior, adherence to treatment, psychosocial stress, food deserts, and fragmented care. Outcomes of interest are those that are of clinical significance in the treatment of chronic diseases, including standard risk factors such as cholesterol, blood pressure, and glucose, and clinical outcomes such as hospitalizations, functional limitations, excess morbidity, quality of life, and mortality. This link between behavior and chronic disease requires innovative clinical trial methods not only from the behavioral sciences but also from medicine, epidemiology, and biostatistics. This integration does not exist in any current book, or in any training program, in either the behavioral sciences or medicine.

Clinical Trial Biostatistics And Biopharmaceutical Applications

DOWNLOAD
Author : Walter R. Young
language : en
Publisher: CRC Press
Release Date : 2014-11-20

Clinical Trial Biostatistics And Biopharmaceutical Applications written by Walter R. Young and has been published by CRC Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2014-11-20 with Mathematics categories.

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.

Multiple Analyses In Clinical Trials

DOWNLOAD
Author : Lemuel A. Moyé
language : en
Publisher: Springer Science & Business Media
Release Date : 2006-05-17

Multiple Analyses In Clinical Trials written by Lemuel A. Moyé and has been published by Springer Science & Business Media this book supported file pdf, txt, epub, kindle and other format this book has been release on 2006-05-17 with Medical categories.

One of the most challenging issues for clinical trial investigators, sponsors, and regulatory officials is the interpretation of experimental results that are composed of the results of multiple statistical analyses. These analyses may include the effect of therapy on multiple endpoints, the assessment of a subgroup analysis, and the evaluation of a dose-response relationship in complex mixtures. Multiple Analyses in Clinical Trials: Fundamentals for Clinical Investigators is an essentially nonmathematical discussion of the problems posed by the execution of multiple analyses in clinical trials. It concentrates on the rationale for the analyses, the difficulties posed by their interpretation, easily understood solutions, and useful problem sets. This text will help clinical investigators understand multiple analysis procedures and the key issues when designing their own work or reviewing the research of others. This book is written for advanced medical students, clinical investigators at all levels, research groups within the pharmaceutical industry, regulators at the local, state, and federal level, and biostatisticians. Only a basic background in health care and introductory statistics is required. Dr. Lemuel A. Moyé, M.D., Ph.D. is a physician and Professor of Biometry at the University of Texas School of Public Health. He has been Co-Principal Investigator of two multinational clinical trials examining the role of innovative therapy in post myocardial infarction survival (SAVE) and the use of cholesterol reducing agents in post myocardial infarction survival in patients with normal cholesterol levels (CARE). He has authored over one hundred articles in journals such as the Journal of the American Medical Association, the New England Journal of Medicine, Statistics in Medicine, and Controlled Clinical Trials. From the reviews: From the reviews: "A quick scan of the book indicates that it is not a typical statistics book...You can jumpin almost anywhere and just start reading...I like the book’s organization. There is a chapter on clinical trials. Then there are several chapters that explain the situations that arise from the occurrence of multiple analyses. Particular emphasis is given to multiple endpoints, situations where one continues a study to follow up on unanticipated results, and to subgroup analyses, interventions that impact only a fraction of the subjects in a study. The author is equally adept at describing clinical trials for the statistician as at explaining statistics to the clinical investigator. I enjoyed leafing through this book and would certainly enjoy have the opportunity to sit down and read it." Technometrics, August 2004 "Moyé’s background as a statistician and MD makes him especially qualified to write this book...The clinical trial examples are a major strength of the book...His medical background and extensive clinical trials experience shine through." Statistics in Medicine, 2004, 23:3551-3559 "The many examples from well known clinical trials are clearly one of the strengths of this book. It is also fascinating to share the author's experience with the FDA where he attended many meetings of Advisory Committees."Biometrics, December 2005 "According to the preface, this book is written for clinical investigators and research groups within the pharmaceutical industry, medical students and regulators. ... I admire the eloquency of the author. ... The author does a remarkable job ... . Without any doubt, the book is a valuable source of ideas for the intended audience. For statisticians it is an interesting source of experimental setups, that are actually used in practice and that consequently are worth while to be studied." (dr H. W. M. Hendriks, Kwantitatieve Methoden, Issue 72B41, 2005) "The book is entertaining and informative, sufficiently informal to recruit and retain the intended non-statisticalreadership, but sufficiently formal to detail methods. The author effectively sets up each issue with examples and conceptual discussion, grounding all in realistic examples." (T. A. Louis, Short Book Reviews, Vol. 24 (2), 2004) "Here in this book is a special situation for clinical trials in which the investigator must do statistical analyses for a number of different response measurements. ... The author felt that the clinical investigators needed a book that they could understand. This book is his effort to reach that audience. ... I like the book’s organization. ... I enjoyed leafing through this book and would certainly enjoy having the opportunity to sit down and read it." (Eric R. Ziegel, Technometrics, Vol. 46 (3), August, 2004) "This book is written primarily for clinical researchers who are interested in designing and analysing clinical trials. It concentrates on elucidating problems arising from multiple analyses in clinical trials ... . In general, the book is well written and easy to follow. ... Although this book is written for clinical investigators, I find it a useful reference book for statisticians in the pharmaceutical industry ... . Furthermore, senior statisticians may find the non-technical discussions and practical examples useful." (Shuying Yang, Pharmaceutical Statistics, Issue 3, 2004) "Without any doubt, the book is a valuable source of ideas for the intendend audience. For statisticians, it is an interesting source of experimental setups, that are actually used in practice and that consequently are worthwhile to be studied." (Dr. H.W.M. Hendriks, Kwantitatieve Methoden)

Design And Analysis Of Quality Of Life Studies In Clinical Trials

DOWNLOAD
Author : Diane L. Fairclough
language : en
Publisher: CRC Press
Release Date : 2010-01-07

Design And Analysis Of Quality Of Life Studies In Clinical Trials written by Diane L. Fairclough and has been published by CRC Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2010-01-07 with Mathematics categories.

Design Principles and Analysis Techniques for HRQoL Clinical TrialsSAS, R, and SPSS examples realistically show how to implement methods Focusing on longitudinal studies, Design and Analysis of Quality of Life Studies in Clinical Trials, Second Edition addresses design and analysis aspects in enough detail so that readers can apply statistical meth

Translational Surgery

DOWNLOAD
Author : Adam E.M. Eltorai
language : en
Publisher: Elsevier
Release Date : 2023-03-22

Translational Surgery written by Adam E.M. Eltorai and has been published by Elsevier this book supported file pdf, txt, epub, kindle and other format this book has been release on 2023-03-22 with Science categories.

Translational Surgery covers the principles of evidence-based medicine and applies these principles to the design of translational investigations. The reader will come to fully understand important concepts including case-control studies, prospective cohort studies, randomized trials, and reliability studies. Investigators will benefit from greater confidence in their ability to initiate and execute their own investigations, avoid common pitfalls in surgical research, and know what is needed for collaboration. Further, this title is an indispensable tool in grant writing and funding efforts. The practical, straightforward approach helps the translational research navigate challenging considerations in study design and implementation. The book provides valuable discussions of the critical appraisal of published studies in surgery, allowing the reader to learn how to evaluate the quality of such studies. Thus, they will improve at measuring outcomes; making effective use of all types of evidence in patient care. In short, this practical guidebook will be of interest to every surgeon or surgical researcher who has ever had a good clinical idea, but not the knowledge of how to test it. - Focuses on translational research in Surgery, covering the principles of evidence-based medicine and applying those principles to the design of translational investigations - Provides a practical, straightforward approach to help surgeons and researchers navigate challenging aspects of study design and implementation - Details valuable discussions on the critical appraisal of published studies in Surgery, allowing the reader to effectively use all types of evidence for patient care

Methods And Applications Of Sample Size Calculation And Recalculation In Clinical Trials

DOWNLOAD
Author : Meinhard Kieser
language : en
Publisher: Springer Nature
Release Date : 2020-11-19

Methods And Applications Of Sample Size Calculation And Recalculation In Clinical Trials written by Meinhard Kieser and has been published by Springer Nature this book supported file pdf, txt, epub, kindle and other format this book has been release on 2020-11-19 with Mathematics categories.

This book provides an extensive overview of the principles and methods of sample size calculation and recalculation in clinical trials. Appropriate calculation of the required sample size is crucial for the success of clinical trials. At the same time, a sample size that is too small or too large is problematic due to ethical, scientific, and economic reasons. Therefore, state-of-the art methods are required when planning clinical trials. Part I describes a general framework for deriving sample size calculation procedures. This enables an understanding of the common principles underlying the numerous methods presented in the following chapters. Part II addresses the fixed sample size design, where the required sample size is determined in the planning stage and is not changed afterwards. It covers sample size calculation methods for superiority, non-inferiority, and equivalence trials, as well as comparisons between two and more than two groups. A wide range of further topics is discussed, including sample size calculation for multiple comparisons, safety assessment, and multi-regional trials. There is often some uncertainty about the assumptions to be made when calculating the sample size upfront. Part III presents methods that allow to modify the initially specified sample size based on new information that becomes available during the ongoing trial. Blinded sample size recalculation procedures for internal pilot study designs are considered, as well as methods for sample size reassessment in adaptive designs that use unblinded data from interim analyses. The application is illustrated using numerous clinical trial examples, and software code implementing the methods is provided. The book offers theoretical background and practical advice for biostatisticians and clinicians from the pharmaceutical industry and academia who are involved in clinical trials. Covering basic as well as more advanced and recently developed methods, it is suitable for beginners, experienced applied statisticians, and practitioners. To gain maximum benefit, readers should be familiar with introductory statistics. The content of this book has been successfully used for courses on the topic.