Download or read book Statistical Aspects Of The Design And Analysis Of Clinical Trials Revised Edition written by Brian S Everitt and published by World Scientific. This book was released on 2004-02-26 with total page 338 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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./a

Download or read book Small Clinical Trials written by Institute of Medicine and published by National Academies Press. This book was released on 2001-01-01 with total page 221 pages. Available in PDF, EPUB and Kindle. Book excerpt: Clinical trials are used to elucidate the most appropriate preventive, diagnostic, or treatment options for individuals with a given medical condition. Perhaps the most essential feature of a clinical trial is that it aims to use results based on a limited sample of research participants to see if the intervention is safe and effective or if it is comparable to a comparison treatment. Sample size is a crucial component of any clinical trial. A trial with a small number of research participants is more prone to variability and carries a considerable risk of failing to demonstrate the effectiveness of a given intervention when one really is present. This may occur in phase I (safety and pharmacologic profiles), II (pilot efficacy evaluation), and III (extensive assessment of safety and efficacy) trials. Although phase I and II studies may have smaller sample sizes, they usually have adequate statistical power, which is the committee's definition of a "large" trial. Sometimes a trial with eight participants may have adequate statistical power, statistical power being the probability of rejecting the null hypothesis when the hypothesis is false. Small Clinical Trials assesses the current methodologies and the appropriate situations for the conduct of clinical trials with small sample sizes. This report assesses the published literature on various strategies such as (1) meta-analysis to combine disparate information from several studies including Bayesian techniques as in the confidence profile method and (2) other alternatives such as assessing therapeutic results in a single treated population (e.g., astronauts) by sequentially measuring whether the intervention is falling above or below a preestablished probability outcome range and meeting predesigned specifications as opposed to incremental improvement.

Download or read book Statistical Design Monitoring and Analysis of Clinical Trials written by Weichung Joe Shih and published by CRC Press. This book was released on 2021-10-25 with total page 405 pages. Available in PDF, EPUB and Kindle. Book excerpt: Statistical Design, Monitoring, and Analysis of Clinical Trials, Second Edition concentrates on the biostatistics component of clinical trials. This new edition is updated throughout and includes five new chapters. Developed from the authors’ courses taught to public health and medical students, residents, and fellows during the past 20 years, the text shows how biostatistics in clinical trials is an integration of many fundamental scientific principles and statistical methods. 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, phase 2/3 seamless design and trials with predictive biomarkers, exploit multiple testing procedures, and explain the concept of estimand, intercurrent events, and different missing data processes, and describe how to analyze incomplete data by proper multiple imputations. This text reflects the academic research, commercial development, and public health aspects of clinical trials. It gives students and practitioners a multidisciplinary understanding of the concepts and techniques involved in designing, monitoring, and analyzing various types of trials. The book’s balanced set of homework assignments and in-class exercises are appropriate for students and researchers in (bio)statistics, epidemiology, medicine, pharmacy, and public health.

Download or read book Statistical Issues in Drug Development written by Stephen S. Senn and published by John Wiley & Sons. This book was released on 2008-02-28 with total page 523 pages. Available in PDF, EPUB and Kindle. Book excerpt: Drug development is the process of finding and producingtherapeutically useful pharmaceuticals, turning them into safe andeffective medicine, and producing reliable information regardingthe appropriate dosage and dosing intervals. With regulatoryauthorities demanding increasingly higher standards in suchdevelopments, statistics has become an intrinsic and criticalelement in the design and conduct of drug development programmes. Statistical Issues in Drug Development presents anessential and thought provoking guide to the statistical issues andcontroversies involved in drug development. This highly readable second edition has been updated toinclude: Comprehensive coverage of the design and interpretation ofclinical trials. Expanded sections on missing data, equivalence, meta-analysisand dose finding. An examination of both Bayesian and frequentist methods. A new chapter on pharmacogenomics and expanded coverage ofpharmaco-epidemiology and pharmaco-economics. Coverage of the ICH guidelines, in particular ICH E9,Statistical Principles for Clinical Trials. It is hoped that the book will stimulate dialogue betweenstatisticians and life scientists working within the pharmaceuticalindustry. The accessible and wide-ranging coverage make itessential reading for both statisticians and non-statisticiansworking in the pharmaceutical industry, regulatory bodies andmedical research institutes. There is also much to benefitundergraduate and postgraduate students whose courses include amedical statistics component.

Download or read book Introduction to Statistical Methods for Clinical Trials written by Thomas D. Cook and published by CRC Press. This book was released on 2007-11-19 with total page 465 pages. Available in PDF, EPUB and Kindle. Book excerpt: Clinical trials have become essential research tools for evaluating the benefits and risks of new interventions for the treatment and prevention of diseases, from cardiovascular disease to cancer to AIDS. Based on the authors’ collective experiences in this field, Introduction to Statistical Methods for Clinical Trials presents various statistical topics relevant to the design, monitoring, and analysis of a clinical trial. After reviewing the history, ethics, protocol, and regulatory issues of clinical trials, the book provides guidelines for formulating primary and secondary questions and translating clinical questions into statistical ones. It examines designs used in clinical trials, presents methods for determining sample size, and introduces constrained randomization procedures. The authors also discuss how various types of data must be collected to answer key questions in a trial. In addition, they explore common analysis methods, describe statistical methods that determine what an emerging trend represents, and present issues that arise in the analysis of data. The book concludes with suggestions for reporting trial results that are consistent with universal guidelines recommended by medical journals. Developed from a course taught at the University of Wisconsin for the past 25 years, this textbook provides a solid understanding of the statistical approaches used in the design, conduct, and analysis of clinical trials.

Download or read book The Prevention and Treatment of Missing Data in Clinical Trials written by National Research Council and published by National Academies Press. This book was released on 2010-12-21 with total page 163 pages. Available in PDF, EPUB and Kindle. Book excerpt: Randomized clinical trials are the primary tool for evaluating new medical interventions. Randomization provides for a fair comparison between treatment and control groups, balancing out, on average, distributions of known and unknown factors among the participants. Unfortunately, these studies often lack a substantial percentage of data. This missing data reduces the benefit provided by the randomization and introduces potential biases in the comparison of the treatment groups. Missing data can arise for a variety of reasons, including the inability or unwillingness of participants to meet appointments for evaluation. And in some studies, some or all of data collection ceases when participants discontinue study treatment. Existing guidelines for the design and conduct of clinical trials, and the analysis of the resulting data, provide only limited advice on how to handle missing data. Thus, approaches to the analysis of data with an appreciable amount of missing values tend to be ad hoc and variable. The Prevention and Treatment of Missing Data in Clinical Trials concludes that a more principled approach to design and analysis in the presence of missing data is both needed and possible. Such an approach needs to focus on two critical elements: (1) careful design and conduct to limit the amount and impact of missing data and (2) analysis that makes full use of information on all randomized participants and is based on careful attention to the assumptions about the nature of the missing data underlying estimates of treatment effects. In addition to the highest priority recommendations, the book offers more detailed recommendations on the conduct of clinical trials and techniques for analysis of trial data.

Download or read book Strategy and Statistics in Clinical Trials written by Joseph Tal and published by Academic Press. This book was released on 2011-07-14 with total page 279 pages. Available in PDF, EPUB and Kindle. Book excerpt: Delineates the statistical building blocks and concepts of clinical trials.

Download or read book Design and Analysis of Quality of Life Studies in Clinical Trials written by Diane L. Fairclough and published by CRC Press. This book was released on 2002-03-28 with total page 332 pages. Available in PDF, EPUB and Kindle. Book excerpt: More and more frequently, clinical trials include the evaluation of Health-Related Quality of Life (HRQoL), yet many investigators remain unaware of the unique measurement and analysis issues associated with the assessment of HRQoL. At the end of a study, clinicians and statisticians often face challenging and sometimes insurmountable analytic problems. Design and Analysis of Quality of Life Studies in Clinical Trials details these issues and presents a range of solutions. Written from the author's extensive experience in the field, it focuses on the very specific features of QoL data: its longitudinal nature, multidimensionality, and the problem of missing data. The author uses three real clinical trials throughout her discussions to illustrate practical implementation of the strategies and analytic methods presented. As Quality of Life becomes an increasingly important aspect of clinical trials, it becomes essential for clinicians, statisticians, and designers of these studies to understand and meet the challenges this kind of data present. In this book, SAS and S-PLUS programs, checklists, numerous figures, and a clear, concise presentation combine to provide readers with the tools and skills they need to successfully design, conduct, analyze, and report their own studies.

Download or read book Design of Experiments and Advanced Statistical Techniques in Clinical Research written by Basavarajaiah D. M. and published by Springer Nature. This book was released on 2020-11-05 with total page 380 pages. Available in PDF, EPUB and Kindle. Book excerpt: Recent Statistical techniques are one of the basal evidence for clinical research, a pivotal in handling new clinical research and in evaluating and applying prior research. This book explores various choices of statistical tools and mechanisms, analyses of the associations among different clinical attributes. It uses advanced statistical methods to describe real clinical data sets, when the clinical processes being examined are still in the process. This book also discusses distinct methods for building predictive and probability distribution models in clinical situations and ways to assess the stability of these models and other quantitative conclusions drawn by realistic experimental data sets. Design of experiments and recent posthoc tests have been used in comparing treatment effects and precision of the experimentation. This book also facilitates clinicians towards understanding statistics and enabling them to follow and evaluate the real empirical studies (formulation of randomized control trial) that pledge insight evidence base for clinical practices. This book will be a useful resource for clinicians, postgraduates scholars in medicines, clinical research beginners and academicians to nurture high-level statistical tools with extensive scope.

Download or read book Sharing Clinical Trial Data written by Institute of Medicine and published by National Academies Press. This book was released on 2015-04-20 with total page 236 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Download or read book Handbook of Statistics in Clinical Oncology written by John Crowley and published by CRC Press. This book was released on 2005-12-01 with total page 642 pages. Available in PDF, EPUB and Kindle. Book excerpt: A compendium of cutting-edge statistical approaches to solving problems in clinical oncology, Handbook of Statistics in Clinical Oncology, Second Edition focuses on clinical trials in phases I, II, and III, proteomic and genomic studies, complementary outcomes and exploratory methods. Cancer Forum called the first edition a

Download or read book Study Design and Statistical Analysis written by Mitchell Katz and published by Cambridge University Press. This book was released on 2006-06-22 with total page 229 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book takes the reader through the entire research process: choosing a question, designing a study, collecting the data, using univariate, bivariate and multivariable analysis, and publishing the results. It does so by using plain language rather than complex derivations and mathematical formulae. It focuses on the nuts and bolts of performing research by asking and answering the most basic questions about doing research studies. Making good use of numerous tables, graphs and tips, this book helps to demystify the process. A generous number of up-to-date examples from the clinical literature give an illustrated and practical account of how to use multivariable analysis.

Download or read book Statistical Design and Analysis of Stability Studies written by Shein-Chung Chow and published by CRC Press. This book was released on 2007-05-30 with total page 351 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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 sta

Download or read book Statistical Thinking in Clinical Trials written by Michael A. Proschan and published by CRC Press. This book was released on 2021-11-24 with total page 276 pages. Available in PDF, EPUB and Kindle. Book excerpt: Statistical Thinking in Clinical Trials combines a relatively small number of key statistical principles and several instructive clinical trials to gently guide the reader through the statistical thinking needed in clinical trials. Randomization is the cornerstone of clinical trials and randomization-based inference is the cornerstone of this book. Read this book to learn the elegance and simplicity of re-randomization tests as the basis for statistical inference (the analyze as you randomize principle) and see how re-randomization tests can save a trial that required an unplanned, mid-course design change. Other principles enable the reader to quickly and confidently check calculations without relying on computer programs. The `EZ’ principle says that a single sample size formula can be applied to a multitude of statistical tests. The `O minus E except after V’ principle provides a simple estimator of the log odds ratio that is ideally suited for stratified analysis with a binary outcome. The same principle can be used to estimate the log hazard ratio and facilitate stratified analysis in a survival setting. Learn these and other simple techniques that will make you an invaluable clinical trial statistician.

Download or read book Analysis of Clinical Trials Using SAS written by Alex Dmitrienko and published by SAS Institute. This book was released on 2017-07-17 with total page 455 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Download or read book Innovative Strategies Statistical Solutions and Simulations for Modern Clinical Trials written by Mark Chang and published by CRC Press. This book was released on 2019-03-20 with total page 255 pages. Available in PDF, EPUB and Kindle. Book excerpt: "This is truly an outstanding book. [It] brings together all of the latest research in clinical trials methodology and how it can be applied to drug development.... Chang et al provide applications to industry-supported trials. This will allow statisticians in the industry community to take these methods seriously." Jay Herson, Johns Hopkins University The pharmaceutical industry's approach to drug discovery and development has rapidly transformed in the last decade from the more traditional Research and Development (R & D) approach to a more innovative approach in which strategies are employed to compress and optimize the clinical development plan and associated timelines. However, these strategies are generally being considered on an individual trial basis and not as part of a fully integrated overall development program. Such optimization at the trial level is somewhat near-sighted and does not ensure cost, time, or development efficiency of the overall program. This book seeks to address this imbalance by establishing a statistical framework for overall/global clinical development optimization and providing tactics and techniques to support such optimization, including clinical trial simulations. Provides a statistical framework for achieve global optimization in each phase of the drug development process. Describes specific techniques to support optimization including adaptive designs, precision medicine, survival-endpoints, dose finding and multiple testing. Gives practical approaches to handling missing data in clinical trials using SAS. Looks at key controversial issues from both a clinical and statistical perspective. Presents a generous number of case studies from multiple therapeutic areas that help motivate and illustrate the statistical methods introduced in the book. Puts great emphasis on software implementation of the statistical methods with multiple examples of software code (both SAS and R). It is important for statisticians to possess a deep knowledge of the drug development process beyond statistical considerations. For these reasons, this book incorporates both statistical and "clinical/medical" perspectives.

Download or read book Adaptive Design Methods in Clinical Trials written by Shein-Chung Chow and published by CRC Press. This book was released on 2011-12-01 with total page 368 pages. Available in PDF, EPUB and Kindle. Book excerpt: With new statistical and scientific issues arising in adaptive clinical trial design, including the U.S. FDA's recent draft guidance, a new edition of one of the first books on the topic is needed. Adaptive Design Methods in Clinical Trials, Second Edition reflects recent developments and regulatory positions on the use of adaptive designs in clini