Sample Size Tables for Clinical Studies


Book Description

This book provides statisticians and researchers with the statistical tools - equations, formulae and numerical tables - to design and plan clinical studies and carry out accurate, reliable and reproducible analysis of the data so obtained. There is no way around this as incorrect procedure in clinical studies means that the researcher's paper will not be accepted by a peer-reviewed journal. Planning and analysing clinical studies is a very complicated business and this book provides indispensible factual information. Please go to http://booksupport.wiley.com and enter 9781405146500 to easily download the supporting materials.




Sample Size Calculations in Clinical Research


Book Description

Praise for the Second Edition: "... this is a useful, comprehensive compendium of almost every possible sample size formula. The strong organization and carefully defined formulae will aid any researcher designing a study." -Biometrics "This impressive book contains formulae for computing sample size in a wide range of settings. One-sample studies and two-sample comparisons for quantitative, binary, and time-to-event outcomes are covered comprehensively, with separate sample size formulae for testing equality, non-inferiority, and equivalence. Many less familiar topics are also covered ..." – Journal of the Royal Statistical Society Sample Size Calculations in Clinical Research, Third Edition presents statistical procedures for performing sample size calculations during various phases of clinical research and development. A comprehensive and unified presentation of statistical concepts and practical applications, this book includes a well-balanced summary of current and emerging clinical issues, regulatory requirements, and recently developed statistical methodologies for sample size calculation. Features: Compares the relative merits and disadvantages of statistical methods for sample size calculations Explains how the formulae and procedures for sample size calculations can be used in a variety of clinical research and development stages Presents real-world examples from several therapeutic areas, including cardiovascular medicine, the central nervous system, anti-infective medicine, oncology, and women’s health Provides sample size calculations for dose response studies, microarray studies, and Bayesian approaches This new edition is updated throughout, includes many new sections, and five new chapters on emerging topics: two stage seamless adaptive designs, cluster randomized trial design, zero-inflated Poisson distribution, clinical trials with extremely low incidence rates, and clinical trial simulation.




Sample Sizes for Clinical Trials


Book Description

Drawing on various real-world applications, Sample Sizes for Clinical Trials takes readers through the process of calculating sample sizes for many types of clinical trials. It provides descriptions of the calculations with a practical emphasis.Focusing on normal, binary, ordinal, and survival data, the book explores a range of trials, including su







Fundamentals of Clinical Trials


Book Description

This classic reference, now updated with the newest applications and results, addresses the fundamentals of such trials based on sound scientific methodology, statistical principles, and years of accumulated experience by the three authors.




Sharing Clinical Trial Data


Book Description

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.




Statistical Rules of Thumb


Book Description

Praise for the First Edition: "For a beginner [this book] is a treasure trove; for an experienced person it can provide new ideas on how better to pursue the subject of applied statistics." —Journal of Quality Technology Sensibly organized for quick reference, Statistical Rules of Thumb, Second Edition compiles simple rules that are widely applicable, robust, and elegant, and each captures key statistical concepts. This unique guide to the use of statistics for designing, conducting, and analyzing research studies illustrates real-world statistical applications through examples from fields such as public health and environmental studies. Along with an insightful discussion of the reasoning behind every technique, this easy-to-use handbook also conveys the various possibilities statisticians must think of when designing and conducting a study or analyzing its data. Each chapter presents clearly defined rules related to inference, covariation, experimental design, consultation, and data representation, and each rule is organized and discussed under five succinct headings: introduction; statement and illustration of the rule; the derivation of the rule; a concluding discussion; and exploration of the concept's extensions. The author also introduces new rules of thumb for topics such as sample size for ratio analysis, absolute and relative risk, ANCOVA cautions, and dichotomization of continuous variables. Additional features of the Second Edition include: Additional rules on Bayesian topics New chapters on observational studies and Evidence-Based Medicine (EBM) Additional emphasis on variation and causation Updated material with new references, examples, and sources A related Web site provides a rich learning environment and contains additional rules, presentations by the author, and a message board where readers can share their own strategies and discoveries. Statistical Rules of Thumb, Second Edition is an ideal supplementary book for courses in experimental design and survey research methods at the upper-undergraduate and graduate levels. It also serves as an indispensable reference for statisticians, researchers, consultants, and scientists who would like to develop an understanding of the statistical foundations of their research efforts. A related website www.vanbelle.org provides additional rules, author presentations and more.




Statistical Power Analysis for the Behavioral Sciences


Book Description

Statistical Power Analysis is a nontechnical guide to power analysis in research planning that provides users of applied statistics with the tools they need for more effective analysis. The Second Edition includes: * a chapter covering power analysis in set correlation and multivariate methods; * a chapter considering effect size, psychometric reliability, and the efficacy of "qualifying" dependent variables and; * expanded power and sample size tables for multiple regression/correlation.




Small Clinical Trials


Book Description

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.




Statistics with Confidence


Book Description

This highly popular introduction to confidence intervals has been thoroughly updated and expanded. It includes methods for using confidence intervals, with illustrative worked examples and extensive guidelines and checklists to help the novice.