Sample Sizes For Clinical Laboratory And Epidemiology Studies

An authoritative resource that offers the statistical tools and software needed to design and plan valid clinical studies Now in its fourth and extended edition, Sample Sizes for Clinical, Laboratory and Epidemiology Studiesincludes the sample size software (SSS) and formulae and numerical tables needed to design valid clinical studies. The text covers clinical as well as laboratory and epidemiology studies and contains the information needed to ensure a study will form a valid contribution to medical research. The authors, noted experts in the field, explain step by step and explore the wide range of considerations necessary to assist investigational teams when deriving an appropriate sample size for their when planned study. The book contains sets of sample size tables with companion explanations and clear worked out examples based on real data. In addition, the text offers bibliography and references sections that are designed to be helpful with guidance on the principles discussed. This revised fourth edition: Offers the only text available to include sample size software for use in designing and planning clinical studies Presents new and extended chapters with many additional and refreshed examples Includes clear explanations of the principles and methodologies involved with relevant practical examples Makes clear a complex but vital topic that is designed to ensure valid methodology and publishable results Contains guidance from an internationally recognised team of medical statistics experts Written for medical researchers from all specialities and medical statisticians, Sample Sizes for Clinical, Laboratory and EpidemiologyStudies offers an updated fourth edition of the important guide for designing and planning reliable and evidence based clinical studies.

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.

A Guide to Sample Size for Animal-based Studies Understand a foundational area of experimental design with this innovative reference Animal-based research is an essential part of basic and preclinical research, but poses a unique set of experimental design challenges. The most important of these are the 3Rs − Replacement, Reduction and Refinement − the principles comprising the ethical framework for humane animal-based studies. However, many researchers have difficulty navigating the design trade-offs necessary to simultaneously minimize animal use, and produce scientific information that is both rigorous and reliable. A Guide to Sample Size for Animal-based Studies meets this need with a thorough, accessible reference work to the subject. This book provides a straightforward systematic approach to “rightsizing” animal-based experiments, with sample size estimates based on the fundamentals of statistical thinking: structured research questions, variation control and appropriate design of experiments. The result is a much-needed guide to planning animal-based experiments to ensure scientifically valid and reliable results. This book offers: Step-by-step guidance in diverse methods for approximating and refining sample size Detailed treatment of research topics specific to animal-based research, including pilot, feasibility and proof-of-concept studies Sample size approximation methods for different types of data − binary, continuous, ordinal, time to event − and different study types − description, comparison, nested designs, reference interval construction and dose-response studies Numerous worked examples, using real data from published papers, together with SAS and R code A Guide to Sample Size for Animal-based Studies is a must-have reference for preclinical and veterinary researchers, as well as ethical oversight committees and policymakers.

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.

This User’s Guide is a resource for investigators and stakeholders who develop and review observational comparative effectiveness research protocols. It explains how to (1) identify key considerations and best practices for research design; (2) build a protocol based on these standards and best practices; and (3) judge the adequacy and completeness of a protocol. Eleven chapters cover all aspects of research design, including: developing study objectives, defining and refining study questions, addressing the heterogeneity of treatment effect, characterizing exposure, selecting a comparator, defining and measuring outcomes, and identifying optimal data sources. Checklists of guidance and key considerations for protocols are provided at the end of each chapter. The User’s Guide was created by researchers affiliated with AHRQ’s Effective Health Care Program, particularly those who participated in AHRQ’s DEcIDE (Developing Evidence to Inform Decisions About Effectiveness) program. Chapters were subject to multiple internal and external independent reviews. More more information, please consult the Agency website: www.effectivehealthcare.ahrq.gov)

First edition published in 2002. Second edition published in 2008.

Randomised Clinical Trials: Design, Practice and Reporting provides a detailed overview of the methodology for conducting clinical trials, including developing protocols, data capture, randomisation, analysis and reporting. Assuming no prior background, this user-friendly resource describes the statistical, regulatory, and practical components required for conducting randomised clinical trials. Numerous examples and case studies from industry, academia, and the research literature help readers understand each stage of the clinical trial process. This second edition contains extensively revised material throughout, including new chapters covering designs for repeated measures, non-inferiority, cluster and stepped wedge trials. Other new chapters describe data and safety monitoring, biomarker studies, and feasibility studies. Updated and expanded sections discuss situations where multiple organs, different body locations or competing risks are involved, subgroup analysis, and multiple outcomes. Written by an author team with extensive experience in conducting clinical trials, this book: Provides comprehensive coverage of randomised clinical trials, ranging from basic to advanced Features several new chapters, updated case studies and examples, and references to changes in regulations Explains basic randomised trials, including the parallel two-group controlled trial with a single outcome measure Covers paired trial designs and trials with more than two interventions Includes a chapter on miscellaneous topics such as adaptive designs, large simple trials, Bayesian methods for very small trials, alpha-spending functions and the predictive probability test Randomised Clinical Trials is essential reading for clinicians, nurses, data managers, and medical statisticians involved in clinical trials, and for health practitioners responsible for direct patient care in a clinical trial setting.