Evidence from randomized controlled clinical trials is widely accepted as the only sound basis for assessing the efficacy of new medical treatments. Statistical methods play a key role in all stages of these trials, including their justification, design, and analysis. This second edition of Introduction to Randomized Controlled Clinical Trials prov

Evidence from randomized controlled clinical trials is widely accepted as the only sound basis for assessing the efficacy of new medical treatments. Statistical methods play a key role in all stages of these trials, including their justification, design, and analysis. This second edition of Introduction to Randomized Controlled Clinical Trials provides a concise presentation of the principles applied in this area. It details the concepts behind randomization and methods for designing and analyzing trials and also includes information on meta-analysis and specialized designs, such as cross-over trials, cluster-randomized designs, and equivalence studies. This latest edition features new and revised references, examples, exercises, and a new chapter dedicated to binary outcomes and survival analysis. It also presents numerous examples taken from the medical literature, contains exercises at the end of each chapter, and offers solutions in an appendix. The author uses Minitab and R software throughout the text for implementing the methods that are presented. Comprehensive and accessible, Introduction to Randomized Controlled Clinical Trials is well-suited for those familiar with elementary statistical ideas and methods who want to further their knowledge of the subject.

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.

This is an open access title available under the terms of a CC BY-NC 4.0 International licence. It is free to read at Oxford Scholarship Online and offered as a free PDF download from OUP and selected open access locations. Before new interventions are released into disease control programmes, it is essential that they are carefully evaluated in field trials'. These may be complex and expensive undertakings, requiring the follow-up of hundreds, or thousands, of individuals, often for long periods. Descriptions of the detailed procedures and methods used in the trials that have been conducted have rarely been published. A consequence of this, individuals planning such trials have few guidelines available and little access to knowledge accumulated previously, other than their own. In this manual, practical issues in trial design and conduct are discussed fully and in sufficient detail, that Field Trials of Health Interventions may be used as a toolbox' by field investigators. It has been compiled by an international group of over 30 authors with direct experience in the design, conduct, and analysis of field trials in low and middle income countries and is based on their accumulated knowledge and experience. Available as an open access book via Oxford Medicine Online, this new edition is a comprehensive revision, incorporating the new developments that have taken place in recent years with respect to trials, including seven new chapters on subjects ranging from trial governance, and preliminary studies to pilot testing.

This book provides a practical guide to the analysis of data from randomized controlled trials (RCT). It gives an answer to the question of how to estimate the intervention effect in an appropriate way. This problem is examined for different RCT designs, such as RCTs with one follow-up measurement, RCTs with more than one follow-up measurement, cluster RCTs, cross-over trials, stepped wedge trials, and N-of-1 trials. The statistical methods are explained in a non-mathematical way and are illustrated by extensive examples. All datasets used in the book are available for download, so readers can reanalyse the examples to gain a better understanding of the methods used. Although most examples are taken from epidemiological and clinical studies, this book is also highly recommended for researchers working in other fields.

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.

Principles of Research Design and Drug Literature Evaluation is a unique resource that provides a balanced approach covering critical elements of clinical research, biostatistical principles, and scientific literature evaluation techniques for evidence-based medicine. This accessible text provides comprehensive course content that meets and exceeds the curriculum standards set by the Accreditation Council for Pharmacy Education (ACPE). Written by expert authors specializing in pharmacy practice and research, this valuable text will provide pharmacy students and practitioners with a thorough understanding of the principles and practices of drug literature evaluation with a strong grounding in research and biostatistical principles. Principles of Research Design and Drug Literature Evaluation is an ideal foundation for professional pharmacy students and a key resource for pharmacy residents, research fellows, practitioners, and clinical researchers. FEATURES * Chapter Pedagogy: Learning Objectives, Review Questions, References, and Online Resources * Instructor Resources: PowerPoint Presentations, Test Bank, and an Answer Key * Student Resources: a Navigate Companion Website, including Crossword Puzzles, Interactive Flash Cards, Interactive Glossary, Matching Questions, and Web Links From the Foreword: "This book was designed to provide and encourage practitioner’s development and use of critical drug information evaluation skills through a deeper understanding of the foundational principles of study design and statistical methods. Because guidance on how a study’s limited findings should not be used is rare, practitioners must understand and evaluate for themselves the veracity and implications of the inherently limited primary literature findings they use as sources of drug information to make evidence-based decisions together with their patients. The editors organized the book into three supporting sections to meet their pedagogical goals and address practitioners’ needs in translating research into practice. Thanks to the editors, authors, and content of this book, you can now be more prepared than ever before for translating research into practice." L. Douglas Ried, PhD, FAPhA Editor-in-Chief Emeritus, Journal of the American Pharmacists Association Professor and Associate Dean for Academic Affairs, College of Pharmacy, University of Texas at Tyler, Tyler, Texas