Download or read book Randomization Based Inference for Treatment Modifying Factors in Randomized Trials with Noncompliance written by Pasi Korhonen and published by . This book was released on 1998 with total page 27 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Download or read book Randomization in Clinical Trials written by William F. Rosenberger and published by John Wiley & Sons. This book was released on 2004-03-24 with total page 286 pages. Available in PDF, EPUB and Kindle. Book excerpt: A unique overview that melds the concepts of conditionalprobability and stochastic processes into real-lifeapplications The role of randomization techniques in clinical trials has becomeincreasingly important. This comprehensive guide combines both theapplied aspects of randomization in clinical trials with aprobabilistic treatment of properties of randomization. Taking anunabashedly non-Bayesian and nonparametric approach to inference,the book focuses on the linear rank test under a randomizationmodel, with added discussion on likelihood-based inference as itrelates to sufficiency and ancillarity. Developments in stochasticprocesses and applied probability are also given where appropriate.Intuition is stressed over mathematics, but not without a cleardevelopment of the latter in the context of the former. Providing a consolidated review of the field, the book includesrelevant and practical discussions of: * The benefits of randomization in terms of reduction of bias * Randomization as a basis for inference * Covariate-adaptive and response-adaptive randomization * Current philosophies, controversies, and new developments With ample problem sets, theoretical exercises, and short computersimulations using SAS, Randomization in Clinical Trials: Theory andPractice is equally useful as a standard textbook in biostatisticsgraduate programs as well as a reliable reference forbiostatisticians in practice.
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 Clinical Trials in Neurology written by Bernard Ravina and published by Cambridge University Press. This book was released on 2012-04-12 with total page 387 pages. Available in PDF, EPUB and Kindle. Book excerpt: Translating laboratory discoveries into successful therapeutics can be difficult. Clinical Trials in Neurology aims to improve the efficiency of clinical trials and the development of interventions in order to enhance the development of new treatments for neurologic diseases. It introduces the reader to the key concepts underpinning trials in the neurosciences. This volume tackles the challenges of developing therapies for neurologic disorders from measurement of agents in the nervous system to the progression of clinical signs and symptoms through illustrating specific study designs and their applications to different therapeutic areas. Clinical Trials in Neurology covers key issues in Phase I, II and III clinical trials, as well as post-marketing safety surveillance. Topics addressed include regulatory and implementation issues, outcome measures and common problems in drug development. Written by a multidisciplinary team, this comprehensive guide is essential reading for neurologists, psychiatrists, neurosurgeons, neuroscientists, statisticians and clinical researchers in the pharmaceutical industry.
Download or read book Principles and Practice of Clinical Trials written by Steven Piantadosi and published by Springer Nature. This book was released on 2022-07-19 with total page 2573 pages. Available in PDF, EPUB and Kindle. Book excerpt: This is a comprehensive major reference work for our SpringerReference program covering clinical trials. Although the core of the Work will focus on the design, analysis, and interpretation of scientific data from clinical trials, a broad spectrum of clinical trial application areas will be covered in detail. This is an important time to develop such a Work, as drug safety and efficacy emphasizes the Clinical Trials process. Because of an immense and growing international disease burden, pharmaceutical and biotechnology companies continue to develop new drugs. Clinical trials have also become extremely globalized in the past 15 years, with over 225,000 international trials ongoing at this point in time. Principles in Practice of Clinical Trials is truly an interdisciplinary that will be divided into the following areas: 1) Clinical Trials Basic Perspectives 2) Regulation and Oversight 3) Basic Trial Designs 4) Advanced Trial Designs 5) Analysis 6) Trial Publication 7) Topics Related Specific Populations and Legal Aspects of Clinical Trials The Work is designed to be comprised of 175 chapters and approximately 2500 pages. The Work will be oriented like many of our SpringerReference Handbooks, presenting detailed and comprehensive expository chapters on broad subjects. The Editors are major figures in the field of clinical trials, and both have written textbooks on the topic. There will also be a slate of 7-8 renowned associate editors that will edit individual sections of the Reference.
Download or read book Statistical Models in Epidemiology the Environment and Clinical Trials written by M.Elizabeth Halloran and published by Springer Science & Business Media. This book was released on 1999-10-29 with total page 300 pages. Available in PDF, EPUB and Kindle. Book excerpt: This IMA Volume in Mathematics and its Applications STATISTICAL MODELS IN EPIDEMIOLOGY, THE ENVIRONMENT,AND CLINICAL TRIALS is a combined proceedings on "Design and Analysis of Clinical Trials" and "Statistics and Epidemiology: Environment and Health. " This volume is the third series based on the proceedings of a very successful 1997 IMA Summer Program on "Statistics in the Health Sciences. " I would like to thank the organizers: M. Elizabeth Halloran of Emory University (Biostatistics) and Donald A. Berry of Duke University (Insti tute of Statistics and Decision Sciences and Cancer Center Biostatistics) for their excellent work as organizers of the meeting and for editing the proceedings. I am grateful to Seymour Geisser of University of Minnesota (Statistics), Patricia Grambsch, University of Minnesota (Biostatistics); Joel Greenhouse, Carnegie Mellon University (Statistics); Nicholas Lange, Harvard Medical School (Brain Imaging Center, McLean Hospital); Barry Margolin, University of North Carolina-Chapel Hill (Biostatistics); Sandy Weisberg, University of Minnesota (Statistics); Scott Zeger, Johns Hop kins University (Biostatistics); and Marvin Zelen, Harvard School of Public Health (Biostatistics) for organizing the six weeks summer program. I also take this opportunity to thank the National Science Foundation (NSF) and the Army Research Office (ARO), whose financial support made the workshop possible. Willard Miller, Jr.
Download or read book Evaluating AIDS Prevention Programs written by National Research Council and published by National Academies Press. This book was released on 1991-02-01 with total page 391 pages. Available in PDF, EPUB and Kindle. Book excerpt: With insightful discussion of program evaluation and the efforts of the Centers for Disease Control, this book presents a set of clear-cut recommendations to help ensure that the substantial resources devoted to the fight against AIDS will be used most effectively. This expanded edition of Evaluating AIDS Prevention Programs covers evaluation strategies and outcome measurements, including a realistic review of the factors that make evaluation of AIDS programs particularly difficult. Randomized field experiments are examined, focusing on the use of alternative treatments rather than placebo controls. The book also reviews nonexperimental techniques, including a critical examination of evaluation methods that are observational rather than experimentalâ€"a necessity when randomized experiments are infeasible.
Download or read book Causal Inference in Statistics Social and Biomedical Sciences written by Guido W. Imbens and published by Cambridge University Press. This book was released on 2015-04-06 with total page 647 pages. Available in PDF, EPUB and Kindle. Book excerpt: This text presents statistical methods for studying causal effects and discusses how readers can assess such effects in simple randomized experiments.
Download or read book Binary Data Analysis of Randomized Clinical Trials with Noncompliance written by Kung-Jong Lui and published by John Wiley & Sons. This book was released on 2011-03-31 with total page 217 pages. Available in PDF, EPUB and Kindle. Book excerpt: It is quite common in a randomized clinical trial (RCT) to encounter patients who do not comply with their assigned treatment. Since noncompliance often occurs non-randomly, the commonly-used approaches, including both the as-treated (AT) and as-protocol (AP) analysis, and the intent-to-treat (ITT) (or as-randomized) analysis, are all well known to possibly produce a biased inference of the treatment efficacy. This book provides a systematic and organized approach to analyzing data for RCTs with noncompliance under the most frequently-encountered situations. These include parallel sampling, stratified sampling, cluster sampling, parallel sampling with subsequent missing outcomes, and a series of dependent Bernoulli sampling for repeated measurements. The author provides a comprehensive approach by using contingency tables to illustrate the latent probability structure of observed data. Using real-life examples, computer-simulated data and exercises in each chapter, the book illustrates the underlying theory in an accessible, and easy to understand way. Key features: Consort-flow diagrams and numerical examples are used to illustrate the bias of commonly used approaches, such as, AT analysis, AP analysis and ITT analysis for a RCT with noncompliance. Real-life examples are used throughout the book to explain the practical usefulness of test procedures and estimators. Each chapter is self-contained, allowing the book to be used as a reference source. Includes SAS programs which can be easily modified in calculating the required sample size. Biostatisticians, clinicians, researchers and data analysts working in pharmaceutical industries will benefit from this book. This text can also be used as supplemental material for a course focusing on clinical statistics or experimental trials in epidemiology, psychology and sociology.
Download or read book Developing a Protocol for Observational Comparative Effectiveness Research A User s Guide written by Agency for Health Care Research and Quality (U.S.) and published by Government Printing Office. This book was released on 2013-02-21 with total page 236 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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)
Download or read book Registries for Evaluating Patient Outcomes written by Agency for Healthcare Research and Quality/AHRQ and published by Government Printing Office. This book was released on 2014-04-01 with total page 385 pages. Available in PDF, EPUB and Kindle. Book excerpt: This User’s Guide is intended to support the design, implementation, analysis, interpretation, and quality evaluation of registries created to increase understanding of patient outcomes. For the purposes of this guide, a patient registry is an organized system that uses observational study methods to collect uniform data (clinical and other) to evaluate specified outcomes for a population defined by a particular disease, condition, or exposure, and that serves one or more predetermined scientific, clinical, or policy purposes. A registry database is a file (or files) derived from the registry. Although registries can serve many purposes, this guide focuses on registries created for one or more of the following purposes: to describe the natural history of disease, to determine clinical effectiveness or cost-effectiveness of health care products and services, to measure or monitor safety and harm, and/or to measure quality of care. Registries are classified according to how their populations are defined. For example, product registries include patients who have been exposed to biopharmaceutical products or medical devices. Health services registries consist of patients who have had a common procedure, clinical encounter, or hospitalization. Disease or condition registries are defined by patients having the same diagnosis, such as cystic fibrosis or heart failure. 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.
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 Targeted Learning written by Mark J. van der Laan and published by Springer Science & Business Media. This book was released on 2011-06-17 with total page 628 pages. Available in PDF, EPUB and Kindle. Book excerpt: The statistics profession is at a unique point in history. The need for valid statistical tools is greater than ever; data sets are massive, often measuring hundreds of thousands of measurements for a single subject. The field is ready to move towards clear objective benchmarks under which tools can be evaluated. Targeted learning allows (1) the full generalization and utilization of cross-validation as an estimator selection tool so that the subjective choices made by humans are now made by the machine, and (2) targeting the fitting of the probability distribution of the data toward the target parameter representing the scientific question of interest. This book is aimed at both statisticians and applied researchers interested in causal inference and general effect estimation for observational and experimental data. Part I is an accessible introduction to super learning and the targeted maximum likelihood estimator, including related concepts necessary to understand and apply these methods. Parts II-IX handle complex data structures and topics applied researchers will immediately recognize from their own research, including time-to-event outcomes, direct and indirect effects, positivity violations, case-control studies, censored data, longitudinal data, and genomic studies.
Download or read book Handbook of Economic Field Experiments written by Abhijit Vinayak Banerjee and published by . This book was released on 2017 with total page 0 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Download or read book Experimental and Quasi experimental Designs for Generalized Causal Inference written by William R. Shadish and published by Cengage Learning. This book was released on 2002 with total page 664 pages. Available in PDF, EPUB and Kindle. Book excerpt: Sections include: experiments and generalised causal inference; statistical conclusion validity and internal validity; construct validity and external validity; quasi-experimental designs that either lack a control group or lack pretest observations on the outcome; quasi-experimental designs that use both control groups and pretests; quasi-experiments: interrupted time-series designs; regresssion discontinuity designs; randomised experiments: rationale, designs, and conditions conducive to doing them; practical problems 1: ethics, participation recruitment and random assignment; practical problems 2: treatment implementation and attrition; generalised causal inference: a grounded theory; generalised causal inference: methods for single studies; generalised causal inference: methods for multiple studies; a critical assessment of our assumptions.
Download or read book Advances in Clinical Trial Biostatistics written by Nancy L. Geller and published by CRC Press. This book was released on 2003-10-21 with total page 308 pages. Available in PDF, EPUB and Kindle. Book excerpt: From aspects of early trials to complex modeling problems, Advances in Clinical Trial Biostatistics summarizes current methodologies used in the design and analysis of clinical trials. Its chapters, contributed by internationally renowned methodologists experienced in clinical trials, address topics that include Bayesian methods for phase I clinical trials, adaptive two-stage clinical trials, and the design and analysis of cluster randomization trials, trials with multiple endpoints, and therapeutic equivalence trials. Other discussions explore Bayesian reporting, methods incorporating compliance in treatment evaluation, and statistical issues emerging from clinical trials in HIV infection.
Download or read book A Practical Introduction to Regression Discontinuity Designs written by Matias D. Cattaneo and published by Cambridge University Press. This book was released on 2020-02-13 with total page 114 pages. Available in PDF, EPUB and Kindle. Book excerpt: In this Element and its accompanying second Element, A Practical Introduction to Regression Discontinuity Designs: Extensions, Matias Cattaneo, Nicolás Idrobo, and Rocıìo Titiunik provide an accessible and practical guide for the analysis and interpretation of regression discontinuity (RD) designs that encourages the use of a common set of practices and facilitates the accumulation of RD-based empirical evidence. In this Element, the authors discuss the foundations of the canonical Sharp RD design, which has the following features: (i) the score is continuously distributed and has only one dimension, (ii) there is only one cutoff, and (iii) compliance with the treatment assignment is perfect. In the second Element, the authors discuss practical and conceptual extensions to this basic RD setup.
