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Author: National Research Council
Publisher: National Academies Press
ISBN: 030918651X
Category : Medical
Languages : en
Pages : 163
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Book Description
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.
Author: National Research Council
Publisher: National Academies Press
ISBN: 030918651X
Category : Medical
Languages : en
Pages : 163
Get Book Here
Book Description
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.
Author: Deborah G. Mayo
Publisher: Cambridge University Press
ISBN: 1108563309
Category : Mathematics
Languages : en
Pages : 503
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Book Description
Mounting failures of replication in social and biological sciences give a new urgency to critically appraising proposed reforms. This book pulls back the cover on disagreements between experts charged with restoring integrity to science. It denies two pervasive views of the role of probability in inference: to assign degrees of belief, and to control error rates in a long run. If statistical consumers are unaware of assumptions behind rival evidence reforms, they can't scrutinize the consequences that affect them (in personalized medicine, psychology, etc.). The book sets sail with a simple tool: if little has been done to rule out flaws in inferring a claim, then it has not passed a severe test. Many methods advocated by data experts do not stand up to severe scrutiny and are in tension with successful strategies for blocking or accounting for cherry picking and selective reporting. Through a series of excursions and exhibits, the philosophy and history of inductive inference come alive. Philosophical tools are put to work to solve problems about science and pseudoscience, induction and falsification.
Author: Judea Pearl
Publisher: John Wiley & Sons
ISBN: 1119186862
Category : Mathematics
Languages : en
Pages : 162
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Book Description
CAUSAL INFERENCE IN STATISTICS A Primer Causality is central to the understanding and use of data. Without an understanding of cause–effect relationships, we cannot use data to answer questions as basic as "Does this treatment harm or help patients?" But though hundreds of introductory texts are available on statistical methods of data analysis, until now, no beginner-level book has been written about the exploding arsenal of methods that can tease causal information from data. Causal Inference in Statistics fills that gap. Using simple examples and plain language, the book lays out how to define causal parameters; the assumptions necessary to estimate causal parameters in a variety of situations; how to express those assumptions mathematically; whether those assumptions have testable implications; how to predict the effects of interventions; and how to reason counterfactually. These are the foundational tools that any student of statistics needs to acquire in order to use statistical methods to answer causal questions of interest. This book is accessible to anyone with an interest in interpreting data, from undergraduates, professors, researchers, or to the interested layperson. Examples are drawn from a wide variety of fields, including medicine, public policy, and law; a brief introduction to probability and statistics is provided for the uninitiated; and each chapter comes with study questions to reinforce the readers understanding.
Author: Judea Pearl
Publisher: Createspace Independent Publishing Platform
ISBN: 9781507894293
Category : Causation
Languages : en
Pages : 0
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Book Description
This paper summarizes recent advances in causal inference and underscores the paradigmatic shifts that must be undertaken in moving from traditional statistical analysis to causal analysis of multivariate data. Special emphasis is placed on the assumptions that underly all causal inferences, the languages used in formulating those assumptions, the conditional nature of all causal and counterfactual claims, and the methods that have been developed for the assessment of such claims. These advances are illustrated using a general theory of causation based on the Structural Causal Model (SCM) described in Pearl (2000a), which subsumes and unifies other approaches to causation, and provides a coherent mathematical foundation for the analysis of causes and counterfactuals. In particular, the paper surveys the development of mathematical tools for inferring (from a combination of data and assumptions) answers to three types of causal queries: (1) queries about the effects of potential interventions, (also called "causal effects" or "policy evaluation") (2) queries about probabilities of counterfactuals, (including assessment of "regret," "attribution" or "causes of effects") and (3) queries about direct and indirect effects (also known as "mediation"). Finally, the paper defines the formal and conceptual relationships between the structural and potential-outcome frameworks and presents tools for a symbiotic analysis that uses the strong features of both. The tools are demonstrated in the analyses of mediation, causes of effects, and probabilities of causation. -- p. 1.
Author: D. R. Cox
Publisher: Cambridge University Press
ISBN: 1139459139
Category : Mathematics
Languages : en
Pages : 227
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Book Description
In this definitive book, D. R. Cox gives a comprehensive and balanced appraisal of statistical inference. He develops the key concepts, describing and comparing the main ideas and controversies over foundational issues that have been keenly argued for more than two-hundred years. Continuing a sixty-year career of major contributions to statistical thought, no one is better placed to give this much-needed account of the field. An appendix gives a more personal assessment of the merits of different ideas. The content ranges from the traditional to the contemporary. While specific applications are not treated, the book is strongly motivated by applications across the sciences and associated technologies. The mathematics is kept as elementary as feasible, though previous knowledge of statistics is assumed. The book will be valued by every user or student of statistics who is serious about understanding the uncertainty inherent in conclusions from statistical analyses.
Author: Mark J. van der Laan
Publisher: Springer Science & Business Media
ISBN: 1441997822
Category : Mathematics
Languages : en
Pages : 628
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Book Description
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.
Author: Bradley Efron
Publisher: Cambridge University Press
ISBN: 1108107958
Category : Mathematics
Languages : en
Pages : 496
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Book Description
The twenty-first century has seen a breathtaking expansion of statistical methodology, both in scope and in influence. 'Big data', 'data science', and 'machine learning' have become familiar terms in the news, as statistical methods are brought to bear upon the enormous data sets of modern science and commerce. How did we get here? And where are we going? This book takes us on an exhilarating journey through the revolution in data analysis following the introduction of electronic computation in the 1950s. Beginning with classical inferential theories - Bayesian, frequentist, Fisherian - individual chapters take up a series of influential topics: survival analysis, logistic regression, empirical Bayes, the jackknife and bootstrap, random forests, neural networks, Markov chain Monte Carlo, inference after model selection, and dozens more. The distinctly modern approach integrates methodology and algorithms with statistical inference. The book ends with speculation on the future direction of statistics and data science.
Author: Guido W. Imbens
Publisher: Cambridge University Press
ISBN: 0521885884
Category : Business & Economics
Languages : en
Pages : 647
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Book Description
This text presents statistical methods for studying causal effects and discusses how readers can assess such effects in simple randomized experiments.
Author: University of Michigan. College of Literature, Science, and the Arts
Publisher: UM Libraries
ISBN:
Category :
Languages : en
Pages : 316
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Book Description
Author: Geert Molenberghs
Publisher: CRC Press
ISBN: 1439854610
Category : Mathematics
Languages : en
Pages : 600
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Book Description
Missing data affect nearly every discipline by complicating the statistical analysis of collected data. But since the 1990s, there have been important developments in the statistical methodology for handling missing data. Written by renowned statisticians in this area, Handbook of Missing Data Methodology presents many methodological advances and the latest applications of missing data methods in empirical research. Divided into six parts, the handbook begins by establishing notation and terminology. It reviews the general taxonomy of missing data mechanisms and their implications for analysis and offers a historical perspective on early methods for handling missing data. The following three parts cover various inference paradigms when data are missing, including likelihood and Bayesian methods; semi-parametric methods, with particular emphasis on inverse probability weighting; and multiple imputation methods. The next part of the book focuses on a range of approaches that assess the sensitivity of inferences to alternative, routinely non-verifiable assumptions about the missing data process. The final part discusses special topics, such as missing data in clinical trials and sample surveys as well as approaches to model diagnostics in the missing data setting. In each part, an introduction provides useful background material and an overview to set the stage for subsequent chapters. Covering both established and emerging methodologies for missing data, this book sets the scene for future research. It provides the framework for readers to delve into research and practical applications of missing data methods.
