A fundamental statistical framework for the analysis of complex longitudinal data is provided in this book. It provides the first comprehensive description of optimal estimation techniques based on time-dependent data structures. The techniques go beyond standard statistical approaches and can be used to teach masters and Ph.D. students. The text is ideally suitable for researchers in statistics with a strong interest in the analysis of complex longitudinal data.
An observational study infers the effects caused by a treatment, policy, program, intervention, or exposure in a context in which randomized experimentation is unethical or impractical. One task in an observational study is to adjust for visible pretreatment differences between the treated and control groups. Multivariate matching and weighting are two modern forms of adjustment. This handbook provides a comprehensive survey of the most recent methods of adjustment by matching, weighting, machine learning and their combinations. Three additional chapters introduce the steps from association to causation that follow after adjustments are complete. When used alone, matching and weighting do not use outcome information, so they are part of the design of an observational study. When used in conjunction with models for the outcome, matching and weighting may enhance the robustness of model-based adjustments. The book is for researchers in medicine, economics, public health, psychology, epidemiology, public program evaluation, and statistics who examine evidence of the effects on human beings of treatments, policies or exposures.
This book summarizes current knowledge regarding the theory of estimation for semiparametric models with missing data, in an organized and comprehensive manner. It starts with the study of semiparametric methods when there are no missing data. The description of the theory of estimation for semiparametric models is both rigorous and intuitive, relying on geometric ideas to reinforce the intuition and understanding of the theory. These methods are then applied to problems with missing, censored, and coarsened data with the goal of deriving estimators that are as robust and efficient as possible.
This second edition of Design of Observational Studies is both an introduction to statistical inference in observational studies and a detailed discussion of the principles that guide the design of observational studies. An observational study is an empiric investigation of effects caused by treatments when randomized experimentation is unethical or infeasible. Observational studies are common in most fields that study the effects of treatments on people, including medicine, economics, epidemiology, education, psychology, political science and sociology. The quality and strength of evidence provided by an observational study is determined largely by its design. Design of Observational Studies is organized into five parts. Chapters 2, 3, and 5 of Part I cover concisely many of the ideas discussed in Rosenbaum’s Observational Studies (also published by Springer) but in a less technical fashion. Part II discusses the practical aspects of using propensity scores and other tools to create a matched comparison that balances many covariates, and includes an updated chapter on matching in R. In Part III, the concept of design sensitivity is used to appraise the relative ability of competing designs to distinguish treatment effects from biases due to unmeasured covariates. Part IV is new to this edition; it discusses evidence factors and the computerized construction of more than one comparison group. Part V discusses planning the analysis of an observational study, with particular reference to Sir Ronald Fisher’s striking advice for observational studies: "make your theories elaborate." This new edition features updated exploration of causal influence, with four new chapters, a new R package DOS2 designed as a companion for the book, and discussion of several of the latest matching packages for R. In particular, DOS2 allows readers to reproduce many analyses from Design of Observational Studies.
This edition contains a large number of additions and corrections scattered throughout the text, including the incorporation of a new chapter on state-space models. The companion diskette for the IBM PC has expanded into the software package ITSM: An Interactive Time Series Modelling Package for the PC, which includes a manual and can be ordered from Springer-Verlag. * We are indebted to many readers who have used the book and programs and made suggestions for improvements. Unfortunately there is not enough space to acknowledge all who have contributed in this way; however, special mention must be made of our prize-winning fault-finders, Sid Resnick and F. Pukelsheim. Special mention should also be made of Anthony Brockwell, whose advice and support on computing matters was invaluable in the preparation of the new diskettes. We have been fortunate to work on the new edition in the excellent environments provided by the University of Melbourne and Colorado State University. We thank Duane Boes particularly for his support and encouragement throughout, and the Australian Research Council and National Science Foundation for their support of research related to the new material. We are also indebted to Springer-Verlag for their constant support and assistance in preparing the second edition. Fort Collins, Colorado P. J. BROCKWELL November, 1990 R. A. DAVIS * /TSM: An Interactive Time Series Modelling Package for the PC by P. J. Brockwell and R. A. Davis. ISBN: 0-387-97482-2; 1991.
This book covers recent developments in correlated data analysis. It utilizes the class of dispersion models as marginal components in the formulation of joint models for correlated data. This enables the book to cover a broader range of data types than the traditional generalized linear models. The reader is provided with a systematic treatment for the topic of estimating functions, and both generalized estimating equations (GEE) and quadratic inference functions (QIF) are studied as special cases. In addition to the discussions on marginal models and mixed-effects models, this book covers new topics on joint regression analysis based on Gaussian copulas.
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.
Comparative effectiveness research (CER) is the generation and synthesis of evidence that compares the benefits and harms of alternative methods to prevent, diagnose, treat, and monitor a clinical condition or to improve the delivery of care (IOM 2009). CER is conducted to develop evidence that will aid patients, clinicians, purchasers, and health policy makers in making informed decisions at both the individual and population levels. CER encompasses a very broad range of types of studies—experimental, observational, prospective, retrospective, and research synthesis. This volume covers the main areas of quantitative methodology for the design and analysis of CER studies. The volume has four major sections—causal inference; clinical trials; research synthesis; and specialized topics. The audience includes CER methodologists, quantitative-trained researchers interested in CER, and graduate students in statistics, epidemiology, and health services and outcomes research. The book assumes a masters-level course in regression analysis and familiarity with clinical research.
The last twenty years have witnessed a significant growth of interest in optimal factorial designs, under possible model uncertainty, via the minimum aberration and related criteria. This book gives, for the first time in book form, a comprehensive and up-to-date account of this modern theory. Many major classes of designs are covered in the book. While maintaining a high level of mathematical rigor, it also provides extensive design tables for research and practical purposes. Apart from being useful to researchers and practitioners, the book can form the core of a graduate level course in experimental design.
Over the last few decades, important progresses in the methods of sampling have been achieved. This book draws up an inventory of new methods that can be useful for selecting samples. Forty-six sampling methods are described in the framework of general theory. The algorithms are described rigorously, which allows implementing directly the described methods. This book is aimed at experienced statisticians who are familiar with the theory of survey sampling.
