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 Assessing Treatment Effect Heterogeneity for Binary Outcomes written by Edward Joseph Mascha and published by . This book was released on 2005 with total page 360 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Download or read book Examining the Foundations of Methods That Assess Treatment Effect Heterogeneity Across Intermediate Outcomes written by Avi Feller and published by . This book was released on 2015 with total page 7 pages. Available in PDF, EPUB and Kindle. Book excerpt: The goal of this study is to better understand how methods for estimating treatment effects of latent groups operate. In particular, the authors identify where violations of assumptions can lead to biased estimates, and explore how covariates can be critical in the estimation process. For each set of approaches, the authors first review the assumptions necessary for identification and discuss practical issues that arise in estimation; second, they then examine how covariates allow for improved estimation, and determine the conditions necessary for using covariates to identify causal effects in latent groups; and third, they then compare the different methods using simulation studies built from datasets constructed by imputing missing class membership and potential outcomes from real-world studies. This allows for examining the performance of the different techniques under a variety of plausible circumstances. Analyzed is data from the Job Search Intervention Study (JOBS II), a randomized evaluation of an intervention for unemployed workers consisting of a series of training sessions and also the Head Start Impact Study, a large-scale randomized evaluation of the Head Start program in which children randomized to treatment were offered a seat in a classroom in a Head Start program. The authors conclude that, in practice, randomized trials should attempt to collect such covariates by, for example, having expert assessment of likelihood of compliance collected at baseline and that for identification, many methods require assumptions that are quite strong.
Download or read book Assessing Treatment Effect Heterogeneity written by Konstantinos Papangelou and published by . This book was released on 2021 with total page pages. Available in PDF, EPUB and Kindle. Book excerpt:
Download or read book Experimental Methods in Survey Research written by Paul J. Lavrakas and published by John Wiley & Sons. This book was released on 2019-10-08 with total page 544 pages. Available in PDF, EPUB and Kindle. Book excerpt: A thorough and comprehensive guide to the theoretical, practical, and methodological approaches used in survey experiments across disciplines such as political science, health sciences, sociology, economics, psychology, and marketing This book explores and explains the broad range of experimental designs embedded in surveys that use both probability and non-probability samples. It approaches the usage of survey-based experiments with a Total Survey Error (TSE) perspective, which provides insight on the strengths and weaknesses of the techniques used. Experimental Methods in Survey Research: Techniques that Combine Random Sampling with Random Assignment addresses experiments on within-unit coverage, reducing nonresponse, question and questionnaire design, minimizing interview measurement bias, using adaptive design, trend data, vignettes, the analysis of data from survey experiments, and other topics, across social, behavioral, and marketing science domains. Each chapter begins with a description of the experimental method or application and its importance, followed by reference to relevant literature. At least one detailed original experimental case study then follows to illustrate the experimental method’s deployment, implementation, and analysis from a TSE perspective. The chapters conclude with theoretical and practical implications on the usage of the experimental method addressed. In summary, this book: Fills a gap in the current literature by successfully combining the subjects of survey methodology and experimental methodology in an effort to maximize both internal validity and external validity Offers a wide range of types of experimentation in survey research with in-depth attention to their various methodologies and applications Is edited by internationally recognized experts in the field of survey research/methodology and in the usage of survey-based experimentation —featuring contributions from across a variety of disciplines in the social and behavioral sciences Presents advances in the field of survey experiments, as well as relevant references in each chapter for further study Includes more than 20 types of original experiments carried out within probability sample surveys Addresses myriad practical and operational aspects for designing, implementing, and analyzing survey-based experiments by using a Total Survey Error perspective to address the strengths and weaknesses of each experimental technique and method Experimental Methods in Survey Research: Techniques that Combine Random Sampling with Random Assignment is an ideal reference for survey researchers and practitioners in areas such political science, health sciences, sociology, economics, psychology, public policy, data collection, data science, and marketing. It is also a very useful textbook for graduate-level courses on survey experiments and survey methodology.
Download or read book Comparative Effectiveness Review Methods written by U. S. Department of Health and Human Services and published by Createspace Independent Pub. This book was released on 2013-05-17 with total page 226 pages. Available in PDF, EPUB and Kindle. Book excerpt: The Agency for Healthcare Research and Quality (AHRQ) commissioned the RTI International–University of North Carolina at Chapel Hill (RTI-UNC) Evidence-based Practice Center (EPC) to explore how systematic review groups have dealt with clinical heterogeneity and to seek out best practices for addressing clinical heterogeneity in systematic reviews (SRs) and comparative effectiveness reviews (CERs). Such best practices, to the extent they exist, may enable AHRQ's EPCs to address critiques from patients, clinicians, policymakers, and other proponents of health care about the extent to which “average” estimates of the benefits and harms of health care interventions apply to individual patients or to small groups of patients sharing similar characteristics. Such users of reviews often assert that EPC reviews typically focus on broad populations and, as a result, often lack information relevant to patient subgroups that are of particular concern to them. More important, even when EPCs evaluate literature on homogeneous groups, there may be varying individual treatment for no apparent reason, indicating that average treatment effect does not point to the best treatment for any given individual. Thus, the health care community is looking for better ways to develop information that may foster better medical care at a “personal” or “individual” level. To address our charge for this methods project, the EPC set out to answer six key questions (KQ). Key questions for methods report on clinical heterogeneity include: 1. What is clinical heterogeneity? a. How has it been defined by various groups? b. How is it distinct from statistical heterogeneity? c. How does it fit with other issues that have been addressed by the AHRQ Methods Manual for CERs? 2. How have systematic reviews dealt with clinical heterogeneity in the key questions? a. What questions have been asked? b. How have they pre-identified population subgroups with common clinical characteristics that modify their intervention-outcome association? c. What are best practices in key questions and how these subgroups have been identified? 3. How have systematic reviews dealt with clinical heterogeneity in the review process? a. What do guidance documents of various systematic review groups recommend? b. How have EPCs handled clinical heterogeneity in their reviews? c. What are best practices in searching for and interpreting results for particular subgroups with common clinical characteristics that may modify their intervention-outcome association? 4. What are critiques in how systematic reviews handle clinical heterogeneity? a. What are critiques from specific reviews (peer and public) on how EPCs handled clinical heterogeneity? b. What general critiques (in the literature) have been made against how systematic reviews handle clinical heterogeneity? 5. What evidence is there to support how to best address clinical heterogeneity in a systematic review? 6. What questions should an EPC work group on clinical heterogeneity address? Heterogeneity (of any type) in EPC reviews is important because its appearance suggests that included studies differed on one or more dimensions such as patient demographics, study designs, coexisting conditions, or other factors. EPCs then need to clarify for clinical and other audiences, collectively referred to as stakeholders, what are the potential causes of the heterogeneity in their results. This will allow the stakeholders to understand whether and to what degree they can apply this information to their own patients or constituents. Of greatest importance for this project was clinical heterogeneity, which we define as the variation in study population characteristics, coexisting conditions, cointerventions, and outcomes evaluated across studies included in an SR or CER that may influence or modify the magnitude of the intervention measure of effect (e.g., odds ratio, risk ratio, risk difference).
Download or read book Doing Meta Analysis with R written by Mathias Harrer and published by CRC Press. This book was released on 2021-09-15 with total page 500 pages. Available in PDF, EPUB and Kindle. Book excerpt: Doing Meta-Analysis with R: A Hands-On Guide serves as an accessible introduction on how meta-analyses can be conducted in R. Essential steps for meta-analysis are covered, including calculation and pooling of outcome measures, forest plots, heterogeneity diagnostics, subgroup analyses, meta-regression, methods to control for publication bias, risk of bias assessments and plotting tools. Advanced but highly relevant topics such as network meta-analysis, multi-three-level meta-analyses, Bayesian meta-analysis approaches and SEM meta-analysis are also covered. A companion R package, dmetar, is introduced at the beginning of the guide. It contains data sets and several helper functions for the meta and metafor package used in the guide. The programming and statistical background covered in the book are kept at a non-expert level, making the book widely accessible. Features • Contains two introductory chapters on how to set up an R environment and do basic imports/manipulations of meta-analysis data, including exercises • Describes statistical concepts clearly and concisely before applying them in R • Includes step-by-step guidance through the coding required to perform meta-analyses, and a companion R package for the book
Download or read book Cochrane Handbook for Systematic Reviews of Interventions written by Julian P. T. Higgins and published by Wiley. This book was released on 2008-11-24 with total page 672 pages. Available in PDF, EPUB and Kindle. Book excerpt: Healthcare providers, consumers, researchers and policy makers are inundated with unmanageable amounts of information, including evidence from healthcare research. It has become impossible for all to have the time and resources to find, appraise and interpret this evidence and incorporate it into healthcare decisions. Cochrane Reviews respond to this challenge by identifying, appraising and synthesizing research-based evidence and presenting it in a standardized format, published in The Cochrane Library (www.thecochranelibrary.com). The Cochrane Handbook for Systematic Reviews of Interventions contains methodological guidance for the preparation and maintenance of Cochrane intervention reviews. Written in a clear and accessible format, it is the essential manual for all those preparing, maintaining and reading Cochrane reviews. Many of the principles and methods described here are appropriate for systematic reviews applied to other types of research and to systematic reviews of interventions undertaken by others. It is hoped therefore that this book will be invaluable to all those who want to understand the role of systematic reviews, critically appraise published reviews or perform reviews themselves.
Download or read book Nonparametric Tests for Treatment Effect Heterogeneity written by and published by . This book was released on 2006 with total page 31 pages. Available in PDF, EPUB and Kindle. Book excerpt: A large part of the recent literature on program evaluation has focused on estimation of the average effect of the treatment under assumptions of unconfoundedness or ignorability following the seminal work by Rubin (1974) and Rosenbaum and Rubin (1983). In many cases however, researchers are interested in the effects of programs beyond estimates of the overall average or the average for the subpopulation of treated individuals. It may be of substantive interest to investigate whether there is any subpopulation for which a program or treatment has a nonzero average effect, or whether there is heterogeneity in the effect of the treatment. The hypothesis that the average effect of the treatment is zero for all subpopulations is also important for researchers interested in assessing assumptions concerning the selection mechanism. In this paper we develop two nonparametric tests. The first test is for the null hypothesis that the treatment has a zero average effect for any subpopulation defined by covariates. The second test is for the null hypothesis that the average effect conditional on the covariates is identical for all subpopulations, in other words, that there is no heterogeneity in average treatment effects by covariates. Sacrificing some generality by focusing on these two specific null hypotheses we derive tests that are straightforward to implement
Download or read book Finding What Works in Health Care written by Institute of Medicine and published by National Academies Press. This book was released on 2011-07-20 with total page 267 pages. Available in PDF, EPUB and Kindle. Book excerpt: Healthcare decision makers in search of reliable information that compares health interventions increasingly turn to systematic reviews for the best summary of the evidence. Systematic reviews identify, select, assess, and synthesize the findings of similar but separate studies, and can help clarify what is known and not known about the potential benefits and harms of drugs, devices, and other healthcare services. Systematic reviews can be helpful for clinicians who want to integrate research findings into their daily practices, for patients to make well-informed choices about their own care, for professional medical societies and other organizations that develop clinical practice guidelines. Too often systematic reviews are of uncertain or poor quality. There are no universally accepted standards for developing systematic reviews leading to variability in how conflicts of interest and biases are handled, how evidence is appraised, and the overall scientific rigor of the process. In Finding What Works in Health Care the Institute of Medicine (IOM) recommends 21 standards for developing high-quality systematic reviews of comparative effectiveness research. The standards address the entire systematic review process from the initial steps of formulating the topic and building the review team to producing a detailed final report that synthesizes what the evidence shows and where knowledge gaps remain. Finding What Works in Health Care also proposes a framework for improving the quality of the science underpinning systematic reviews. This book will serve as a vital resource for both sponsors and producers of systematic reviews of comparative effectiveness research.
Download or read book Methods in Comparative Effectiveness Research written by Constantine Gatsonis and published by CRC Press. This book was released on 2017-02-24 with total page 547 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.
Download or read book Prevention of Coronary Heart Disease written by Ira S. Ockene and published by Little, Brown Medical Division. This book was released on 1992 with total page 632 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Download or read book Heterogeneous Treatment Effects in the Presence of Self Selection written by Xiang Zhou and published by . This book was released on 2018 with total page 34 pages. Available in PDF, EPUB and Kindle. Book excerpt: An essential feature common to all empirical social research is variability across units of analysis. Individuals differ not only in background characteristics, but also in how they respond to a particular treatment, intervention, or stimulation. Moreover, individuals may self-select into treatment on the basis of their anticipated treatment effects. To study heterogeneous treatment effects in the presence of self-selection, Heckman and Vytlacil (1999, 2001a, 2005, 2007b) have developed a structural approach that builds on the marginal treatment effect (MTE). In this paper, we extend the MTE-based approach through a redefinition of MTE. Specifically, we redefine MTE as the expected treatment effect conditional on the propensity score (instead of all observed covariates) as well as a latent variable representing unobserved resistance to treatment. The redefined MTE improves upon the original MTE in a number of aspects. First, while it is conditional on a unidimensional summary of covariates, it is sufficient to capture all of the treatment effect heterogeneity that is consequential for selection bias. Second, the new MTE is a bivariate function, and thus is easier to visualize than the original MTE. Third, as with the original MTE, the new MTE can also be used as a building block for evaluating standard causal estimands such as ATE and TT. However, the weights associated with the new MTE are simpler, more intuitive, and easier to compute. Finally, the redefined MTE immediately reveals treatment effect heterogeneity among individuals who are at the margin of treatment. As a result, it can be used to evaluate a wide range of policy changes with little analytical twist, and to design policy interventions that optimize the marginal benefits of treatment.
Download or read book Theory of U Statistics written by Vladimir S. Korolyuk and published by Springer Science & Business Media. This book was released on 2013-03-09 with total page 558 pages. Available in PDF, EPUB and Kindle. Book excerpt: The theory of U-statistics goes back to the fundamental work of Hoeffding [1], in which he proved the central limit theorem. During last forty years the interest to this class of random variables has been permanently increasing, and thus, the new intensively developing branch of probability theory has been formed. The U-statistics are one of the universal objects of the modem probability theory of summation. On the one hand, they are more complicated "algebraically" than sums of independent random variables and vectors, and on the other hand, they contain essential elements of dependence which display themselves in the martingale properties. In addition, the U -statistics as an object of mathematical statistics occupy one of the central places in statistical problems. The development of the theory of U-statistics is stipulated by the influence of the classical theory of summation of independent random variables: The law of large num bers, central limit theorem, invariance principle, and the law of the iterated logarithm we re proved, the estimates of convergence rate were obtained, etc.
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 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 Can Variation in Subgroups Average Treatment Effects Explain Treatment Effect Heterogeneity written by Marianne P. Bitler and published by . This book was released on 2014 with total page 30 pages. Available in PDF, EPUB and Kindle. Book excerpt: In this paper, we assess whether welfare reform affects earnings only through mean impacts that are constant within but vary across subgroups. This is important because researchers interested in treatment effect heterogeneity typically restrict their attention to estimating mean impacts that are only allowed to vary across subgroups. Using a novel approach to simulating treatment group earnings under the constant mean-impacts within subgroup model, we find that this model does a poor job of capturing the treatment effect heterogeneity for Connecticut's Jobs First welfare reform experiment using quantile treatment effects. Notably, ignoring within-group heterogeneity would lead one to miss evidence that the Jobs First experiment's effects are consistent with central predictions of basic labor supply theory.
