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Statistical Topics in Health Economics and Outcomes Research

Statistical Topics in Health Economics and Outcomes Research
Author: Demissie Alemayehu, PhD
Publisher: CRC Press
Total Pages: 274
Release: 2017-11-22
Genre: Mathematics
ISBN: 1351252674

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With ever-rising healthcare costs, evidence generation through Health Economics and Outcomes Research (HEOR) plays an increasingly important role in decision-making about the allocation of resources. Accordingly, it is now customary for health technology assessment and reimbursement agencies to request for HEOR evidence, in addition to data from clinical trials, to inform decisions about patient access to new treatment options. While there is a great deal of literature on HEOR, there is a need for a volume that presents a coherent and unified review of the major issues that arise in application, especially from a statistical perspective. Statistical Topics in Health Economics and Outcomes Research fulfils that need by presenting an overview of the key analytical issues and best practice. Special attention is paid to key assumptions and other salient features of statistical methods customarily used in the area, and appropriate and relatively comprehensive references are made to emerging trends. The content of the book is purposefully designed to be accessible to readers with basic quantitative backgrounds, while providing an in-depth coverage of relatively complex statistical issues. The book will make a very useful reference for researchers in the pharmaceutical industry, academia, and research institutions involved with HEOR studies. The targeted readers may include statisticians, data scientists, epidemiologists, outcomes researchers, health economists, and healthcare policy and decision-makers.


Analysis of Observational Health Care Data Using SAS

Analysis of Observational Health Care Data Using SAS
Author: Douglas E. Faries
Publisher: SAS Press
Total Pages: 0
Release: 2010
Genre: Medical care
ISBN: 9781607642275

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This book guides researchers in performing and presenting high-quality analyses of all kinds of non-randomized studies, including analyses of observational studies, claims database analyses, assessment of registry data, survey data, pharmaco-economic data, and many more applications. The text is sufficiently detailed to provide not only general guidance, but to help the researcher through all of the standard issues that arise in such analyses. Just enough theory is included to allow the reader to understand the pros and cons of alternative approaches and when to use each method. The numerous contributors to this book illustrate, via real-world numerical examples and SAS code, appropriate implementations of alternative methods. The end result is that researchers will learn how to present high-quality and transparent analyses that will lead to fair and objective decisions from observational data. This book is part of the SAS Press program.


Measuring Racial Discrimination

Measuring Racial Discrimination
Author: National Research Council
Publisher: National Academies Press
Total Pages: 335
Release: 2004-07-24
Genre: Social Science
ISBN: 0309091268

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Many racial and ethnic groups in the United States, including blacks, Hispanics, Asians, American Indians, and others, have historically faced severe discriminationâ€"pervasive and open denial of civil, social, political, educational, and economic opportunities. Today, large differences among racial and ethnic groups continue to exist in employment, income and wealth, housing, education, criminal justice, health, and other areas. While many factors may contribute to such differences, their size and extent suggest that various forms of discriminatory treatment persist in U.S. society and serve to undercut the achievement of equal opportunity. Measuring Racial Discrimination considers the definition of race and racial discrimination, reviews the existing techniques used to measure racial discrimination, and identifies new tools and areas for future research. The book conducts a thorough evaluation of current methodologies for a wide range of circumstances in which racial discrimination may occur, and makes recommendations on how to better assess the presence and effects of discrimination.


Statistical Inference for Costs and Incremental Cost-effectiveness Ratios with Censored Data

Statistical Inference for Costs and Incremental Cost-effectiveness Ratios with Censored Data
Author: Shuai Chen
Publisher:
Total Pages:
Release: 2012
Genre:
ISBN:

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Cost-effectiveness analysis is widely conducted in the economic evaluation of new treatment options. In many clinical and observational studies of costs, data are often censored. Censoring brings challenges to both medical cost estimation and cost-effectiveness analysis. Although methods have been proposed for estimating the mean costs with censored data, they are often derived from theory and it is not always easy to understand how these methods work. We provide an alternative method for estimating the mean cost more efficiently based on a replace-from-the-right algorithm, and show that this estimator is equivalent to an existing estimator based on the inverse probability weighting principle and semiparametric efficiency theory. Therefore, we provide an intuitive explanation to a theoretically derived mean cost estimator. In many applications, it is also important to estimate the survival function of costs. We propose a generalized redistribute-to-the right algorithm for estimating the survival function of costs with censored data, and show that it is equivalent to a simple weighted survival estimator of costs based on inverse probability weighting techniques. Motivated by this redistribute-to-the-right principle, we also develop a more efficient survival estimator for costs, which has the desirable property of being monotone, and more efficient, although not always consistent. We conduct simulation to compare our method with some existing survival estimators for costs, and find the bias seems quite small. Thus, it may be considered as a candidate for survival estimator for costs in a real setting when the censoring is heavy and cost history information is available. Finally, we consider one special situation in conducting cost-effectiveness analysis, when the terminating events for survival time and costs are different. Traditional methods for statistical inference cannot deal with such data. We propose a new method for deriving the confidence interval for the incremental cost-effectiveness ratio under this situation, based on counting process and the general theory for missing data process. The simulation studies show that our method performs very well for some practical settings. Our proposed method has a great potential of being applied to a real setting when different terminating events exist for survival time and costs.


Cost Effectiveness in Health and Medicine

Cost Effectiveness in Health and Medicine
Author: Peter J. Neumann
Publisher: Oxford University Press
Total Pages: 537
Release: 2017
Genre: Business & Economics
ISBN: 0190492937

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CEAs (cost-effectiveness analyses) are used by decision makers in the health sector to make enlightened evaluations and this book provides an in depth look at how to evaluate the evaluator. The book is aimed specifically at Public health specialists.


Statistical Analysis of Cost-Effectiveness Data

Statistical Analysis of Cost-Effectiveness Data
Author: Andrew R. Willan
Publisher: John Wiley & Sons
Total Pages: 210
Release: 2006-08-14
Genre: Mathematics
ISBN: 0470856270

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The statistical analysis of cost-effectiveness data is becoming increasingly important within health and medical research. Statistical Analysis of Cost-Effectiveness Data provides a practical book that synthesises the huge amount of research that has taken place in the area over the last two decades. Comprising an up-to-date overview of the statistical analysis of cost-effectiveness data, the book is supported by numerous worked examples from the author’s own experience. It has been written in a style suitable for medical statisticians and health care professionals alike. Key features include: an overview of statistical methods used in the analysis of cost-effectiveness data. coverage of Bayesian methodology. illustrated throughout by worked examples using real data. suitability for health care professionals with limited statistical knowledge. discussion of software used for data analysis. An essential reference for biostatisticians and health economists engaged in cost-effectiveness analysis of health-care interventions, both in academia and industry. Also of interest to graduate students of biostatistics, public health and economics.


Modeling Approaches for Cost and Cost-effectiveness Estimation Using Observational Data

Modeling Approaches for Cost and Cost-effectiveness Estimation Using Observational Data
Author: Jiaqi Li
Publisher:
Total Pages: 150
Release: 2016
Genre:
ISBN:

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The estimation of treatment effects on medical costs and cost effectiveness measures is complicated by the need to account for non-independent censoring, skewness and the effects of confounders. In this dissertation, we develop several cost and cost-effectiveness tools that account for these issues. Since medical costs are often collected from observational claims data, we investigate propensity score methods such as covariate adjustment, stratification, inverse probability weighting and doubly robust weighting. We also propose several doubly robust estimators for common cost effectiveness measures. Lastly, we explore the role of big data tools and machine learning algorithms in cost estimation. We show how these modern techniques can be applied to big data manipulation, cost prediction and dimension reduction.


Developing a Protocol for Observational Comparative Effectiveness Research: A User's Guide

Developing a Protocol for Observational Comparative Effectiveness Research: A User's Guide
Author: Agency for Health Care Research and Quality (U.S.)
Publisher: Government Printing Office
Total Pages: 236
Release: 2013-02-21
Genre: Medical
ISBN: 1587634236

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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)


Small Clinical Trials

Small Clinical Trials
Author: Institute of Medicine
Publisher: National Academies Press
Total Pages: 221
Release: 2001-01-01
Genre: Medical
ISBN: 0309171148

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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.