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 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 Toward Precision Medicine written by National Research Council and published by National Academies Press. This book was released on 2012-01-16 with total page 142 pages. Available in PDF, EPUB and Kindle. Book excerpt: Motivated by the explosion of molecular data on humans-particularly data associated with individual patients-and the sense that there are large, as-yet-untapped opportunities to use this data to improve health outcomes, Toward Precision Medicine explores the feasibility and need for "a new taxonomy of human disease based on molecular biology" and develops a potential framework for creating one. The book says that a new data network that integrates emerging research on the molecular makeup of diseases with clinical data on individual patients could drive the development of a more accurate classification of diseases and ultimately enhance diagnosis and treatment. The "new taxonomy" that emerges would define diseases by their underlying molecular causes and other factors in addition to their traditional physical signs and symptoms. The book adds that the new data network could also improve biomedical research by enabling scientists to access patients' information during treatment while still protecting their rights. This would allow the marriage of molecular research and clinical data at the point of care, as opposed to research information continuing to reside primarily in academia. Toward Precision Medicine notes that moving toward individualized medicine requires that researchers and health care providers have access to very large sets of health- and disease-related data linked to individual patients. These data are also critical for developing the information commons, the knowledge network of disease, and ultimately the new taxonomy.

Download or read book Economic Dimensions of Personalized and Precision Medicine written by Ernst R. Berndt and published by University of Chicago Press. This book was released on 2019-04-22 with total page 361 pages. Available in PDF, EPUB and Kindle. Book excerpt: Personalized and precision medicine (PPM)—the targeting of therapies according to an individual’s genetic, environmental, or lifestyle characteristics—is becoming an increasingly important approach in health care treatment and prevention. The advancement of PPM is a challenge in traditional clinical, reimbursement, and regulatory landscapes because it is costly to develop and introduces a wide range of scientific, clinical, ethical, and socioeconomic issues. PPM raises a multitude of economic issues, including how information on accurate diagnosis and treatment success will be disseminated and who will bear the cost; changes to physician training to incorporate genetics, probability and statistics, and economic considerations; questions about whether the benefits of PPM will be confined to developed countries or will diffuse to emerging economies with less developed health care systems; the effects of patient heterogeneity on cost-effectiveness analysis; and opportunities for PPM’s growth beyond treatment of acute illness, such as prevention and reversal of chronic conditions. This volume explores the intersection of the scientific, clinical, and economic factors affecting the development of PPM, including its effects on the drug pipeline, on reimbursement of PPM diagnostics and treatments, and on funding of the requisite underlying research; and it examines recent empirical applications of PPM.

Download or read book Handbook of Statistical Methods for Precision Medicine written by Eric Laber and published by CRC Press. This book was released on 2024-10-23 with total page 482 pages. Available in PDF, EPUB and Kindle. Book excerpt: The statistical study and development of analytic methodology for individualization of treatments is no longer in its infancy. Many methods of study design, estimation, and inference exist, and the tools available to the analyst are ever growing. This handbook introduces the foundations of modern statistical approaches to precision medicine, bridging key ideas to active lines of current research in precision medicine. The contributions in this handbook vary in their level of assumed statistical knowledge; all contributions are accessible to a wide readership of statisticians and computer scientists including graduate students and new researchers in the area. Many contributions, particularly those that are more comprehensive reviews, are suitable for epidemiologists and clinical researchers with some statistical training. The handbook is split into three sections: Study Design for Precision Medicine, Estimation of Optimal Treatment Strategies, and Precision Medicine in High Dimensions. The first focuses on designed experiments, in many instances, building and extending on the notion of sequential multiple assignment randomized trials. Dose finding and simulation-based designs using agent-based modelling are also featured. The second section contains both introductory contributions and more advanced methods, suitable for estimating optimal adaptive treatment strategies from a variety of data sources including non-experimental (observational) studies. The final section turns to estimation in the many-covariate setting, providing approaches suitable to the challenges posed by electronic health records, wearable devices, or any other settings where the number of possible variables (whether confounders, tailoring variables, or other) is high. Together, these three sections bring together some of the foremost leaders in the field of precision medicine, offering new insights and ideas as this field moves towards its third decade.

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 Advanced Medical Statistics 2nd Edition written by Ying Lu and published by World Scientific. This book was released on 2015-06-29 with total page 1471 pages. Available in PDF, EPUB and Kindle. Book excerpt: The book aims to provide both comprehensive reviews of the classical methods and an introduction to new developments in medical statistics. The topics range from meta analysis, clinical trial design, causal inference, personalized medicine to machine learning and next generation sequence analysis. Since the publication of the first edition, there have been tremendous advances in biostatistics and bioinformatics. The new edition tries to cover as many important emerging areas and reflect as much progress as possible. Many distinguished scholars, who greatly advanced their research areas in statistical methodology as well as practical applications, also have revised several chapters with relevant updates and written new ones from scratch.The new edition has been divided into four sections, including, Statistical Methods in Medicine and Epidemiology, Statistical Methods in Clinical Trials, Statistical Genetics, and General Methods. To reflect the rise of modern statistical genetics as one of the most fertile research areas since the publication of the first edition, the brand new section on Statistical Genetics includes entirely new chapters reflecting the state of the art in the field.Although tightly related, all the book chapters are self-contained and can be read independently. The book chapters intend to provide a convenient launch pad for readers interested in learning a specific topic, applying the related statistical methods in their scientific research and seeking the newest references for in-depth research.

Download or read book The Theory of Probability written by Hans Reichenbach and published by Univ of California Press. This book was released on 1971 with total page 516 pages. Available in PDF, EPUB and Kindle. Book excerpt:

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 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 Precision Medicine and Artificial Intelligence written by Michael Mahler and published by Academic Press. This book was released on 2021-03-12 with total page 302 pages. Available in PDF, EPUB and Kindle. Book excerpt: Precision Medicine and Artificial Intelligence: The Perfect Fit for Autoimmunity covers background on artificial intelligence (AI), its link to precision medicine (PM), and examples of AI in healthcare, especially autoimmunity. The book highlights future perspectives and potential directions as AI has gained significant attention during the past decade. Autoimmune diseases are complex and heterogeneous conditions, but exciting new developments and implementation tactics surrounding automated systems have enabled the generation of large datasets, making autoimmunity an ideal target for AI and precision medicine. More and more diagnostic products utilize AI, which is also starting to be supported by regulatory agencies such as the Food and Drug Administration (FDA). Knowledge generation by leveraging large datasets including demographic, environmental, clinical and biomarker data has the potential to not only impact the diagnosis of patients, but also disease prediction, prognosis and treatment options. - Allows the readers to gain an overview on precision medicine for autoimmune diseases leveraging AI solutions - Provides background, milestone and examples of precision medicine - Outlines the paradigm shift towards precision medicine driven by value-based systems - Discusses future applications of precision medicine research using AI - Other aspects covered in the book include regulatory insights, data analytics and visualization, types of biomarkers as well as the role of the patient in precision medicine

Download or read book Application of Novel Statistical and Machine learning Methods to High dimensional Clinical Cancer and Multi Omics data written by Chao Xu and published by Frontiers Media SA. This book was released on 2022-02-02 with total page 136 pages. Available in PDF, EPUB and Kindle. Book excerpt:

Download or read book Causal Inference in Statistics written by Judea Pearl and published by John Wiley & Sons. This book was released on 2016-01-25 with total page 162 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Download or read book Sharing Clinical Trial Data written by Institute of Medicine and published by National Academies Press. This book was released on 2015-04-20 with total page 236 pages. Available in PDF, EPUB and Kindle. Book excerpt: Data sharing can accelerate new discoveries by avoiding duplicative trials, stimulating new ideas for research, and enabling the maximal scientific knowledge and benefits to be gained from the efforts of clinical trial participants and investigators. At the same time, sharing clinical trial data presents risks, burdens, and challenges. These include the need to protect the privacy and honor the consent of clinical trial participants; safeguard the legitimate economic interests of sponsors; and guard against invalid secondary analyses, which could undermine trust in clinical trials or otherwise harm public health. Sharing Clinical Trial Data presents activities and strategies for the responsible sharing of clinical trial data. With the goal of increasing scientific knowledge to lead to better therapies for patients, this book identifies guiding principles and makes recommendations to maximize the benefits and minimize risks. This report offers guidance on the types of clinical trial data available at different points in the process, the points in the process at which each type of data should be shared, methods for sharing data, what groups should have access to data, and future knowledge and infrastructure needs. Responsible sharing of clinical trial data will allow other investigators to replicate published findings and carry out additional analyses, strengthen the evidence base for regulatory and clinical decisions, and increase the scientific knowledge gained from investments by the funders of clinical trials. The recommendations of Sharing Clinical Trial Data will be useful both now and well into the future as improved sharing of data leads to a stronger evidence base for treatment. This book will be of interest to stakeholders across the spectrum of research-from funders, to researchers, to journals, to physicians, and ultimately, to patients.

Download or read book Precision medicine approaches for heterogeneous conditions such as autism spectrum disorders The need for a biomarker exploration phase in clinical trials Phase 2m written by David Quentin Beversdorf and published by Frontiers Media SA. This book was released on 2023-04-17 with total page 153 pages. Available in PDF, EPUB and Kindle. Book excerpt: Many therapeutic interventions for autism spectrum disorder fail when they are examined in a clinical trial. Frequently, there is a subset of patients that responds very well to the intervention, while others do not, and the overall result does not yield a positive result. As autism spectrum disorder is highly heterogeneous in its underlying genetics and other etiological risk factors, as well as its heterogeneous phenotypic manifestation, this variability in response to any specific treatment is not entirely surprising. However, it remains a challenge to meaningfully subtype this heterogeneity for targeted treatment. The purpose of this research topic is to solicit articles that address the heterogeneity in autism spectrum disorder in a manner that may meaningfully contribute to targeted treatment approaches. Studies that address the heterogeneity of autism that could theoretically lead to targeted treatment, and studies that more directly address the use of a marker in association with response to a treatment, are both aspects that will contribute to this purpose. It is hoped that this Research Topic will yield articles that can help advance the field towards precision medicine in autism spectrum disorders. Manuscripts that contribute to the specification of the heterogeneity of autism spectrum disorder in a manner that could theoretically lead to targeted treatment would be appropriate for this research topic. Additionally, articles that utilize subtyping in relation to response to treatment would be appropriate for this research topic.

Download or read book Statistics in Precision Health written by Yichuan Zhao and published by Springer Nature. This book was released on with total page 545 pages. Available in PDF, EPUB and Kindle. Book excerpt:

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 634 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.