Download or read book Clinical and Statistical Considerations in Personalized Medicine written by Claudio Carini and published by CRC Press. This book was released on 2014-03-27 with total page 368 pages. Available in PDF, EPUB and Kindle. Book excerpt: Clinical and Statistical Considerations in Personalized Medicine explores recent advances related to biomarkers and their translation into clinical development. Leading clinicians, biostatisticians, regulators, commercial professionals, and researchers address the opportunities and challenges in successfully applying biomarkers in drug discovery and preclinical and clinical development. Robust Biomarkers for Drug Development and Disease Treatment The first four chapters discuss biomarker development from a clinical perspective. Coverage ranges from an introduction to biomarkers to advances in RNAi screens, epigenetics, and rare diseases as targets for personalized medicine approaches. Subsequent chapters examine the statistical considerations in applying a personalized medicine approach, including multiplicity in pharmacogenomics. The last chapter assesses the regulatory issues involved in using biomarkers. Improve Patient Care and Reduce Costs and Side Effects Despite the vast amount of literature on biomarkers, there is no comprehensive book that integrates the clinical and statistical components. This book is one of the first to incorporate both the clinical and statistical aspects of biomarkers in the personalized medicine paradigm. Covering a wide spectrum of personalized medicine-related topics, it presents state-of-the-art techniques for advancing the application of biomarkers in drug discovery and development.

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 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 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 Clinical and Statistical Considerations in Personalized Medicine written by Claudio Carini and published by . This book was released on 2014-01-01 with total page 368 pages. Available in PDF, EPUB and Kindle. Book excerpt: The Future of Clinical Research and Health Care: From Empirical to Precision Medicine Clinical and Statistical Considerations in Personalized Medicine explores recent advances related to biomarkers and their translation into clinical development. Leading clinicians, biostatisticians, regulators, commercial professionals, and researchers address the opportunities and challenges in successfully applying biomarkers in drug discovery and preclinical and clinical development. Robust Biomarkers for Drug Development and Disease Treatment The first four chapters discuss biomarker development from a clinical perspective. Coverage ranges from an introduction to biomarkers to advances in RNAi screens, epigenetics, and rare diseases as targets for personalized medicine approaches. Subsequent chapters examine the statistical considerations in applying a personalized medicine approach, including multiplicity in pharmacogenomics. The last chapter assesses the regulatory issues involved in using biomarkers. Improve Patient Care and Reduce Costs and Side Effects Despite the vast amount of literature on biomarkers, there is no comprehensive book that integrates the clinical and statistical components. This book is one of the first to incorporate both the clinical and statistical aspects of biomarkers in the personalized medicine paradigm. Covering a wide spectrum of personalized medicine-related topics, it presents state-of-the-art techniques for advancing the application of biomarkers in drug discovery and development.

Download or read book Greek Medicine from Hippocrates to Galen written by Jacques Jouanna and published by BRILL. This book was released on 2012-07-25 with total page 424 pages. Available in PDF, EPUB and Kindle. Book excerpt: This volume makes available in English translation a selection of Jacques Jouanna's papers on Greek and Roman medicine, ranging from the early beginnings of Greek medicine to late antiquity.

Download or read book Genomic and Precision Medicine written by Geoffrey S. Ginsburg and published by Academic Press. This book was released on 2017-03-30 with total page 372 pages. Available in PDF, EPUB and Kindle. Book excerpt: Genomic and Precision Medicine: Primary Care, Third Edition is an invaluable resource on the state-of-the-art tools, technologies and policy issues that are required to fully realize personalized health care in the area of primary care. One of the major areas where genomic and personalized medicine is most active is the realm of the primary care practitioner. Risk, family history, personal genomics and pharmacogenomics are becoming increasingly important to the PCP and their patients, and this book discusses the implications as they relate to primary care practitioners. - Presents a comprehensive volume for primary care providers - Provides succinct commentary and key learning points that will assist providers with their local needs for the implementation of genomic and personalized medicine - Includes a current overview on major opportunities for genomic and personalized medicine in practice - Highlights case studies that illustrate the practical use of genomics in the management in patients

Download or read book Fundamentals of Clinical Data Science written by Pieter Kubben and published by Springer. This book was released on 2018-12-21 with total page 219 pages. Available in PDF, EPUB and Kindle. Book excerpt: This open access book comprehensively covers the fundamentals of clinical data science, focusing on data collection, modelling and clinical applications. Topics covered in the first section on data collection include: data sources, data at scale (big data), data stewardship (FAIR data) and related privacy concerns. Aspects of predictive modelling using techniques such as classification, regression or clustering, and prediction model validation will be covered in the second section. The third section covers aspects of (mobile) clinical decision support systems, operational excellence and value-based healthcare. Fundamentals of Clinical Data Science is an essential resource for healthcare professionals and IT consultants intending to develop and refine their skills in personalized medicine, using solutions based on large datasets from electronic health records or telemonitoring programmes. The book’s promise is “no math, no code”and will explain the topics in a style that is optimized for a healthcare audience.

Download or read book Beyond the HIPAA Privacy Rule written by Institute of Medicine and published by National Academies Press. This book was released on 2009-03-24 with total page 334 pages. Available in PDF, EPUB and Kindle. Book excerpt: In the realm of health care, privacy protections are needed to preserve patients' dignity and prevent possible harms. Ten years ago, to address these concerns as well as set guidelines for ethical health research, Congress called for a set of federal standards now known as the HIPAA Privacy Rule. In its 2009 report, Beyond the HIPAA Privacy Rule: Enhancing Privacy, Improving Health Through Research, the Institute of Medicine's Committee on Health Research and the Privacy of Health Information concludes that the HIPAA Privacy Rule does not protect privacy as well as it should, and that it impedes important health research.

Download or read book Lung Cancer and Personalized Medicine written by Aamir Ahmad and published by Springer. This book was released on 2015-12-14 with total page 236 pages. Available in PDF, EPUB and Kindle. Book excerpt: This, the first of two volumes on personalized medicine in lung cancer, touches on the core issues related to the understanding of lung cancer—statistics and epidemiology of lung cancer—along with the incidence of lung cancer in non-smokers. A major focus of this volume is the state of current therapies against lung cancer—immune, targeted therapies against EGFR TKIs, KRAS, ALK, angiogenesis; the associated challenges, especially resistance mechanisms; and recent progress in targeted drug development based on metal chemistry. Chapters are written by some of the leading experts in the field, who provide a better understanding of lung cancer, the factors that make it lethal, and current research focused on developing personalized treatment plans. With a unique mix of topics, this volume summarizes the current state-of-knowledge on lung cancer and the available therapies.

Download or read book Preventive and Predictive Genetics Towards Personalised Medicine written by Godfrey Grech and published by Springer. This book was released on 2015-06-24 with total page 388 pages. Available in PDF, EPUB and Kindle. Book excerpt: Pharmacogenomics supports personalized medicine by translating genome-based knowledge into clinical practice, offering enhanced benefit for patients and health-care systems at large. Current routine practice for diagnosing and treating patients is conducted by correlating parameters such as age, gender and weight with risks and expected treatment outcomes. In the new era of personalized medicine the healthcare provider is equipped with improved ability to prevent, diagnose, treat and predict outcomes on the basis of complex information sources, including genetic and genomic data. Targeted therapy and reliable prediction of expected outcomes offer patients access to better healthcare management, by way of identifying the therapies effective for the relevant patient group, avoiding prescription of unnecessary treatment and reducing the likelihood of developing adverse drug reactions.

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 Personalized Hip and Knee Joint Replacement written by Charles Rivière and published by Springer Nature. This book was released on 2020-06-30 with total page 346 pages. Available in PDF, EPUB and Kindle. Book excerpt: This open access book describes and illustrates the surgical techniques, implants, and technologies used for the purpose of personalized implantation of hip and knee components. This new and flourishing treatment philosophy offers important benefits over conventional systematic techniques, including component positioning appropriate to individual anatomy, improved surgical reproducibility and prosthetic performance, and a reduction in complications. The techniques described in the book aim to reproduce patients’ native anatomy and physiological joint laxity, thereby improving the prosthetic hip/knee kinematics and functional outcomes in the quest of the forgotten joint. They include kinematically aligned total knee/total hip arthroplasty, partial knee replacement, and hip resurfacing. The relevance of available and emerging technological tools for these personalized approaches is also explained, with coverage of, for example, robotics, computer-assisted surgery, and augmented reality. Contributions from surgeons who are considered world leaders in diverse fields of this novel surgical philosophy make this open access book will invaluable to a wide readership, from trainees at all levels to consultants practicing lower limb surgery

Download or read book Clinical Prediction Models written by Ewout W. Steyerberg and published by Springer. This book was released on 2019-07-22 with total page 574 pages. Available in PDF, EPUB and Kindle. Book excerpt: The second edition of this volume provides insight and practical illustrations on how modern statistical concepts and regression methods can be applied in medical prediction problems, including diagnostic and prognostic outcomes. Many advances have been made in statistical approaches towards outcome prediction, but a sensible strategy is needed for model development, validation, and updating, such that prediction models can better support medical practice. There is an increasing need for personalized evidence-based medicine that uses an individualized approach to medical decision-making. In this Big Data era, there is expanded access to large volumes of routinely collected data and an increased number of applications for prediction models, such as targeted early detection of disease and individualized approaches to diagnostic testing and treatment. Clinical Prediction Models presents a practical checklist that needs to be considered for development of a valid prediction model. Steps include preliminary considerations such as dealing with missing values; coding of predictors; selection of main effects and interactions for a multivariable model; estimation of model parameters with shrinkage methods and incorporation of external data; evaluation of performance and usefulness; internal validation; and presentation formatting. The text also addresses common issues that make prediction models suboptimal, such as small sample sizes, exaggerated claims, and poor generalizability. The text is primarily intended for clinical epidemiologists and biostatisticians. Including many case studies and publicly available R code and data sets, the book is also appropriate as a textbook for a graduate course on predictive modeling in diagnosis and prognosis. While practical in nature, the book also provides a philosophical perspective on data analysis in medicine that goes beyond predictive modeling. Updates to this new and expanded edition include: • A discussion of Big Data and its implications for the design of prediction models • Machine learning issues • More simulations with missing ‘y’ values • Extended discussion on between-cohort heterogeneity • Description of ShinyApp • Updated LASSO illustration • New case studies

Download or read book Innovative Strategies Statistical Solutions and Simulations for Modern Clinical Trials written by Mark Chang and published by CRC Press. This book was released on 2019-03-20 with total page 376 pages. Available in PDF, EPUB and Kindle. Book excerpt: "This is truly an outstanding book. [It] brings together all of the latest research in clinical trials methodology and how it can be applied to drug development.... Chang et al provide applications to industry-supported trials. This will allow statisticians in the industry community to take these methods seriously." Jay Herson, Johns Hopkins University The pharmaceutical industry's approach to drug discovery and development has rapidly transformed in the last decade from the more traditional Research and Development (R & D) approach to a more innovative approach in which strategies are employed to compress and optimize the clinical development plan and associated timelines. However, these strategies are generally being considered on an individual trial basis and not as part of a fully integrated overall development program. Such optimization at the trial level is somewhat near-sighted and does not ensure cost, time, or development efficiency of the overall program. This book seeks to address this imbalance by establishing a statistical framework for overall/global clinical development optimization and providing tactics and techniques to support such optimization, including clinical trial simulations. Provides a statistical framework for achieve global optimization in each phase of the drug development process. Describes specific techniques to support optimization including adaptive designs, precision medicine, survival-endpoints, dose finding and multiple testing. Gives practical approaches to handling missing data in clinical trials using SAS. Looks at key controversial issues from both a clinical and statistical perspective. Presents a generous number of case studies from multiple therapeutic areas that help motivate and illustrate the statistical methods introduced in the book. Puts great emphasis on software implementation of the statistical methods with multiple examples of software code (both SAS and R). It is important for statisticians to possess a deep knowledge of the drug development process beyond statistical considerations. For these reasons, this book incorporates both statistical and "clinical/medical" perspectives.

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 Personalized Psychiatry written by Bernhard Baune and published by Academic Press. This book was released on 2019-10-16 with total page 594 pages. Available in PDF, EPUB and Kindle. Book excerpt: Personalized Psychiatry presents the first book to explore this novel field of biological psychiatry that covers both basic science research and its translational applications. The book conceptualizes personalized psychiatry and provides state-of-the-art knowledge on biological and neuroscience methodologies, all while integrating clinical phenomenology relevant to personalized psychiatry and discussing important principles and potential models. It is essential reading for advanced students and neuroscience and psychiatry researchers who are investigating the prevention and treatment of mental disorders. - Combines neurobiology with basic science methodologies in genomics, epigenomics and transcriptomics - Demonstrates how the statistical modeling of interacting biological and clinical information could transform the future of psychiatry - Addresses fundamental questions and requirements for personalized psychiatry from a basic research and translational perspective