Download or read book Evaluation of Biomarkers and Surrogate Endpoints in Chronic Disease written by Institute of Medicine and published by National Academies Press. This book was released on 2010-06-25 with total page 335 pages. Available in PDF, EPUB and Kindle. Book excerpt: Many people naturally assume that the claims made for foods and nutritional supplements have the same degree of scientific grounding as those for medication, but that is not always the case. The IOM recommends that the FDA adopt a consistent scientific framework for biomarker evaluation in order to achieve a rigorous and transparent process.

Download or read book Rare Diseases and Orphan Products written by Institute of Medicine and published by National Academies Press. This book was released on 2011-04-03 with total page 442 pages. Available in PDF, EPUB and Kindle. Book excerpt: Rare diseases collectively affect millions of Americans of all ages, but developing drugs and medical devices to prevent, diagnose, and treat these conditions is challenging. The Institute of Medicine (IOM) recommends implementing an integrated national strategy to promote rare diseases research and product development.

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 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 Rare Disease Drug Development written by Raymond A. Huml and published by Springer Nature. This book was released on 2021-11-08 with total page 418 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book provides a broad overview of rare disease drug development. It offers unique insights from various perspectives, including third-party capital providers, caregivers, patient advocacy groups, drug development professionals, marketing and commercial experts, and patients. A unique reference, the book begins with narratives on the many challenges faced by rare disease patient and their caregivers. Subsequent chapters underscore the critical, multidimensional role of patient advocacy groups and the novel approaches to related clinical trials, investment decisions, and the optimization of rare disease registries. The book addresses various rare disease drug development processes by disciplines such as oncology, hematology, pediatrics, and gene therapy. Chapters then address the operational aspects of drug development, including approval processes, development accelerations, and market access strategies. The book concludes with reflections on the authors' case for real-world data and evidence generation in orphan medicinal drug development. Rare Disease Drug Development is an expertly written text optimized for biopharmaceutical R&D experts, commercial experts, third-party capital providers, patient advocacy groups, patients, and caregivers.

Download or read book Illuminating Natural History written by Henrietta McBurney and published by Paul Mellon Centre. This book was released on 2021-06-22 with total page 384 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book explores the life and work of the 18th-century English artist, explorer, naturalist, and author Mark Catesby (1683-1749). During Catesby's lifetime, science was poised to shift from a world of amateur virtuosi to one of professional experts. He worked against a backdrop of global travel that incorporated collecting and direct observation of nature. Catesby spent two prolonged periods in the New World--in Virginia (1712-19) and South Carolina and the Bahamas (1722-26)--which he documented in Natural History of Carolina, Florida and the Bahama Islands, the first large-format, color-plate book on the natural history of North America. Interweaving elements of art history, history of science, natural history illustration, painting materials, book history, paper studies, garden history, and colonial history, this volume brings together a wealth of unpublished images as well as previously unpublished letters by Catesby, with contemporary accounts of his collecting and encounters in the wild, and details of the materials and techniques of packing and transporting plants and animals across the Atlantic.

Download or read book Exploring Novel Clinical Trial Designs for Gene Based Therapies written by National Academies of Sciences, Engineering, and Medicine and published by National Academies Press. This book was released on 2020-08-27 with total page 127 pages. Available in PDF, EPUB and Kindle. Book excerpt: Recognizing the potential design complexities and ethical issues associated with clinical trials for gene therapies, the Forum on Regenerative Medicine of the National Academies of Sciences, Engineering, and Medicine held a 1-day workshop in Washington, DC, on November 13, 2019. Speakers at the workshop discussed patient recruitment and selection for gene-based clinical trials, explored how the safety of new therapies is assessed, reviewed the challenges involving dose escalation, and spoke about ethical issues such as informed consent and the role of clinicians in recommending trials as options to their patients. The workshop also included discussions of topics related to gene therapies in the context of other available and potentially curative treatments, such as bone marrow transplantation for hemoglobinopathies. This publication summarizes the presentation and discussion of the workshop.

Download or read book The Role of Purchasers and Payers in the Clinical Research Enterprise written by Institute of Medicine and published by National Academies Press. This book was released on 2002-06-14 with total page 120 pages. Available in PDF, EPUB and Kindle. Book excerpt: In a workshop organized by the Clinical Research roundtable, representatives from purchaser organizations (employers), payer organizations (health plans and insurance companies), and other stakeholder organizations (voluntary health associations, clinical researchers, research organizations, and the technology community) came together to explore: What do purchasers and payers need from the Clinical Research Enterprise? How have current efforts in clinical research met their needs? What are purchasers, payers, and other stakeholders willing to contribute to the enterprise? This book documents these discussions and summarizes what employers and insurers need from and are willing to contribute to clinical research from both a business and a national health care perspective.

Download or read book The Future of Natural History Museums written by Eric Dorfman and published by Routledge. This book was released on 2017-10-12 with total page 381 pages. Available in PDF, EPUB and Kindle. Book excerpt: Natural history museums are changing, both because of their own internal development and in response to changes in context. Historically, the aim of collecting from nature was to develop encyclopedic assemblages to satisfy human curiosity and build a basis for taxonomic information. Today, with global biodiversity in rapid decline, there are new reasons to build and maintain collections, while audiences are more diverse, numerous, and technically savvy. Institutions must learn to embrace new technology while retaining the authenticity of their stories and the value placed on their objects. The Future of Natural History Museums begins to develop a cohesive discourse that balances the disparate issues that our institutions will face over the next decades. It disassembles the topic into various key elements and, through commentary and synthesis, explores a cohesive picture of the trajectory of the natural history museum sector. This book contributes to the study of collections, teaching and learning, ethics, and running non-profit businesses and will be of interest to museum and heritage professionals and academics and senior students in Biological Sciences and Museum Studies.

Download or read book Natural Experiments of History written by Jared Diamond and published by Harvard University Press. This book was released on 2012-10-01 with total page 290 pages. Available in PDF, EPUB and Kindle. Book excerpt: Some central questions in the natural and social sciences can't be answered by controlled laboratory experiments, often considered to be the hallmark of the scientific method. This impossibility holds for any science concerned with the past. In addition, many manipulative experiments, while possible, would be considered immoral or illegal. One has to devise other methods of observing, describing, and explaining the world. In the historical disciplines, a fruitful approach has been to use natural experiments or the comparative method. This book consists of eight comparative studies drawn from history, archeology, economics, economic history, geography, and political science. The studies cover a spectrum of approaches, ranging from a non-quantitative narrative style in the early chapters to quantitative statistical analyses in the later chapters. The studies range from a simple two-way comparison of Haiti and the Dominican Republic, which share the island of Hispaniola, to comparisons of 81 Pacific islands and 233 areas of India. The societies discussed are contemporary ones, literate societies of recent centuries, and non-literate past societies. Geographically, they include the United States, Mexico, Brazil, western Europe, tropical Africa, India, Siberia, Australia, New Zealand, and other Pacific islands. In an Afterword, the editors discuss how to cope with methodological problems common to these and other natural experiments of history.

Download or read book Deep Things Out of Darkness written by John G. T. Anderson and published by Univ of California Press. This book was released on 2013 with total page 362 pages. Available in PDF, EPUB and Kindle. Book excerpt: Natural history, the deliberate observation of the environment, is arguably the oldest science. From purely practical beginnings as a way of finding food and shelter, natural history evolved into the holistic, systematic study of plants, animals, and the landscape. This book chronicles the rise, decline, and ultimate revival of natural history within the realms of science and public discourse. It charts the journey of the naturalist's endeavour from prehistory to the present, underscoring the need for natural history in an era of dynamic environmental change.

Download or read book The Natural History of Alcoholism Revisited written by George E. Vaillant and published by Harvard University Press. This book was released on 1995-05-25 with total page 468 pages. Available in PDF, EPUB and Kindle. Book excerpt: When The Natural History of Alcoholism was first published in 1983, it was acclaimed in the press as the single most important contribution to the literature on alcoholism since the first edition of Alcoholic Anonymous’s Big Book. George Vaillant took on the crucial questions of whether alcoholism is a symptom or a disease, whether it is progressive, whether alcoholics differ from others before the onset of their alcoholism, and whether alcoholics can safely drink. Based on an evaluation of more than 600 individuals followed for over forty years, Vaillant’s monumental study offered new and authoritative answers to all of these questions. In this updated version of his classic book, Vaillant returns to the same subjects with the perspective gained from fifteen years of further follow-up. Alcoholics who had been studied to age 50 in the earlier book have now reached age 65 and beyond, and Vaillant reassesses what we know about alcoholism in light of both their experiences and the many new studies of the disease by other researchers. The result is a sharper focus on the nature and course of this devastating disorder as well as a sounder foundation for the assessment of various treatments.

Download or read book A Natural History of Rape written by Randy Thornhill and published by MIT Press. This book was released on 2001-02-23 with total page 274 pages. Available in PDF, EPUB and Kindle. Book excerpt: A biologist and an anthropologist use evolutionary biology to explain the causes and inform the prevention of rape. In this controversial book, Randy Thornhill and Craig Palmer use evolutionary biology to explain the causes of rape and to recommend new approaches to its prevention. According to Thornhill and Palmer, evolved adaptation of some sort gives rise to rape; the main evolutionary question is whether rape is an adaptation itself or a by-product of other adaptations. Regardless of the answer, Thornhill and Palmer note, rape circumvents a central feature of women's reproductive strategy: mate choice. This is a primary reason why rape is devastating to its victims, especially young women. Thornhill and Palmer address, and claim to demolish scientifically, many myths about rape bred by social science theory over the past twenty-five years. The popular contention that rapists are not motivated by sexual desire is, they argue, scientifically inaccurate. Although they argue that rape is biological, Thornhill and Palmer do not view it as inevitable. Their recommendations for rape prevention include teaching young males not to rape, punishing rape more severely, and studying the effectiveness of "chemical castration." They also recommend that young women consider the biological causes of rape when making decisions about dress, appearance, and social activities. Rape could cease to exist, they argue, only in a society knowledgeable about its evolutionary causes. The book includes a useful summary of evolutionary theory and a comparison of evolutionary biology's and social science's explanations of human behavior. The authors argue for the greater explanatory power and practical usefulness of evolutionary biology. The book is sure to stir up discussion both on the specific topic of rape and on the larger issues of how we understand and influence human behavior.

Download or read book Ainu written by William W. Fitzhugh and published by University of Washington Press. This book was released on 1999 with total page 424 pages. Available in PDF, EPUB and Kindle. Book excerpt: "Some 55 scholars, mostly Japanese but with a considerable number from the US and Europe, write about the ethnicity, theories of origin, history, economies, art, religious beliefs, mythology, and other aspects of the culture of the Ainu, the indigenous people of Japan, now principally found in Hokkaido and smaller far northern islands. Hundreds of photographs and paintings, mostly in excellent quality color, show a wide variety of Ainu people, as well as clothing, jewelry, and various artifacts."--"Choice". "The most in-depth treatise available on Ainu prehistory, material culture, and ethnohistory." - "Library Journal".--Amazon.com (2001 ed, book description).

Download or read book A Natural History of Pragmatism written by Joan Richardson and published by Cambridge University Press. This book was released on 2006-12-21 with total page 14 pages. Available in PDF, EPUB and Kindle. Book excerpt: Joan Richardson provides a fascinating and compelling account of the emergence of the quintessential American philosophy: pragmatism. She demonstrates pragmatism's engagement with various branches of the natural sciences and traces the development of Jamesian pragmatism from the late nineteenth century through modernism, following its pointings into the present. Richardson combines strands from America's religious experience with scientific information to offer interpretations that break new ground in literary and cultural history. This book exemplifies the value of interdisciplinary approaches to producing literary criticism. In a series of highly original readings of Edwards, Emerson, William and Henry James, Stevens, and Stein, A Natural History of Pragmatism tracks the interplay of religious motive, scientific speculation, and literature in shaping an American aesthetic. Wide-ranging and bold, this groundbreaking book will be essential reading for all students and scholars of American literature.

Download or read book Clinical Trials written by Tom Brody and published by Academic Press. This book was released on 2016-02-19 with total page 897 pages. Available in PDF, EPUB and Kindle. Book excerpt: Clinical Trials, Second Edition, offers those engaged in clinical trial design a valuable and practical guide. This book takes an integrated approach to incorporate biomedical science, laboratory data of human study, endpoint specification, legal and regulatory aspects and much more with the fundamentals of clinical trial design. It provides an overview of the design options along with the specific details of trial design and offers guidance on how to make appropriate choices. Full of numerous examples and now containing actual decisions from FDA reviewers to better inform trial design, the 2nd edition of Clinical Trials is a must-have resource for early and mid-career researchers and clinicians who design and conduct clinical trials. - Contains new and fully revised material on key topics such as biostatistics, biomarkers, orphan drugs, biosimilars, drug regulations in Europe, drug safety, regulatory approval and more - Extensively covers the "study schema" and related features of study design - Incorporates laboratory data from studies on human patients to provide a concrete tool for understanding the concepts in the design and conduct of clinical trials - Includes decisions made by FDA reviewers when granting approval of a drug as real world learning examples for readers

Download or read book Concepts of Epidemiology written by Raj S. Bhopal and published by Oxford University Press. This book was released on 2016 with total page 481 pages. Available in PDF, EPUB and Kindle. Book excerpt: First edition published in 2002. Second edition published in 2008.