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 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 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 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 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 396 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 Natural History Studies written by John Arthur Thomson and published by . This book was released on 1921 with total page 264 pages. Available in PDF, EPUB and Kindle. Book excerpt:
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 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 248 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 A Natural History of Infixation written by Alan C. L. Yu and published by OUP Oxford. This book was released on 2007-07-05 with total page 280 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book presents the first cross-linguistic study of the phenomenon of infixation, typically associated in English with words like "im-bloody-possible", and found in all the world's major linguistic families. Infixation is a central puzzle in prosodic morphology: Professor Yu explores its prosodic, phonological, and morphological characteristics, considers its diverse functions, and formulates a general theory to explain the rules and constraints by which it is governed. He examines 154 infixation patterns from over a hundred languages, including examples from Asia, Europe, Africa, New Guinea, and South America. He compares the formal properties of different kinds of infix, explores the range of diachronic pathways that lead to them, and considers the processes by which they are acquired in first language learning. A central argument of the book concerns the idea that the typological tendencies of language may be traced back to its origins and to the mechanisms of language transmission. The book thus combines the history of infixation with an exploration of the role diachronic and functional factors play in synchronic argumentation: it is an exemplary instance of the holistic approach to linguistic explanation. Alan Yu's pioneering study will interest phonologists and morphologists of all theoretical persuasions, as well as typologists and historical linguists.
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 University of Iowa Studies in Natural History written by and published by . This book was released on 1933 with total page 370 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Download or read book Critical Thinking in Clinical Research written by Felipe Fregni and published by Oxford University Press. This book was released on 2018 with total page 537 pages. Available in PDF, EPUB and Kindle. Book excerpt: Critical Thinking in Clinical Research explains the fundamentals of clinical research in a case-based approach. The core concept is to combine a clear and concise transfer of information and knowledge with an engagement of the reader to develop a mastery of learning and critical thinking skills. The book addresses the main concepts of clinical research, basics of biostatistics, advanced topics in applied biostatistics, and practical aspects of clinical research, with emphasis on clinical relevance across all medical specialties.
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.
Download or read book A Natural History of Human Thinking written by Michael Tomasello and published by Harvard University Press. This book was released on 2018-04-09 with total page 193 pages. Available in PDF, EPUB and Kindle. Book excerpt: Tool-making or culture, language or religious belief: ever since Darwin, thinkers have struggled to identify what fundamentally differentiates human beings from other animals. Michael Tomasello weaves his twenty years of comparative studies of humans and great apes into a compelling argument that cooperative social interaction is the key to our cognitive uniqueness. Tomasello maintains that our prehuman ancestors, like today's great apes, were social beings who could solve problems by thinking. But they were almost entirely competitive, aiming only at their individual goals. As ecological changes forced them into more cooperative living arrangements, early humans had to coordinate their actions and communicate their thoughts with collaborative partners. Tomasello's "shared intentionality hypothesis" captures how these more socially complex forms of life led to more conceptually complex forms of thinking. In order to survive, humans had to learn to see the world from multiple social perspectives, to draw socially recursive inferences, and to monitor their own thinking via the normative standards of the group. Even language and culture arose from the preexisting need to work together and coordinate thoughts. A Natural History of Human Thinking is the most detailed scientific analysis to date of the connection between human sociality and cognition.
Download or read book Worlds of Natural History written by Helen Anne Curry and published by Cambridge University Press. This book was released on 2018-11-22 with total page 683 pages. Available in PDF, EPUB and Kindle. Book excerpt: Explores the development of natural history since the Renaissance and contextualizes current discussions of biodiversity.
Download or read book A Democracy of Facts written by Andrew J. Lewis and published by University of Pennsylvania Press. This book was released on 2011-04-05 with total page 216 pages. Available in PDF, EPUB and Kindle. Book excerpt: Chronicles the story of American naturalists who came of age and stumbled toward a profession in the years after the American Revolution. --from publisher description.
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 2009-07-01 with total page 463 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.
