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Author: Manuel Posada de la Paz Publisher: Springer ISBN: 3319671448 Category : Medical Languages : en Pages : 667
Book Description
The fields of rare diseases research and orphan products development continue to expand with more products in research and development status. In recent years, the role of the patient advocacy groups has evolved into a research partner with the academic research community and the bio-pharmaceutical industry. Unique approaches to research and development require epidemiological data not previously available to assist in protocol study design and patient recruitment for clinical trials required by regulatory agencies prior to approval for access by patents and practicing physicians.
Author: Manuel Posada de la Paz Publisher: Springer ISBN: 3319671448 Category : Medical Languages : en Pages : 667
Book Description
The fields of rare diseases research and orphan products development continue to expand with more products in research and development status. In recent years, the role of the patient advocacy groups has evolved into a research partner with the academic research community and the bio-pharmaceutical industry. Unique approaches to research and development require epidemiological data not previously available to assist in protocol study design and patient recruitment for clinical trials required by regulatory agencies prior to approval for access by patents and practicing physicians.
Author: Manuel Posada de la Paz Publisher: Springer ISBN: 9789048194841 Category : Medical Languages : en Pages : 542
Book Description
In our etiologic research, we epidemiologists need to leave behind the concepts of ‘cohort’ study and ‘case–control’ study and adopt that of the etiologic study as the singular substitute for these. With this sentence, the famous epidemiologist Professor Olli S. Miettinen began his personal re ection on the future of the epidemiology [1]. He sought to highlight the fact that the role of the epidemiologist should be mainly focused on aetiological research. Nevertheless, the widespread idea still exists that epidemiology is limited to purely providing gures and descriptive data on the frequency and distribution of disease. Indeed, it is more than likely that the precise aim of those rst classic epidemiological steps, i. e. , methods essentially based on describing the distri- tion of a given disease, is still not all that well understood by many scientists, let alone the general public. Such descriptions seek to generate hypotheses and afford explanations for key factors (be these risk factors or the presumable causes th- selves), which might justify differences in terms of persons, time or place and, in turn, ultimately serve to develop preventive measures and/or gain quality-adjusted life years. To restrict the goals of epidemiology to activities exclusively concerned with reporting gures or even complex statistical results is a great mistake, one that renders it dif cult to take full advantage of the epidemiologist’s true role, which is “to study disease determinants and to assess the actual impact of factors involved in their development, distribution and dissemination”.
Author: Institute of Medicine Publisher: National Academies Press ISBN: 0309158060 Category : Medical Languages : en Pages : 442
Book Description
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.
Author: Manuel Posada de la Paz Publisher: Springer ISBN: 9789400733381 Category : Medical Languages : en Pages : 542
Book Description
In our etiologic research, we epidemiologists need to leave behind the concepts of ‘cohort’ study and ‘case–control’ study and adopt that of the etiologic study as the singular substitute for these. With this sentence, the famous epidemiologist Professor Olli S. Miettinen began his personal re ection on the future of the epidemiology [1]. He sought to highlight the fact that the role of the epidemiologist should be mainly focused on aetiological research. Nevertheless, the widespread idea still exists that epidemiology is limited to purely providing gures and descriptive data on the frequency and distribution of disease. Indeed, it is more than likely that the precise aim of those rst classic epidemiological steps, i. e. , methods essentially based on describing the distri- tion of a given disease, is still not all that well understood by many scientists, let alone the general public. Such descriptions seek to generate hypotheses and afford explanations for key factors (be these risk factors or the presumable causes th- selves), which might justify differences in terms of persons, time or place and, in turn, ultimately serve to develop preventive measures and/or gain quality-adjusted life years. To restrict the goals of epidemiology to activities exclusively concerned with reporting gures or even complex statistical results is a great mistake, one that renders it dif cult to take full advantage of the epidemiologist’s true role, which is “to study disease determinants and to assess the actual impact of factors involved in their development, distribution and dissemination”.
Author: Agency for Healthcare Research and Quality/AHRQ Publisher: Government Printing Office ISBN: 1587634333 Category : Medical Languages : en Pages : 396
Book Description
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.
Author: National Organization for Rare Disorders Publisher: Lippincott Williams & Wilkins ISBN: 9780781730631 Category : Medical Languages : en Pages : 982
Book Description
NORD Guide to Rare Disorders is a comprehensive, practical, authoritative guide to the diagnosis and management of more than 800 rare diseases. The diseases are discussed in a uniform, easy-to-follow format--a brief description, signs and symptoms, etiology, related disorders, epidemiology, standard treatment, investigational treatment, resources, and references.The book includes a complete directory of orphan drugs, a full-color atlas of visual diagnostic signs, and a Master Resource List of support groups and helpful organizations. An index of symptoms and key words offers physicians valuable assistance in finding the information they need quickly.
Author: Tarun Weeramanthri Publisher: Frontiers Media SA ISBN: 2889455017 Category : Languages : en Pages : 149
Book Description
Precision Public Health is a new and rapidly evolving field, that examines the application of new technologies to public health policy and practice. It draws on a broad range of disciplines including genomics, spatial data, data linkage, epidemiology, health informatics, big data, predictive analytics and communications. The hope is that these new technologies will strengthen preventive health, improve access to health care, and reach disadvantaged populations in all areas of the world. But what are the downsides and what are the risks, and how can we ensure the benefits flow to those population groups most in need, rather than simply to those individuals who can afford to pay? This is the first collection of theoretical frameworks, analyses of empirical data, and case studies to be assembled on this topic, published to stimulate debate and promote collaborative work.
Author: Muin Khoury Publisher: Oxford University Press ISBN: 0195398440 Category : Medical Languages : en Pages : 701
Book Description
This text describes the role that epidemiologic methods play in the continuum from gene discovery to the development and application of genetic tests. It provides a foundation that should help researchers, policy makers and practitioners integrate genomics into medical and public health practice.
Author: National Research Council Publisher: National Academies Press ISBN: 0309255716 Category : Medical Languages : en Pages : 424
Book Description
In the late 1980s, the National Cancer Institute initiated an investigation of cancer risks in populations near 52 commercial nuclear power plants and 10 Department of Energy nuclear facilities (including research and nuclear weapons production facilities and one reprocessing plant) in the United States. The results of the NCI investigation were used a primary resource for communicating with the public about the cancer risks near the nuclear facilities. However, this study is now over 20 years old. The U.S. Nuclear Regulatory Commission requested that the National Academy of Sciences provide an updated assessment of cancer risks in populations near USNRC-licensed nuclear facilities that utilize or process uranium for the production of electricity. Analysis of Cancer Risks in Populations near Nuclear Facilities: Phase 1 focuses on identifying scientifically sound approaches for carrying out an assessment of cancer risks associated with living near a nuclear facility, judgments about the strengths and weaknesses of various statistical power, ability to assess potential confounding factors, possible biases, and required effort. The results from this Phase 1 study will be used to inform the design of cancer risk assessment, which will be carried out in Phase 2. This report is beneficial for the general public, communities near nuclear facilities, stakeholders, healthcare providers, policy makers, state and local officials, community leaders, and the media.
Author: Manuel Posada de la Paz
Author: Manuel Posada de la Paz
Author: Manuel Posada de la Paz
Author: Institute of Medicine
Author: Manuel Posada de la Paz
Author: Agency for Healthcare Research and Quality/AHRQ
Author: National Organization for Rare Disorders
Author: Tarun Weeramanthri
Author: Raj S. Bhopal
Author: Muin Khoury
Author: National Research Council