Author : Institute of Medicine
Publisher : National Academies Press
Page : 442 pages
File Size : 18,74 MB
Release : 2011-04-03
Category : Medical
ISBN : 0309158060
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
Page : 675 pages
File Size : 48,28 MB
Release : 2017-12-06
Category : Medical
ISBN : 3319671448
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 : United States. Congress. Senate. Committee on Health, Education, Labor, and Pensions
Publisher :
Page : 14 pages
File Size : 40,96 MB
Release : 2001
Category : Orphan drugs
ISBN :
Author : Manuel Posada de la Paz
Publisher : Springer
Page : 542 pages
File Size : 44,35 MB
Release : 2010-09-18
Category : Medical
ISBN : 9789048194841
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 : Raymond A. Huml
Publisher : Springer Nature
Page : 418 pages
File Size : 28,80 MB
Release : 2021-11-08
Category : Medical
ISBN : 3030786056
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.
Author : David Pryde
Publisher : Royal Society of Chemistry
Page : 487 pages
File Size : 20,32 MB
Release : 2014-07-30
Category : Science
ISBN : 1782624201
Orphan drugs are designated drug substances that are intended to treat rare or ‘orphan’ diseases. More than 7000 rare diseases are known that collectively affect some 6-7% of the developed world’s population; however, individually, any single, rare disease may only affect a handful of people making them commercially unattractive for the biopharmaceutical industry to target. Ground breaking legislation, starting with the Orphan Drug Act that was passed in the US in 1983 to provide financial incentives for companies to develop orphan drugs, has sparked ever increasing interest from biopharmaceutical companies to tackle rare diseases. These developments have made rare diseases, and the orphan drugs that treat them, sufficiently attractive to pharmaceutical development and many pharmaceutical companies now have research units dedicated to this area of research. It is therefore timely to review the area of orphan drugs and some of the basic science, drug discovery and regulatory factors that underpin this important, and growing, area of biomedical research. Written by a combination of academic and industry experts working in the field, this text brings together expert authors in the regulatory, drug development, genetics, biochemistry, patient advocacy group, medicinal chemistry and commercial domains to create a unique and timely reference for all biomedical researchers interested in finding out more about orphan drugs and the rare diseases they treat. Providing an up-to-date monograph, this book covers the basic science, drug discovery and regulatory elements behind orphan drugs and will appeal to medicinal and pharmaceutical chemists, biochemists and anyone working within the fields of rare disease research and drug development or pharmaceuticals in industry or academia.
Author : Agency for Healthcare Research and Quality/AHRQ
Publisher : Government Printing Office
Page : 385 pages
File Size : 13,75 MB
Release : 2014-04-01
Category : Medical
ISBN : 1587634333
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 : J-F. Cordier
Publisher : European Respiratory Society
Page : 376 pages
File Size : 18,21 MB
Release : 2011-12-01
Category : Medical
ISBN : 1849840148
This book has been replaced by "Rare Diseases of the Respiratory System", ISBN 978-1-84984-167-2 A respiratory physician is only likely to see a few orphan lung diseases each year or even during their career. However, it is essential that specialists are able identify, confirm and diagnosis orphan lung disease in a patient. This Monograph comprehensively covers the most common and/or complex orphan lung diseases. It should be seen as a solid companion to the respiratory specialist each time they need to consider a diagnosis of an orphan disease.
Author : Farid A. Badria
Publisher : BoD – Books on Demand
Page : 236 pages
File Size : 39,52 MB
Release : 2020-12-02
Category : Medical
ISBN : 1839685204
Drug repurposing or drug repositioning is a new approach to presenting new indications for common commercial and clinically approved existing drugs. For example, chloroquine, an old antimalarial drug, showed promising results for treating COVID-19, interfering with MDR in several types of cancer, and chemosensitizing human leukemic cells.This book focuses on the hypothesis, risk/benefits, and economic impacts of drug repurposing on drug discovery in dermatology, infectious diseases, neurological disorders, cancer, and orphan diseases. It brings together up-to-date research to provide readers with an informative, illustrative, and easy-to-read book useful for students, clinicians, and the pharmaceutical industry.
Author : Isao Kamae
Publisher : Springer Nature
Page : 282 pages
File Size : 31,28 MB
Release : 2019-09-03
Category : Business & Economics
ISBN : 9811357935
Representing the first book on the topic, this work offers the reader an introduction to the Japanese systems for health technology assessment (HTA) officially introduced by the Ministry of Health, Labour and Welfare (MHLW) in 2016. Policy and guidelines are discussed, with the relevant methods and conditions of cost-effectiveness analysis explained alongside. Numerous instructive examples and exercises, ranging from basic to advanced, impart valuable knowledge and insight on the quantitative methods for economic evaluation, which will appeal to both beginners and experts. This guidebook is authored by Japan’s foremost expert in HTA and pharmacoeconomics, with a view to strengthening the reader’s expertise in value-based healthcare and decision-making. The methods presented are essential to informing regulatory, local and patient decisions; as such, the book is equally recommended to industry and government, as well as academia, and anyone with an interest in Japanese HTA.
