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Rare Diseases and Orphan Drugs

Rare Diseases and Orphan Drugs
  • Author : Jules J. Berman
  • Publisher :Unknown
  • Release Date :2014-05-26
  • Total pages :400
  • ISBN : 9780124200098
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Summary : Rare Diseases and Orphan Drugs shows that much of what we now know about common diseases has been achieved by studying rare diseases. It proposes that future advances in the prevention, diagnosis, and treatment of common diseases will come as a consequence of our accelerating progress in the field of rare diseases. Understanding the complex steps in the development of common diseases, such as cancer, cardiovascular disease, and metabolic diseases, has proven a difficult problem. Rare diseases, however, are often caused by aberrations of a single gene. In rare diseases, we may study how specific genetic defects can trigger a series of events that lead to the expression of a particular disease. Often, the disease process manifested in a certain rare disease is strikingly similar to the disease process observed in a common disease. This work ties the lessons learned about rare diseases to our understanding of common ones. Chapters covering the number of common diseases are minimized, while rare diseases are introduced as single diseases or as members of diseases classes. After reading this book, readers will appreciate how further research into the rare diseases may lead to new methods for preventing, diagnosing, and treating all diseases, rare or common. Makes rare diseases relevant to clinicians and researchers by tying lessons learned about the rare diseases to our understanding of the common diseases Stresses basic pathologic mechanisms that account for human disease (e.g., disorders of cell development, replication, maintenance, function and structure), that can be understood without prior training in pathology Discusses advanced concepts in molecular biology and genetics in a simple, functional context appropriate for medical trainees and new researchers Offers insights into how further research into rare diseases may lead to new methods for preventing, diagnosing, and treating all diseases.

Rare Diseases and Orphan Products

Rare Diseases and Orphan Products
  • Author : Institute of Medicine,Board on Health Sciences Policy,Committee on Accelerating Rare Diseases Research and Orphan Product Development
  • Publisher :Unknown
  • Release Date :2011-04-03
  • Total pages :442
  • ISBN : 9780309158060
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Summary : 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.

Orphan Drugs and Rare Diseases

Orphan Drugs and Rare Diseases
  • Author : David C Pryde,Michael J Palmer
  • Publisher :Unknown
  • Release Date :2014
  • Total pages :486
  • ISBN : 9781849738064
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Summary : This book provides an up-to-date monograph on the drug discovery and regulatory elements of therapeutics used to treat rare or "orphan" diseases.

Orphan Drugs

Orphan Drugs
  • Author : Elizabeth Hernberg-Ståhl,Miroslav Reljanović
  • Publisher :Unknown
  • Release Date :2013-11-15
  • Total pages :334
  • ISBN : 9781908818393
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Summary : This authoritative and comprehensive book makes the reader familiar with the processes of bringing orphan drugs to the global market. There are between 5,000 and 7,000 rare diseases and the number of patients suffering from them is estimated to be more than 50 million in the US and Europe. Before the orphan drug legislation enacted in the US in 1983, there was a limited interest from industry to develop treatment for very small patient groups. One of the difficulties is, of course, that similar levels of investment are needed from a pharmaceutical company to bring a drug to the market for both small and large patient groups. The journey from application of an orphan drug designation to a reimbursed market- approved drug is long and many obstacles occur during the journey. After reading the book, readers will: Understand who the players/stakeholders are in the rare orphan disease field and their specific needs and concerns: patients and patient organizations, researchers and treating physicians within the field, industry, regulatory and reimbursement bodies* Understand the strong partnership between the different players and the various initiatives to improve and increase access to treatment for patients; minimizing the gap between numbers of known diseases, orphan designations, approved drugs and paid drugs. The book also provides short practical case stories from patients and researchers, as well as representatives from industry and authorities on the challenges they came across in developing orphan drugs or getting access to orphan drugs. A comprehensive overview of strategy, key activities and considerations of how to bring an orphan drug from concept to the market and make it available to patients A source of updated information, news and trends for those who are already active in this fast-evolving field Covers the global definitions and the criteria for getting an orphan drug designation in, for example, the US and Europe

Innovative Methods for Rare Disease Drug Development

Innovative Methods for Rare Disease Drug Development
  • Author : Shein-Chung Chow
  • Publisher :Unknown
  • Release Date :2020-11-11
  • Total pages :306
  • ISBN : 9781000208337
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Summary : In the United States, a rare disease is defined by the Orphan Drug Act as a disorder or condition that affects fewer than 200,000 persons. For the approval of "orphan" drug products for rare diseases, the traditional approach of power analysis for sample size calculation is not feasible because there are only limited number of subjects available for clinical trials. In this case, innovative approaches are needed for providing substantial evidence meeting the same standards for statistical assurance as drugs used to treat common conditions. Innovative Methods for Rare Disease Drug Development focuses on biostatistical applications in terms of design and analysis in pharmaceutical research and development from both regulatory and scientific (statistical) perspectives. Key Features: Reviews critical issues (e.g., endpoint/margin selection, sample size requirements, and complex innovative design). Provides better understanding of statistical concepts and methods which may be used in regulatory review and approval. Clarifies controversial statistical issues in regulatory review and approval accurately and reliably. Makes recommendations to evaluate rare diseases regulatory submissions. Proposes innovative study designs and statistical methods for rare diseases drug development, including n-of-1 trial design, adaptive trial design, and master protocols like platform trials. Provides insight regarding current regulatory guidance on rare diseases drug development like gene therapy.

Rare Diseases

Rare Diseases
  • Author : Nicolas Sireau
  • Publisher :Unknown
  • Release Date :2017-09-08
  • Total pages :278
  • ISBN : 9781351278386
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Summary : There are 7,000 rare diseases affecting 6%–8% of the global population. That's 3.5 million people in the UK alone. Yet only 200 rare diseases have approved treatments. In recent years, there has been a surge of interest from business and social entrepreneurs in the field of health – including looking at ways to treat rare disease patients better and faster. This book presents some of the latest developments in the world of rare disease entrepreneurship from a global group of experts. It examines the topic from the business angle, considering the drug development process and providing case studies of successful orphan drug enterprises. It also looks at rare diseases from the perspective of the patient, analysing the growing rare disease patient movement, a successful patient group that uses social enterprise techniques, and chapters on key requirements for helping patients with rare diseases through registries and centres of excellence. The book will be an essential toolkit for social and business entrepreneurs who are interested in the world of rare/orphan diseases. It has the rigour of an academic publication, along with the clarity of a lay publication. An original and timely book, Rare Diseases will help to add knowledge and awareness to a vastly under-published subject.

Orphan Lung Diseases

Orphan Lung Diseases
  • Author : J.-F. Cordier
  • Publisher :Unknown
  • Release Date :2014-05-14
  • Total pages :376
  • ISBN : 9781849840149
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Summary : Orphan lung diseases differ from the more common pulmonary disorders, due to the fact that the respiratory physician will only see a few of them each year or even during their career. However, as a specialist, it is necessary to identify and confirm such a diagnosis in a patient. This Monograph comprehensively covers the most common and/or complex of these orphan lung diseases. This Monograph should be seen as a solid companion for the respiratory specialist each time they need to consider a diagnosis of one of these orphan diseases.

Orphan Diseases and Orphan Drugs

Orphan Diseases and Orphan Drugs
  • Author : I. Herbert Scheinberg,John Michael Walshe
  • Publisher :Unknown
  • Release Date :1986
  • Total pages :228
  • ISBN : 0719022959
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Summary :

NORD Guide to Rare Disorders

NORD Guide to Rare Disorders
  • Author : National Organization for Rare Disorders
  • Publisher :Unknown
  • Release Date :2003
  • Total pages :895
  • ISBN : 0781730635
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Summary : 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.

Introduction to Basics of Pharmacology and Toxicology

Introduction to Basics of Pharmacology and Toxicology
  • Author : Gerard Marshall Raj,Ramasamy Raveendran
  • Publisher :Unknown
  • Release Date :2019-11-16
  • Total pages :417
  • ISBN : 9789813297791
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Summary : This book illustrates, in a comprehensive manner, the most crucial principles involved in pharmacology and allied sciences. The title begins by discussing the historical aspects of drug discovery, with up to date knowledge on Nobel Laureates in pharmacology and their significant discoveries. It then examines the general pharmacological principles - pharmacokinetics and pharmacodynamics, with in-depth information on drug transporters and interactions. In the remaining chapters, the book covers a definitive collection of topics containing essential information on the basic principles of pharmacology and how they are employed for the treatment of diseases. Readers will learn about special topics in pharmacology that are hard to find elsewhere, including issues related to environmental toxicology and the latest information on drug poisoning and treatment, analytical toxicology, toxicovigilance, and the use of molecular biology techniques in pharmacology. The book offers a valuable resource for researchers in the fields of pharmacology and toxicology, as well as students pursuing a degree in or with an interest in pharmacology.

Rare Diseases Epidemiology

Rare Diseases Epidemiology
  • Author : Manuel Posada de la Paz,Stephen C. Groft
  • Publisher :Unknown
  • Release Date :2012-11-07
  • Total pages :542
  • ISBN : 9400733380
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Summary : 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”.

Registries for Evaluating Patient Outcomes

Registries for Evaluating Patient Outcomes
  • Author : Agency for Healthcare Research and Quality/AHRQ
  • Publisher :Unknown
  • Release Date :2014-04-01
  • Total pages :356
  • ISBN : 9781587634338
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Summary : 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.

Rare Cancer Agenda 2030

Rare Cancer Agenda 2030
  • Author : Rare Cancers Europe
  • Publisher :Unknown
  • Release Date :2020-02-24
  • Total pages :229
  • ISBN : 9788831642712
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Summary : RARE CANCER AGENDA 2030 Ten Recommendations from the EU Joint Action on Rare Cancers 1. Rare cancers are the rare diseases of oncology 2. Rare cancers should be monitored 3. Health systems should exploit networking 4. Medical education should exploit and serve healthcare networking 5. Research should be fostered by networking and should take into account an expected higher degree of uncertainty 6. Patient-physician shared clinical decision-making should be especially valued 7. Appropriate state-of-the-art instruments should be developed in rare cancer 8. Regulation on rare cancers should tolerate a higher degree of uncertainty 9. Policy strategies on rare cancers and sustainability of interventions should be based on networking 10. Rare cancer patients should be engaged

Health Services Restructuring in Canada

Health Services Restructuring in Canada
  • Author : Charles M. Beach,Richard Chaykowski,John Deutsch Institute for the Study of Economic Policy,Sam Shortt
  • Publisher :Unknown
  • Release Date :2006
  • Total pages :378
  • ISBN : 1553390768
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Summary : (John Deutsch Institute for the Study of Economic Policy)Queen's Univ., Kingston, Canada. Presents proceedings of a conference held at Queen's Univ., on November, 17-18, 2005. Covers health-care reform, drugs for rare diseases, issues in delivery of health care, and more. For public health personnel. Softcover, hardcover also available.

Role of Biomarkers in Medicine

Role of Biomarkers in Medicine
  • Author : Mu Wang,Frank Witzmann
  • Publisher :Unknown
  • Release Date :2016-08-17
  • Total pages :260
  • ISBN : 9789535125051
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Summary : The use of biomarkers in basic and clinical research has become routine in many areas of medicine. They are accepted as molecular signatures that have been well characterized and repeatedly shown to be capable of predicting relevant disease states or clinical outcomes. In Role of Biomarkers in Medicine, expert researchers in their individual field have reviewed many biomarkers or potential biomarkers in various types of diseases. The topics address numerous aspects of medicine, demonstrating the current conceptual status of biomarkers as clinical tools and as surrogate endpoints in clinical research. This book highlights the current state of biomarkers and will aid scientists and clinicians to develop better and more specific biomarkers for disease management.

The Cambridge History of Medicine

The Cambridge History of Medicine
  • Author : Roy Porter
  • Publisher :Unknown
  • Release Date :2006-06-05
  • Total pages :408
  • ISBN : 9780521864268
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Summary : The Cambridge History of Medicine surveys the rise of medicine in the West from classical times to the present. Covering both the social and scientific history of medicine, this 2006 volume traces the chronology of key developments and events, engaging with the issues, discoveries, and controversies that have characterized medical progress.

Breakthrough Business Models

Breakthrough Business Models
  • Author : Institute of Medicine,Board on Health Sciences Policy,Forum on Drug Discovery, Development, and Translation,Robert Giffin,Sally Robinson,Theresa Wizemann
  • Publisher :Unknown
  • Release Date :2009-02-17
  • Total pages :150
  • ISBN : 0309178169
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Summary : The process for developing new drug and biologic products is extraordinarily expensive and time-consuming. Although large pharmaceutical companies may be able to afford the cost of development because they can expect a large return on investment, organizations developing drugs to treat rare and neglected diseases are unable to rely on such returns. On June 23, 2008, the Institute of Medicine's Forum on Drug Discovery, Development, and Translation held a public workshop, "Breakthrough Business Models: Drug Development for Rare and Neglected Diseases and Individualized Therapies," which sought to explore new and innovative strategies for developing drugs for rare and neglected diseases.

Innovation Policy and the Economy

Innovation Policy and the Economy
  • Author : Adam B. Jaffe,Josh Lerner,Scott Stern
  • Publisher :Unknown
  • Release Date :2006-08-01
  • Total pages :215
  • ISBN : 9780262101189
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Summary : Leading economists discuss how economic policy can stimulate technological innovation.

Genomic and Precision Medicine

Genomic and Precision Medicine
  • Author : Geoffrey S. Ginsburg,Huntington F Willard,Sean P. David
  • Publisher :Unknown
  • Release Date :2017-03-30
  • Total pages :370
  • ISBN : 9780128006542
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Summary : 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

Innovation Policy and the Economy, 2018

Innovation Policy and the Economy, 2018
  • Author : Josh Lerner,Scott Stern
  • Publisher :Unknown
  • Release Date :2019-02-21
  • Total pages :250
  • ISBN : 022664524X
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Summary : This volume highlights the interaction between public policy and innovation. The first chapter documents the dramatic globalization of R&D and how this development has affected the efforts of U.S. multinationals to operate on the global technology frontier. The next chapter synthesizes research on the impact of trade shocks on innovation and explains how these shocks’ effects depend on the firms, industries, and countries affected. The third chapter examines the Advanced Research Projects Agency (ARPA) model of research management—an approach to funding and managing high-risk R&D—and offers a method for diagnosing which research efforts are “ARPA-able.” Next is a study of the Orphan Drug Act and the key changes in the U.S. healthcare landscape and in drug discovery and development since its passage in 1983. The next two chapters focus on artificial intelligence (AI). One describes how AI diffuses through the economy and discusses implications for economic inequality, antitrust, and intellectual property. The other investigates issues surrounding firm competition and labor force participation, such as data portability and a Universal Basic Income, and evaluates ways to address these issues.

OECD Health Policy Studies Pharmaceutical Innovation and Access to Medicines

OECD Health Policy Studies Pharmaceutical Innovation and Access to Medicines
  • Author : OECD
  • Publisher :Unknown
  • Release Date :2018-11-29
  • Total pages :192
  • ISBN : 9789264307391
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Summary : This report reviews the important role of medicines in health sytems, describes recent trends in pharmaceutical expenditure and financing, and summarises the approaches used by OECD countries to determine coverage and pricing.