June 16, 2021

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Rare Diseases and Orphan Drugs

Rare Diseases and Orphan Drugs
Author : Jules J. Berman
Publisher : Academic Press
Release Date : 2014-06-02
Category : Medical
Total pages :380
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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.

Orphan Diseases and Orphan Drugs

Orphan Diseases and Orphan Drugs
Author : I. Herbert Scheinberg,J. M. Walshe
Publisher : Manchester University Press
Release Date : 1989-04
Category : Chemotherapy
Total pages :228
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Rare Diseases and Orphan Drugs

Rare Diseases and Orphan Drugs
Author : Anonim
Publisher : Unknown
Release Date : 2007
Category :
Total pages :129
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Proceedings of 4th World Congress on Rare Diseases and Orphan Drugs 2018

Proceedings of 4th World Congress on Rare Diseases and Orphan Drugs 2018
Author : ConferenceSeries
Publisher : ConferenceSeries
Release Date : 2021
Category :
Total pages :80
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June 11-12, 2018 | Dublin, Ireland Key Topics : Neglected Tropical Diseases, Rare Pulmonary Diseases, Rare Diseases in Neurology, Rare Genetic Diseases, Scope of Orphan Drugs, Rare diseases of Endocrine System, Rare diseases of Immune System, Rare Cardiac Diseases, Rare Eye and Ear Diseases, Orphan Drugs Treatment for Rare Diseases, Rare Oral Diseases, Rare Hepatic Diseases, Rare Gastrointestinal Diseases, Rare Bacterial, Viral and Fungal infections, Rare diseases of Genitourinary System, Rare diseases in Nephrology, Rare Skin Diseases, Clinical Research on Orphan Drugs, Rare Morphological Diseases, Development of Orphan Products, Rare Diseases in Oncology, Rare Diseases in Anaesthesiology, Rare Diseases in Haematology, Orphan Drugs Market Research, Rare Gynaecological and Obstetrical Diseases, Pediatric Rare Diseases, Current Rare Diseases Research, Rare Diseases of Sexual Health, Rare Hereditary Diseases, Diagnosis and Treatment for Rare Diseases, Clinical case studies on Rare Diseases, Imaging of Rare Diseases, Other Rare Diseases,

Orphan Drugs and Rare Diseases

Orphan Drugs and Rare Diseases
Author : David C Pryde,Michael J Palmer
Publisher : Royal Society of Chemistry
Release Date : 2014-07-30
Category : Science
Total pages :350
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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.

Rare Diseases Epidemiology: Update and Overview

Rare Diseases Epidemiology: Update and Overview
Author : Manuel Posada de la Paz,Domenica Taruscio,Stephen C. Groft
Publisher : Springer
Release Date : 2017-12-06
Category : Medical
Total pages :667
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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.

Orphan Drugs

Orphan Drugs
Author : Elizabeth Hernberg-Ståhl,Miroslav Reljanović
Publisher : Elsevier
Release Date : 2013-11-15
Category : Medical
Total pages :334
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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

Orphan Drugs in Development for Rare Diseases

Orphan Drugs in Development for Rare Diseases
Author : Anonim
Publisher : Unknown
Release Date : 2011
Category :
Total pages :129
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Value Assessment of Orphan Drugs and Treatments for Rare Diseases

Value Assessment of Orphan Drugs and Treatments for Rare Diseases
Author : Catherine M. Lockhart
Publisher : Unknown
Release Date : 2016
Category :
Total pages :69
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OBJECTIVES: In 1983 the US Orphan Drug Act was passed to facilitate commercialization of drugs to treat rare diseases. The market value for orphan drugs in the US reached $90 billion annually in 2014, with worldwide sales forecast at $176 billion. Payers and policymakers need robust methodology for evaluation of health technology in this growing landscape of expensive treatments for rare diseases. Here I present a systematic review of current practices in value-based evaluation of orphan drugs from a global perspective. I also propose a potential new framework to be developed as new metric for assessing the value of orphan drugs, the Orphan Drug Index Estimate (ODIE). METHODS: For the systematic review, searches were conducted in December 2015 in PubMed®, EMBASE®, and Web of Science® databases using the following keywords: orphan drug, rare disease treatment, economics, resource utilization, cost, cost effectiveness, questionnaire, and value. Only references published in English were included. Manuscripts that solely reported one of the following were excluded: clinical or patient care, policy or legislation on orphan drugs particularly relating to research incentive, opinion or editorial, preclinical studies, drug-development, unrelated to rare diseases or healthcare, reviews other than systematic reviews for health technology assessment. RESULTS: A total of 2513 unique references were obtained, and screened by title and abstract according to exclusion criteria. After exclusion, 333 references remained for full evaluation. Of those, an additional 296 were excluded, but 51 additional studies were included from the reference lists of included articles. A total of 88 articles were included in the complete analysis. Overall, the methodology employed for conducting cost-effectiveness assessments followed traditional techniques including decision analysis and Markov modeling techniques. The reported incremental cost effectiveness ratios (ICERs) ranged from dominant treatments to a high of €6.1 million per quality adjusted life year (QALY). Interpretation of the results was more challenging, with 43% of studies reporting ICERs that would not be considered cost-effective under a willingness-to-pay threshold of $50,000 per QALY. In spite of the lack of cost-effectiveness, the majority of authors agreed that since the treatment under review is for a rare condition, there is an obligation to cover the costs. In light of these analyses, there is an evident need for a method of analysis that is more comprehensive than the ICER, and more appropriate for addressing the uniqueness of orphan drugs, including variables related to the rarity and severity of disease, and a broader societal perspective on costs, including societal burden and identifiable opportunity costs. In response, here I propose a potential new metric based on multicriteria decision analysis (MCDA) techniques to provide a more comprehensive evaluation of orphan drugs. CONCLUSIONS: There is a global consensus of a need to develop appropriate methodology, analysis techniques, and related policies to address management of expensive treatments. It is not yet clear how best to evaluate the value of orphan drugs. More thorough evaluation and validation of novel modeling techniques, analytic rationale and proactive policy changes are needed to redefine the status quo of health technology assessment of rare disease treatments. I propose a new metric to overcome some limitations of the ICER in evaluation of rare diseases. Continued research is needed in detailed development of a valid, quantifiable, and reproducible metric; however, the work presented here provides a foundation for the development process.

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 : National Academies Press
Release Date : 2011-04-03
Category : Medical
Total pages :442
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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.

Rare Diseases in the Age of Health 2.0

Rare Diseases in the Age of Health 2.0
Author : Rajeev K. Bali,Lodewijk Bos,Michael Christopher Gibbons,Simon Ibell
Publisher : Springer Science & Business Media
Release Date : 2013-10-21
Category : Technology & Engineering
Total pages :292
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This text focuses on various factors associated with orphan diseases and the influence and role of health information technologies. Orphan diseases have not been adopted by the pharmaceutical industry because they provide little financial incentive to treat or prevent it. It is estimated that 6,000-7,000 orphan diseases exist today; as medical knowledge continues to expand, this number is likely to become much greater. The book highlights the opportunities and challenges in this increasingly important area. The book explores new avenues which are opened by information technologies and Health 2.0, and highlights also economic opportunities of orphan disease medicine. The editors of this new book have international experience and competencies in the key areas of patient empowerment, healthcare and clinical knowledge management, healthcare inequalities and disparities, rare diseases and patient advocacy.

Rare Diseases and Orphan Drugs

Rare Diseases and Orphan Drugs
Author : Istituto superiore di sanità (Italy)
Publisher : Unknown
Release Date : 2007
Category :
Total pages :184
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The Social Construction of the Orphan Drug Industry

The Social Construction of the Orphan Drug Industry
Author : Qing Ying Low (Timothy)
Publisher : Unknown
Release Date : 2018
Category : Orphan drugs
Total pages :70
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"Sociologists have noted that markets are not always formed "naturally", and the creation of markets would require social actors such as the legislator to legitimate it and policies by the state to support its establishment. Using legitimation and choice-within-constraints framework, this would also seem to be the case for the orphan drug industry. Due to the nature of orphan drug being used to treat a small population of patients with rare diseases, legislators in the United States have created new justifications to pass the Orphan Drug Act in 1983. While the act has encouraged pharmaceutical companies to develop orphan drugs, scholars have noted that little has been done to reduce orphan drug prices to ensure that patients have access to these drugs. This paper will argue that during the legitimation of the Orphan Drug Act, legislators have been committed and constrained to a set of "free market" ideas when debating and proposing solutions. The justifications for the orphan drug act had generally been embedded within "free market" ideas. While there were instances that legislators used arguments such as "basic human right" to pass the act, these justifications became less relevant in future debates. Justification tied to "free market" ideas that were created for the orphan drug act has also become constraints to future legislators, resulting in Congress to pay little attention to the issue of high orphan drug prices and access of orphan drug for rare disease patient" -- Abstract.

Workshop

Workshop
Author : Marco Salvatore,Domenica Taruscio
Publisher : Unknown
Release Date : 2007
Category :
Total pages :134
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Physicians' Guide to Rare Diseases

Physicians' Guide to Rare Diseases
Author : Jess G. Thoene,National Organization for Rare Disorders
Publisher : Dowden Publishing Company
Release Date : 1995
Category : Medical
Total pages :915
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This classic reference, now with almost 900 disease descriptions, is edited by nationally known pediatrician Jess G. Thoene, M.D. & provides a quick, organized approach to identifying puzzling & unusual symptoms, giving patients the opportunity for early diagnosis & treatment. Special features include: * A full color atlas of visual clues, cross referenced to disease listings. * Thirteen separate specialty sections with an expert overview to introduce each section. Eminent clinicians from the outstanding research centers in rare diseases give you the current status of research & practical approaches to diagnosis & therapy of the rare diseases in that specialty. * Full reference to more than 250 orphan drugs. Each drug is cross referenced to the appropriate disease description. * Clear language for the Layperson. Though written for physicians, the descriptions of diseases, symptoms & treatments are clear & understandable for patients, educators & others outside the medical profession. Each disease description provides symptomatology, etiology, affected population & a comparison of related disorders. Each disease description contains a list of other resources to consult: voluntary agencies, units of the National Institutes of Health, research centers around the country, etc. Addresses & phone numbers are provided in a central reference. Library Journal: "...PHYSICIAN'S GUIDE...is recommended for medical collections & public libraries with consumer health collections."