November 24, 2020

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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 and Rare Diseases

Orphan Drugs and Rare Diseases
Author : David C Pryde,Michael J Palmer
Publisher : Royal Society of Chemistry
Release Date : 2014
Category : Medical
Total pages :486
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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 Diseases and Orphan Drugs

Orphan Diseases and Orphan Drugs
Author : I. Herbert Scheinberg,J. M. Walshe
Publisher : Manchester University Press
Release Date : 1986
Category : Chemotherapy
Total pages :228
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Orphan drugs

Orphan drugs
Author : United States. Congress. House. Committee on Interstate and Foreign Commerce. Subcommittee on Health and the Environment
Publisher : Unknown
Release Date : 1980
Category : Drugs
Total pages :129
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Rare Diseases and Orphan Drugs

Rare Diseases and Orphan Drugs
Author : Jules J. Berman
Publisher : Academic Press
Release Date : 2014-05-26
Category : Medical
Total pages :400
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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 drugs in epilepsy

Orphan drugs in epilepsy
Author : M. Nikanorova,P. Genton,S. I. Johannessen,C. Johannessen Landmark
Publisher : John Libbey Eurotext
Release Date : 2013-09-18
Category :
Total pages :112
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All the necessary information on 6 molecules called “orphan drugs” used in the treatment of some epileptic syndromes. More than half of epilepsies start before the age of 20 years and nearly 25% of them are refractory. Two problems arise: - It is essential to treat them so that brain development continues as normally as possible. - There are few drugs available. This work outlines 6 molecules in detail that are specifically used in epileptic encephalopathies. Every aspect of these molecules is discussed, including drug development, indication, efficacy, cost, etc. Drafted by international experts in the field of epileptology, this book provides all the necessary information on orphan drugs and their clinical use.

Pharmaceutical Innovation and Orphan Drugs

Pharmaceutical Innovation and Orphan Drugs
Author : Natalie Denise Reaves
Publisher : Unknown
Release Date : 1996
Category : Drug development
Total pages :80
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Orphan Drug Amendments of 1991

Orphan Drug Amendments of 1991
Author : United States,United States. Congress. Senate. Committee on Labor and Human Resources
Publisher : Unknown
Release Date : 1992
Category : Government publications
Total pages :183
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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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Cooperative Approaches to Research and Development of Orphan Drugs

Cooperative Approaches to Research and Development of Orphan Drugs
Author : Eunyong Chung
Publisher : Unknown
Release Date : 1985
Category : Drug Evaluation
Total pages :204
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Orphan Drugs

Orphan Drugs
Author : Karen Miller Allen
Publisher : Unknown
Release Date : 1983
Category : Drugs
Total pages :12
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Orphan Drug Act

Orphan Drug Act
Author : United States. Congress. House. Committee on Energy and Commerce. Subcommittee on Health and the Environment
Publisher : Unknown
Release Date : 1990
Category : Competition
Total pages :261
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Orphan Drug Law Matures into Medical Mainstay

Orphan Drug Law Matures into Medical Mainstay
Author : Anonim
Publisher : DIANE Publishing
Release Date : 2020
Category :
Total pages :129
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Opportunities in Orphan Drugs

Opportunities in Orphan Drugs
Author : Peter Thornton
Publisher : Unknown
Release Date : 2013
Category :
Total pages :107
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Orphan drugs present opportunities to pharma companies looking to move into niche indications with high unmet needs. Although many successful, profitable orphan drugs (Gleevec, NovoSeven) orphan drug development must be approached carefully.

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.