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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

Orphan Drugs

Orphan Drugs
  • Author : Elizabeth Hernberg-Ståhl,Miroslav Reljanovic
  • Publisher :Unknown
  • Release Date :2018-11-13
  • Total pages :334
  • ISBN : 0081013590
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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

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.

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.

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
  • Total pages :229
  • ISBN : UOM:39015004895994
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Summary :

Drug Regulation Reform--oversight: Orphan drugs

Drug Regulation Reform--oversight: Orphan drugs
  • Author : United States. Congress. House. Committee on Interstate and Foreign Commerce. Subcommittee on Health and the Environment
  • Publisher :Unknown
  • Release Date :1980
  • Total pages :229
  • ISBN : SRLF:AA0000513267
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Summary :

Report of the National Conference on Orphan Drugs

Report of the National Conference on Orphan Drugs
  • Author : Anonim
  • Publisher :Unknown
  • Release Date :1989
  • Total pages :12
  • ISBN : UCR:31210023584608
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Summary :

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 Drug Reauthorization

Orphan Drug Reauthorization
  • Author : United States,United States. Congress. House. Committee on Energy and Commerce. Subcommittee on Health and the Environment
  • Publisher :Unknown
  • Release Date :1994
  • Total pages :117
  • ISBN : UCR:31210013511751
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Summary :

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 :

Report of the National Commission on Orphan Diseases

Report of the National Commission on Orphan Diseases
  • Author : National Commission on Orphan Diseases (U.S.)
  • Publisher :Unknown
  • Release Date :1989
  • Total pages :106
  • ISBN : UCR:31210024950451
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Summary :

Orphan Drugs

Orphan Drugs
  • Author : Carolyn H. Asbury
  • Publisher :Unknown
  • Release Date :1985
  • Total pages :219
  • ISBN : UOM:39015009529093
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Summary :

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
  • Total pages :204
  • ISBN : UOM:39015009554349
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Summary :

Orphan Drugs

Orphan Drugs
  • Author : Karen Miller Allen
  • Publisher :Unknown
  • Release Date :1983
  • Total pages :12
  • ISBN : STANFORD:36105039619114
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Summary :

Orphan Drugs and Orphan Diseases

Orphan Drugs and Orphan Diseases
  • Author : George J. Brewer
  • Publisher :Unknown
  • Release Date :1983
  • Total pages :263
  • ISBN : UOM:39015004420660
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Summary :

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.

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
  • Total pages :70
  • ISBN : OCLC:1124575368
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Summary : "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.

Cardiology Drug Facts

Cardiology Drug Facts
  • Author : Ezra A. Amsterdam,Jonathan Abrams
  • Publisher :Unknown
  • Release Date :1989
  • Total pages :573
  • ISBN : UOM:39015017720999
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Summary :

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.

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.

Pediatric Drug Development

Pediatric Drug Development
  • Author : Andrew E. Mulberg,Dianne Murphy,Julia Dunne,Lisa L. Mathis
  • Publisher :Unknown
  • Release Date :2013-05-20
  • Total pages :624
  • ISBN : 9781118312056
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Summary : Pediatric Drug Development, Second Edition, encompasses the new regulatory initiatives across EU, US and ROW designed to encourage improved access to safe and effective medicines for children. It includes new developments in biomarkers and surrogate endpoints, developmental pharmacology and other novel aspects of pediatric drug development.