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Orphan Drugs

Orphan Drugs
Understanding the Rare Disease Market and its Dynamics

by Elizabeth Hernberg-Ståhl,Miroslav Reljanović

  • Publisher : Elsevier
  • Release : 2013-11-15
  • Pages : 334
  • ISBN : 1908818395
  • Language : En, Es, Fr & De
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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

Rare Diseases and Orphan Drugs

Rare Diseases and Orphan Drugs
Keys to Understanding and Treating the Common Diseases

by Jules J. Berman

  • Publisher : Academic Press
  • Release : 2014-05-26
  • Pages : 400
  • ISBN : 0124200095
  • Language : En, Es, Fr & De
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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

Orphan drugs
A Book

by United States. Congress. House. Committee on Interstate and Foreign Commerce. Subcommittee on Health and the Environment

  • Publisher : Unknown Publisher
  • Release : 1980
  • Pages : 329
  • ISBN : 9876543210XXX
  • Language : En, Es, Fr & De
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Orphan Drugs and Rare Diseases

Orphan Drugs and Rare Diseases

by David C Pryde,Michael J Palmer

  • Publisher : Royal Society of Chemistry
  • Release : 2014
  • Pages : 486
  • ISBN : 1849738068
  • Language : En, Es, Fr & De
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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.

Drug Regulation Reform--oversight: Orphan drugs

Drug Regulation Reform--oversight: Orphan drugs
A Book

by United States. Congress. House. Committee on Interstate and Foreign Commerce. Subcommittee on Health and the Environment

  • Publisher : Unknown Publisher
  • Release : 1980
  • Pages : 329
  • ISBN : 9876543210XXX
  • Language : En, Es, Fr & De
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Report of the National Conference on Orphan Drugs

Report of the National Conference on Orphan Drugs
The Path to Promise : May 18-19, 1988, George Washington University, Washington

by Anonim

  • Publisher : Unknown Publisher
  • Release : 1989
  • Pages : 12
  • ISBN : 9876543210XXX
  • Language : En, Es, Fr & De
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Rare Diseases and Orphan Products

Rare Diseases and Orphan Products
Accelerating Research and Development

by Institute of Medicine,Board on Health Sciences Policy,Committee on Accelerating Rare Diseases Research and Orphan Product Development

  • Publisher : National Academies Press
  • Release : 2011-04-03
  • Pages : 442
  • ISBN : 0309158060
  • Language : En, Es, Fr & De
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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.

Anticompetitive Abuse of the Orphan Drug Act

Anticompetitive Abuse of the Orphan Drug Act
Invitation to High Prices : Hearing Before the Subcommittee on Antitrust, Monopolies, and Business Rights of the Committee on the Judiciary, United States Senate, One Hundred Second Congress, Second Session, on S. 2060, a Bill to Revise the Orphan Drug Provisions of the Federal Food, Drug, and Cosmetic Act, the Public Health Service Act, and the Orphan Drug Act, and for Other Purposes, January 21, 1992

by United States. Congress. Senate. Committee on the Judiciary. Subcommittee on Antitrust, Monopolies, and Business Rights,United States

  • Publisher : Unknown Publisher
  • Release : 1992
  • Pages : 319
  • ISBN : 9876543210XXX
  • Language : En, Es, Fr & De
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Orphan Drug Reauthorization

Orphan Drug Reauthorization
Hearing Before the Subcommittee on Health and the Environment of the Committee on Energy and Commerce, House of Representatives, One Hundred Third Congress, Second Session on H.R. 4160, a Bill to Amend the Federal Food, Drug, and Cosmetic Act, the Public Health Service Act, and the Orphan Drug Act to Revise the Provisions of Such Acts Relating to Orphan Drugs, June 16, 1994

by United States,United States. Congress. House. Committee on Energy and Commerce. Subcommittee on Health and the Environment

  • Publisher : Unknown Publisher
  • Release : 1994
  • Pages : 117
  • ISBN : 9876543210XXX
  • Language : En, Es, Fr & De
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Orphan Diseases and Orphan Drugs

Orphan Diseases and Orphan Drugs
A Book

by I. Herbert Scheinberg,J. M. Walshe

  • Publisher : Manchester University Press
  • Release : 1986
  • Pages : 228
  • ISBN : 9780719022968
  • Language : En, Es, Fr & De
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Pharmaceutical Innovation and Orphan Drugs

Pharmaceutical Innovation and Orphan Drugs
The Effects of the 1983 Orphan Drug Act

by Natalie Denise Reaves

  • Publisher : Unknown Publisher
  • Release : 1996
  • Pages : 80
  • ISBN : 9876543210XXX
  • Language : En, Es, Fr & De
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Orphan Drugs

Orphan Drugs
Medical Versus Market Value

by Carolyn H. Asbury

  • Publisher : Free Press
  • Release : 1985
  • Pages : 219
  • ISBN : 9876543210XXX
  • Language : En, Es, Fr & De
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Cooperative Approaches to Research and Development of Orphan Drugs

Cooperative Approaches to Research and Development of Orphan Drugs
Proceedings of a Conference in New York, New York, April 9 and 10, 1984

by Eunyong Chung

  • Publisher : Unknown Publisher
  • Release : 1985
  • Pages : 204
  • ISBN : 9876543210XXX
  • Language : En, Es, Fr & De
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Orphan Drugs and Orphan Diseases

Orphan Drugs and Orphan Diseases
Clinical Realities and Public Policy : Proceedings of a Conference Held in Ann Arbor, Michigan, September 27-29, 1982

by George J. Brewer

  • Publisher : Unknown Publisher
  • Release : 1983
  • Pages : 263
  • ISBN : 9876543210XXX
  • Language : En, Es, Fr & De
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Orphan Drugs

Orphan Drugs
A Bibliography of Publications about Drugs of Limited Commercial Value

by Karen Miller Allen

  • Publisher : Unknown Publisher
  • Release : 1983
  • Pages : 12
  • ISBN : 9876543210XXX
  • Language : En, Es, Fr & De
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Rare Diseases and Orphan Drugs

Rare Diseases and Orphan Drugs
Keys to Understanding and Treating the Common Diseases

by Jules J. Berman

  • Publisher : Academic Press
  • Release : 2014-05-26
  • Pages : 400
  • ISBN : 0124200095
  • Language : En, Es, Fr & De
GET BOOK

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.

Innovative Methods for Rare Disease Drug Development

Innovative Methods for Rare Disease Drug Development
A Book

by Shein-Chung Chow

  • Publisher : CRC Press
  • Release : 2020-11-12
  • Pages : 306
  • ISBN : 1000208214
  • Language : En, Es, Fr & De
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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.

Orphan Drugs and Rare Diseases

Orphan Drugs and Rare Diseases

by David C Pryde,Michael J Palmer

  • Publisher : Royal Society of Chemistry
  • Release : 2014
  • Pages : 486
  • ISBN : 1849738068
  • Language : En, Es, Fr & De
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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 drugs in epilepsy

Orphan drugs in epilepsy

by M. Nikanorova,P. Genton,S. I. Johannessen,C. Johannessen Landmark

  • Publisher : John Libbey Eurotext
  • Release : 2013-09-18
  • Pages : 112
  • ISBN : 2742012354
  • Language : En, Es, Fr & De
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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.

Cardiology Drug Facts

Cardiology Drug Facts
A Book

by Ezra A. Amsterdam,Jonathan Abrams

  • Publisher : Facts & Comparisons
  • Release : 1989
  • Pages : 573
  • ISBN : 9876543210XXX
  • Language : En, Es, Fr & De
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