Orphan Drugs

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

Produk Detail:

  • Author : Elizabeth Hernberg-Ståhl
  • Publisher : Elsevier
  • Pages : 334 pages
  • ISBN : 1908818395
  • Rating : 4/5 from 21 reviews
CLICK HERE TO GET THIS BOOKOrphan Drugs

Orphan Drugs

Orphan Drugs
  • Author : Elizabeth Hernberg-Ståhl,Miroslav Reljanović
  • Publisher : Elsevier
  • Release : 15 November 2013
GET THIS BOOKOrphan Drugs

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

Rare Diseases and Orphan Drugs

Rare Diseases and Orphan Drugs
  • Author : Jules J. Berman
  • Publisher : Academic Press
  • Release : 26 May 2014
GET THIS BOOKRare Diseases and Orphan Drugs

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

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 : 03 April 2011
GET THIS BOOKRare Diseases and Orphan Products

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 in epilepsy

Orphan drugs in epilepsy
  • Author : M. Nikanorova,P. Genton,S. I. Johannessen,C. Johannessen Landmark
  • Publisher : John Libbey Eurotext
  • Release : 18 September 2013
GET THIS BOOKOrphan drugs in epilepsy

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

Rare Diseases and Orphan Drugs

Rare Diseases and Orphan Drugs
  • Author : Jules J. Berman
  • Publisher : Academic Press
  • Release : 02 June 2014
GET THIS BOOKRare Diseases and Orphan Drugs

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

Orphan Drugs and Rare Diseases

Orphan Drugs and Rare Diseases
  • Author : David C Pryde,Michael J Palmer
  • Publisher : Royal Society of Chemistry
  • Release : 30 July 2014
GET THIS BOOKOrphan Drugs and Rare Diseases

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

Searching for Magic Bullets

Searching for Magic Bullets
  • Author : Lisa A Basara,Michael E Montagne
  • Publisher : CRC Press
  • Release : 10 August 1994
GET THIS BOOKSearching for Magic Bullets

Searching for Magic Bullets reveals the quest of consumers, health professionals, and drug developers to find safer and faster methods of bringing new medications to the marketplace. Authors Basara and Montagne explore the current drug development and approval processes, their strengths and weaknesses, and the mechanisms by which patients and organizations evade these processes. Readers learn about the fundamentals of traditional and nontraditional drug discovery and development as they occur in the U.S., as well as the views of

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 Publisher
  • Release : 16 June 2021
GET THIS BOOKValue Assessment of Orphan Drugs and Treatments for Rare Diseases

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

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 : 16 June 2021
GET THIS BOOKProceedings of 4th World Congress on Rare Diseases and Orphan Drugs 2018

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

The Social Construction of the Orphan Drug Industry

The Social Construction of the Orphan Drug Industry
  • Author : Qing Ying Low (Timothy)
  • Publisher : Unknown Publisher
  • Release : 16 June 2021
GET THIS BOOKThe Social Construction of the Orphan Drug Industry

"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