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Orphan Drugs

Orphan Drugs Book
Author : Elizabeth Hernberg-Ståhl,Miroslav Reljanović
Publisher : Elsevier
Release : 2013-11-15
ISBN : 1908818395
Language : En, Es, Fr & De

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Book Description :

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 Book
Author : Jules J. Berman
Publisher : Academic Press
Release : 2014-05-26
ISBN : 0124200095
Language : En, Es, Fr & De

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Book Description :

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.

Rare Diseases and Orphan Products

Rare Diseases and Orphan Products Book
Author : 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
ISBN : 0309158060
Language : En, Es, Fr & De

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Book Description :

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 Diseases and Orphan Drugs

Orphan Diseases and Orphan Drugs Book
Author : I. Herbert Scheinberg,J. M. Walshe
Publisher : Manchester University Press
Release : 1989-04
ISBN : 9780719022968
Language : En, Es, Fr & De

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Book Description :

Download Orphan Diseases and Orphan Drugs book written by I. Herbert Scheinberg,J. M. Walshe, available in PDF, EPUB, and Kindle, or read full book online anywhere and anytime. Compatible with any devices.

Global Pediatric Development of Drugs Biologics and Medical Devices

Global Pediatric Development of Drugs  Biologics  and Medical Devices Book
Author : Jocelyn Jennings,Linda McBride
Publisher : Unknown
Release : 2021-11-26
ISBN : 9781947493735
Language : En, Es, Fr & De

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Book Description :

Download Global Pediatric Development of Drugs Biologics and Medical Devices book written by Jocelyn Jennings,Linda McBride, available in PDF, EPUB, and Kindle, or read full book online anywhere and anytime. Compatible with any devices.

Orphan Drugs and Rare Diseases

Orphan Drugs and Rare Diseases Book
Author : David C Pryde,Michael J Palmer
Publisher : Royal Society of Chemistry
Release : 2014-07-30
ISBN : 1782624201
Language : En, Es, Fr & De

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Book Description :

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.

Pediatric Drug Development

Pediatric Drug Development Book
Author : Andrew E. Mulberg,Dianne Murphy,Julia Dunne,Lisa L. Mathis
Publisher : John Wiley & Sons
Release : 2013-05-20
ISBN : 1118312058
Language : En, Es, Fr & De

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Book Description :

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.

Breakthrough Business Models

Breakthrough Business Models Book
Author : Institute of Medicine,Board on Health Sciences Policy,Forum on Drug Discovery, Development, and Translation,Robert Giffin,Sally Robinson,Theresa Wizemann
Publisher : National Academies Press
Release : 2009-02-17
ISBN : 0309178169
Language : En, Es, Fr & De

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Book Description :

The process for developing new drug and biologic products is extraordinarily expensive and time-consuming. Although large pharmaceutical companies may be able to afford the cost of development because they can expect a large return on investment, organizations developing drugs to treat rare and neglected diseases are unable to rely on such returns. On June 23, 2008, the Institute of Medicine's Forum on Drug Discovery, Development, and Translation held a public workshop, "Breakthrough Business Models: Drug Development for Rare and Neglected Diseases and Individualized Therapies," which sought to explore new and innovative strategies for developing drugs for rare and neglected diseases.

Innovative Methods for Rare Disease Drug Development

Innovative Methods for Rare Disease Drug Development Book
Author : Shein-Chung Chow
Publisher : CRC Press
Release : 2020-11-11
ISBN : 1000208338
Language : En, Es, Fr & De

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Book Description :

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 Drug Amendments of 1991

Orphan Drug Amendments of 1991 Book
Author : United States,United States. Congress. Senate. Committee on Labor and Human Resources
Publisher : Unknown
Release : 1992
ISBN : 0987650XXX
Language : En, Es, Fr & De

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Book Description :

Download Orphan Drug Amendments of 1991 book written by United States,United States. Congress. Senate. Committee on Labor and Human Resources, available in PDF, EPUB, and Kindle, or read full book online anywhere and anytime. Compatible with any devices.

Anticompetitive Abuse of the Orphan Drug Act

Anticompetitive Abuse of the Orphan Drug Act Book
Author : United States,United States. Congress. Senate. Committee on the Judiciary. Subcommittee on Antitrust, Monopolies, and Business Rights
Publisher : Unknown
Release : 1992
ISBN : 0987650XXX
Language : En, Es, Fr & De

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Book Description :

Download Anticompetitive Abuse of the Orphan Drug Act book written by United States,United States. Congress. Senate. Committee on the Judiciary. Subcommittee on Antitrust, Monopolies, and Business Rights, available in PDF, EPUB, and Kindle, or read full book online anywhere and anytime. Compatible with any devices.

Drug Repurposing

Drug Repurposing Book
Author : Farid A. Badria
Publisher : BoD – Books on Demand
Release : 2020-12-02
ISBN : 1839685204
Language : En, Es, Fr & De

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Book Description :

Drug repurposing or drug repositioning is a new approach to presenting new indications for common commercial and clinically approved existing drugs. For example, chloroquine, an old antimalarial drug, showed promising results for treating COVID-19, interfering with MDR in several types of cancer, and chemosensitizing human leukemic cells.This book focuses on the hypothesis, risk/benefits, and economic impacts of drug repurposing on drug discovery in dermatology, infectious diseases, neurological disorders, cancer, and orphan diseases. It brings together up-to-date research to provide readers with an informative, illustrative, and easy-to-read book useful for students, clinicians, and the pharmaceutical industry.

Rare Diseases

Rare Diseases Book
Author : Nicolas Sireau
Publisher : Routledge
Release : 2017-09-08
ISBN : 135127838X
Language : En, Es, Fr & De

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Book Description :

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.

Orphan Drug Act

Orphan Drug Act Book
Author : United States. Congress. House. Committee on Energy and Commerce. Subcommittee on Health and the Environment
Publisher : Unknown
Release : 1990
ISBN : 0987650XXX
Language : En, Es, Fr & De

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Book Description :

Download Orphan Drug Act book written by United States. Congress. House. Committee on Energy and Commerce. Subcommittee on Health and the Environment, available in PDF, EPUB, and Kindle, or read full book online anywhere and anytime. Compatible with any devices.

Orphan Drug Reauthorization

Orphan Drug Reauthorization Book
Author : United States,United States. Congress. House. Committee on Energy and Commerce. Subcommittee on Health and the Environment
Publisher : Unknown
Release : 1994
ISBN : 0987650XXX
Language : En, Es, Fr & De

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Book Description :

Download Orphan Drug Reauthorization book written by United States,United States. Congress. House. Committee on Energy and Commerce. Subcommittee on Health and the Environment, available in PDF, EPUB, and Kindle, or read full book online anywhere and anytime. Compatible with any devices.

Introduction to Basics of Pharmacology and Toxicology

Introduction to Basics of Pharmacology and Toxicology Book
Author : Gerard Marshall Raj,Ramasamy Raveendran
Publisher : Springer Nature
Release : 2019-11-16
ISBN : 9813297794
Language : En, Es, Fr & De

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Book Description :

This book illustrates, in a comprehensive manner, the most crucial principles involved in pharmacology and allied sciences. The title begins by discussing the historical aspects of drug discovery, with up to date knowledge on Nobel Laureates in pharmacology and their significant discoveries. It then examines the general pharmacological principles - pharmacokinetics and pharmacodynamics, with in-depth information on drug transporters and interactions. In the remaining chapters, the book covers a definitive collection of topics containing essential information on the basic principles of pharmacology and how they are employed for the treatment of diseases. Readers will learn about special topics in pharmacology that are hard to find elsewhere, including issues related to environmental toxicology and the latest information on drug poisoning and treatment, analytical toxicology, toxicovigilance, and the use of molecular biology techniques in pharmacology. The book offers a valuable resource for researchers in the fields of pharmacology and toxicology, as well as students pursuing a degree in or with an interest in pharmacology.

Orphan Drug Law Matures into Medical Mainstay

Orphan Drug Law Matures into Medical Mainstay Book
Author : Anonim
Publisher : DIANE Publishing
Release : 2022-08-08
ISBN : 9781422326770
Language : En, Es, Fr & De

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Book Description :

Download Orphan Drug Law Matures into Medical Mainstay book written by , available in PDF, EPUB, and Kindle, or read full book online anywhere and anytime. Compatible with any devices.

Orphan Lung Diseases

Orphan Lung Diseases Book
Author : J.-F. Cordier
Publisher : European Respiratory Society
Release : 2014-05-14
ISBN : 1849840148
Language : En, Es, Fr & De

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Book Description :

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.

The Waxman Report

The Waxman Report Book
Author : Henry Waxman
Publisher : Twelve
Release : 2009-07-02
ISBN : 0446545678
Language : En, Es, Fr & De

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Book Description :

At a time when some of the most sweeping national initiatives in decades are being debated, Congressman Henry Waxman offers a fascinating inside account of how Congress really works by describing the subtleties and complexities of the legislative process. For four decades, Waxman has taken visionary and principled positions on crucial issues and been a driving force for change. Because of legislation he helped champion, our air is cleaner, our food is safer, and our medical care better. Thanks to his work as a top watchdog in Congress, crucial steps have been taken to curb abuses on Wall Street, to halt wasteful spending in Iraq, and to ban steroids from Major League Baseball. Few legislators can match his accomplishments or his insights on how good work gets done in Washington. In this book, Waxman affords readers a rare glimpse into how this is achieved-the strategy, the maneuvering, the behind-the-scenes deals. He shows how the things we take for granted (clear information about tobacco's harmfulness, accurate nutritional labeling, important drugs that have saved countless lives) started out humbly-derided by big business interests as impossible or even destructive. Sometimes, the most dramatic breakthroughs occur through small twists of fate or the most narrow voting margin. Waxman's stories are surprising because they illustrate that while government's progress may seem glacial, much is happening, and small battles waged over years can yield great results. At a moment when so much has been written about what's wrong with Congress-the grid­lock, the partisanship, the influence of interest groups-Henry Waxman offers sophisticated, concrete examples of how govern­ment can (and should) work.

Rare Diseases Epidemiology

Rare Diseases Epidemiology Book
Author : Manuel Posada de la Paz,Stephen C. Groft
Publisher : Springer
Release : 2012-11-07
ISBN : 9789400733381
Language : En, Es, Fr & De

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Book Description :

In our etiologic research, we epidemiologists need to leave behind the concepts of ‘cohort’ study and ‘case–control’ study and adopt that of the etiologic study as the singular substitute for these. With this sentence, the famous epidemiologist Professor Olli S. Miettinen began his personal re ection on the future of the epidemiology [1]. He sought to highlight the fact that the role of the epidemiologist should be mainly focused on aetiological research. Nevertheless, the widespread idea still exists that epidemiology is limited to purely providing gures and descriptive data on the frequency and distribution of disease. Indeed, it is more than likely that the precise aim of those rst classic epidemiological steps, i. e. , methods essentially based on describing the distri- tion of a given disease, is still not all that well understood by many scientists, let alone the general public. Such descriptions seek to generate hypotheses and afford explanations for key factors (be these risk factors or the presumable causes th- selves), which might justify differences in terms of persons, time or place and, in turn, ultimately serve to develop preventive measures and/or gain quality-adjusted life years. To restrict the goals of epidemiology to activities exclusively concerned with reporting gures or even complex statistical results is a great mistake, one that renders it dif cult to take full advantage of the epidemiologist’s true role, which is “to study disease determinants and to assess the actual impact of factors involved in their development, distribution and dissemination”.