Rare Diseases and Orphan Drugs

Book Rare Diseases and Orphan Drugs Cover

Read or download book entitled Rare Diseases and Orphan Drugs written by Jules J. Berman and published by Academic Press in PDF, EPUB and Kindle Format. Click Get This Book button to download or read online books. Join over 650.000 happy Readers and READ as many books as you like. We cannot guarantee that Rare Diseases and Orphan Drugs book is available in the library.

  • Publisher : Academic Press
  • Release : 26 May 2014
  • ISBN : 9780124200098
  • Page : 400 pages
  • Rating : 4.5/5 from 103 voters

Download Rare Diseases and Orphan Drugs in PDF, Epub and Kindle

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.

GET THIS BOOK

Rare Diseases and Orphan Drugs

Rare Diseases and Orphan Drugs
  • Author : Jules J. Berman
  • Publisher : Academic Press
  • Release Date : 2014-05-26
  • ISBN : 9780124200098
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 Date : 2014
  • ISBN : 9781849738064
GET THIS BOOKOrphan Drugs and Rare Diseases

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 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
  • ISBN : 9780309158060
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

Orphan Drugs
  • Author : Elizabeth Hernberg-Ståhl,Miroslav Reljanović
  • Publisher : Elsevier
  • Release Date : 2013-11-15
  • ISBN : 9781908818393
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

Innovative Methods for Rare Disease Drug Development

Innovative Methods for Rare Disease Drug Development
  • Author : Shein-Chung Chow
  • Publisher : CRC Press
  • Release Date : 2020-11-11
  • ISBN : 9781000208337
GET THIS BOOKInnovative Methods for Rare Disease Drug Development

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

Rare Disease Drug Development

Rare Disease Drug Development
  • Author : Raymond A. Huml, MS, DVM
  • Publisher : Springer
  • Release Date : 2021-10-09
  • ISBN : 3030786048
GET THIS BOOKRare Disease Drug Development

This book provides a broad overview of rare disease drug development. It offers unique insights from various perspectives, including third-party capital providers, caregivers, patient advocacy groups, drug development professionals, marketing and commercial experts, and patients. A unique reference, the book begins with narratives on the many challenges faced by rare disease patient and their caregivers. Subsequent chapters underscore the critical, multidimensional role of patient advocacy groups and the novel approaches to related clinical trials, investment decisions, and the optimization of

Rare Diseases Epidemiology: Update and Overview

Rare Diseases Epidemiology: Update and Overview
  • Author : Manuel Posada de la Paz,Domenica Taruscio,Stephen C. Groft
  • Publisher : Springer
  • Release Date : 2017-12-06
  • ISBN : 9783319671444
GET THIS BOOKRare Diseases Epidemiology: Update and Overview

The fields of rare diseases research and orphan products development continue to expand with more products in research and development status. In recent years, the role of the patient advocacy groups has evolved into a research partner with the academic research community and the bio-pharmaceutical industry. Unique approaches to research and development require epidemiological data not previously available to assist in protocol study design and patient recruitment for clinical trials required by regulatory agencies prior to approval for access by

NORD Guide to Rare Disorders

NORD Guide to Rare Disorders
  • Author : National Organization for Rare Disorders
  • Publisher : Lippincott Williams & Wilkins
  • Release Date : 2003
  • ISBN : 0781730635
GET THIS BOOKNORD Guide to Rare Disorders

NORD Guide to Rare Disorders is a comprehensive, practical, authoritative guide to the diagnosis and management of more than 800 rare diseases. The diseases are discussed in a uniform, easy-to-follow format--a brief description, signs and symptoms, etiology, related disorders, epidemiology, standard treatment, investigational treatment, resources, and references.The book includes a complete directory of orphan drugs, a full-color atlas of visual diagnostic signs, and a Master Resource List of support groups and helpful organizations. An index of symptoms and key words

Rare Diseases

Rare Diseases
  • Author : Nicolas Sireau
  • Publisher : Routledge
  • Release Date : 2017-09-08
  • ISBN : 9781351278386
GET THIS BOOKRare Diseases

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

Orphan Lung Diseases

Orphan Lung Diseases
  • Author : J.-F. Cordier
  • Publisher : European Respiratory Society
  • Release Date : 2014-05-14
  • ISBN : 9781849840149
GET THIS BOOKOrphan Lung Diseases

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

Global Pediatric Development of Drugs, Biologics, and Medical Devices

Global Pediatric Development of Drugs, Biologics, and Medical Devices
  • Author : Jocelyn Jennings,Linda McBride
  • Publisher : Unknown
  • Release Date : 2021-11-26
  • ISBN : 1947493736
GET THIS BOOKGlobal Pediatric Development of Drugs, Biologics, and Medical Devices

Rare Diseases Epidemiology

Rare Diseases Epidemiology
  • Author : Manuel Posada de la Paz,Stephen C. Groft
  • Publisher : Springer
  • Release Date : 2012-11-07
  • ISBN : 9400733380
GET THIS BOOKRare Diseases Epidemiology

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

Introduction to Basics of Pharmacology and Toxicology

Introduction to Basics of Pharmacology and Toxicology
  • Author : Gerard Marshall Raj,Ramasamy Raveendran
  • Publisher : Springer Nature
  • Release Date : 2019-11-16
  • ISBN : 9789813297791
GET THIS BOOKIntroduction to Basics of Pharmacology and Toxicology

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

Orphan Drugs and Rare Diseases

Orphan Drugs and Rare Diseases
  • Author : David C Pryde,Michael J Palmer
  • Publisher : Royal Society of Chemistry
  • Release Date : 2014-07-30
  • ISBN : 9781782624202
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

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
  • Release Date : 2016
  • ISBN : OCLC:954774773
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