Orphan Drugs and Rare Diseases Book

Orphan Drugs and Rare Diseases


  • Author : David C Pryde
  • Publisher : Royal Society of Chemistry
  • Release Date : 2014
  • Genre: Medical
  • Pages : 488
  • ISBN 10 : 9781849738064

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Orphan Drugs and Rare Diseases Excerpt :

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 Book

Rare Diseases and Orphan Drugs


  • Author : Jules J. Berman
  • Publisher : Academic Press
  • Release Date : 2014-05-26
  • Genre: Medical
  • Pages : 400
  • ISBN 10 : 9780124200098

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Rare Diseases and Orphan Drugs Excerpt :

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 Book

Rare Diseases and Orphan Products


  • Author : Institute of Medicine
  • Publisher : National Academies Press
  • Release Date : 2011-04-03
  • Genre: Medical
  • Pages : 442
  • ISBN 10 : 9780309158060

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Rare Diseases and Orphan Products Excerpt :

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 Book

Orphan Drugs


  • Author : Elizabeth Hernberg-Ståhl
  • Publisher : Woodhead Publishing
  • Release Date : 2018-11-13
  • Genre: Medical
  • Pages : 334
  • ISBN 10 : 0081013590

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Orphan Drugs Excerpt :

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

Innovative Methods for Rare Disease Drug Development Book

Innovative Methods for Rare Disease Drug Development


  • Author : Shein-Chung Chow
  • Publisher : CRC Press
  • Release Date : 2020-11-12
  • Genre: Mathematics
  • Pages : 306
  • ISBN 10 : 9781000208214

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Innovative Methods for Rare Disease Drug Development Excerpt :

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.

Rare Disease Drug Development Book

Rare Disease Drug Development


  • Author : Raymond A. Huml
  • Publisher : Unknown
  • Release Date : 2021
  • Genre: Uncategoriezed
  • Pages : 0
  • ISBN 10 : 3030786064

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Rare Disease Drug Development Excerpt :

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 disease registries. The book addresses various rare disease drug development processes by disciplines such as oncology, hematology, pediatrics, and gene therapy. Chapters then address the operational aspects of drug development, including approval processes, development accelerations, and market access strategies. The book concludes with reflections on the authors' case for real-world data and evidence generation in orphan medicinal drug development. Rare Disease Drug Development is an expertly written text optimized for biopharmaceutical R&D experts, commercial experts, third-party capital providers, patient advocacy groups, patients, and caregivers.

Orphan Drugs Book

Orphan Drugs


  • Author : Elizabeth Hernberg-Ståhl
  • Publisher : Elsevier
  • Release Date : 2013-11-15
  • Genre: Medical
  • Pages : 334
  • ISBN 10 : 9781908818393

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Orphan Drugs Excerpt :

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

NORD Guide to Rare Disorders Book
Score: 3
From 1 Ratings

NORD Guide to Rare Disorders


  • Author : National Organization for Rare Disorders
  • Publisher : Lippincott Williams & Wilkins
  • Release Date : 2003
  • Genre: Medical
  • Pages : 982
  • ISBN 10 : 0781730635

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NORD Guide to Rare Disorders Excerpt :

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 offers physicians valuable assistance in finding the information they need quickly.

Rare Diseases Epidemiology  Update and Overview Book

Rare Diseases Epidemiology Update and Overview


  • Author : Manuel Posada de la Paz
  • Publisher : Springer
  • Release Date : 2017-12-06
  • Genre: Medical
  • Pages : 667
  • ISBN 10 : 9783319671444

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Rare Diseases Epidemiology Update and Overview Excerpt :

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 patents and practicing physicians.

Rare Diseases Book

Rare Diseases


  • Author : Nicolas Sireau
  • Publisher : Routledge
  • Release Date : 2017-09-08
  • Genre: Business & Economics
  • Pages : 278
  • ISBN 10 : 9781351278386

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Rare Diseases Excerpt :

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 Lung Diseases Book

Orphan Lung Diseases


  • Author : J.-F. Cordier
  • Publisher : European Respiratory Society
  • Release Date : 2014-05-14
  • Genre: Medical
  • Pages : 376
  • ISBN 10 : 9781849840149

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Orphan Lung Diseases Excerpt :

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 Epidemiology Book

Rare Diseases Epidemiology


  • Author : Manuel Posada de la Paz
  • Publisher : Springer
  • Release Date : 2012-11-07
  • Genre: Medical
  • Pages : 542
  • ISBN 10 : 9400733380

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Rare Diseases Epidemiology Excerpt :

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”.

Introduction to Basics of Pharmacology and Toxicology Book

Introduction to Basics of Pharmacology and Toxicology


  • Author : Gerard Marshall Raj
  • Publisher : Springer Nature
  • Release Date : 2019-11-16
  • Genre: Medical
  • Pages : 417
  • ISBN 10 : 9789813297791

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Introduction to Basics of Pharmacology and Toxicology Excerpt :

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 Diseases and Orphan Drugs Book

Orphan Diseases and Orphan Drugs


  • Author : I. Herbert Scheinberg
  • Publisher : Manchester University Press
  • Release Date : 1989-04
  • Genre: Chemotherapy
  • Pages : 244
  • ISBN 10 : 0719022967

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Orphan Diseases and Orphan Drugs Excerpt :