Rare Diseases And Orphan Products
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Rare Diseases And Orphan Products
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Author : Institute of Medicine
language : en
Publisher: National Academies Press
Release Date : 2011-04-03
Rare Diseases And Orphan Products written by Institute of Medicine and has been published by National Academies Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2011-04-03 with Medical categories.
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.
Rare Diseases And Orphan Drugs
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Author : Jules J. Berman
language : en
Publisher: Academic Press
Release Date : 2014-05-26
Rare Diseases And Orphan Drugs written by Jules J. Berman and has been published by Academic Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2014-05-26 with Medical categories.
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
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Author : Elizabeth Hernberg-Ståhl
language : en
Publisher: Elsevier
Release Date : 2013-11-15
Orphan Drugs written by Elizabeth Hernberg-Ståhl and has been published by Elsevier this book supported file pdf, txt, epub, kindle and other format this book has been release on 2013-11-15 with Medical categories.
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
Orphan Drugs And Rare Diseases
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Author : David C Pryde
language : en
Publisher: Royal Society of Chemistry
Release Date : 2014
Orphan Drugs And Rare Diseases written by David C Pryde and has been published by Royal Society of Chemistry this book supported file pdf, txt, epub, kindle and other format this book has been release on 2014 with Medical categories.
This book provides an up-to-date monograph on the drug discovery and regulatory elements of therapeutics used to treat rare or "orphan" diseases.
Report Of The National Commission On Orphan Diseases
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Author : National Commission on Orphan Diseases (U.S.)
language : en
Publisher:
Release Date : 1989
Report Of The National Commission On Orphan Diseases written by National Commission on Orphan Diseases (U.S.) and has been published by this book supported file pdf, txt, epub, kindle and other format this book has been release on 1989 with Federal aid to medical research categories.
Orphan Diseases And Orphan Drugs
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Author : I. Herbert Scheinberg
language : en
Publisher: Manchester University Press
Release Date : 1986
Orphan Diseases And Orphan Drugs written by I. Herbert Scheinberg and has been published by Manchester University Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 1986 with Medical categories.
Innovative Methods For Rare Disease Drug Development
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Author : Shein-Chung Chow
language : en
Publisher: CRC Press
Release Date : 2020-11-11
Innovative Methods For Rare Disease Drug Development written by Shein-Chung Chow and has been published by CRC Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2020-11-11 with Mathematics categories.
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.
Global Pediatric Development Of Drugs Biologics And Medical Devices
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Author : Jocelyn Jennings
language : en
Publisher:
Release Date : 2021-11-26
Global Pediatric Development Of Drugs Biologics And Medical Devices written by Jocelyn Jennings and has been published by this book supported file pdf, txt, epub, kindle and other format this book has been release on 2021-11-26 with categories.
Rare Diseases Epidemiology Update And Overview
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Author : Manuel Posada de la Paz
language : en
Publisher: Springer
Release Date : 2017-12-06
Rare Diseases Epidemiology Update And Overview written by Manuel Posada de la Paz and has been published by Springer this book supported file pdf, txt, epub, kindle and other format this book has been release on 2017-12-06 with Medical categories.
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 Disease Drug Development
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Author : Raymond A. Huml
language : en
Publisher: Springer Nature
Release Date : 2021-11-08
Rare Disease Drug Development written by Raymond A. Huml and has been published by Springer Nature this book supported file pdf, txt, epub, kindle and other format this book has been release on 2021-11-08 with Medical categories.
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.