Rare Diseases and Orphan Drugs

Rare Diseases and Orphan Drugs
Author: Jules J. Berman
Publsiher: Academic Press
Total Pages: 400
Release: 2014-05-26
ISBN 10: 0124200095
ISBN 13: 9780124200098
Language: EN, FR, DE, ES & NL

Rare Diseases and Orphan Drugs Book Review:

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 and Rare Diseases

Orphan Drugs and Rare Diseases
Author: David C Pryde,Michael J Palmer
Publsiher: Royal Society of Chemistry
Total Pages: 486
Release: 2014
ISBN 10: 1849738068
ISBN 13: 9781849738064
Language: EN, FR, DE, ES & NL

Orphan Drugs and Rare Diseases Book Review:

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
Publsiher: National Academies Press
Total Pages: 442
Release: 2011-04-03
ISBN 10: 0309158060
ISBN 13: 9780309158060
Language: EN, FR, DE, ES & NL

Rare Diseases and Orphan Products Book Review:

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
Publsiher: Elsevier
Total Pages: 334
Release: 2013-11-15
ISBN 10: 1908818395
ISBN 13: 9781908818393
Language: EN, FR, DE, ES & NL

Orphan Drugs Book Review:

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 Diseases and Orphan Drugs

Orphan Diseases and Orphan Drugs
Author: I. Herbert Scheinberg,J. M. Walshe
Publsiher: Manchester University Press
Total Pages: 228
Release: 1989-04
ISBN 10: 9780719022968
ISBN 13: 0719022967
Language: EN, FR, DE, ES & NL

Orphan Diseases and Orphan Drugs Book Review:

Orphan Drugs and Rare Diseases

Orphan Drugs and Rare Diseases
Author: David C Pryde,Michael J Palmer
Publsiher: Royal Society of Chemistry
Total Pages: 350
Release: 2014-07-30
ISBN 10: 1782624201
ISBN 13: 9781782624202
Language: EN, FR, DE, ES & NL

Orphan Drugs and Rare Diseases Book Review:

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.

Rare Diseases and Orphan Drugs

Rare Diseases and Orphan Drugs
Author: Anonim
Publsiher: Unknown
Total Pages: 135
Release: 2007
ISBN 10: 1928374650XXX
ISBN 13: OCLC:500740977
Language: EN, FR, DE, ES & NL

Rare Diseases and Orphan Drugs Book Review:

Value Assessment of Orphan Drugs and Treatments for Rare Diseases

Value Assessment of Orphan Drugs and Treatments for Rare Diseases
Author: Catherine M. Lockhart
Publsiher: Unknown
Total Pages: 69
Release: 2016
ISBN 10: 1928374650XXX
ISBN 13: OCLC:954774773
Language: EN, FR, DE, ES & NL

Value Assessment of Orphan Drugs and Treatments for Rare Diseases Book Review:

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 a potential new framework to be developed as new metric for assessing the value of orphan drugs, the Orphan Drug Index Estimate (ODIE). METHODS: For the systematic review, searches were conducted in December 2015 in PubMed®, EMBASE®, and Web of Science® databases using the following keywords: orphan drug, rare disease treatment, economics, resource utilization, cost, cost effectiveness, questionnaire, and value. Only references published in English were included. Manuscripts that solely reported one of the following were excluded: clinical or patient care, policy or legislation on orphan drugs particularly relating to research incentive, opinion or editorial, preclinical studies, drug-development, unrelated to rare diseases or healthcare, reviews other than systematic reviews for health technology assessment. RESULTS: A total of 2513 unique references were obtained, and screened by title and abstract according to exclusion criteria. After exclusion, 333 references remained for full evaluation. Of those, an additional 296 were excluded, but 51 additional studies were included from the reference lists of included articles. A total of 88 articles were included in the complete analysis. Overall, the methodology employed for conducting cost-effectiveness assessments followed traditional techniques including decision analysis and Markov modeling techniques. The reported incremental cost effectiveness ratios (ICERs) ranged from dominant treatments to a high of €6.1 million per quality adjusted life year (QALY). Interpretation of the results was more challenging, with 43% of studies reporting ICERs that would not be considered cost-effective under a willingness-to-pay threshold of $50,000 per QALY. In spite of the lack of cost-effectiveness, the majority of authors agreed that since the treatment under review is for a rare condition, there is an obligation to cover the costs. In light of these analyses, there is an evident need for a method of analysis that is more comprehensive than the ICER, and more appropriate for addressing the uniqueness of orphan drugs, including variables related to the rarity and severity of disease, and a broader societal perspective on costs, including societal burden and identifiable opportunity costs. In response, here I propose a potential new metric based on multicriteria decision analysis (MCDA) techniques to provide a more comprehensive evaluation of orphan drugs. CONCLUSIONS: There is a global consensus of a need to develop appropriate methodology, analysis techniques, and related policies to address management of expensive treatments. It is not yet clear how best to evaluate the value of orphan drugs. More thorough evaluation and validation of novel modeling techniques, analytic rationale and proactive policy changes are needed to redefine the status quo of health technology assessment of rare disease treatments. I propose a new metric to overcome some limitations of the ICER in evaluation of rare diseases. Continued research is needed in detailed development of a valid, quantifiable, and reproducible metric; however, the work presented here provides a foundation for the development process.

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
Publsiher: ConferenceSeries
Total Pages: 80
Release: 2022
ISBN 10: 1928374650XXX
ISBN 13: 9182736450XXX
Language: EN, FR, DE, ES & NL

Proceedings of 4th World Congress on Rare Diseases and Orphan Drugs 2018 Book Review:

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 Morphological Diseases, Development of Orphan Products, Rare Diseases in Oncology, Rare Diseases in Anaesthesiology, Rare Diseases in Haematology, Orphan Drugs Market Research, Rare Gynaecological and Obstetrical Diseases, Pediatric Rare Diseases, Current Rare Diseases Research, Rare Diseases of Sexual Health, Rare Hereditary Diseases, Diagnosis and Treatment for Rare Diseases, Clinical case studies on Rare Diseases, Imaging of Rare Diseases, Other Rare Diseases,

Innovative Methods for Rare Disease Drug Development

Innovative Methods for Rare Disease Drug Development
Author: Shein-Chung Chow
Publsiher: CRC Press
Total Pages: 306
Release: 2020-11-12
ISBN 10: 1000208214
ISBN 13: 9781000208214
Language: EN, FR, DE, ES & NL

Innovative Methods for Rare Disease Drug Development Book Review:

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 Diseases Epidemiology Update and Overview

Rare Diseases Epidemiology  Update and Overview
Author: Manuel Posada de la Paz,Domenica Taruscio,Stephen C. Groft
Publsiher: Springer
Total Pages: 667
Release: 2017-12-06
ISBN 10: 3319671448
ISBN 13: 9783319671444
Language: EN, FR, DE, ES & NL

Rare Diseases Epidemiology Update and Overview Book Review:

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.

Orphan Drugs in Development for Rare Diseases

Orphan Drugs in Development for Rare Diseases
Author: Anonim
Publsiher: Unknown
Total Pages: 135
Release: 2011
ISBN 10: 1928374650XXX
ISBN 13: OCLC:847743900
Language: EN, FR, DE, ES & NL

Orphan Drugs in Development for Rare Diseases Book Review:

NORD Guide to Rare Disorders

NORD Guide to Rare Disorders
Author: National Organization for Rare Disorders
Publsiher: Lippincott Williams & Wilkins
Total Pages: 895
Release: 2003
ISBN 10: 9780781730631
ISBN 13: 0781730635
Language: EN, FR, DE, ES & NL

NORD Guide to Rare Disorders Book Review:

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.

Orphan Lung Diseases

Orphan Lung Diseases
Author: J.-F. Cordier
Publsiher: European Respiratory Society
Total Pages: 376
Release: 2014-05-14
ISBN 10: 1849840148
ISBN 13: 9781849840149
Language: EN, FR, DE, ES & NL

Orphan Lung Diseases Book Review:

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 in the Age of Health 2 0

Rare Diseases in the Age of Health 2 0
Author: Rajeev K. Bali,Lodewijk Bos,Michael Christopher Gibbons,Simon Ibell
Publsiher: Springer Science & Business Media
Total Pages: 292
Release: 2013-10-21
ISBN 10: 3642386431
ISBN 13: 9783642386435
Language: EN, FR, DE, ES & NL

Rare Diseases in the Age of Health 2 0 Book Review:

This text focuses on various factors associated with orphan diseases and the influence and role of health information technologies. Orphan diseases have not been adopted by the pharmaceutical industry because they provide little financial incentive to treat or prevent it. It is estimated that 6,000-7,000 orphan diseases exist today; as medical knowledge continues to expand, this number is likely to become much greater. The book highlights the opportunities and challenges in this increasingly important area. The book explores new avenues which are opened by information technologies and Health 2.0, and highlights also economic opportunities of orphan disease medicine. The editors of this new book have international experience and competencies in the key areas of patient empowerment, healthcare and clinical knowledge management, healthcare inequalities and disparities, rare diseases and patient advocacy.

Rare Disease Drug Development

Rare Disease Drug Development
Author: Raymond A. Huml, MS, DVM
Publsiher: Springer
Total Pages: 409
Release: 2021-10-09
ISBN 10: 9783030786045
ISBN 13: 3030786048
Language: EN, FR, DE, ES & NL

Rare Disease Drug Development Book Review:

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.

Drug Repurposing

Drug Repurposing
Author: Farid A. Badria
Publsiher: BoD – Books on Demand
Total Pages: 234
Release: 2020-12-02
ISBN 10: 1839685204
ISBN 13: 9781839685200
Language: EN, FR, DE, ES & NL

Drug Repurposing Book Review:

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.

Communicating Rare Diseases and Disorders in the Digital Age

Communicating Rare Diseases and Disorders in the Digital Age
Author: Costa, Liliana Vale,Oliveira, Sónia
Publsiher: IGI Global
Total Pages: 412
Release: 2020-01-03
ISBN 10: 1799820890
ISBN 13: 9781799820895
Language: EN, FR, DE, ES & NL

Communicating Rare Diseases and Disorders in the Digital Age Book Review:

A primary concern of rare disease diagnosis is the lack of accurate information that may lead to delayed interventions, administering inaccurate treatments, and social consequences. Health communication continues to be one-way and rely on the expertise from the health practitioner. In such a broad spectrum of rare diseases, patients may find it difficult to obtain timely information, accurate diagnosis, and appropriate treatments, surgeries, medications, or psychological counseling in their own countries. The use of information and communication technologies can create new communication channels that address this lack of knowledge. Communicating Rare Diseases and Disorders in the Digital Age is an essential reference source that uses computer-mediated communication to improve patient knowledge when afflicted or dealing with rare health conditions. Featuring research on topics such as support networking, eHealth management, and social computing, this book is ideally designed for health practitioners, physicians, patients, medical administrators, nurses, surgeons, infectious disease educators, hospital directors, world health organizations, academicians, students, and researchers seeking coverage on current advances in health communication, computer science, and epidemiology.

Orphan Drug Law Matures into Medical Mainstay

Orphan Drug Law Matures into Medical Mainstay
Author: Anonim
Publsiher: DIANE Publishing
Total Pages: 135
Release: 2022
ISBN 10: 9781422326770
ISBN 13: 1422326772
Language: EN, FR, DE, ES & NL

Orphan Drug Law Matures into Medical Mainstay Book Review:

Rare Diseases

Rare Diseases
Author: Nicolas Sireau
Publsiher: Routledge
Total Pages: 278
Release: 2017-09-08
ISBN 10: 135127838X
ISBN 13: 9781351278386
Language: EN, FR, DE, ES & NL

Rare Diseases Book Review:

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.