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Orphan Drugs

Orphan Drugs
  • Author : Elizabeth Hernberg-Ståhl,Miroslav Reljanović
  • Publisher :Unknown
  • Release Date :2013-11-15
  • Total pages :334
  • ISBN : 9781908818393
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Summary : 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
  • Publisher :Unknown
  • Release Date :1989-04
  • Total pages :228
  • ISBN : 0719022967
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Summary :

Rare Diseases and Orphan Drugs

Rare Diseases and Orphan Drugs
  • Author : Jules J. Berman
  • Publisher :Unknown
  • Release Date :2014-05-26
  • Total pages :400
  • ISBN : 9780124200098
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Summary : 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 in epilepsy

Orphan drugs in epilepsy
  • Author : M. Nikanorova,P. Genton,S. I. Johannessen,C. Johannessen Landmark
  • Publisher :Unknown
  • Release Date :2013-09-18
  • Total pages :112
  • ISBN : 9782742012350
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Summary : All the necessary information on 6 molecules called “orphan drugs” used in the treatment of some epileptic syndromes. More than half of epilepsies start before the age of 20 years and nearly 25% of them are refractory. Two problems arise: - It is essential to treat them so that brain development continues as normally as possible. - There are few drugs available. This work outlines 6 molecules in detail that are specifically used in epileptic encephalopathies. Every aspect of these molecules is discussed, including drug development, indication, efficacy, cost, etc. Drafted by international experts in the field of epileptology, this book provides all the necessary information on orphan drugs and their clinical use.

Orphan Drugs and Rare Diseases

Orphan Drugs and Rare Diseases
  • Author : David C Pryde,Michael J Palmer
  • Publisher :Unknown
  • Release Date :2014
  • Total pages :486
  • ISBN : 9781849738064
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Summary : 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 :Unknown
  • Release Date :2011-04-03
  • Total pages :442
  • ISBN : 9780309158060
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Summary : 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

Rare Diseases and Orphan Drugs
  • Author : Jules J. Berman
  • Publisher :Unknown
  • Release Date :2014-06-02
  • Total pages :380
  • ISBN : 0124199887
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Summary : 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
  • Publisher :Unknown
  • Release Date :2014-07-30
  • Total pages :350
  • ISBN : 9781782624202
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Summary : 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.

Searching for Magic Bullets

Searching for Magic Bullets
  • Author : Lisa A Basara,Michael E Montagne
  • Publisher :Unknown
  • Release Date :1994-08-10
  • Total pages :266
  • ISBN : 1560248580
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Summary : Searching for Magic Bullets reveals the quest of consumers, health professionals, and drug developers to find safer and faster methods of bringing new medications to the marketplace. Authors Basara and Montagne explore the current drug development and approval processes, their strengths and weaknesses, and the mechanisms by which patients and organizations evade these processes. Readers learn about the fundamentals of traditional and nontraditional drug discovery and development as they occur in the U.S., as well as the views of consumers, patients, and health professionals. Specific case studies of non-traditional drug development and acquisition strategies are highlighted, including AIDS medications, orphan drugs, and patient importation of medications. Basara and Montagne establish the differences in both knowledge and opinions of health consumers and health professionals regarding drug development, as well as how these differences often lead to frustration, dissatisfaction, and misappropriation of resources. The authors pinpoint the need for consumers and patients to know much more about the discovery and development of medicines, and for health professionals and students to understand patients’concerns, needs and beliefs, including their reasons for considering alternative methods of drug development and acquisition. Searching for Magic Bullets is a springboard from which consumers, health professionals, and students can discuss, debate, and resolve these issues and begin to develop more capable drug development and approval systems. This groundbreaking new book enlightens health professionals about patients’views regarding medication discovery and development and informs consumers and patients about the sometimes conflicting views of health professionals. It is divided into three sections: drug development and approval in the U.S., a case study of orphan drugs, and risky and sometimes illegal ways in which consumers evade the traditional drug development and approval systems. An Overview of the Chapters: A Review of the Drug Development Process of the Pharmaceutical Industry: Presents the steps that must be taken when researching and developing a new medication. The Food and Drug Administration and the Drug Approval Process: Describes the history and scope of the FDA, the steps involved in acquiring drug approval, and the various stages of clinical testing. Orphan Drug Legislation: A review of the Orphan Drug Act of 1983 and the changes that have recently been proposed by Congress. The impact of the Act is highlighted through a description of products that have been made available since the legislation was enacted. Issues of controversy are also highlighted. Non-traditional Methods of Drug Development: The role of patients and consumers in drug development and evaluation is discussed, with an emphasis on the perceived shortcomings of the formal system. Patient Influence on Drug Development and Regulation: The influence of patient advocacy groups and consumers is discussed in relation to the development and approval of orphan drugs, the fast-tracking of specific medications, and the use of unapproved and alternative therapies. Prescription Drug Importation: Clarifies the current drug importation regulations, as well as provides specific directions for patients wishing to receive such products or learn more about FDA importation laws. The final chapter summarizes safe and rational techniques that empower consumers in their search for beneficial drug therapies. Resources and strategies for obtaining and using information are provided as a reference for readers. A glossary of terms, acronyms, and a directory of supplemental information sources strengthens the reader's understanding of the information presented. Who Benefits From This Book? Consumers and patients can use Searching for Magic Bullets as an accurate source of information about significant but often confusing medical issues. The FDA and the way medications are developed are easily misunderstood, while alternative therapies and medication sources are often believed to be the only options. Patients will learn the viewpoints of the pharmaceutical industry, the government, and their health care professionals; the rationale for various steps in the drug development process; the risks and benefits of participation in clinical trials; how to obtain the highest quality care, make informed health decisions, and reduce health care costs; and finally, how to cope with a rare disease and/or limited access to approved medications. The result is an informed, influential, and active patient. For health professionals, this book reviews the steps of drug development and approval and provides explanations for drug development decisions; drug approval time lag; and patient frustrations, misinterpretations, and expectations. It is critical for health professionals to understand the needs of patients and to determine how they can work with patients to find acceptable solutions. The literature references and medical information sources are invaluable in this regard. Pharmaceutical industry executives, product managers, clinical researchers, and sales representatives will find a concise and timely examination of the ways in which medications are discovered, developed, marketed, and used by patients. Discussions of orphan drug development, biotechnology products, and patient issues may also provide new insights into these often misunderstood areas. Pharmacy, medical, nursing, and other students will find this book a consolidated reference source and guidebook for information about the primary issues surrounding drug development and the FDA approval process. Patients’knowledge of alternative medical therapies will only increase and health care curricula must include material that helps students understand patients’perceptions of the medication development and approval systems, as well as the importance of patients in health care decisionmaking. The disadvantages of current drug development and approval systems are described with the hope that future health professionals can amend these processes and ultimately enhance patient care.

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
  • Total pages :69
  • ISBN : OCLC:954774773
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Summary : 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.

Orphan Drugs

Orphan Drugs
  • Author : Carolyn H. Asbury
  • Publisher :Unknown
  • Release Date :1985
  • Total pages :219
  • ISBN : UOM:39015009529093
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Summary :

Orphan Drug Law Matures into Medical Mainstay

Orphan Drug Law Matures into Medical Mainstay
  • Author : Anonim
  • Publisher :Unknown
  • Release Date :2021
  • Total pages :229
  • ISBN : 1422326772
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Summary :

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
  • Publisher :Unknown
  • Release Date :2021
  • Total pages :80
  • ISBN : 1230987654XX
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Summary : 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,

Cooperative Approaches to Research and Development of Orphan Drugs

Cooperative Approaches to Research and Development of Orphan Drugs
  • Author : Eunyong Chung
  • Publisher :Unknown
  • Release Date :1985
  • Total pages :204
  • ISBN : UOM:39015009554349
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Summary :

Orphan Drugs

Orphan Drugs
  • Author : Timothy R. Coté
  • Publisher :Unknown
  • Release Date :2017-12-29
  • Total pages :352
  • ISBN : 1119113741
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Summary :

Pharmaceutical Innovation and Orphan Drugs

Pharmaceutical Innovation and Orphan Drugs
  • Author : Natalie Denise Reaves
  • Publisher :Unknown
  • Release Date :1996
  • Total pages :80
  • ISBN : STANFORD:36105060443293
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Summary :

Orphan Drugs

Orphan Drugs
  • Author : Timothy R. Cot_
  • Publisher :Unknown
  • Release Date :2011-04-22
  • Total pages :352
  • ISBN : 047055133X
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Summary : This book is a "how to" book that shows people with ideas how to meet the scientific, financial, and regulatory challenges of orphan drug development. It offers an insider's view of the drug development pathway from thought to therapy. Written by the Director of FDA's Office of Orphan Products Development with contributing authors both inside and outside government, this book relates the history of the 1983 Orphan Drug Act and the 1,955 orphan-designated drugs it has spawned, some 334 of which have received marketing approval for the treatment of rare diseases.

Orphan Drugs

Orphan Drugs
  • Author : Fred Karch
  • Publisher :Unknown
  • Release Date :1982
  • Total pages :210
  • ISBN : UOM:39015004349604
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Summary :

Orphan Drugs

Orphan Drugs
  • Author : Daniel Hagn,Oliver Schöffski
  • Publisher :Unknown
  • Release Date :2005
  • Total pages :160
  • ISBN : 3936863059
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Summary : "The promotion and development of medicinal products for the treatment of rare diseases require our particular attention, as health is our most valuable possession," said Ursula Schleicher, former Vice-President of the European Parliament. According to estimates, around 25 million people in Europe are suffering from 5,000 to 8,000 orphan diseases. Many of them are still waiting for medicines that will allow them to at least be able to lead a pain-free life, if not always a normal life. From a purely business-management viewpoint, the development of drugs for rare diseases is not always economical for the pharmaceutical industry. Nor can sufferers raise the funds themselves for research into the drugs that for them are vitally necessary. It is, therefore, a question of moral values and national priorities as to what extent society is prepared to help those affected. In 1999 the European Union adopted the Orphan Medicinal Products Regulation. This offers the biopharmaceutical and pharmaceutical industry incentives to develop orphan drugs. This book presents the European Orphan Medicinal Products Regulation and compares it with the rules of other countries. It also discusses the possibilities of creating further incentives.

Orphan Drugs

Orphan Drugs
  • Author : Karen Miller Allen
  • Publisher :Unknown
  • Release Date :1983
  • Total pages :12
  • ISBN : STANFORD:36105039619114
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Summary :

Pediatric Drug Development

Pediatric Drug Development
  • Author : Andrew E. Mulberg,Dianne Murphy,Julia Dunne,Lisa L. Mathis
  • Publisher :Unknown
  • Release Date :2013-05-20
  • Total pages :624
  • ISBN : 9781118312056
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Summary : 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.