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Record Nr. |
UNINA9910960118803321 |
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Titolo |
Rare diseases and orphan products : accelerating research and development / / Committee on Accelerating Rare Diseases Research and Orphan Product Development, Board on Health Sciences Policy ; Marilyn J. Field and Thomas F. Boat, editors ; Institute of Medicine of the National Academies |
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Pubbl/distr/stampa |
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Washington, D.C. : , : National Academies Press, , 2010 |
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©2010 |
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ISBN |
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9786613044563 |
9780309209915 |
0309209919 |
9781283044561 |
1283044560 |
9780309158077 |
0309158079 |
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Edizione |
[1st ed.] |
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Descrizione fisica |
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1 online resource (xxi, 420 pages) : illustrations |
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Altri autori (Persone) |
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FieldMarilyn J (Marilyn Jane) |
BoatThomas F |
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Disciplina |
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Soggetti |
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Orphan drugs |
Drugs - Design |
Rare diseases - Treatment |
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Lingua di pubblicazione |
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Formato |
Materiale a stampa |
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Livello bibliografico |
Monografia |
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Note generali |
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Description based upon print version of record. |
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Nota di bibliografia |
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Includes bibliographical references (p. 249-282) and index. |
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Nota di contenuto |
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""Front Matter""; ""Acknowledgments""; ""Reviewers""; ""Preface""; ""Contents""; ""Boxes, Figures, and Tables""; ""Summary""; ""1 Introduction""; ""2 Profile of Rare Diseases""; ""3 Regulatory Framework for Drugs for Rare Diseases""; ""4 Discovery Research for Rare Diseases and Orphan Product Development""; ""5 Development of New Therapeutic Drugs and Biologics for Rare Diseases""; ""6 Coverage and Reimbursement: Incentives and Disincentives for Product Development""; ""7 Medical Devices: Research and Development for Rare Diseases"" |
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""8 Toward an Integrated Approach to Accelerating Research and Product Development for Rare Diseases""; ""References""; ""Appendix A: Study Activities""; ""Appendix B: Innovation and the Orphan Drug Act, 1983-2009: Regulatory and Clinical Characteristics of Approved Orphan Drugs""; ""Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs""; ""Appendix D: Glossary, Abbreviations, and Public Laws""; ""Appendix E: Rare Diseases Clinical Research Network""; ""Appendix F: Advocacy Group Approaches to Accelerating Research and Product Development: Illustrative Examples""; ""Appendix G: Committee and Staff Biographies""; ""Index"" |
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Sommario/riassunto |
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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. |
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