03387nam 2200649Ia 450 991045686860332120200520144314.01-283-04456-097866130445630-309-15807-9(CKB)2550000000031967(EBL)3378753(SSID)ssj0000472566(PQKBManifestationID)12212326(PQKBTitleCode)TC0000472566(PQKBWorkID)10453295(PQKB)10164132(MiAaPQ)EBC3378753(Au-PeEL)EBL3378753(CaPaEBR)ebr10459089(CaONFJC)MIL304456(OCoLC)923282998(EXLCZ)99255000000003196720101215d2010 uy 0engurcn|||||||||txtccrRare diseases and orphan products[electronic resource] 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 AcademiesWashington, D.C. National Academies Pressc20101 online resource (443 p.)Description based upon print version of record.0-309-15806-0 Includes bibliographical references (p. 249-282) and index.""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""""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""Orphan drugsDrugsDesignRare diseasesTreatmentElectronic books.Orphan drugs.DrugsDesign.Rare diseasesTreatment.615.19Field Marilyn J(Marilyn Jane)855032Boat Thomas F910464Institute of Medicine (U.S.).Committee on Accelerating Rare Diseases Research and Orphan Product Development.National Academies Press (U.S.)MiAaPQMiAaPQMiAaPQBOOK9910456868603321Rare diseases and orphan products2486767UNINA