03626oam 2200733Ia 450 991096011880332120251017110058.09786613044563978030920991503092099199781283044561128304456097803091580770309158079(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(Perlego)4740295(DNLM)1549696(EXLCZ)99255000000003196720101215h20102010 uy 0engurcn|||||||||txtrdacontentcrdamediacrrdacarrierRare 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 Academies1st ed.Washington, D.C. :National Academies Press,2010.©20101 online resource (xxi, 420 pages) illustrationsDescription based upon print version of record.9780309158060 0309158060 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 diseasesTreatmentOrphan drugs.DrugsDesign.Rare diseasesTreatment.615.19Field Marilyn J(Marilyn Jane)1804195Boat Thomas F1803632Institute of Medicine (U.S.).Committee on Accelerating Rare Diseases Research and Orphan Product Development.National Academies Press (U.S.)MiAaPQMiAaPQMiAaPQBOOK9910960118803321Rare diseases and orphan products4358436UNINA