03994oam 2200757Ia 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(BIP)32459735(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""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 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