LEADER 03994oam  2200757Ia 450 
001 9910960118803321
005 20251017110058.0
010   $a9786613044563
010   $a9780309209915
010   $a0309209919
010   $a9781283044561
010   $a1283044560
010   $a9780309158077
010   $a0309158079
035   $a(CKB)2550000000031967
035   $a(EBL)3378753
035   $a(SSID)ssj0000472566
035   $a(PQKBManifestationID)12212326
035   $a(PQKBTitleCode)TC0000472566
035   $a(PQKBWorkID)10453295
035   $a(PQKB)10164132
035   $a(MiAaPQ)EBC3378753
035   $a(Au-PeEL)EBL3378753
035   $a(CaPaEBR)ebr10459089
035   $a(CaONFJC)MIL304456
035   $a(OCoLC)923282998
035   $a(Perlego)4740295
035   $a(DNLM)1549696
035   $a(BIP)32459735
035   $a(EXLCZ)992550000000031967
100   $a20101215h20102010  uy         0
101 0 $aeng
135   $aurcn|||||||||
181   $ctxt$2rdacontent
182   $cc$2rdamedia
183   $acr$2rdacarrier
200 00$aRare diseases and orphan products $eaccelerating research and development /$fCommittee 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
205   $a1st ed.
210  1$aWashington, D.C. :$cNational Academies Press,$d2010.
210  4$dİ2010
215   $a1 online resource (xxi, 420 pages) $cillustrations
300   $aDescription based upon print version of record.
311 0 $a9780309158060 
311 0 $a0309158060 
320   $aIncludes bibliographical references (p. 249-282) and index.
327   $a""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""
327   $a""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""
330   $aRare 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.
606   $aOrphan drugs
606   $aDrugs$xDesign
606   $aRare diseases$xTreatment
615  0$aOrphan drugs.
615  0$aDrugs$xDesign.
615  0$aRare diseases$xTreatment.
676   $a615.19
701   $aField$b Marilyn J$g(Marilyn Jane)$01804195
701   $aBoat$b Thomas F$01803632
712 02$aInstitute of Medicine (U.S.).$bCommittee on Accelerating Rare Diseases Research and Orphan Product Development.
712 02$aNational Academies Press (U.S.)
801  0$bMiAaPQ
801  1$bMiAaPQ
801  2$bMiAaPQ
906   $aBOOK
912   $a9910960118803321
996   $aRare diseases and orphan products$94358436
997   $aUNINA