1.

Record Nr.

UNINA9910825782703321

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

Pubbl/distr/stampa

Washington, D.C. : , : National Academies Press, , 2010

©2010

ISBN

0-309-20991-9

1-283-04456-0

9786613044563

0-309-15807-9

Edizione

[1st ed.]

Descrizione fisica

1 online resource (xxi, 420 pages) : illustrations

Altri autori (Persone)

FieldMarilyn J (Marilyn Jane)

BoatThomas F

Disciplina

615.19

Soggetti

Orphan drugs

Drugs - Design

Rare diseases - Treatment

Lingua di pubblicazione

Inglese

Formato

Materiale a stampa

Livello bibliografico

Monografia

Note generali

Description based upon print version of record.

Nota di bibliografia

Includes bibliographical references (p. 249-282) and index.

Nota di contenuto

""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""