Rare diseases and orphan products: accelerating research and development
Gespeichert in:
| Format: | Buch |
|---|---|
| Sprache: | English |
| Veröffentlicht: |
Washington, DC
National Academies Press
2010
|
| Schlagworte: | |
| Online-Zugang: | Inhaltsverzeichnis |
| Beschreibung: | Includes bibliographical references and index |
| Beschreibung: | XXI, 420 S. graph. Darst. |
| ISBN: | 0309158060 9780309158060 |
Internformat
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| 245 | 1 | 0 | |a Rare diseases and orphan products |b accelerating research and development |c Committee on Accelerating Rare Diseases Research and Orphan Product Development, Board on Health Sciences Policy. Marilyn J. Field and Thomas F. Boat, editors |
| 264 | 1 | |a Washington, DC |b National Academies Press |c 2010 | |
| 300 | |a XXI, 420 S. |b graph. Darst. | ||
| 336 | |b txt |2 rdacontent | ||
| 337 | |b n |2 rdamedia | ||
| 338 | |b nc |2 rdacarrier | ||
| 500 | |a Includes bibliographical references and index | ||
| 650 | 4 | |a Orphan drugs | |
| 650 | 4 | |a Drugs |x Design | |
| 650 | 4 | |a Rare diseases |x Treatment | |
| 650 | 4 | |a Orphan Drug Production |v Evaluation Studies | |
| 650 | 4 | |a Rare Diseases |x drug therapy |v Evaluation Studies | |
| 650 | 4 | |a Drug Discovery |v Evaluation Studies | |
| 650 | 4 | |a Drug Evaluation |v Evaluation Studies | |
| 650 | 4 | |a Health Policy |v Evaluation Studies | |
| 650 | 4 | |a Policy Making |v Evaluation Studies | |
| 650 | 0 | 7 | |a Orphan Drug |0 (DE-588)7558516-9 |2 gnd |9 rswk-swf |
| 650 | 0 | 7 | |a Seltene Krankheit |0 (DE-588)4180919-1 |2 gnd |9 rswk-swf |
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| 689 | 0 | 1 | |a Orphan Drug |0 (DE-588)7558516-9 |D s |
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| 700 | 1 | |a Field, Marilyn J. |e Sonstige |4 oth | |
| 700 | 1 | |a Boat, Thomas F. |e Sonstige |4 oth | |
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Datensatz im Suchindex
| _version_ | 1804145625725403136 |
|---|---|
| adam_text | Titel: Rare diseases and orphan products
Autor: Field, Marilyn J.
Jahr: 2010
SUMMARY 1
1 INTRODUCTION 15
Overview of Rare Diseases Research and Product
Development: Challenges and Opportunities, 19
Historical and Policy Context, 22
Study Origins and Focus, 30
Concepts and Definitions, 32
2 PROFILE OF RARE DISEASES 41
Epidemiology of Rare Diseases, 43
Causes of Rare Diseases, 51
Prevention, Diagnosis, and Treatment, 55
Impact of Rare Diseases on Patients, Families, and
Communities, 69
3 REGULATORY FRAMEWORK FOR DRUGS FOR RARE
DISEASES 73
General Framework for the Regulation of Drugs and
Biologies, 75
Regulatory Policy to Promote Innovation and Development
of Orphan Drugs and Biologies, 85
FDA Resources and Organization, 101
Recommendations, 105
xv
xvi CONTENTS
4 DISCOVERY RESEARCH FOR RARE DISEASES AND
ORPHAN PRODUCT DEVELOPMENT 111
Target Discovery, 114
Therapeutics Discovery, 118
Infrastructure for Basic Research and Drug Discovery for Rare
Diseases, 125
Innovation Platforms for Target and Drug Discovery, 137
Recommendations, 142
5 DEVELOPMENT OF NEW THERAPEUTIC DRUGS AND
BIOLOGICS FOR RARE DISEASES 147
Preclinical Development, 149
Phase I Clinical Trials: Safety, 152
Phase II Clinical Trials: Proof of Concept or Efficacy, 154
Phase III Clinical Trials: Regulatory Proof, 154
Phase IV Postmarketing Studies, 156
Infrastructure for Drug Development, 156
Innovation Platforms for Drug Development, 165
Recommendations, 173
6 COVERAGE AND REIMBURSEMENT: INCENTIVES AND
DISINCENTIVES FOR PRODUCT DEVELOPMENT 179
Medicare and Medicaid Coverage of FDA-Approved Drugs, 184
Private Health Plan Coverage of FDA-Approved Drugs, 197
Other Means of Financial Access to Orphan Drugs, 198
Public and Private Health Plan Coverage of Certain Costs
in Clinical Trials, 200
Recommendations, 201
7 MEDICAL DEVICES: RESEARCH AND DEVELOPMENT
FOR RARE DISEASES 205
Differences Between Medical Devices and Drugs, 206
Regulation of Medical Devices, 209
Coverage and Reimbursement for HDE Medical Devices, 224
Medical Device Research and Development, 225
Device Innovation and the HDE Option, 234
Recommendations, 237
8 TOWARD AN INTEGRATED APPROACH TO
ACCELERATING RESEARCH AND PRODUCT
DEVELOPMENT FOR RARE DISEASES 241
Elements of an Integrated National Strategy, 242
Recommendation, 246
CONTENTS XVll
REFERENCES 249
APPENDIXES
A Study Activities 283
B Innovation and the Orphan Drug Act, 1983-2009: Regulatory
and Clinical Characteristics of Approved Orphan Drugs 291
C Medicare Part D Coverage and Reimbursement of
Orphan Drugs 309
D Glossary, Abbreviations, and Public Laws 345
E Rare Diseases Clinical Research Network 365
F Advocacy Group Approaches to Accelerating Research and
Product Development: Illustrative Examples 371
G Committee and Staff Biographies 387
INDEX 395
|
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| spelling | 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 Washington, DC National Academies Press 2010 XXI, 420 S. graph. Darst. txt rdacontent n rdamedia nc rdacarrier Includes bibliographical references and index Orphan drugs Drugs Design Rare diseases Treatment Orphan Drug Production Evaluation Studies Rare Diseases drug therapy Evaluation Studies Drug Discovery Evaluation Studies Drug Evaluation Evaluation Studies Health Policy Evaluation Studies Policy Making Evaluation Studies Orphan Drug (DE-588)7558516-9 gnd rswk-swf Seltene Krankheit (DE-588)4180919-1 gnd rswk-swf Seltene Krankheit (DE-588)4180919-1 s Orphan Drug (DE-588)7558516-9 s DE-604 Field, Marilyn J. Sonstige oth Boat, Thomas F. Sonstige oth Erscheint auch als Online-Ausgabe, PDF 978-0-309-15807-7 HBZ Datenaustausch application/pdf http://bvbr.bib-bvb.de:8991/F?func=service&doc_library=BVB01&local_base=BVB01&doc_number=022493673&sequence=000004&line_number=0001&func_code=DB_RECORDS&service_type=MEDIA Inhaltsverzeichnis |
| spellingShingle | Rare diseases and orphan products accelerating research and development Orphan drugs Drugs Design Rare diseases Treatment Orphan Drug Production Evaluation Studies Rare Diseases drug therapy Evaluation Studies Drug Discovery Evaluation Studies Drug Evaluation Evaluation Studies Health Policy Evaluation Studies Policy Making Evaluation Studies Orphan Drug (DE-588)7558516-9 gnd Seltene Krankheit (DE-588)4180919-1 gnd |
| subject_GND | (DE-588)7558516-9 (DE-588)4180919-1 |
| title | Rare diseases and orphan products accelerating research and development |
| title_auth | Rare diseases and orphan products accelerating research and development |
| title_exact_search | Rare diseases and orphan products accelerating research and development |
| title_full | 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 |
| title_fullStr | 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 |
| title_full_unstemmed | 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 |
| title_short | Rare diseases and orphan products |
| title_sort | rare diseases and orphan products accelerating research and development |
| title_sub | accelerating research and development |
| topic | Orphan drugs Drugs Design Rare diseases Treatment Orphan Drug Production Evaluation Studies Rare Diseases drug therapy Evaluation Studies Drug Discovery Evaluation Studies Drug Evaluation Evaluation Studies Health Policy Evaluation Studies Policy Making Evaluation Studies Orphan Drug (DE-588)7558516-9 gnd Seltene Krankheit (DE-588)4180919-1 gnd |
| topic_facet | Orphan drugs Drugs Design Rare diseases Treatment Orphan Drug Production Evaluation Studies Rare Diseases drug therapy Evaluation Studies Drug Discovery Evaluation Studies Drug Evaluation Evaluation Studies Health Policy Evaluation Studies Policy Making Evaluation Studies Orphan Drug Seltene Krankheit |
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