Rare Diseases and Orphan Products : Accelerating Research and Development

by Field, Marilyn J.

• ISBN13: 9780309158060
• ISBN10: 0309158060
• Edition: 1st
• Format: Paperback
• Copyright: 2011-01-01
• Publisher: NATL ACADEMY OF SCIENCE

Summary

Presents a report from Institute of Medicine committee recommending a coordinated, national, and global effort to plan and begin systematically implementing new strategies for addressing the needs of patients with rare diseases. Topics include profile of rare diseases and development of new therapeutic drugs and biologics for rare diseases. For researchers. Softcover.

Table of Contents

Summary	p. 1
Introduction	p. 15
Overview of Rare Diseases Research and Product Development: Challenges and Opportunities	p. 19
Historical and Policy Context	p. 22
Study Origins and Focus	p. 30
Concepts and Definitions	p. 32
Profile of Rare Diseases	p. 41
Epidemiology of Rare Diseases	p. 43
Causes of Rare Diseases	p. 51
Prevention, Diagnosis, and Treatment	p. 55
Impact of Rare Diseases on Patients, Families, and Communities	p. 69
Regulatory Framework for Drugs for Rare Diseases	p. 73
General Framework for the Regulation of Drugs and Biologies	p. 75
Regulatory Policy to Promote Innovation and Development of Orphan Drugs and Biologies	p. 85
FDA Resources and Organization	p. 101
Recommendations	p. 105
Discovery Research for Rare Diseases and Orphan Product Development	p. 111
Target Discovery	p. 114
Therapeutics Discovery	p. 118
Infrastructure for Basic Research and Drug Discovery for Diseases	p. 125
Innovation Platforms for Target and Drug Discovery	p. 137
Recommendations	p. 142
Development of New Therapeutic Drugs and Biologics for Rare Diseases	p. 147
Preclinical Development	p. 149
Clinical Trials: Safety	p. 152
Clinical Trials: Proof of Concept or Efficacy	p. 154
Clinical Trials: Regulatory Proof	p. 154
Postmarketing Studies	p. 156
Infrastructure for Drug Development	p. 156
Innovation Platforms for Drug Development	p. 165
Recommendations	p. 173
Coverage and Reimbursement: Incentives and Disincentives for Product Development	p. 179
Medicare and Medicaid Coverage of FDA-Approved Drugs	p. 184
Private Health Plan Coverage of FDA-Approved Drugs	p. 197
Other Means of Financial Access to Orphan Drugs	p. 198
Public and Private Health Plan Coverage of Certain Costs in Clinical Trials	p. 200
Recommendations	p. 201
Medical Devices: Research and Development for Rare Diseases	p. 205
Differences Between Medical Devices and Drugs	p. 206
Regulation of Medical Devices	p. 209
Coverage and Reimbursement for HDE Medical Devices	p. 224
Medical Device Research and Development	p. 225
Device Innovation and the HDE Option	p. 234
Recommendations	p. 237
Toward an Integrated Approach to Accelerating Research and Product Development for Rare Diseases	p. 241
Elements of an Integrated National Strategy	p. 242
Recommendation	p. 246
References	p. 249
Appendixes
Study Activities	p. 283
Innovation and the Orphan Drug Act, 1983-2009: Regulatory and Clinical Characteristics of Approved Orphan Drugs	p. 291
Medicare Part D Coverage and Reimbursement of Orphan Drugs	p. 309
Glossary, Abbreviations, and Public Laws	p. 345
Rare Diseases Clinical Research Network	p. 365
Advocacy Group Approaches to Accelerating Research and Product Development: Illustrative Examples	p. 371
Committee and Staff Biographies	p. 387
Index	p. 395
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