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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