Agriculture, Rural Development, Food and Drug Administration, and Related Agencies Appropriations Act of 2010, 100

Albright hereditary osteodystrophy, 131

Alglucosidase alfa (Myozyme), 96, 159, 198

Allele, defined, 345, 346

Alpers syndrome, 368

Alpha-1 antitrypsin deficiency, 52, 131, 375

Alpha-1 Foundation, 373, 375-377

Alzheimer disease, 15, 141, 147, 167

Amanita phalloides (“death cap” mushroom), 54

Amatoxin poisoning, 54

Ambrisentan (Letairis), 302, 303 n.29, 330, 334

Amebiasis, 318, 323

Amendments to the Patent and Trademark Act of 1980 (Bayh-Dole Act), 114

American Academy of Pediatrics, 68

American Association of Poison Control Centers, 46

American College of Medical Genetics (ACMG), 57-58, 114

American College of Physicians, 68

American Heart Association, 384

American Institute for Medical and Biological Engineering, 230

American Partnership for Eosinophilic Disorders, 287

American Recovery and Reinvestment Act, 131, 132

American Society of Health-System Pharmacists, 199 n.10

American Society of Hematology, 66-67

Aminoglycoside-induced deafness, 368

Aminolevulinate dehydratase deficiency porphyria, 368

Amyloidosis, 131

Amyotrophic lateral sclerosis (ALS), 16, 28, 64, 85, 166, 184, 310, 325

Amyris Biotechnologies, 173

Andersen-Tawil syndrome, 366

Anencephaly, 56

Angelman, Rett, and Prader-Willi Syndromes Consortium, 365

Angelman syndrome, 115-116, 365

Animal models and studies

access to, 6, 7, 128, 139, 143, 380-381

of biologics, 152

carcinogenicity studies, 150

cystic fibrosis, 127

defined, 345, 355

development, 127-128, 380-381

exploratory IND studies, 75, 153

funding for, 132

genetically modified animals, 115, 116, 127-128

Huntington disease, 127

infrastructure for, 127-128

Marfan syndrome, 113, 162

medical devices, 210

naturally occurring diseases, 127

preclinical studies, 120, 149, 150, 152, 153

repositories, 6, 7, 143, 347

spinal muscular atrophy, 380-381

Antibiotics, 43, 53, 86 n.6, 293

Anthracycline, 55

Anthrax, 81

Antibodies, 113, 120, 121, 137, 149-150, 151, 152, 300, 339-340, 346, 380, 382

(see also Monoclonal antibodies)

resources, 382

Aortic aneurysm, 113, 118, 129, 162

Aplastic anemias, 51

Approval process for orphan drugs and biologics

(see also Center for Drug Evaluation and Research;

Investigational New Drug)

accelerated approval, 80, 81, 82, 87, 97, 156, 157

access to clinical data supporting/denying approvals, 83-84, 98, 164-165, 166-167

basics of, 75-77

defined, 346

evidence of safety and efficacy accepted by FDA, 4, 76-77, 95-98

fast track, 80, 81, 87

funding, 26

generic products, 76, 78, 89, 93

guidance for research sponsors, 4, 13, 27, 75-76, 98, 99-100

guidance for reviewers, 6, 27, 98

inconsistencies in FDA reviews, 4-5, 13, 95, 99-100, 158-159

issues/criticisms of, 4, 95-101

medical device approval process compared to, 216-220

neglected tropical disease drugs, 82, 86 n.7

Front Matter (R1-R22)

Summary (1-14)

1 Introduction (15-40)

2 Profile of Rare Diseases (41-72)

3 Regulatory Framework for Drugs for Rare Diseases (73-110)

4 Discovery Research for Rare Diseases and Orphan Product Development (111-146)

5 Development of New Therapeutic Drugs and Biologics for Rare Diseases (147-178)

6 Coverage and Reimbursement: Incentives and Disincentives for Product Development (179-204)

7 Medical Devices: Research and Development for Rare Diseases (205-240)

8 Toward an Integrated Approach to Accelerating Research and Product Development for Rare Diseases (241-248)

References (249-282)

Appendix A: Study Activities (283-290)

Appendix B: Innovation and the Orphan Drug Act, 1983-2009: Regulatory and Clinical Characteristics of Approved Orphan Drugs (291-308)

Appendix C: Medicare Part D Coverage and Reimbursement of Orphan Drugs (309-344)

Appendix D: Glossary, Abbreviations, and Public Laws (345-364)

Appendix E: Rare Diseases Clinical Research Network (365-370)

Appendix F: Advocacy Group Approaches to Accelerating Research and Product Development: Illustrative Examples (371-386)

Appendix G: Committee and Staff Biographies (387-394)

Index (395-420)