surrogate endpoints as, 53, 129–130

use of, in pediatric studies, 12, 52–53, 129, 130–133, 132b, 194

American Academy of Pediatrics, 30, 43–44, 274

Amlodipine besylate, 74

Amlodipine maleate, 74

Ampicillin, 164–165

Anakinra, 329t, 361t

Animal studies, 55

Antihemophilic factor, 124, 147, 329–330t, 373t

Anti-infective agents, 141, 273t, 279

Antithrombin, 222–223b, 331t, 373t

Antithymocyte globulin, 217, 331t, 374t

Approval letters, 188–189, 190

for biologics, 218

Aripiprazole, 61, 120b, 127, 132b, 200–201b, 202b

Arrhythmias, 56–57, 122, 149, 199b

Artemether/lumefantrine, 213–214

Arthritis, juvenile rheumatoid. See Rheumatoid arthritis

Assent to research participation, 94

Association of Am. Physicians & Surgeons, Inc. v. FDA, 33

Asthma, 21, 23b, 48, 51, 120b, 121, 132–133, 198b, 202b

Atopic dermatitis, 302–303

Attention deficit hyperactivity disorder, 48, 69, 122n, 125, 199b

Autism, 151n

Autologous cultured chondrocytes, 331t, 374t

Azithromycin, 164

B

Basilixmab, 332t, 361t

Becaplermin, 224b, 226, 227, 332t, 361t

Belmont Report, 30–31

Benzyl alcohol, 30

Besifloxacin ophthalmic, 158–159

Best Pharmaceuticals for Children Act

age range covered by, 4–5

biologics coverage added, 3, 7–8, 16, 27, 34, 207

biologics with possible promise for pediatric study, 226

clinical reviews, 112–114

cost-benefit analysis, 109

data sources for analysis of, 5–6, 112

effects on clinical practice, 25

ethical implementation, 89, 99–108

extrapolation studies under, 137–139, 137t, 138t

FDA administrative structure for, 83–84

incentive program, 62, 70–74

labeling changes supported by studies under, 14, 178, 194, 195t

neonatal assessments under, 14, 141, 148, 149–151, 165–167

NIH role, 8–9, 14, 35, 76–77, 96, 163–165, 166–167, 188, 226–227

objectives, 1, 8–9, 19, 111, 271

one-year safety reviews, 125–126

outcomes to date, in pharmaceutical safety and effectiveness, 2–3, 6–8, 26–27

Pediatric Research Equity Act and, 34, 81

potential of requests to generate useful information, 183–187, 186b

proposal for permanent reauthorization, 86–87

public access to studies conducted under, 3, 11, 84–86, 100–102, 109, 112, 272

reauthorizations, 3, 7, 9, 27, 87

requests for studies of off-patent products, 76–77

safeguards for pediatric research, 10

salient issues in analysis of, 1, 5, 24, 28, 261–262

scope and outcomes of studies under, 2, 14–16, 22b, 23b, 34, 74–76, 177–183, 178f, 182f, 203

sources of requests, 182, 226

strategies for improving, 3, 166–167

suggestions for neonatal assessments under, 14, 166–167

types of labeling changes made under, 197–203

Betamethasone, 164

Better Pharmaceutical for Children, 32

Bevacizumab, 309, 332t, 362t

Biologics

access to application and review data, 215

approval letters, 218



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