Table 2.
Characteristics of 142 efficacy trials supporting FDA approval of NDAs for neuropsychiatric conditions, 2005–2014
| No. (%) (n = 142) | No. registered (%) | No. results reported (%) | No. published (%) | |
|---|---|---|---|---|
| FDAAA applicability | ||||
| Pre-FDAAA | 101 (71%) | 65 (64%) | 10 (10%) | 91 (90%) |
| Post-FDAAA | 41 (29%) | 41 (100%) | 41 (100%) | 41 (100%) |
| Pivotal status | ||||
| Pivotal | 92 (65%) | 78 (85%) | 40 (43%) | 90 (98%) |
| Non-pivotal | 50 (35%) | 28 (56%) | 11 (22%) | 8 (84%) |
| Study location | ||||
| All USA | 65 (46%) | 50 (77%) | 14 (22%) | 61 (94%) |
| Some USA | 54 (38%) | 41 (76%) | 29 (54%) | 48 (89%) |
| None USA | 23 (16%) | 15 (65%) | 8 (35%) | 23 (100%) |
| Study phase | ||||
| Phase II | 33 (23%) | 17 (52%) | 7 (21%) | 28 (85%) |
| Phase III | 105 (74%) | 87 (83%) | 44 (42%) | 102 (97%) |
| Phase II/III | 4 (3%) | 2 (50%) | 0 (0%) | 2 (50%) |
| Randomization | ||||
| NA (single-group) | 5 (4%) | 2 (40%) | 0 (0%) | 5 (100%) |
| Randomized | 136 (96%) | 103 (76%) | 51 (37%) | 126 (93%) |
| Non-randomized | 1 (1%) | 1 (100%) | 0 (0%) | 1 (100%) |
| Blinding | ||||
| Double-blinded | 135 (95%) | 102 (76%) | 51 (37%) | 125 (92%) |
| Open-label | 7 (5%) | 4 (57%) | 0 (0%) | 7 (100%) |
| Comparators | ||||
| Placebo only | 94 (66%) | 70 (74%) | 42 (45%) | 86 (91%) |
| Active comparator only | 8 (6%) | 5 (63%) | 1 (13%) | 7 (88%) |
| Placebo and active comparator | 29 (20%) | 23 (79%) | 6 (21%) | 28 (97%) |
| Lower-dose comparator only | 4 (3%) | 4 (100%) | 2 (50%) | 4 (100%) |
| No comparator | 7 (5%) | 4 (57%) | 0 (0%) | 7 (100%) |