Table II.
Pediatric sub-specialty trial characteristics
| Pediatric (*) CV | Adult (*) CV | Pediatric (*) ID | Pediatric (*) Hematology/ oncology | Pediatric (*) Mental health | Pediatric (*) Other therapeutic areas | |
|---|---|---|---|---|---|---|
|
| ||||||
| (N = 213) | (N=5256) | (N = 1176) | (N = 346) | (N = 664) | (N = 2636) | |
| Age eligible for enrollment (†), n (%) | ||||||
| Neonates/infants (0–1 yr) | 130 (61%) | NA | 839 (71%) | 236 (68%) | 23 (4%) | 1122 (43%) |
| Children (1–12 yrs) | 144 (68%) | NA | 835 (71%) | 304 (88%) | 601 (91%) | 2038 (77%) |
| Adolescents (12–18 yrs) | 103 (48%) | NA | 288 (25%) | 233 (67%) | 405 (61%) | 1247 (47%) |
| Study duration (yrs) (‡) | ||||||
| Median (25th, 75th) | 2.2 (1.4, 3.3) | 2.0 (1.1, 3.1) | 1.3 (0.7, 2.3) | 2.9 (1.6, 4.7) | 2.4 (1.4, 3.9) | 1.9 (1.0, 3.2) |
| Study duration missing, n (%) | 55 (26%) | 1278 (24.3%) | 314 (27%) | 89 (26%) | 162 (24%) | 625 (24%) |
| Enrollment (No. of subjects) (‡) | ||||||
| Median (25th, 75th) | 68 (36, 186) | 115 (46, 304) | 305 (120, 751) | 60.0 (26, 205) | 80.0 (40.0, 192.0) | 88 (40, 200) |
| 0 to 100 | 133 (64%) | 2463 (48%) | 260 (22%) | 206 (62%) | 372 (57%) | 1461(57%) |
| 101 to 500 | 62 (30%) | 1804 (35%) | 487 (43%) | 92 (28%) | 253 (39%) | 855 (33%) |
| 501 to 1000 | 8 (4%) | 393 (7%) | 177 (16%) | 14 (4%) | 15 (2%) | 113 (4%) |
| >1000 | 4 (1.9%) | 454 (9%) | 218 (19%) | 20 (6%) | 10 (2%) | 142 (6%) |
| Enrollment missing | 6 (3%) | 142 (3%) | 34 (3%) | 14 (4%) | 14 (2%) | 65 (3%) |
| Phase, n (%) | ||||||
| Early (<= 2) | 64 (30%) | 1232 (23%) | 351 (30%) | 139 (40%) | 180 (27%) | 615 (23%) |
| Late phase (>= 2/3) | 77 (36%) | 2412 (46%) | 676 (58%) | 111 (32%) | 309 (47%) | 1063 (40%) |
| NA | 72 (34%) | 1612 (31%) | 149 (13%) | 96 (28%) | 175 (26%) | 958 (36%) |
| Intervention type, n (%) | ||||||
| Procedure/Device | 25 (12%) | 1858 (35%) | 63 (5%) | 76 (22%) | 24 (4%) | 458 (17%) |
| Drug/Biological | 144 (68%) | 2559 (49%) | 1030 (88%) | 224 (65%) | 366 (55%) | 1320 (50%) |
| Behavioral | 22 (10%) | 387 (7%) | 32 (3%) | 19 (6%) | 215 (32%) | 438 (17%) |
| Other (||) | 22 (10%) | 452 (9%) | 51 (4%) | 27 (8%) | 59 (9%) | 420 (16%) |
| Endpoint classification, n (%) | ||||||
| Safety | 12 (7%) | 283 (6.3%) | 142 (13%) | 15 (6%) | 27 (5%) | 135 (6%) |
| Efficacy | 66 (37%) | 1693 (37.7%) | 321 (30%) | 92 (38%) | 283 (49%) | 1001 (46%) |
| Safety/Efficacy | 79 (45%) | 2294 (51.1%) | 539 (50%) | 120 (49%) | 250 (44%) | 912 (42%) |
| Pharmacometrics | 20 (11%) | 223 (5%) | 68 (6%) | 18 (7%) | 14 (2%) | 135 (6%) |
| Endpoint classification missing | 36 (17%) | 763 (15%) | 106 (9%) | 101 (29%) | 90 (14%) | 453 (17%) |
| Location of study facilities, n (%) | ||||||
| U.S. only | 84 (44%) | 1621 (34%) | 227 (21%) | 132 (42%) | 408 (69%) | 1154 (48%) |
| Foreign only | 71 (37%) | 2824 (59%) | 769 (72%) | 150 (48%) | 162 (27%) | 1110 (47%) |
| Both U.S. and Foreign | 38 (20%) | 383 (8%) | 69 (7%) | 34 (11%) | 23 (4%) | 121 (5%) |
| Locations unknown | 20 (9%) | 428 (8%) | 111 (9%) | 30 (9%) | 71 (11%) | 251 (10%) |
| Derived funding source (§), n (%) | ||||||
| Industry | 69 (32%) | 2356 (45%) | 708 (60%) | 83 (24%) | 227 (34%) | 839 (32%) |
| NIH | 24 (11%) | 295 (6%) | 84 (7%) | 57 (17%) | 149 (22%) | 305 (12%) |
| Other | 120 (56%) | 2605 (50%) | 384 (33%) | 206 (60%) | 288 (43%) | 1492 (57%) |
Restricted to trials excluding participants > 18 years for pediatric trials and trials excluding patients <18 years for adult CV trials.
Studies may enroll participants in > 1 age group and could be included in multiple columns.
Includes actual and anticipated duration or enrollment for completed and ongoing studies, respectively.
Funding source derived from submitted lead sponsor and collaborator information.
Other interventions represent radiation, genetic (including gene transfer, stem cell and recombinant DNA), or dietary supplement (e.g., vitamins, minerals), as well as trials where interventions were identified as “other” (i.e., not any of the other listed intervention types.
Data are rounded to the nearest % and may sum to <> 100%.