TABLE 4.
Trial Characteristics and Quality Indicators Among Randomized Controlled Pediatric and Adult Drug Trials
| Domain | Measure | All Trials (N = 1636) | Pediatric Trials (N = 207) | Adult Trials (N = 1429) | Pa |
|---|---|---|---|---|---|
| Trial design | Primary funding source, n (%) | <.001 | |||
| Industry | 812 (49.6) | 61 (29.5) | 751 (52.6) | ||
| Government | 100 (6.1) | 16 (7.7) | 84 (5.9) | ||
| Nonprofit | 724 (44.2) | 130 (62.8) | 593 (41.5) | ||
| Study phase, n (%) | <.001 | ||||
| Phases 1, 2, 2/3 | 608 (37.2) | 41 (19.8) | 567 (39.7) | ||
| Phases 3, 4 | 866 (52.9) | 128 (61.8) | 738 (51.6) | ||
| Unknown phase | 162 (9.9) | 38 (18.4) | 124 (8.7) | ||
| Comparator type, n (%) | .41 | ||||
| Active agent | 770 (47.1) | 103 (49.8) | 666 (46.6) | ||
| Placebo | 866 (52.9) | 104 (50.2) | 768 (53.4) | ||
| Masking, n (%) | .18 | ||||
| Double-blind | 1217 (74.4) | 144 (69.6) | 1073 (75.1) | ||
| Single blind | 72 (4.4) | 9 (4.4) | 62 (4.3) | ||
| No blinding | 347 (21.2) | 54 (26.1) | 293 (20.5) | ||
| Inclusion of primary safety outcome, n (%) | 261 (16.0) | 20 (9.7) | 241 (16.9) | .008 | |
| Scope, n (%) | .007 | ||||
| Multicenter | 938 (57.3) | 102 (49.3) | 835 (58.5) | ||
| Single center | 600 (36.7) | 96 (46.4) | 504 (35.3) | ||
| Unknown | 98 (6.0) | 9 (4.4) | 89 (6.2) | ||
| Anticipated sample size, median (IQR)b | 142 (50–390) | 220 (90–450) | 124 (50–380) | <.001 | |
| Length of trial conduct, y, median (IQR)c | 1.7 (1.0–2.7) | 1.8 (1.0–2.7) | 1.7 (1.0–2.7) | .53 | |
| Trial conduct | Registration before study start, N (%) | 659 (40.3) | 97 (46.9) | 562 (39.3) | .04 |
| Registration before study completion, n (%)c | 1359 (95.4) | 170 (92.9) | 1189 (95.7) | .09 | |
| Attainment of ≥75% anticipated study sample, n (%)d | 180 (90.0) | 34 (89.5) | 146 (90.1) | .90 | |
| Provision of study results, n (%) | 120 (7.3) | 16 (7.7) | 104 (7.3) | .82 | |
| Results reporting | Outcomed,e | .62 | |||
| Positive | 121 (67.2) | 22 (71.0) | 99 (66.4) | ||
| Negative | 59 (32.8) | 9 (29.0) | 50 (33.6) |
IQR, interquartile range.
a
P value based on χ2 for categorical variables, Student t test for mean values, and Kruskal-Wallis test for median values.
b
Based on 1622 trials with enrollment data.
c
Based on 1425 trials with available completion dates.
d
Based on 200 trials with publications.
e
Five publications included results pooled from 2 studies; 15 trials designated as neither positive or negative.