Table 1.
Characteristics of included study protocols
| Trial Characteristic | Phase II (n = 15) | Phase III (n = 24) | All Trials (n = 39) |
|---|---|---|---|
| Sponsor | |||
| Pharmaceutical companyb | 12 (80%) | 23 (96%) | 35 (90%) |
| Top 21 pharmaceutical company | 9 (60%) | 13 (54%) | 22 (56%) |
| Planned sample size | |||
| Mean (±standard deviation) | 141 (±125) | 651 (±992) | 455 (±815) |
| Median (minimum, maximum) | 99 (30, 500) | 232 (15, 4126) | 180 (15, 4126) |
| Therapeutic areaa | |||
| Blood or blood‐forming organs | 0 (0.0%) | 3 (12.5%) | 3 (7.7%) |
| Endocrine, nutritional, or metabolic diseases | 0 (0.0%) | 2 (8.3%) | 2 (5.1%) |
| Mental, behavioral, or neurodevelopmental disorders | 0 (0.0%) | 2 (8.3%) | 2 (5.1%) |
| Nervous system | 0 (0.0%) | 3 (12.5%) | 3 (7.7%) |
| Visual system | 1 (6.7%) | 2 (8.3%) | 3 (7.7%) |
| Ear or mastoid process | 1 (6.7%) | 0 (0.0%) | 1 (2.6%) |
| Circulatory/cardiovascular system | 0 (0.0%) | 2 (8.3%) | 2 (5.1%) |
| Respiratory system | 6 (40.0%) | 6 (25.0%) | 12 (30.8%) |
| Digestive System | 3 (20.0%) | 1 (4.2%) | 4 (10.3%) |
| Skin | 1 (6.7%) | 1 (4.2%) | 2 (5.1%) |
| Immune system | 2 (13.3%) | 1 (4.2%) | 3 (7.7%) |
| Genitourinary system | 1 (6.7%) | 0 (0.0%) | 1 (2.6%) |
| Developmental abnomalies | 0 (0.0%) | 1 (4.2%) | 1 (2.6%) |
a
Only ICD‐11 (20) superior categories 01 to 20 are considered since categories 21 to 26 do not represent therapeutic areas.
b
Including the top 21 pharmaceutical companies.