Table 2 Characteristics of early phase studies and associations with phase III study results
Study characteristics | All early phase studies (n = 59) | True neutral (n = 37) | False positive (n = 22) | aOR [95%-CI] | P |
|---|---|---|---|---|---|
No. of subjects, median (IQR) | 92.0 (46.0-176.0) | 100.0 (46.0-157.0) | 91.5 (49.3-197.5) | 1.00 [0.99-1.00] | 0.713 |
Industry sponsored, n (%) | 36 (61.0) | 25 (67.6) | 11 (50.0) | 1.73 [0.40-7.54] | 0.465 |
Multicentered, n (%) | 41 (69.5) | 28 (75.7) | 13 (59.1) | 1.48 [0.28-7.87] | 0.643 |
Randomized, n (%) | 57 (96.6) | 35 (94.6) | 22 (100.0) | 0.00 [0.00-∞] | 1.000 |
Blinded outcome assessment, n (%) | 52 (88.1) | 34 (91.9) | 18 (81.8) | 3.95 [0.26-60.30] | 0.323 |
Dose–response investigated, n (%) | 16 (27.1) | 12 (32.4) | 4 (18.2) | 1.25 [0.26-6.07] | 0.786 |
Use of imaging endpoint, n (%) | 15 (25.4) | 8 (21.6) | 7 (31.8) | 0.46 [0.12-1.81] | 0.263 |
Duration of follow-up for endpoints*, median (IQR), days | 87.0 (30.0-90.0) | 90.0* (30.0-90.0) | 41.0 (28.0-90.0) | 1.00 [0.99-1.01] | 0.722 |
Same characteristics in phase I/II studies as in subsequent phase III studies | |||||
Same therapeutic time window, n (%) | 19 (32.2) | 10 (27.0) | 9 (40.9) | 0.69 [0.18-2.68] | 0.588 |
Same dose, n (%) | 29 (49.2) | 18 (48.6) | 11 (50.0) | 0.94 [0.28-3.14] | 0.916 |
Same route of administration, n (%) | 50 (84.7) | 34 (91.9) | 16 (72.7) | 3.33 [0.59-18.86] | 0.174 |