Table 1 Characteristics of selected published trials (N = 275) and trials leading to an FDA approval (N = 77), according to the availability of assessable post-progression data
PUBLISHED TRIALS | TRIALS LEADING TO AN FDA APPROVAL | |||||
|---|---|---|---|---|---|---|
Total | Post-Progression data a NO | Post-Progression data YES | Total | Post-Progression data a NO | Post-Progression data YES | |
275 | 175 (63.6%) | 100 (36.4%) | 77 | 40 (51.9%) | 37 (48.1%) | |
Sponsor: | ||||||
Any industry involvment | 237 | 143 (60.3%) | 94 (39.6%) | NA b | NA b | NA b |
No industry involvment | 38 | 32 (84.2%) | 6 (15.8%) | NA b | NA b | NA b |
Phase c | ||||||
≤ 2 | 71 | 58 (81.7%) | 13 (18.3%) | 4 | 4 (100%) | 0 (0%) |
≥ 3 | 204 | 117 (57.4%) | 87 (42.6%) | 73 | 36 (49.3%) | 37 (50.7%) |
Design | ||||||
Blind | 91 | 52 (57.1%) | 39 (42.9%) | 31 | 15 (48.4%) | 16 (51.6%) |
Open | 184 | 123 (66.8%) | 61 (33.2%) | 46 | 25 (54.3%) | 21 (45.7%) |
Tumor Type | ||||||
Hematologic malignancy | 64 | 54 (84.4%) | 10 (15.6%) | 18 | 15 (83.3%) | 3 (16.7%) |
Solid Tumor | 211 | 121 (57.3%) | 90 (42.7%) | 59 | 25 (42.4%) | 34 (57.6%) |
OS Results | ||||||
Positive | 71 | 29 (40.8%) | 42 (59.2%) | 36 | 10 (27.8%) | 26 (72.2%) |
Others d | 204 | 146 (71.6%) | 58 (28.4%) | 41 | 30 (73.2%) | 11 (26.8%) |
Setting: | ||||||
First line | 128 | 74 (57.8%) | 54 (42.2%) | 39 | 15 (38.5%) | 24 (61.5%) |
Second line or subsequent | 92 | 61 (66.3%) | 31 (33.7%) | 15 | 7 (46.7%) | 8 (53.3%) |
Third line or subsequent | 14 | 8 (57.1%) | 6 (42.9%) | 7 | 6 (85.7%) | 1 (14.3%) |
Fourth or fifth or subsequent | 2 | 2 (100%) | 0 | 2 | 2 (100%) | 0 |
Maintenance | 22 | 16 (72.7%) | 6 (27.3%) | 7 | 4 (57.1%) | 3 (42.9%) |
Mixed | 17 | 14 (82.4%) | 3 (17.6%) | 7 | 6 (85.7%) | 1 (14.3%) |