Table 1. Trial Characteristics and Outcomes by Study Perioda.
| Characteristic or outcome | 1986-1996 (n = 36) | 1997-2006 (n = 62) | 2007-2016 (n = 52) | P value |
|---|---|---|---|---|
| Trial characteristics | ||||
| Trial size, median (IQR), No. of participants | 308 (198.5-430.5) | 477.5 (291.0-820.0) | 404 (248.0-756.0) | NA |
| Line of therapy | ||||
| First | 34 (94.4) | 47 (75.8) | 28 (53.8) | <.001 |
| Second | 2 (5.6) | 13 (21.0) | 9 (17.3) | |
| Third and beyond | 0 | 2 (3.2) | 15 (28.8) | |
| Primary end point | ||||
| OS | 14 (38.9) | 16 (25.8) | 16 (30.8) | <.001 |
| PFSb | 5 (13.9) | 29 (46.8) | 23 (44.2) | |
| Response rate | 13 (36.1) | 10 (16.1) | 1 (1.9) | |
| Otherc | 4 (11.2) | 7 (11.3) | 12 (23.1) | |
| Treatment type | ||||
| Cytotoxic | 18 (51.4) | 32 (51.6) | 6 (11.5) | <.001 |
| Targeted | 0 | 2 (3.2) | 20 (38.5) | |
| Other | 4 (11.4) | 0 | 1 (1.9) | |
| Cytotoxic and targeted | 0 | 21 (33.9) | 22 (42.3) | |
| Cytotoxic and other | 13 (37.1) | 7 (11.3) | 3 (5.8) | |
| Placebo | ||||
| Yes | 3 (8.3) | 8 (12.9) | 18 (34.6) | .002 |
| No | 33 (91.7) | 54 (87.1) | 34 (65.4) | |
| Blinding | ||||
| Yes | 4 (11.1) | 7 (11.3) | 19 (36.5) | .001 |
| No | 32 (88.9) | 55 (88.7) | 33 (63.5) | |
| Funding source | ||||
| Pharmaceutical company | 8 (22.2) | 27 (43.5) | 30 (57.7) | <.001 |
| Nonprofit organization | 11 (30.6) | 12 (19.4) | 12 (23.1) | |
| Pharmaceutical company and nonprofit organization | 17 (47.2) | 23 (37.1) | 10 (19.2) | |
| Sites | ||||
| North America | 7 (19.4) | 8 (12.9) | 4 (7.7) | .001 |
| Europe | 19 (52.8) | 30 (48.4) | 16 (30.8) | |
| Asia | 0 | 1 (1.6) | 12 (23.1) | |
| Multiregion | 10 (27.8) | 21 (33.9) | 18 (34.6) | |
| Unknown or not reported | 0 | 2 (3.2) | 2 (3.8) | |
| Trial outcomes | ||||
| OS | ||||
| No improvement | 24 (72.7) | 44 (77.2) | 29/42 (69.0) | .66 |
| At least 2 mo of improvement | 9 (27.3) | 13 (22.8) | 13/42 (30.9) | |
| Positive primary end point | ||||
| Yes | 15 (41.7) | 27 (43.5) | 27/50 (54.0) | .43 |
| No | 21 (58.3) | 35 (56.5) | 23/50 (46.0) |
Abbreviations: NA, not applicable; OS, overall survival; PFS, progression-free survival.
a
Data are presented as number (percentage) of patients unless otherwise indicated.
b
Progression-free survival includes failure-free survival, treatment-free survival, and time to progression.
c
Other end points included survival rate, toxic effects, and quality of life.