Table 1.
Characteristics of included studies (n=222)
| No. (%) | |
|---|---|
| Year of Publication | |
| 2015 | 92 (41) |
| 2014 | 81 (37) |
| 2013 | 49 (22) |
| Object of Study | |
| Drug | 219 (99) |
| Device | 3 (1) |
| Phase | |
| II | 54 (24) |
| III | 143 (64) |
| II / III | 1 (0.5) |
| Not applicablea | 23 (10) |
| Not specifiedb | 1 (0.5) |
| Journal | |
| Lancet Oncology | 138 (62) |
| European Journal of Cancer |
33 (15) |
| European Urology | 16 (7) |
| Lancet | 13 (6) |
| Journal of the American Medical Association (JAMA) |
7 (3) |
| JAMA Oncology | 4 (2) |
| American Journal of Gastroenterology |
3 (1) |
| Other | 8 (4) |
| Study design | |
| Superiority | 198 (89) |
| Noninferiority | 18 (8) |
| Neither | 2 (1) |
| Safety / Efficacy | 4 (2) |
| Analysis design | |
| Intention-to-treat | 140 (63) |
| Modified intention-to-treat | 77 (35) |
| Neither / per protocol | 5 (2) |
| Placebo Control | 66 (30) |
| Primary endpoint | |
| Overall survival | 52 (23) |
| PFS / DFS | 94 (42) |
| Objective response | 17 (8) |
| Other | 39 (18) |
| Composite | 20 (9) |
| Patients blinded | 70 (32) |
| Clinicians blinded | 69 (31) |
| Outcomes assessors blinded | 63 (28) |
| Primary result | |
| Positive | 118 (53) |
| Negative | 97 (44) |
| Mixed | 7 (3) |
a
Refers to trials of approved drugs or devices, or follow-up studies
b
Refers to preliminary trials of novel compounds for therapy
PFS=progression-free survival; DFS=disease-free survival