Table S1. Characteristics of included trials.
| No. | Author & year | NCT number | P (Exp) | P (Con) | Funding source | Mask | Tumor type | Study design | Primary endpoint (outcome) |
|---|---|---|---|---|---|---|---|---|---|
| 1 | Baselga et al. 2012 (9) | NCT00863655 | 485 | 239 | Pharmaceutical | Double | BC | Super | L-PFS (+) |
| 2 | Piccart et al. 2014 (10) | ||||||||
| 3 | Yardley et al. 2013 (11) | ||||||||
| 4 | Beaver et al. 2012 (12) | ||||||||
| 5 | Cortes et al. 2011 (13) | NCT00388726 | 508 | 254 | Pharmaceutical | Open | BC | Super | OS (+) |
| 6 | Bergh et al. 2012 (14) | NCT00393939 | 296 | 297 | Pharmaceutical | Open | BC | Super | C-PFS (−) |
| 7 | Wu et al. 2014 (15) | NCT01121393 | 242 | 122 | Pharmaceutical | Open | NSCLC | Super | C-PFS (+) |
| 8 | Yang et al. 2015 (16) | ||||||||
| 9 | Miller et al. 2012 (17) | NCT00656136 | 390 | 195 | Pharmaceutical | Double | NSCLC | Super | OS (+) |
| 10 | Gianni et al. 2013 (18) | NCT00391092 | 216 | 208 | Pharmaceutical | Open | BC | Super | L-PFS (−) |
| 11 | Motzer et al. 2013 (19) | NCT00678392 | 361 | 362 | Pharmaceutical | Open | RCC | Super | C-PFS (+) |
| 12 | Rini et al. 2011 (20) | ||||||||
| 13 | Lynch et al. 2010 (21) | NCT00112294 | 338 | 338 | Pharmaceutical | Open | NSCLC | Super | C-PFS (−) |
| 14 | Hauschild et al. 2012 (22) | NCT01227889 | 187 | 63 | Pharmaceutical | Open | M | Super | L-PFS (+) |
| 15 | Motzer et al. 2014 (23) | NCT01223027 | 284 | 286 | Pharmaceutical | Open | RCC | Super | C-PFS (+) |
| 16 | Schwartzentruber et al. 2011 (24) | NCT00019682 | 91 | 94 | Academic | Single | M | Super | ORR (±†) |
| 17 | Von Hoff et al. 2013 (25) | NCT00844649 | 431 | 430 | Pharmaceutical | Open | PT | Super | OS (+) |
| 18 | Wu et al. 2013 (26) | NCT00883779 | 226 | 225 | Pharmaceutical | Double | NSCLC | Super | L-PFS (+) |
| 19 | Motzer et al. 2013 (27) | NCT00720941 | 557 | 553 | Pharmaceutical | Open | RCC | Non | C-PFS (+) |
| 20 | Kaufman et al. 2015 (28) | NCT00337103 | 554 | 548 | Pharmaceutical | Open | BC | Super | C-PFS + OS (−) |
| 21 | Sequist et al. 2013 (29) | NCT00949650 | 230 | 115 | Pharmaceutical | Open | NSCLC | Super | C-PFS (+) |
| 8 | Yang et al. 2015 (16) | ||||||||
| 22 | Crown et al. 2013 (30) | NCT00435409 | 221 | 221 | Pharmaceutical | Open | BC | Super | C-PFS (-) |
| 23 | Paz-Ares et al. 2012 (31) | NCT00449033 | 452 | 452 | Pharmaceutical | Double | NSCLC | Super | OS (−) |
| 24 | Motzer et al. 2013 (32) | NCT01030783 | 260 | 257 | Pharmaceutical | Open | RCC | Super | C-PFS (+) |
| 25 | Socinski et al. 2012 (33) | NCT00540514 | 521 | 531 | Pharmaceutical | Open | NSCLC | Non | C-ORR (+) |
| 26 | Aghajanian et al. 2014 (34) | NCT00434642 | 242 | 242 | Pharmaceutical | Double | OC | Super | L-PFS (+) |
| 27 | Aghajanian et al. 2012 (35) | ||||||||
| 28 | Colombo et al. 2012 (36) | NCT00262990 | 412 | 417 | Pharmaceutical | Open | OC | Super | OS (−) |
| 29 | Monk et al. 2010 (37) | NCT00113607 | 337 | 335 | Pharmaceutical | Open | OC | Super | C-PFS (+) |
| 30 | Monk et al. 2012 (38) | ||||||||
| 31 | Soria et al. 2015 (39) | NCT01523587 | 398 | 397 | Pharmaceutical | Open | NSCLC | Super | C-PFS (+) |
| 32 | Rini et al. 2016 (40) | NCT01265901 | 204 | 135 | Pharmaceutical | Open | RCC | Super | OS (−) |
| 33 | Choueiri et al. 2016 (41) | NCT01865747 | 330 | 328 | Pharmaceutical | Open | RCC | Super | C-PFS (+) |
| 34 | Blay et al. 2015 (42) | NCT00785785 | 324 | 320 | Pharmaceutical | Open | GST | Super | C-PFS (−) |
| 35 | Zhang et al. 2017 (43) | NCT02253459 | 270 | 135 | Pharmaceutical | Open | BC | Super | C-PFS (+) |
†, in one study, objective response rate (ORR) was the primary endpoint: significant difference in central review (P=0.03), not in local assessment (P=0.05). We consider “indeterminate” because we are unable to determine whether central- or local-assessed ORR was the primary endpoint (24). P, patients; Criteria, assessment criteria for tumor response or progression; BC, breast cancer; OC, ovarian cancer; NSCLC, non-small-cell lung cancer; RCC, renal-cell cancer; M, melanoma; PT, pancreatic tumor: GST, gastrointestinal stromal tumor; Super, superiority; Non, noninferiority; C-, central-assessed; L-, local-assessed; ORR, objective response rate; PFS, progression-free survival; OS, overall survival. +, positive; –, negative.