Table 1. Design characteristics and results of phase 3 RCTs in hematological and solid tumours published globally 2014–2017.
| All RCTs n = 694 |
Hematological n = 124 |
Solid tumour n = 570 |
p-value |
|
|---|---|---|---|---|
| N (%) | N (%) | N (%) | ||
| Experimental arm | ||||
| Systemic | 601 (87) | 121 (98) | 480 (84) | 0.002 |
| Radiation | 38 (6) | 1 (1) | 37 (7) | |
| Surgery | 16 (2) | 0 (0) | 16 (3) | |
| Other/combinations | 39 (6) | 2 (2) | 37 (7) | |
| Study design | ||||
| Superiority | 610 (88) | 109 (88) | 501 (88) | 1.00 |
| NI/equivalence | 84 (12) | 12 (12) | 69 (12) | |
| Primary endpoint | ||||
| OS | 215 (31) | 15 (12) | 200 (35) | <0.001 |
| DFS/EFS/RFS | 149 (22) | 24 (19) | 125 (22) | |
| PFS/TTF | 232 (33) | 58 (47) | 174 (31) | |
| Other@ | 98 (14) | 27 (22) | 71 (13) | |
| Country of origin~ | ||||
| HIC | 636 (92) | 119 (96) | 517 (91) | 0.113 |
| UMIC | 51 (7) | 5 (4) | 46 (8) | |
| LMIC | 7 (1) | 0 (0) | 7 (1) | |
| Industry funding | 493 (71) | 97 (78) | 396 (70) | 0.052 |
| Total sample size | ||||
| Median (IQR) | 443 (246–718) | 373 (236–544) | 465 (250–800) | 0.001 |
| Primary endpoint met | ||||
| Yes | 325 (47) | 69 (56) | 256 (45) | 0.037 |
| No | 369 (53) | 55 (44) | 314 (55) | |
| ^ IF | n = 686 | n = 124 | n = 562 | |
| Median (IQR) | 21 (7–27) | 13 (7–27) | 21 (7–28) | 0.768 |
| IF for + RCTs | n = 318 | n = 69 | n = 249 | |
| Median (IQR) | 25 (8–44) | 25 (11–48) | 25 (7–36) | 0.398 |
| IF for − RCTs | n = 368 | n = 55 | n = 313 | |
| Median (IQR) | 18 (6–26) | 12 (5–24) | 18 (6–26) | 0.024 |
| HR for + RCTs | n = 217* | n = 49a | n = 168b | |
| Median (IQR) | 0.68 (0.64–0.74) | 0.67 (0.61–0.72) | 0.69 (0.64–0.75) | 0.161 |
RCTs = randomized controlled trial, OS = overall survival, DFS = disease-free survival, EFS = eventfree survival, RFS = relapse-freeFS = progression free survival, TTF = time to treatment failure, NI = non-inferiority, HIC = High-income country, UMIC = upper-middle income country, LMIC = low-middle-income country. QOL = quality of life
Treatment intent was reported as ‘other’ for one and ‘not stated for another’, therefore n = 692
Other primary endpoints include QOL/toxicity (n = 4 and 17), response rate (n = 20 and 24), other or not specified (n = 3 and 30) for hematological and solid tumours, respectively
Based on the institutional affiliation of the first author
Impact factor not available for 6
Note that percentages may total to 101 due to rounding
p-values are based on the Fisher’s Exact test
RCT = Randomized controlled trial; IQR = interquartile range; HR = hazard ratio
*Only reported for n = 262 positive superiority trials but HR unavailable for 45, n = 217
Only reported for n = 59 positive superiority trials, but HR unavailable for 10, n = 49
Only reported for n = 203 positive superiority trials but HR unavailable for 35, n = 168
p-values are based on the Fisher’s exact test or the Mann-Witney U