Table 2.
Descriptive characteristics and accrual rate among randomised and non-randomised clinical trials supporting oncology US Food and Drug Administration approvals
| Trial characteristics | Randomised | Non-randomised | ||||
| N (%) | Accrual rate (IQR)* | P value† | N (%) | Accrual rate (IQR)* | P value† | |
| Total number | 133 (100) | NA | 61 (100) | NA | ||
| Sample size | NA | NA | ||||
| Median (IQR) | 466 (319–669) | 105 (74–206) | ||||
| Duration of enrolment (months) | NA | NA | ||||
| Median (IQR) | 22 (16–27) | 22 (14–34) | ||||
| Accrual rate | NA | NA | ||||
| Median (IQR) | 22 (15–36) | 5 (3–12) | ||||
| Blinding | ||||||
| Less than double blind | 87 (65) | 20 (13–32) | 0.008 | 61 (100) | 5 (3–12) | NA |
| Double blind and over | 46 (35) | 32 (18–41) | 0 (0) | 0 (0) | ||
| Primary endpoint | ||||||
| OS | 36 (27) | 22 (18–36) | 1 (0) | 16 (16–16) | ||
| PFS | 72 (54) | 21 (14–36) | 0.007 | 2 (3) | 8 (6–10) | <0.001 |
| Response rate | 25 (19) | 23 (13–35) | 58 (97) | 5 (3–11) | ||
| Phase | ||||||
| 1/2 | 13 (11) | 10 (6–15) | 0.005 | 56 (92) | 5 (3–12) | <0.001 |
| 3 | 120 (89) | 24 (17–38) | 5 (8) | 6 (4–7) | ||
| Line of therapy | ||||||
| First or second line | 115 (86) | 24 (17–39) | 47 (77) | 5 (3–8) | ||
| Third line or beyond | 18 (14) | 16 (13–20) | 0.002 | 14 (23) | 5 (2–13) | 0.118 |
| Manufacturer | ||||||
| Pfizer | 12 (9) | 19 (12–33) | 2 (3) | 2 (2–3) | ||
| Roche | 18 (14) | 21 (17–38) | 3 (5) | 5 (4–14) | <0.001 | |
| Novartis | 7 (5) | 24 (16–34) | <0.001 | 5 (8) | 4 (2–9) | |
| Merck | 11 (8) | 35 (18–47) | 9 (15) | 13 (6–19) | ||
| All else | 85 (64) | 21 (15–34) | 42 (69) | 4 (3–8) | ||
| Randomisation ratio | ||||||
| Equal | 95 (71) | 23 (15–36) | 0 (0) | 0 (0) | ||
| Skewed | 38 (29) | 21 (15–35) | 0.03 | 0 (0) | 0 (0) | NA |
| NA‡ | 0 (0) | 0 (0) | 61 (100) | 5 | ||
| Crossover specified | ||||||
| Yes | 55 (41) | 28 (19–41) | 0.03 | 1 (1) | 14 (14–14) | 0.006 |
| No | 78 (59) | 20 (14–32) | 60 (99) | 5 (3–11) | ||
| Single agent response rate | ||||||
| Median (IQR) | 44% (22–65) | NA | 44% (31–62) | NA | ||
| Sites, number | ||||||
| Median (IQR) | 135 (95–189) | NA | 47 (30–67) | NA | ||
| Disease burden§ | ||||||
| Median (IQR) | 23 660 (13 780–43 600) | NA | 20 720 (13 780–34 130) | NA | ||
*Accrual rate: reported as median accrual rate; persons per month accrued.
†P value from Wilcoxen Signed Rank test and x2 test for categorical variables
‡Non-randomised studies do not have a randomisation ratio.
§Disease burden: number of deaths in 1 year per tumour type using data from Surveillance, Epidemiology and End Results.16
NA, not applicable; OS, overall survival; PFS, progression-free survival; RCT, randomised controlled trial.