Table 2.
Categories | Variables | Options | Reimbursement decisions, No. (%) | Test | P-value | |
---|---|---|---|---|---|---|
Positive (n = 76) |
Negative (n = 36) |
|||||
Trial design characteristics | Primary endpoint | Only OS | 8 (10.5) | 11 (30.6) | Pearson's chi-squared test | 0.001 |
Only PFS | 32 (42.1) | 6 (16.7) | ||||
Only ORR | 23 (30.3) | 6 (16.7) | ||||
Other | 10 (13.2) | 7 (19.4) | ||||
Two endpoints | 3 (3.9) | 6 (16.7) | ||||
Phases | I | 3 (3.9) | 1 (2.8) | Fisher's exact test | 0.043 | |
II | 24 (31.6) | 4 (11.1) | ||||
III | 49 (64.5) | 31 (86.1) | ||||
Blinded experiment | Yes | 28 (36.8) | 16 (44.4) | Pearson's chi-squared test | 0.442 | |
No | 48 (63.2) | 20 (55.6) | ||||
Control group | Yes | 53 (69.7) | 28 (77.8) | Pearson's chi-squared test | 0.374 | |
No | 23 (30.3) | 8 (22.2) | ||||
Randomization | Yes | 54 (71.1) | 28 (77.8) | Pearson's chi-squared test | 0.453 | |
No | 22 (28.9) | 8 (22.2) | ||||
Clinical outcomes | HROS data availability | Yes | 30 (39.5) | 19 (52.8) | Pearson's chi-squared test | 0.185 |
No | 46 (60.5) | 17 (47.2) | ||||
Risk benefit in OS | Yes | 17 (22.4) | 18 (50.0) | Pearson's chi-squared test | 0.011 | |
No | 13 (17.1) | 1 (2.8) | ||||
Missing | 46 (60.5) | 17 (47.2) | / | / | ||
Magnitude of risk benefit in OS | HROS, median (interquartile range) | 0.680 [0.530–0.747] | 0.625 [0.428–0.743] | Two-sample t test | 0.627 | |
HRPFS data availability | Yes | 41 (53.9) | 18 (50.0) | Pearson's chi-squared test | 0.696 | |
No | 35 (46.1) | 18 (50.0) | ||||
Risk benefit in PFS | Yes | 37 (48.7) | 16 (44.4) | Pearson's chi-squared test | 1.000 | |
No | 4 (5.3) | 2 (5.6) | ||||
Missing | 35 (46.1) | 18 (50.0) | / | / | ||
Magnitude of risk benefit in PFS | HRPFS, median (interquartile range) | 0.377 [0.280–0.563] | 0.588 [0.280–0.735] | Two-sample t test | 0.087 | |
Magnitude of survival benefit | Survival benefit for mOS, median | 2.6 [1.875–5.875] | 4.95 [1.430-6.680] | Mann-Whitney U Test | 0.545 | |
Survival benefit for mPFS, median | 5.00 [2.075–6.975] | 2.10 [0.080–8.075] | Mann-Whitney U Test | 0.189 |