Table 3.
Patient characteristics and multivariable predictors of therapeutic proportion
| Variable | Total No. of patients (proportion of total, %) | No. of therapeutic patients (therapeutic proportion, %) | Rate ratio (95% CI) |
|---|---|---|---|
| Trial start year | |||
| 2005 | 3676 (11.3) | 107 (2.9) | 1 (Referent) |
| 2006 | 5269 (16.2) | 41 (0.8) | 0.21 (0.05 to 0.84) |
| 2007 | 5196 (15.9) | 19 (0.4) | 0.10 (0.02 to 0.67) |
| 2008 | 4529 (13.9) | 98 (2.2) | 0.58 (0.13 to 2.6) |
| 2009 | 5740 (17.6) | 45 (0.8) | 0.26 (0.05 to 1.3) |
| 2010 | 8172 (25.1) | 76 (0.9) | 0.28 (0.04 to 1.8) |
| Sponsorship | |||
| Nonindustry | 12 509 (38.4) | 87 (0.7) | 1 (Referent) |
| Industry | 20 073 (61.6) | 299 (1.5) | 2.92 (0.72 to 12.14) |
| No. of drugs | |||
| Monotherapy: 1 drug | 16 845 (51.7) | 311 (1.8) | 1 (Referent) |
| Combination: ≥2 drugs | 15 737 (48.3) | 75 (0.5) | 0.09 (0.01 to 0.68) |
| Approval status | |||
| Previously approved | 16 542 (50.8) | 142 (0.9) | 1 (Referent) |
| Novel treatment | 16 040 (49.2) | 244 (1.5) | 3.18 (0.63 to 16.00) |
| Biomarker enrichment | |||
| No | 29 482 (90.5) | 282 (1.0) | 1 (Referent) |
| Yes | 3100 (9.5) | 104 (3.4) | 4.49 (1.53 to 13.23) |
| Cancer indication | |||
| Hematological | 5797 (17.8) | 108 (1.9) | 1 (Referent) |
| Solid tumor | 26 785 (82.2) | 278 (1.1) | 0.82 (0.30 to 2.29) |
| Drug class | |||
| Cytotoxic | 5270 (16.2) | 100 (1.9) | 1 (Referent) |
| Targeted | 22 116 (67.9) | 215 (1.0) | 0.48 (0.13 to 2.44) |
| Immunotherapy | 3661(11.2) | 24 (0.7) | 0.56 (0.02 to 1.78) |
| Other | 1535 (4.7) | 47 (3.1) | 1.00 (0.09 to 10.83) |
| No. of indications tested | |||
| Mixed: ≥2 tested | 17 413 (53.4) | 105 (0.6) | 1 (Referent) |
| Single: 1 tested | 15 169 (46.6) | 281 (1.9) | 3.32 (1.2 to 9.1) |