Table 3.

Patient characteristics and multivariable predictors of therapeutic proportion

VariableTotal No. of patients (proportion of total, %)No. of therapeutic patients (therapeutic proportion, %)Rate ratio (95% CI)
Trial start year
 20053676 (11.3)107 (2.9)1 (Referent)
 20065269 (16.2)41 (0.8)0.21 (0.05 to 0.84)
 20075196 (15.9)19 (0.4)0.10 (0.02 to 0.67)
 20084529 (13.9)98 (2.2)0.58 (0.13 to 2.6)
 20095740 (17.6)45 (0.8)0.26 (0.05 to 1.3)
 20108172 (25.1)76 (0.9)0.28 (0.04 to 1.8)
Sponsorship
 Nonindustry12 509 (38.4)87 (0.7)1 (Referent)
 Industry20 073 (61.6)299 (1.5)2.92 (0.72 to 12.14)
No. of drugs
 Monotherapy: 1 drug16 845 (51.7)311 (1.8)1 (Referent)
 Combination: ≥2 drugs15 737 (48.3)75 (0.5)0.09 (0.01 to 0.68)
Approval status
 Previously approved16 542 (50.8)142 (0.9)1 (Referent)
 Novel treatment16 040 (49.2)244 (1.5)3.18 (0.63 to 16.00)
Biomarker enrichment
 No29 482 (90.5)282 (1.0)1 (Referent)
 Yes3100 (9.5)104 (3.4)4.49 (1.53 to 13.23)
Cancer indication
 Hematological5797 (17.8)108 (1.9)1 (Referent)
 Solid tumor26 785 (82.2)278 (1.1)0.82 (0.30 to 2.29)
Drug class
 Cytotoxic5270 (16.2)100 (1.9)1 (Referent)
 Targeted22 116 (67.9)215 (1.0)0.48 (0.13 to 2.44)
 Immunotherapy3661(11.2)24 (0.7)0.56 (0.02 to 1.78)
 Other1535 (4.7)47 (3.1)1.00 (0.09 to 10.83)
No. of indications tested
 Mixed: ≥2 tested17 413 (53.4)105 (0.6)1 (Referent)
 Single: 1 tested15 169 (46.6)281 (1.9)3.32 (1.2 to 9.1)
VariableTotal No. of patients (proportion of total, %)No. of therapeutic patients (therapeutic proportion, %)Rate ratio (95% CI)
Trial start year
 20053676 (11.3)107 (2.9)1 (Referent)
 20065269 (16.2)41 (0.8)0.21 (0.05 to 0.84)
 20075196 (15.9)19 (0.4)0.10 (0.02 to 0.67)
 20084529 (13.9)98 (2.2)0.58 (0.13 to 2.6)
 20095740 (17.6)45 (0.8)0.26 (0.05 to 1.3)
 20108172 (25.1)76 (0.9)0.28 (0.04 to 1.8)
Sponsorship
 Nonindustry12 509 (38.4)87 (0.7)1 (Referent)
 Industry20 073 (61.6)299 (1.5)2.92 (0.72 to 12.14)
No. of drugs
 Monotherapy: 1 drug16 845 (51.7)311 (1.8)1 (Referent)
 Combination: ≥2 drugs15 737 (48.3)75 (0.5)0.09 (0.01 to 0.68)
Approval status
 Previously approved16 542 (50.8)142 (0.9)1 (Referent)
 Novel treatment16 040 (49.2)244 (1.5)3.18 (0.63 to 16.00)
Biomarker enrichment
 No29 482 (90.5)282 (1.0)1 (Referent)
 Yes3100 (9.5)104 (3.4)4.49 (1.53 to 13.23)
Cancer indication
 Hematological5797 (17.8)108 (1.9)1 (Referent)
 Solid tumor26 785 (82.2)278 (1.1)0.82 (0.30 to 2.29)
Drug class
 Cytotoxic5270 (16.2)100 (1.9)1 (Referent)
 Targeted22 116 (67.9)215 (1.0)0.48 (0.13 to 2.44)
 Immunotherapy3661(11.2)24 (0.7)0.56 (0.02 to 1.78)
 Other1535 (4.7)47 (3.1)1.00 (0.09 to 10.83)
No. of indications tested
 Mixed: ≥2 tested17 413 (53.4)105 (0.6)1 (Referent)
 Single: 1 tested15 169 (46.6)281 (1.9)3.32 (1.2 to 9.1)
Table 3.

Patient characteristics and multivariable predictors of therapeutic proportion

VariableTotal No. of patients (proportion of total, %)No. of therapeutic patients (therapeutic proportion, %)Rate ratio (95% CI)
Trial start year
 20053676 (11.3)107 (2.9)1 (Referent)
 20065269 (16.2)41 (0.8)0.21 (0.05 to 0.84)
 20075196 (15.9)19 (0.4)0.10 (0.02 to 0.67)
 20084529 (13.9)98 (2.2)0.58 (0.13 to 2.6)
 20095740 (17.6)45 (0.8)0.26 (0.05 to 1.3)
 20108172 (25.1)76 (0.9)0.28 (0.04 to 1.8)
Sponsorship
 Nonindustry12 509 (38.4)87 (0.7)1 (Referent)
 Industry20 073 (61.6)299 (1.5)2.92 (0.72 to 12.14)
No. of drugs
 Monotherapy: 1 drug16 845 (51.7)311 (1.8)1 (Referent)
 Combination: ≥2 drugs15 737 (48.3)75 (0.5)0.09 (0.01 to 0.68)
Approval status
 Previously approved16 542 (50.8)142 (0.9)1 (Referent)
 Novel treatment16 040 (49.2)244 (1.5)3.18 (0.63 to 16.00)
Biomarker enrichment
 No29 482 (90.5)282 (1.0)1 (Referent)
 Yes3100 (9.5)104 (3.4)4.49 (1.53 to 13.23)
Cancer indication
 Hematological5797 (17.8)108 (1.9)1 (Referent)
 Solid tumor26 785 (82.2)278 (1.1)0.82 (0.30 to 2.29)
Drug class
 Cytotoxic5270 (16.2)100 (1.9)1 (Referent)
 Targeted22 116 (67.9)215 (1.0)0.48 (0.13 to 2.44)
 Immunotherapy3661(11.2)24 (0.7)0.56 (0.02 to 1.78)
 Other1535 (4.7)47 (3.1)1.00 (0.09 to 10.83)
No. of indications tested
 Mixed: ≥2 tested17 413 (53.4)105 (0.6)1 (Referent)
 Single: 1 tested15 169 (46.6)281 (1.9)3.32 (1.2 to 9.1)
VariableTotal No. of patients (proportion of total, %)No. of therapeutic patients (therapeutic proportion, %)Rate ratio (95% CI)
Trial start year
 20053676 (11.3)107 (2.9)1 (Referent)
 20065269 (16.2)41 (0.8)0.21 (0.05 to 0.84)
 20075196 (15.9)19 (0.4)0.10 (0.02 to 0.67)
 20084529 (13.9)98 (2.2)0.58 (0.13 to 2.6)
 20095740 (17.6)45 (0.8)0.26 (0.05 to 1.3)
 20108172 (25.1)76 (0.9)0.28 (0.04 to 1.8)
Sponsorship
 Nonindustry12 509 (38.4)87 (0.7)1 (Referent)
 Industry20 073 (61.6)299 (1.5)2.92 (0.72 to 12.14)
No. of drugs
 Monotherapy: 1 drug16 845 (51.7)311 (1.8)1 (Referent)
 Combination: ≥2 drugs15 737 (48.3)75 (0.5)0.09 (0.01 to 0.68)
Approval status
 Previously approved16 542 (50.8)142 (0.9)1 (Referent)
 Novel treatment16 040 (49.2)244 (1.5)3.18 (0.63 to 16.00)
Biomarker enrichment
 No29 482 (90.5)282 (1.0)1 (Referent)
 Yes3100 (9.5)104 (3.4)4.49 (1.53 to 13.23)
Cancer indication
 Hematological5797 (17.8)108 (1.9)1 (Referent)
 Solid tumor26 785 (82.2)278 (1.1)0.82 (0.30 to 2.29)
Drug class
 Cytotoxic5270 (16.2)100 (1.9)1 (Referent)
 Targeted22 116 (67.9)215 (1.0)0.48 (0.13 to 2.44)
 Immunotherapy3661(11.2)24 (0.7)0.56 (0.02 to 1.78)
 Other1535 (4.7)47 (3.1)1.00 (0.09 to 10.83)
No. of indications tested
 Mixed: ≥2 tested17 413 (53.4)105 (0.6)1 (Referent)
 Single: 1 tested15 169 (46.6)281 (1.9)3.32 (1.2 to 9.1)
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