Table 1. Comparison of Characteristics of Cancer Drug Submissions Recommended vs Not Recommended for Reimbursement by Pan-Canadian Oncology Drug Review Between 2011 and 2020.
| Characteristic | Final reimbursement recommendation, No. (%) | P value | |
|---|---|---|---|
| Positive (n = 78) | Negative (n = 26) | ||
| General submission characteristics | |||
| Time to decision, median (IQR), d | 213.0 (193.5-242.0) | 229.5 (221.0-248.8) | .06 |
| Type of submission | |||
| New drug | 30 (38.5) | 11 (42.3) | .94 |
| New indication | 41 (52.6) | 13 (50.) | |
| Resubmission | 7 (9.0) | 2 (7.7) | |
| Tumor type | |||
| Lung | 18 (23.1) | 7 (26.9) | .16 |
| Gastrointestinal | 10 (12.8) | 8 (30.8) | |
| Breast | 10 (12.8) | 4 (15.4) | |
| Genitourinary | 12 (15.4) | 2 (7.7) | |
| Melanoma | 14 (18.0) | 0 | |
| Gynecological | 5 (6.4) | 1 (3.9) | |
| Other | 9 (11.5) | 4 (15.4) | |
| Drug class | |||
| Small molecule | 33 (42.3) | 15 (57.7) | .28 |
| Immune checkpoint inhibitor | 20 (25.6) | 3 (11.5) | |
| Monoclonal antibody | 10 (12.8) | 4 (15.4) | |
| Cytotoxic | 4 (5.1) | 3 (11.5) | |
| Hormonal | 6 (7.7) | 0 | |
| Other | 5 (6.4) | 1 (3.9) | |
| Treatment setting | |||
| Neoadjuvant | 0 | 1 (3.8) | .16 |
| Adjuvant | 4 (5.1) | 2 (7.7) | |
| Advanced/metastatic | 74 (94.9) | 23 (88.5) | |
| Line of therapya | |||
| First line | 33 (44.6) | 4 (17.4) | .06 |
| Second line and beyond | 37 (50.0) | 18 (78.3) | |
| Maintenance | 4 (5.4) | 1 (4.4) | |
| Trial characteristics | |||
| Trial design | |||
| Phase | |||
| 1 | 1 (1.3) | 4 (15.4) | <.001 |
| 2 | 5 (6.4) | 8 (30.8) | |
| 3 | 72 (92.3) | 14 (53.8) | |
| Randomization | |||
| No | 6 (7.7) | 11 (42.3) | <.001 |
| Yes | 72 (92.3) | 15 (57.7) | |
| Masking | |||
| No | 35 (48.6) | 4 (26.7) | .15 |
| Yes | 37 (51.4) | 11 (73.3) | |
| Primary end point | |||
| OS | 25 (32.1) | 6 (23.1) | <.001 |
| PFS | 42 (53.9) | 6 (23.1) | |
| RR | 5 (6.4) | 10 (38.5) | |
| Other | 6 (7.7) | 4 (15.4) | |
| Clinical characteristics | |||
| PFS data available | |||
| No | 10 (12.8) | 12 (46.2) | <.001 |
| Yes | 68 (87.2) | 14 (53.9) | |
| PFS improvement | |||
| No | 11 (16.2) | 2 (14.3) | .69 |
| Yes | 57 (83.8) | 12 (85.7) | |
| PFS gain, median (IQR)b | 4.7 (2.8-8.2) | 2.9 (0.8-4.0) | .06 |
| OS data available | |||
| No | 13 (16.7) | 12 (46.2) | .01 |
| Yes | 65 (83.3) | 14 (53.9) | |
| OS improvement | |||
| No | 26 (40.0) | 6 (42.9) | .99 |
| Yes | 39 (60.0) | 8 (57.1) | |
| OS gain, median (IQR)b | 3.7 (2.7-6.5) | 1.9 (1.4-4.5) | .06 |
| Substantial clinical benefit by the ESMO-MCBS | |||
| No | 30 (38.5) | 21 (80.8) | <.001 |
| Yes | 48 (61.5) | 5 (19.2) | |
Abbreviations: ESMO-MCBS, European Society for Medical Oncology-Magnitude of Clinical Benefit Scale; IQR, interquartile range; OS, overall survival; PFS, progression-free survival; RR, response rate.
a
If advanced or metastatic treatment setting.
b
Excluding values of 0 (ie, no difference between intervention and comparator) and those where neither the intervention nor comparator had reached its median value.