Table 2.
Comparison of study variables in DURDs and DORDs submissions
| Variable | DURD N = 14 | DORD N = 46 | Effect size (95% CI) | P value |
|---|---|---|---|---|
| Molecular structure | ||||
| Biologic, n (%) | 11 (78.6) | 17 (37.0) | OR = 6.06 (1.25 to 38.58) | 0.012 |
| Small molecule, n (%) | 3 (21.4) | 29 (63.0) | ||
| Missing data, n (%) | 0 (0) | 0 (0) | ||
| Number of clinical studies considered, median (range) | 3 (3) | 2 (13) | Median difference = 0 (0 to 1) | 0.305 |
| Missing data, n (%) | 1 (7.1) | 1 (2.2) | ||
| Level of evidence considered | ||||
| At least one double blinded RCT, n (%) | 8 (57.1) | 42 (91.3) | OR = 0.13 (0.02 to 0.70) | 0.007 |
| At least one open label RCT, n (%) | 0 (0) | 3 (6.5) | ||
| At least one non-randomized uncontrolled trial, n (%) | 6 (42.9) | 1 (2.2) | ||
| Missing data, n (%) | 0 (0) | 0 (0) | ||
| Size of largest study, median, n of patients (range) | 59 (156) | 167 (1134) | Median difference = −108 (−234 to −50) | 0.0001 |
| Missing data, n (%) | 0 (0) | 5 (10.9) | ||
| Clinical study comparator | ||||
| No control, n (%) | 5 (35.7) | 1 (2.2) | OR = 23.11 (2.23 to 1207.19) | 0.0019 |
| Historical control, n (%) | 1 (7.1) | 0 (0) | ||
| placebo, n (%) | 7 (50.0) | 36 (78.3) | ||
| Active control, n (%) | 1 (7.1) | 9 (19.6) | ||
| Missing data, n (%) | 0 (0) | 0 (0) | ||
| Annual treatment cost per patient, median (range) | CAN$330,395 (CAN$934,000) | CAN$52,596 (CAN$429,858.5) | Median difference = CAN$243,787.75 (83,396 to 329,050) | P < 0.0001 |
| Missing data, n (%) | 1 (7.1) | 17 (37.0) | ||
| Incremental cost per quality adjusted life-year, median (range) | CAN$2,680,000 (CAN$560,000) | CAN$ 165,923.5 (CAN$4,574,241) | NA | NA |
| Missing data, n (%) | 12 (85.7) | 24 (52.2) | ||
| Recommendation | ||||
| Positive recommendation, n (%) | 5 (35.7) | 33 (71.7) | OR = 0.22 (0.05 to 0.91) | 0.025 |
| Negative recommendation, n (%) | 9 (64.3) | 13 (28.3) | ||
| Missing data, n (%) | 0 (0) | 0 (0) | ||
*Bold P value indicates statistical significance