Table 2.
Comparison of Characteristics and Review Types of Orphan and Non-Orphan Drugs for Non-Oncology Indications
| Characteristic | Orphan non-oncology drugs | Non-orphan non-oncology drugs | p value |
|---|---|---|---|
| Total number | 73 | 135 | |
| Drug characteristics | |||
| Indications per drug | |||
| Number (median, IQR) | 1 (1,2) | 2 (2,3) | < 0.0001, Mann-Whitney test |
| First in class | |||
| Yes (%) | 37 (50.7) | 38 (28.1) | 0.0006, chi-square test |
| No (%) | 29 (39.7) | 91 (67.4) | |
| No information (%) | 7 (9.6) | 6 (4.4) | |
| Type of drug | |||
| Small molecule (%) | 45 (61.6) | 102 (75.6) | 0.0519, chi-square test |
| Biologic (%) | 28 (38.4) | 33 (24.4) | |
| Clinical trial characteristics | |||
| Pivotal trials | |||
| Number per drug, median (IQR) | 1 (1,1) | 1 (1,1) | 0.7957, Mann-Whitney test |
| Pivotal trial phase used for approval | |||
| 1 (%) | 0 (0) | 2 (1.5) | 0.4285, chi-square test |
| 2 (%) | 6 (8.2) | 6 (4.4) | |
| 3 (%) | 58 (79.5) | 105 (77.8) | |
| No information (%) | 9 (12.3) | 22 (16.3) | |
| Number of arms per trial | |||
| 1 (%) | 0 (0) | 7 (5.2) | 0.077, chi-square test |
| 2 (%) | 6 (8.2) | 74 (54.8) | |
| 3 (%) | 58 (79.5) | 29 (21.5) | |
| 4+ (%) | 9 (12.3) | 11 (8.1) | |
| No information (%) | 0 (0) | 14 (10.4) | |
| Patients enrolled per trial | |||
| Median (IQR) | 137 (52, 260) | 870 (590, 1605) | < 0.0001, Mann-Whitney test |
| Outcome used | |||
| Clinical (%) | 35 (47.9) | 46 (34.1) | < 0.0291, Fisher exact test |
| Surrogate (%) | 37 (50.7) | 89 (65.9) | |
| No information (%) | 1 (1.4) | 0 (0) | |
| Randomized | |||
| Yes (%) | 60 (82.2) | 124 (91.9) | 0.0111, Fisher exact test |
| No (%) | 12 (16.4) | 6 (4.4) | |
| No information (%) | 1 (1.4) | 5 (3.7) | |
| Control | |||
| Placebo (%) | 40 (54.8) | 88 (65.2) | 0.0145, chi-square test |
| Active (%) | 12 (16.4) | 33 (24.4) | |
| No (%) | 15 (20.5) | 10 (7.4) | |
| Other (%) | 3 (4.1) | 3 (2.2) | |
| No information (%) | 3 (4.1) | 1 (0.7) | |
| Blinded | |||
| Yes (%) | 43 (58.9) | 109 (80.7) | < 0.0001, chi-square test |
| No (%) | 24 (32.9) | 12 (8.9) | |
| No information (%) | 6 (8.2) | 14 (10.4) | |
| Review type | |||
| Type of review | |||
| Standard (300 days) | 29 (39.7) | 115 (85.2) | < 0.0001, chi-square test |
| Priority (180 days) | 41 (56.2) | 17 (12.6) | |
| Notice of compliance with conditions/priority + notice of compliance with conditions (conditional approval subject to results of confirmatory studies) | 3 (4.1) | 3 (2.2) | |
| Therapeutic value | |||
| Major (%) | 12 (22.2) | 11 (9.7) | p = 0.0821, chi-square test |
| Moderate (%) | 11 (20.4) | 23 (20.4) | |
| Little to none (%) | 31 (57.4) | 79 (69.9) | |