Table 2.
Regulatory Approval Characteristics of Targeted Multi-Indication Drugs Across the US (FDA), EU (EMA), Canada (HC), and Australia (TGA)
|
US (FDA) |
EU (EMA) |
Canada (HC) |
Australia (TGA) |
P-value | |||||
|---|---|---|---|---|---|---|---|---|---|
| No | (%) | No | (%) | No | (%) | No | (%) | ||
| Approval Type | < .001 | ||||||||
| Standard | 52 | (54.2%) | 74 | (80.4%) | 41 | (47.7%) | 72 | (86.8%) | |
| Conditional Approval | 28 | (29.2%) | 12 | (13.1%) | 23 | (26.7%) | 3 | (3.6%) | |
| Priority Review | 16 | (16.6%) | 6 | (6.5%) | 22 | (25.6%) | 8 | (9.6%) | |
| Orphan Designation | < .001 | ||||||||
| No | 49 | (51.0%) | 70 | (76.1%) | NA | NA | 75 | (90.4%) | |
| Yes | 47 | (49.0%) | 22 | (23.9%) | NA | NA | 8 | (9.6%) | |
| MA Supporting Trial † | < .001 | ||||||||
| No | 57 | (59.4%) | 25 | (27.2%) | 48 | (55.8%) | 50 | (60.2%) | |
| Phase 1 | 7 | (7.3%) | 14 | (15.2%) | 5 | (5.8%) | 2 | (2.4%) | |
| Phase 2 | 15 | (15.6%) | 28 | (30.4%) | 13 | (15.1%) | 19 | (22.9%) | |
| Phase 3 | 17 | (17.7%) | 25 | (27.2%) | 20 | (23.3%) | 12 | (14.5%) | |
| Pivotal Trial Design ‡ | 0.822 | ||||||||
| Phase 1 | 4 | (4.2%) | 3 | (3.2%) | 5 | (5.8%) | 3 | (3.6%) | |
| Phase 2 | 25 | (26.0%) | 18 | (19.6%) | 23 | (26.8%) | 19 | (21.7%) | |
| Phase 3 | 67 | (70.8%) | 71 | (77.2%) | 58 | (67.4%) | 61 | (74.7%) | |
| No. of Observations | 96 | (100%) | 92 | (100%) | 86 | (100%) | 83 | (100%) | |
P-values calculated based on χ2-tests
FDA US Food and Drug Administration, EMA European Medicines Agency, HC Health Canada, TGA Therapeutics Goods Administration, NA Not Applicable
† Highest phase of supporting trials disclosed in the regulatory approval report (No supporting trial: 0, Phase 1: 1, Phase 2: 2, Phase 3: 3)
‡ Highest phase pivotal trial disclosed in the regulatory approval report (Phase 1: 1, Phase 2: 2, Phase 3: 3)