From: Clinical evidence for orphan medicinal products-a cause for concern?
 | Number of pivotal studies (%) |
---|---|
Study phase | |
 Phase III | 50 (46.3%) |
 Phase II-III | 9 (8.3%) |
 Phase II | 39 (36.1%) |
 Phase I | 2 (1.9%) |
 Not reported/Not applicable | 8 (7.4%) |
Center characteristics | |
 Multicenter | 91 (84.3%) |
 Mono-center | 5 (4.6%) |
 Not reported | 12 (11.1%) |
International character of the study | |
 Multinational | 15 (13.9%) |
 Mono-national | 65 (60.2%) |
 Not reported | 28 (25.9%) |
Primary endpoint | |
 At least one hard endpoint | 21 (19.4%) |