Skip to main content

Table 2 Characteristics of the pivotal studies (n = 108)

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%)