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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%)