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Table 6 Characteristics of the study population in the pivotal studies (n = 108)

From: Clinical evidence for orphan medicinal products-a cause for concern?

  Number of pivotal studies (%)
Study population represents the patient population?
 Yes 53 (49.1%)
 No 11 (10.2%)
 Don’t know 44 (40.7%)
A priori power calculations
 Yes, and the required number of inclusions was achieved 51 (47.2%)
 Yes, but the required number of inclusions was not achieved 7 (6.5%)
 Yes, but it is unclear if the required number of inclusions was achieved 3 (2.8%)
 No / Not reported 47 (43.5%)