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%) |