From: Clinical evidence for orphan medicinal products-a cause for concern?
Number of pivotal studies (%) | |
---|---|
Appropriate statistical analysis | |
Yes | 48 (44.4%) |
No | 41 (38.0%) |
Don’t know (too few details) | 19 (17.6%) |
Summary statistics at baseline | |
Yes | 71 (65.7%) |
With statistical testing | 7 (6.5%) |
Summary statistics at outcome | |
No | 5 (4.6%) |
Yes, for all endpoints | 44 (40.7%) |
Statistically tested | 14 (13.0%) |
Not statistically tested | 30 (27.8%) |
Yes, partially | 45 (41.7%) |
Statistically tested | 31 (28.7%) |
Not statistically tested | 14 (13.0%) |
Yes, only primary | 14 (13.0%) |
Statistically tested | 10 (9.3%) |
Not statistically tested | 4 (3.7%) |
Intention-to-treat analysis | |
Yes | 74 (68.5%) |
No | 8 (7.4%) |
Not applicable / Not reported | 26 (24.0%) |
Reported with respect to patients lost to follow-up | |
Reasons for loss to follow-up | 70 (64.8%) |
Characteristics | 2 (1.9%) |
Not applicable | 8 (7.4%) |