Table 7 Methodological conduct of the pivotal studies (n = 108)
From: Clinical evidence for orphan medicinal products-a cause for concern?
| Â | Number of pivotal studies (%) |
|---|---|
QoL-related endpoint | |
 No / Not reported | 79 (73.1%) |
 Yes, using a disease-specific QoL scale | 9 (8.3%) |
 Yes, using a generic QoL scale | 16 (14.8%) |
 Yes, using both a disease-specific and a generic QoL scale | 4 (3.7%) |
Improvement in QoL (n = 29) | |
 Yes | 10 (34.5%) |
 No | 14 (13.0%) |
 Not reported | 5 (4.6%) |
Relevant duration of the study | |
 Yes | 86 (79.6%) |
Non-adherence to the study protocol | |
 Yes, by certain subjects | 7 (6.5%) |
 Yes, by the researcher(s) | 15 (13.9%) |
 Yes, by both | 7 (6.5%) |
 No / Not reported | 79 (73.1%) |
Assessment of patient adherence | |
 Yes | 17 (15.7%) |
 No / Not reported | 91 (84.3%) |