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Table 7 Methodological conduct of the pivotal studies (n = 108)

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

  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%)