From: The effect of adding real-world evidence to regulatory submissions on the breadth of population indicated for rare disease medicine treatment by the European Medicines Agency
Evidence profile
Approved vs. proposed indication populations
p-value
Same or narrower
Broader
Total
RCTE
38 (43.68%)
49 (56.32%)
87
0.269
RCTE + RWE
3 (23.08%)
10 (76.92%)
13
41
59
100