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

ConclusionThe proportion of the disease population studied in RWE was greater than that in RCTE. The analysis testing the relationship between additional RWE and broader indicated population would achieve adequate power between 2032 and 2037 at the current RWE adoption rate.
Source: Journal of Pharmaceutical Policy and Practice - Category: Pharmaceuticals Source Type: research