Oncologic orphan drugs approved in the EU – do clinical trial data correspond with real-world effectiveness?

Table 7 Relation between type of primary endpoint and effectiveness

		Effectiveness
		Survival benefit > 3 months	Survival benefit < 3 months, unknown magnitude or no benefit	Total
Primary endpoint	OS or surrogate endpoint for OS	3	1	4
Primary endpoint	PFS, RFS, TTP, ORR	3	3	6
	Total	6	4	10

ISSN: 1750-1172