Inconsistent approaches of the G-BA regarding acceptance of primary study endpoints as being relevant to patients - an analysis of three disease areas: oncological, metabolic, and infectious diseases

Table 3 G-BA acceptance of symptomatic vs asymptomatic morbidity PEPs as (A) patient-relevant and (B) non-patient-relevant

	Oncological diseases	Metabolic diseases	Infectious diseases
(A) Morbidity PEPs accepted as patient-relevant by the G-BA
Symptomatic	Complete durable tumour & symptomatic response^a	6MWT	Overall cure
Symptomatic	≥35 % reduction in spleen volume^a	Reduction in spleen volume^a	-
Asymptomatic	Haematocrit control without phlebotomy^a	HbA1c (Type 1 diabetes)	Viral response (VR, SVR)
	MCR	mUFC	-
	-	Biochemical control (mean GH <2.5 μg/L and normalisation of IGF-1)	-
(B) Morbidity PEPs not accepted as patient-relevant by the G-BA
Symptomatic	Partial durable tumour & symptomatic response^a	-	-
Asymptomatic	PFS^a	HbA1c (Type 2 diabetes)	-
	ORR	Haemoglobin level^a	-
	-	Thrombocyte count^a	-
	-	Reduction in liver volume^a	-
	-	FEV1	-

^aThis PEP was a component of a co-primary endpoint in at least one dossier
6MWT 6-minute walking test, FEV1 Forced expiratory volume in 1 second, G-BA Federal Joint Committee, GH Growth hormone, HbA1c Glycated haemoglobin, IGF-1 Insulin-like growth factor 1, MCR Major cytogenic response, mUFC Median urinary free cortisol, ORR Objective response rate, OS Overall survival, PEP Primary endpoint, PFS Progression-free survival, SVR Sustained viral response, VR Viral response

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