Which Metrics Are Appropriate to Describe the Value of New Cancer Therapies?

Table 1

Endpoints identified in clinical trial reviews.

Category^a	Endpoint reported (% of category) ()
	Included in >1 cancer type	Malignant melanoma only	NSCLC only	RCC only
Clinical response ()	Complication rate (26%); Response rate (23%); Local control/local tumor control (9%); Absolute benefit (8%); Disease control rate (3%); Complete response (1%)	Partial response (3%)	Time to progression (8%); Objective response rate (4%); Tumor response (3%); Symptom improvement (3%); 3-year freedom from local progression (1%); Length of hospital stay (1%); Radiological improvement (1%); Treatment failure rate (1%); Tumor progression (1%); Postoperative complication rate (1%)	Positive margin rate (1%); Surgery success rate (1%)
Disease progression ()	Progression-free survival (36%); Disease-free survival (32%); Recurrence-free survival (23%)	Distant metastasis-free survival (2%); Locoregional PFS (2%)	Asymptomatic survival (2%); Quality-adjusted PFS (2%)
Survival ()	OS^b (82%)		Drug related deaths (6%); 5-year survival (3%); Cause-specific mortality (3%); 1-year survival (2%); Treatment mortality/morbidity (2%); Perioperative morbidity (1%)
Recurrence/relapse ()	Local recurrence rate (38%); Recurrence rate (overall) (14%)		Relapse (14%); Asymptomatic recurrence (5%); Distant recurrence (5%); Remission (5%); Symptom-free period (5%); Systemic recurrence (5%); Time to recurrence (5%); Time to relapse (5%)
QoL ()	Health-related QoL (8%)	QoL (92%)

Review papers may include more than one endpoint category.
^bIncluded review papers of clinical trials that did not consistently report whether the articles they referenced used median or mean OS. When mentioned, the majority of included trial review papers referred to median OS.