Table 1.

Characteristics of accelerated approval trials that have failed post-approval trials and had indication withdrawn/revoked

Characteristics	Failed confirmatory trials (n=18)	Indication withdrawn or revoked* (n=10)
Primary endpoint for accelerated approval	Response rate (15); progression-free survival (2); overall survival in phase II trial (1)	Response rate (8); progression-free survival (1); overall survival in phase II trial (1)
Primary endpoint for post-approval trial	Overall survival, as primary or co-primary endpoint (16); trials not completed (2)	Overall survival, as primary or co-primary endpoint (8); trials not completed (2)
Tumour type	Urothelial (4); haematological (3); lung (3); breast (2); hepatocellular (2); glioblastoma (1); melanoma (1); sarcoma (1); gastrointestinal (1)	Haematological (3); lung (3); urothelial (2); breast (1); sarcoma (1)
Drug type	Checkpoint inhibitor (11); monoclonal antibody (4); targeted therapy (1); chemotherapy (1); antibody drug conjugate (1)	Checkpoint inhibitor (4); monoclonal antibody (3); targeted therapy (1); chemotherapy (1); antibody drug conjugate (1)

^*10 trials that led to withdrawal or revoking of approvals are subset of overall 18 failed confirmatory trials.