Clinical benefit of immune checkpoint inhibitors approved by US Food and Drug Administration

BACKGROUND: We describe the clinical benefit of immune checkpoint inhibitors using the European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS) and ASCO VF. METHODS: We identify all approved indications of immune checkpoint inhibitors based on RCTs between January 1, 2011 and September 30, 2018 by FDA. Information including medians and HR of OS (PFS or DFS) and 95% CI, grade 3 or 4 toxicities in each arm, QOL data, survival probability at fixed time were extracted. RESULTS: Immune checkpoint inhibitors were approved for 18 indications based on RCTs. All the indications meet the ESMO-MCBS 1.1 threshold for meaningful benefit. By the updated ASCO-VF, the median Net Health Benefit (NHB) of these agents was 55.3 (range 17.4–77.1). Two third of the indication gained the bonus points for durable survival benefits by updated ASCO VF. When updated results were incorporated in the assessment, clinical benefit of most approved immune checkpoint inhibitors increased with a median improvement of NHB of 10 (range 2–20). CONCLUSIONS: Approved immune checkpoint inhibitors provided clinical meaningful benefit by ESMO-MCBS 1.1, and most of these agents reach the threshold for bonus points for durable survival in the updated ASCO VF.