OBJECTIVES: To improve the accessibility of immune checkpoint inhibitors (ICIs) for various cancers, the Taiwan National Health Insurance Administration(NHIA) implemented risk-sharing reimbursement with mandatory real-world data registry. This study aims to provide outcomes from that registry.

METHODS: This nationwide, multicenter, retrospective cohort study collected data mainly from the Immune Checkpoint Inhibitor Registry Database (ICIRD), which enrolled cancer patients treated with ICIs (pembrolizumab, nivolumab, atezolizumab and avelumab) that were reimbursed by Taiwan National Health Insurance. The efficacy and safety of the ICI monotherapy were analyzed.

RESULTS: The study enrolled those who used ICIs reimbursed by NHIA from April 2019 to March 2021, and their outcome was followed up to September 2021. A total of 2382 patients with 10 indications of 7 types of cancers were analyzed for efficacy. The most prevalent indications were head and neck squamous cell carcinoma (n=582, 24.4%), and metastatic NSCLC 1st-line (n=456, 19.1%). The overall median progression-free survival (PFS) was 3.7 months (95% CI 3.4-4.3 months). The overall median overall survival (OS) was 10.1 months (95% CI 9.4-11.1 months), while the best and worst OS were observed in classical Hodgkin lymphoma (median and 95% CI not reached) and head and neck squamous cell carcinoma (7.1, 6.2 to 8.1 months), respectively. Of the 2114 patients registered for discontinuation, 164 (7.8%) patients had immune-related adverse events (irAEs). Grade 3 or higher irAEs were rare (3.7%, 78/2114).

CONCLUSIONS: This study overviews the effectiveness and safety of ICI for various cancers in a single-payer reimbursement system. Under the national registry, the RWE could reassess payment regulations to facilitate the optimal use of these high-cost drugs.