OBJECTIVES : Erdafitinib is the first, and, so far, only targeted therapy approved for the treatment of metastatic urothelial carcinoma (mUC) FGFR+. Before, the treatment pattern of mUC for FGFR+ patients consisted of non-specific drugs, such as chemotherapy and anti-PD1/PDL1. Comparative effectiveness and cost data for these therapies are important to inform decision-making processes for healthcare budget allocation. This analysis was aimed to estimate cost per consequence of erdafitinib and anti-PD1/PDL1 therapies from the perspective of Brazilian private healthcare system.

METHODS : Efficacy was assessed with data from erdafitinib (BLC2001), pembrolizumab (Keynote 045), atezolizumab (IMVIGOR 211), nivolumab (CheckMate 275) and durvalumab (MEDI4736) trials. Treatment costs until disease progression were calculated based on each drug label dosages, administration costs and fees. The Brazilian official lists of drugs prices (CMED) and medical materials (SIMPRO) were used as sources.

RESULTS : Objective response rate (ORR), median PFS (months) and monthly costs per patient were: erdafitinib (40,0% - 5,5 - BRL 50,882), pembrolizumab (21,1% - 2,1 - BRL 46,666), atezolizumab (13,4% - 2,2 - 39,742), nivolumab (19,6% - 2,0 - BRL 47,612) and durvalumab (17,6% - 1,5 - BRL 42,967). On average, erdafitinib increased ORR by 2-fold (1,9 to 3,0-fold) compared to immunotherapies. Likewise, erdafitinib has the longest PFS, being 3-times higher (2,6 – 3,7 times) than immunotherapies. Finally, erdafitinib costs on average 13% (8% to 22%) more than its comparators.

CONCLUSIONS : With the introduction of erdafitinib, ORR in mUC for FGFR+ patients has doubled and PFS has tripled versus anti-PD1/PDL1. In addition to that, erdafitinib is the only targeted therapy for mUC FGFR+ patients and it is more convenient for them than other IV alternatives by being orally administered. Such incremental value is delivered with only 13% incremental treatment costs compared to nonspecific therapies such as anti-PD1/PDL1.