Economic Burden of Primary Biliary Cholangitis by Line of Therapy in the United States.

INTRODUCTION: Lack of or delayed treatment of primary biliary cholangitis (PBC) is associated with worsening outcomes. This study assessed the real-world healthcare resource use (HRU) and costs of PBC in the USA to understand its economic burden by line of therapy. METHODS: This retrospective study analyzed IQVIA PharMetrics® Plus claims data (2016-2022) for three non-mutually exclusive cohorts of adults diagnosed with PBC from January 1, 2017: untreated; first-line (1L) treatment with ursodeoxycholic acid (UDCA); second-line or more (2L+) treatment after UDCA. Index date for each cohort was: untreated, date of PBC diagnosis; 1L, 1L treatment initiation; 2L+, 2L treatment initiation. All-cause per-patient per-year (PPPY) HRU and costs during baseline and follow-up periods were compared among groups. RESULTS: Patients in the untreated cohort had higher mean inpatient (IP) admissions (PPPY; 0.93 visits) and longer mean IP length of stay (LOS) (PPPY; 7.45 days) than the 1L (0.16 visits; 1.12 days) and 2L+ (0.19 visits; 1.77 days) cohorts during follow-up. Patients in the untreated cohort had the highest mean IP costs during the follow-up period (untreated: $37,974; 1L: $5854; 2L+: $6898). Compared with baseline, significantly higher HRU during follow-up was observed for the 1L and 2L+ cohorts regarding mean number of outpatient visits (incidence rate ratio [IRR]: 1.07; p = 0.001) and mean IP LOS (IRR: 2.12; p = 0.001), respectively. CONCLUSIONS: These results highlight the importance of initiating timely treatment for PBC to reduce HRU and medical costs. The findings also demonstrate the need for novel, more effective PBC treatments.