Work

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

Year: 2025

Type: article

Abstract: 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. 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. 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. 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. Primary biliary cholangitis is a rare liver disease that gradually worsens over time and can be potentially life-threatening. Treatments are available for primary biliary cholangitis, but they do not work for everyone, and some people experience unpleasant side effects. Therefore, patients may need to try different treatments to determine what works best for them. This study looked at health insurance claims to understand how patients with primary biliary cholangitis use healthcare services and how much these services cost. The study focused on three groups of patients: those who did not receive any treatment for primary biliary cholangitis (untreated group), those who were treated with a medication called ursodeoxycholic acid as their first treatment (first-line group), and those who received additional treatments for primary biliary cholangitis after receiving ursodeoxycholic acid (second-line or more group). The results showed that after diagnosis, untreated patients were admitted to the hospital more often and had longer hospital stays compared with patients who began treatment. These untreated patients also had the highest costs associated with these hospital stays. In addition, patients who started treatments had longer hospital stays after their disease worsened. These findings suggest that timely treatment may reduce the need for hospital care. The study also highlights the need for new primary biliary cholangitis medications for patients who do not improve with initial treatments.

Source: Advances in Therapy

DOI: 10.1007/s12325-025-03439-6