Real-World Clinical and Economic Burden of Primary Biliary Cholangitis in the United States

OBJECTIVES: Primary biliary cholangitis (PBC) is a chronic cholestatic liver disease that can progress to cirrhosis and end-stage liver disease. This study assessed the real-world clinical/economic burden of PBC in the US by line of treatment.

METHODS: Retrospective study used IQVIA PharMetrics® Plus data (2016–2022) for 3 non-mutually exclusive cohorts: 1) patients with newly diagnosed PBC, 2) patients on first-line treatment (1L), 3) patients on second-line treatments (2L+). Index date: PBC diagnosis or 1L/2L+ treatment initiation. Baseline (12-month pre-index) characteristics, treatment sequence and time-to-treatment initiation/discontinuation, per-patient per-year healthcare costs post-index, and time from index date to earliest clinical outcome (eg, liver transplantation, cirrhosis, death) were summarized per cohort.

RESULTS: 1,748, 1,659, and 181 patients were in the newly diagnosed, 1L, and 2L+ cohorts, respectively. Across cohorts, mean age at index was 53.9–54.5 years, and 84.2%–89.0% were female. Common comorbidities were dyslipidemia (43.9%–54.7%), hypertension (41.2%–43.6%), and fatigue (19.9%–24.4%). Median time from PBC diagnosis to 1L initiation was 1.2 months; 34.8% of patients did not initiate treatment by 4 years post-diagnosis (N=609, untreated cohort). Most patients used ursodeoxycholic acid (UDCA; N=1,123) for 1L treatment. Most patients who discontinued UDCA (N=244/250) did not initiate any 2L+ treatment. 97 (5.5%) patients initiated 2L+ treatment and 55 used obeticholic acid + UDCA in 2L+. Cirrhosis was the most common and earliest clinical outcome during follow-up (untreated, 69/115; 1L, 212/239; 2L+, 31/35). Healthcare costs post-index were higher than pre-index: $54,226 vs $15,687, $18,460 vs $14,470, and $71,356 vs $14,288 in the newly diagnosed, 1L, and 2L+ cohorts, respectively.

CONCLUSIONS: This claims data analysis suggests that many patients with PBC remain untreated years after diagnosis despite approved treatments and experience increased comorbidity burden as lines of therapy increase, likely due to disease progression. These findings indicate an unmet need among all patients with PBC, especially in the untreated and 2L+ settings.