OBJECTIVES: Obeticholic acid (OCA) is the only approved second-line therapy for treatment of primary biliary cholangitis (PBC), based on significant reduction in serum levels of alkaline phosphatase, a surrogate marker that is predictive of clinical hepatic outcomes. Real-world studies have reported a 58%–63% reduction in risk of death, liver transplant, and/or hepatic decompensation for patients with PBC treated with OCA vs matched controls. The aim of this study was to estimate the number of hepatic decompensation events, liver transplantations, and liver-related deaths, as well as all-cause mortality, that may be prevented with OCA using published real-world clinical outcomes data.

METHODS: A model was developed to estimate clinical events in patients with PBC who were eligible for second-line therapy following inadequate response to, intolerance of, or discontinuation of ursodeoxycholic acid (UDCA). Patients treated with 5–10 mg OCA with or without UDCA were compared with patients receiving UDCA or no treatment. Event rates per 100 person-years for hospitalization due to hepatic decompensation, liver transplantation, or death were obtained from the real-world HEROES study.

RESULTS: In a modeled cohort of 1000 patients, an estimated 21, 56, and 85 hepatic decompensation events may be prevented in patients treated with OCA for 1, 3, and 5 years, respectively. Additionally, 4, 10, and 16 liver transplants and 9, 26, and 43 deaths may be avoided over 1, 3, and 5 years, per 1000 OCA-treated patients, respectively.

CONCLUSIONS: Although clinical trials in PBC provide an important measure of effectiveness based on liver biochemistries, studies in real world-settings have demonstrated the benefit of OCA on long-term outcomes as the medication has been used in regular clinical practice since its approval in 2016. Using real-world data to estimate clinical events avoided demonstrates the clinical value of OCA for appropriate patients with PBC.