Burden of primary biliary cholangitis on the Italian National Health Service: retrospective analysis from an administrative database

Introduction: Primary biliary cholangitis (PBC) is a rare, chronic, cholestatic, autoimmune, and disabling liver disease whose treatments are aimed at reducing its progression. The aim was to describe patients with PBC, their healthcare resource utilization (HCRU) and costs on the Italian National Health Service (SSN). Methods: From an Italian administrative healthcare database (~5.5 million inhabitants), called ReS Db, patients with PBC in 2021 (prevalent) were selected by specific codes and described by demographics, comorbidities, one-year HCRU, and health sector costs charged to the SSN. The 3-year treatment pattern was evaluated in a cohort of patients newly diagnosed in 2019 (incident). Results: In 2021, 1375 patients with PBC were identified (prevalence: 28.2/100,000; females: 74.2%; mean age: 64 ± 15 years). During one-year follow-up of the prevalent cohort: 73.1% received ursodeoxycholic acid (UDCA); 3.0% obeticholic acid (OCA), and 3.4% off-label drugs; 19.9% were hospitalized, mainly for cirrhosis-related conditions; 61.2% were examined by a specialist. The annual mean cost was €4422/patient; drugs for PBC, concomitant drugs, and hospitalizations accounted for 12.1%, 31.7%, and 43.6% of total cost, respectively. Among the 40 incident patients, 38 (95%) received UDCA as first-line therapy. Conclusion: The study described the real-world impact of PBC in Italy from the perspective of the SSN and highlighted the burden and therapeutic needs of patients treated with UDCA.