OBJECTIVES: Primary biliary cholangitis (PBC) is a rare autoimmune liver disease characterised by progressive cholestasis and biliary fibrosis. PBC symptoms and complications can negatively impact patients’ health-related quality of life (HRQoL) and may lead to substantial healthcare costs and resource use (CRU). A systematic literature review (SLR) was conducted to identify HRQoL and CRU studies in PBC.

METHODS: This SLR was performed in accordance with PRISMA, Cochrane Collaboration and UK NICE guidelines. Searches were conducted in November 2022 in MEDLINE, EMBASE and the Health Technology Assessment (HTA) Database. HTA/economic websites (n=19), congress proceedings (n=8) from 2021–2022 and bibliographies were hand searched.

RESULTS: 2,604 records, identified from database (n=1,480) and supplementary (n=1,124) searches, were screened against pre-determined eligibility criteria. The large number of studies identified in HRQoL (n=63) and CRU (n=33) prompted prioritisation of 5 HRQoL studies reporting EQ-5D utility data and 9 CRU studies reporting data from Europe in the last 10 years.

2/5 HRQoL studies specifically investigated PBC. 4/5 studies reported a substantial negative impact of PBC on patients’ HRQoL; where reported, pruritus (2 studies), as well as fatigue, bone ache and memory and concentration problems (1 study) were key drivers of reduced HRQoL. EQ-5D scores were lower overall and following liver transplant versus age/sex-adjusted mean UK population scores (1 study).

Among studies reporting CRU data, 5/9 reported ursodeoxycholic acid use in 79.3–90.6% of patients. Liver transplants, reported by 4/9 CRU studies, were associated with substantial costs where reported (2 studies in the UK and Italy), exceeding cirrhosis- and cancer-related costs.

CONCLUSIONS: The HRQoL and CRU data captured in this SLR provide insight into the substantial impact of PBC on patients' lives as well as key inputs for cost-utility analyses, which can be used to elucidate potential benefit of investigational treatments for patients and healthcare systems.