A Systematic Literature Review of Economic Evaluations in Primary Biliary Cholangitis

OBJECTIVES: Primary biliary cholangitis (PBC) is a rare autoimmune liver disease. Up to 40% of patients with PBC do not respond to first-line therapy with ursodeoxycholic acid (UDCA). PBC disease progression due to inadequate response to UDCA may lead to increased healthcare resource utilisation and expenditure, due to disease complications and eventual need for liver transplant. There are limited second-line therapies and these may not be cost-effective. A systematic literature review (SLR) was conducted to identify economic evaluations relevant for formulary decisions in PBC.

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

RESULTS: 1,480 and 1,124 records were identified from database and supplementary searches, respectively, which were screened against pre-determined eligibility criteria. Eight unique studies across five countries (UK, US, Ireland, Canada, and Norway) were included. Two types of economic evaluations were reported: cost-utility (6/8) and cost-effectiveness analysis (2/8). The most common types of model used were Markov state-transition (4/8), Markov (1/8), and microsimulation (1/8) models. The model type was not reported in two studies.

Most (5/8) studies evaluated obeticholic acid (OCA), of which 4/5 were submissions to HTA authorities. The cost of OCA was the most substantial factor in determining cost-effectiveness. The remaining 3 studies evaluated UDCA (n=2) and liver transplants (n=1). No studies evaluated fibrates in PBC. No economic evaluations were published since 2017.

CONCLUSIONS: There are limited cost-effectiveness analyses in PBC. The findings from this SLR highlight the need for additional, up-to-date economic evaluations and alternative therapies in PBC, which would drive choice for patients and healthcare systems, and a competitive treatment landscape.