Primary biliary cholangitis (CBP) is a rare autoimmune cholestatic liver disease, inflammation and progressive destruction of small and medium-sized interlobular ducts, progressing to fibrosis, cirrhosis, and death. Currently, the Brazilian public health system (SUS) offers treatment of the symptoms of cirrhosis, and has no medication with indication for CBP.

Scientific technical opinion with systematic review (SR) of available evidence in the databases MEDLINE (Pubmed), LILACS and Cochrane Library (accessed July 2017) on ursodeoxycholic acid (AUDC). Methodological quality was evaluated with AMSTAR and Newcastle Ottawa tools. Meta-analyses were performed in Review Manager® 5.2 in the random effects model. Analysis of the budget impact calculation deterministic model, from the perspective of five years for the SUS.

Ten SRs and three cohorts were included. There was no statistically significant difference between AUDC and placebo in outcome. Overall survival was significantly (P <0.001) higher in the AUDC group compared to that predicted by the Mayo model or placebo. Treatment with UCD showed an increase in the long-term transplant-free survival time from the fifth year of treatment, with statistically significant results for years five, eight and ten (p <0.01). There were no statistically significant differences for safety outcomes. Based on the assumptions adopted, the incremental budgetary impact with the incorporation of the AUDC into SUS would be BRL 11.77 million (EUR 2.68 million) in the first year and BRL 98.52 million (EUR 22.45 million) in the accumulated five years, considering a market share of 10 percent per year.

Despite the uncertainties in the evidence of effectiveness of the AUDC and the probably underestimated budgetary impact, AUDC was incorporated into the SUS because it is the only alternative with indication for CBP and in use for more than two decades, allowing everyone access to the medicine