Ursodeoxycholic acid (UDCA) is a safe and effective treatment for patients
with primary biliary cirrhosis (PBC), but the cost of this drug has raised
concerns regarding cost-effectiveness. The aim of our study was to determin
e the cost-effectiveness of UDCA in PBC. We compared the costs and outcomes
of managing PBC patients with and without UDCA. From two previously publis
hed trials, the effectiveness of UDCA was determined by comparing the annua
l reduction in the development of ascites, varices, variceal bleeding, ence
phalopathy, liver transplantation, and death between the treatment groups.
Average annual costs for each of these events were estimated based on liter
ature and institutional data. Approximately twice as many major events occu
rred in the placebo group compared with the UDCA group. The relative risk (
RR) of liver transplantation (1.95; 95% CI: 1.14-3.68) and development of e
sophageal varices (3.11; 95% CI: 1.57-10.65) were significantly higher in t
he placebo group compared with the UDCA group. There were no significant in
creases in the RR of ascites, variceal bleeding, encephalopathy, or death b
etween the two groups. Based on the estimated annual cost of managing these
events and the annual costs of UDCA ($2,500), there was an annual cost sav
ings per patient of $1,372. Compared with the placebo group, patients recei
ving UDCA had a lower incidence of major complications and lower medical ca
re costs.