Primary Biliary Cholangitis (PBC) in the U.S.: Real World Effectiveness of Obeticholic Acid in TARGET-PBC.

Challenge

OCA was approved for PBC in 2016 but its real-world effectiveness and safety profile in the more advanced, heterogeneous US patient population—which includes higher proportions of cirrhosis and AIH overlap than registration trial populations—had not been established.

Solution

The TARGET-PBC cohort was used to characterize OCA treatment patterns, biochemical response rates, and adverse events—including pruritus-related discontinuation—in 63 OCA-treated patients, comparing real-world effectiveness to the POISE registration trial in a more advanced population.

Impact

Demonstrating that OCA achieves comparable biochemical responses in a more advanced real-world population to those seen in registration trials supports the external validity of clinical trial results and provides real-world effectiveness context for prescriber and payer communications.