Primary Biliary Cholangitis (PBC) in the U.S.: Clinical Characteristics of Patients Enrolled in TARGET-PBC.

Challenge

Drug development and regulatory strategy for PBC second-line therapies required a comprehensive characterization of the real-world US PBC population—demographics, disease severity, treatment patterns, and biomarker profiles—to establish the external validity of registry data for drug development use.

Solution

The first consecutive patients enrolled in TARGET-PBC were characterized across key clinical, demographic, laboratory, and treatment dimensions, providing a comprehensive cross-sectional baseline of the registry population at multiple enrollment stages.

Impact

Establishing the TARGET-PBC baseline population profile validated the registry's representativeness for drug development and demonstrated that it included advanced and complex patients not typically seen in registration trials, supporting its use for naturalistic comparator data in PBC programs.