A Real-World Observational Cohort of Patients with Primary Biliary Cholangitis: TARGET-PBC Study Design and Rationale.

Challenge

Drug development for PBC second-line therapies and anti-pruritic agents lacked a longitudinal, prospective real-world cohort with systematic PRO collection, deep clinical data, and biospecimen infrastructure spanning the full PBC disease spectrum in a diverse US population.

Solution

The TARGET-PBC study was designed as a multi-site, longitudinal observational cohort of PBC patients at US academic and community sites, incorporating medical record abstraction, PBC-specific patient-reported outcome instruments, and a biospecimen repository.

Impact

Establishing the TARGET-PBC registry created the foundational real-world infrastructure for all subsequent PBC natural history, treatment effectiveness, and patient burden studies, directly enabling partnerships with Intercept, GSK, and other sponsors developing second-line PBC therapies.